[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"myelodysplastic-syndromes\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:myelodysplastic-syndromes":27},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,144,0,25,[9,47,82,105,130,160,184,209,238,263,283,304,330,351,374,397,420,454,480,502,524,552,579,623,649],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":15,"acronym":4,"eligibilityCriteria":16,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100554453","phase-3-a-study-of-elritercept-to-treat-anemia-in-adults-with-very-low-low-or-intermediate-risk-myelodysplastic-syndromes-mds-who-need-regular-blood-transfusions-100554453",false,"NCT06499285","A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions","A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elritercept (KER-050) for the Treatment of Transfusion-Dependent Anemia in Adult Participants With Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) (RENEW)","Inclusion Criteria:\n\n* Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information and\u002For protected personal data in accordance with national and local study participant data protections and privacy regulations.\n* Male or female greater than or equal to (≥)18 years of age at the time of signing informed consent.\n* Diagnosis of MDS with or without RS (as determined in an evaluable bone marrow aspirate, read by an independent central reader to confirm diagnosis at Screening) according to the World Health Organization 2016 classification that meets the International Prognostic Scoring System-Revised (IPSS-R) classification of very low, low, or intermediate risk disease.\n* Transfusion dependence assessed in the 16 weeks immediately preceding randomization in two 8-week blocks, classified as either:\n\n  a. Low-transfusion burden (LTB), defined as 4 to 7 red blood cells (RBC) units per 16 weeks; or b. High-transfusion burden (HTB), defined as ≥8 RBC units per 16 weeks; and c. For all participants: i. Only transfusion events for a pretransfusion hemoglobin (Hgb) lesser than (\\\u003C)10 grams per deciliter (g\u002FdL) are counted toward eligibility; ii. At least 1 transfusion event in each 8-week period and a minimum of 2 transfusion events separated by ≥7 days within the 16-week period immediately preceding randomization; and iii. No consecutive 56-day period can be RBC transfusion-free during the 16-week period immediately preceding randomization.\n* Refractory or intolerant to prior erythropoiesis-stimulating agent (ESA) treatment (discontinued ≥4 weeks before randomization), or unlikely to respond to ESA treatment, defined as follows:\n\n  a. Refractory to prior ESA treatment: documentation of nonresponse or a response that was no longer maintained with a prior ESA-containing regimen, either as a single agent or combination (e.g., with granulocyte colony-stimulating factor \\[G-CSF\\]); ESA regimen must have been either: i. Recombinant human erythropoietin (EPO) ≥40,000 international units per week (IU\u002Fweek) for ≥8 doses or equivalent; or ii. Darbepoetin alpha ≥500 micrograms (μg) every 3 weeks for ≥4 doses or equivalent.\n\n  b. Intolerant to prior ESA treatment: documentation of discontinuation of a prior ESA-containing regimen, either as a single agent or combination (e.g., with G-CSF), at any time after introduction due to intolerance or an AE.\n\n  c. Unlikely to respond to ESA treatment: low chance of response to ESA based on an endogenous serum EPO level greater than (\\>)200 units per liter (U\u002FL).\n* Less than 5% blasts in an evaluable bone marrow aspirate collected at Screening, read by an independent central reader.\n* Eastern Cooperative Oncology Group performance status of 0 to 2.\n* Females of childbearing potential and sexually active males must agree to use highly effective methods of contraception.\n* In the opinion of the Investigator, the participant is able and willing to comply with the requirements of the protocol (e.g., all study procedures, return for follow-up visits).\n\nExclusion Criteria:\n\n* Del(5q) MDS or therapy-related (secondary) MDS.\n* Anemia due to any other known cause (e.g., thalassemia, hemolytic anemia, bleeding events, or deficiency of iron, B12, and\u002For folate).\n* Receipt of RBC transfusion for any reason(s) other than underlying MDS within 16 weeks before randomization.\n* Clinically significant cardiovascular disease defined as:\n\n  1. New York Heart Association heart disease class III or IV;\n  2. Fridericia corrected QT (QTcF) interval \\>500 milliseconds during Screening;\n  3. Presence of uncontrolled hypertension defined as mean systolic blood pressure ≥160 millimeters of mercury (mm Hg) or diastolic blood pressure ≥100 mm Hg during Screening; or\n  4. Uncontrolled arrhythmia, myocardial infarction, or unstable angina within 6 months before Screening.\n* Known ejection fraction \\\u003C35%, confirmed by a local echocardiogram performed during Screening, or a previously performed echocardiogram if collected within 6 months before Screening.\n* Child-Pugh class C hepatic impairment.\n* Stroke, deep vein thrombosis, or pulmonary embolism within 6 months before Screening.\n* Any known history of acute myeloid leukemia (AML).\n* Prior history of malignancies, other than MDS, unless participant has been free of the disease (including completion of any treatment, including maintenance, for prior malignancy) for ≥ 5 years. However, participants with a history or concurrent diagnosis of the following conditions are allowed if not requiring systemic therapy:\n\n  1. Basal or squamous cell carcinoma of the skin;\n  2. Carcinoma in situ of the cervix;\n  3. Carcinoma in situ of the breast; and\u002For\n  4. Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, node, metastasis \\[TNM\\] clinical staging system).\n* History of solid organ or bone marrow transplantation.\n* Active infection requiring intravenous treatment (e.g., antibiotics, antifungals, or antivirals) within 28 days, or oral treatment within 14 days before randomization.\n* History of or known active chronic infection with HIV, hepatitis B virus (HBV), or hepatitis C virus (HCV). Participants without known positive history of HIV, HBV, and\u002For HCV do not require further testing, unless testing is mandated per local guidelines.\n* Body mass index ≥ 40 kilograms per meter square (kg\u002Fm\\^2).\n* Major surgery within 28 days before randomization.\n* History of allergy\u002Fanaphylaxis to investigational medicinal product (IMP) excipients (refer to the current elritercept IB for a list of excipients) or recombinant proteins.\n* Prior use of elritercept, luspatercept, or sotatercept.\n* Prior use of hypomethylating agents (HMAs), isocitrate dehydrogenase inhibitor, lenalidomide, imetelstat, or immunosuppressive therapy given for treatment of MDS.\n* Iron chelation therapy initiated within 8 weeks before randomization. Participants on stable doses of iron chelation therapy for ≥ 8 weeks are allowed.\n* Vitamin B12 or folate therapy initiated within 4 weeks before randomization. Participants on stable replacement doses for ≥ 4 weeks and without ongoing concurrent vitamin B12 or folate deficiency are allowed.\n* Androgen use within 8 weeks before randomization. Participants on stable androgen dosing for hypogonadism for ≥ 8 weeks are allowed.\n* High-dose corticosteroid use within 4 weeks before randomization. Participants on stable chronic steroid doses of prednisone lesser than or equal to (≤) 10 mg\u002Fday or corticosteroid equivalent for ≥ 4 weeks are allowed.10 mg\u002Fday or corticosteroid equivalent for ≥ 4 weeks are allowed.\n* Treatment with any investigational drug within 28 days before Screening or, if the half-life of the product is known, within 5 times the half-life before Screening, whichever is longer.\n* Ongoing participation in another interventional clinical study.\n* Serum EPO level \\>500 U\u002FL.\n* Platelet count ≥450 × 10\\^9\u002FL or ≤25 × 10\\^9\u002FL.\n* Absolute neutrophil count ≤ 500\u002FµL.\n* Serum aspartate aminotransferase or alanine aminotransferase ≥3 × the upper limit of normal (ULN).\n* Total bilirubin ≥2 × ULN unless attributable to Gilbert's syndrome.\n* Ferritin ≤ 50 micrograms per litre (μg\u002FL).\n* Folate ≤2.0 nanograms per milliliter (ng\u002FmL).\n* Vitamin B12 ≤200 picograms per milliliter (pg\u002FmL).\n* Estimated glomerular filtration rate \\\u003C30 milliliters per minute per 1.73 meter square (mL\u002Fmin\u002F1.73m\\^2) as determined by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Collaboration equation.\n* Pregnant or lactating female.\n* Any other condition not specifically noted above that, in the opinion of the Investigator, would preclude the participant from participating in the study or could confound interpretation of data from the study.\n* Investigational site staff members directly involved in the conduct of the study and site staff members otherwise supervised by the Investigator, employees of the Sponsor or contract research organization (CRO) directly involved in the conduct of the study, or immediate family members (defined as a spouse, parent, child, or sibling, whether biological or legally adopted).\n* For Participants in France: Persons under court protection, persons not affiliated with a social security system, and protected adults (per applicable French law \\[Art. L. 1121-6, Art. L. 1121-8, Art. L. 1121-8-1\\]).","ALL","18 Years",{"count":20,"type":21},225,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","The main aim of this study is to find out how well elritercept works in lowering the need for RBC transfusions. Other aims are to learn how well elritercept works in reducing the need for RBC transfusions over longer periods of time or in adults with high transfusion needs. The study will also check on how safe elritercept is and how well it is tolerated.",[27],"Myelodysplastic Syndromes",[29,30,31,32,33],"Anemia","Elritercept","Myelodysplastic neoplasms","KER-050","MDS","RECRUITING","2026-08-20",{"date":37,"type":38},"2026-08-21","ACTUAL",{"date":40,"type":38},"2025-05-06",{"date":42,"type":21},"2032-05-01",{"name":44,"class":45},"Takeda","INDUSTRY",177,{"id":48,"slug":49,"hasResults":12,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":12,"sex":17,"minAge":54,"maxAge":55,"enrollmentInfo":56,"targetDuration":4,"studyType":22,"phases":58,"briefSummary":61,"conditions":62,"keywords":63,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":73,"startDateStruct":74,"completionDateStruct":76,"leadSponsor":78,"locationsCount":81},"100539388","pacritinib-a-kinase-inhibitor-of-csf1r-irak1-jak2-and-flt3-in-adults-and-pediatric-participants-12-years-of-age-or-older-with-myelodysplastic-syndromes-or-myelodysplasticmyeloproliferative-neoplasms-100539388","NCT06303193","Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic\u002FMyeloproliferative Neoplasms","Phase I\u002FII Trial of Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic\u002FMyeloproliferative Neoplasms","* INCLUSION CRITERIA:\n* Participants must have histologically or cytologically confirmed MDS or MDS\u002FMPN, including therapy-related MDS or MDS\u002FMPN, and MDS or MDS\u002FMPN with germline predisposition, by the Department of Laboratory Medicine Hematology Laboratory, CC or by the Laboratory of Pathology, NCI as defined according to the 2016 WHO criteria, 2022 WHO criteria, or 2022 International Consensus Classification\n* Age 12-17 years for phase I and age \\>= 18 years for phase II\n* Participants \\>= 18 years of age with HR-MDS must have resistance to hypomethylating agents as defined as failure to show improvement after at least 4 cycles of treatment (primary resistance) or relapse in participants with initial response to long-term treatment (secondary resistance) OR have intolerance to hypomethylating agents OR have a contraindication to hypomethylating agents\n* Participants \\>= 18 years of age with LR-MDS must be refractory to or ineligible to receive standard of care therapies, i.e. erythropoietin-stimulating agents, lenalidomide, luspatercept, and present with one of the following characteristics:\n\n  * Severe neutropenia defined by absolute neutrophils count \\\u003C=0.5(SqrRoot) 10\\^9\u002FL without the use of granulocyte colony-stimulating factors\n  * Symptomatic anemia defined by hemoglobin 16-week average \\\u003C10 g\u002FdL and symptoms that may include fatigue, weakness, reduced exercise tolerance, dyspnea on exertion, palpitations, (orthostatic) hypotension, near syncope and restless legs\n  * Thrombocytopenia defined as platelets \\\u003C20(SqrRoot) 10\\^9\u002FL or platelets \\\u003C50(SqrRoot) 10\\^9\u002FL and a history of clinically relevant non-major or major bleeding according to the ISTH classification\n* Participants 12-17 years of age with MDS must be relapsed\u002Frefractory OR ineligible to receive immunosuppressive therapy and hematopoietic stem cell transplantation\n\n  --Ineligibility to receive hematopoietic stem cell transplantation will include participants who are not anticipated to be candidates to receive transplantation within the next 3 months due to medical comorbidities, lack of appropriate donor, or logistical barriers to transplant\n* Participants with MDS\u002FMPN must be relapsed\u002Frefractory (failed a minimum of 1 standard of care therapy) OR ineligible to receive standard of care OR without known life-prolonging therapy options OR have a diagnosis for which no known standard of care exists\n* Participants 12-17 years of age must weigh \\>= 35 kg\n* If any of the prior therapies noted below were given to the participant, they must have been completed within the following timeframes:\n\n  * 7 days from last dose of short-acting myeloid growth factors (i.e., filgrastim) and \\>= 14 days for long-acting (i.e., pegfilgrastim)\n  * 14 days from last dose of short-acting thrombopoietic growth factors (i.e.,eltrombopag) and \\>= 28 days for long-acting (i.e., romiplostim)\n  * 14 days or 5 pharmacokinetic half-lives from biological therapy agent\n  * 21 days from myelosuppressive chemotherapy\n  * 28 days from last dose of immunosuppressive therapy (e.g., ATG, cyclosporine, steroids greater than physiologic replacement)\n  * 28 days from last dose of lenalidomide\n  * 28 days from last dose of venetoclax\n  * 28 days from any other investigational agent\n  * 42 days from last dose of erythropoiesis stimulating agents\n  * 56 days from last dose of luspatercept\n  * 100 days from stem cell transplant with no evidence of active graft vs. host disease in participants who relapsed following transplant\n  * 150 days from total body irradiation\n* Performance status:\n\n  * For participants \\>= 16 years of age, ECOG performance status \\\u003C 2 (Karnofsky \\>= 60%)\n  * For participants \\\u003C 16 years of age, Lansky \\>= 60%\n* Participants must have adequate organ function as defined below:\n\n  * Total bilirubin: \\\u003C= 1.5 X institutional upper limit of normal OR \\\u003C= 3 x institutional upper limit of normal in participants with Gilbert s syndrome\n  * AST(SGOT)\u002FALT(SGPT): \\\u003C= 2.5 X institutional upper limit of normal\n  * Creatinine clearance: \\>= 50 mL\u002Fmin\u002F1.73 m\\^2 for participants with creatinine levels above institutional normal\n  * PT and PTT: \\\u003C= 1.5 X institutional upper limit of normal, except in the setting of PTT elevation due to lupus anticoagulant, in which case these participants would be exempt from meeting this inclusion criterion\n* Individuals of child-bearing potential (IOCBP) and individuals able to father a child with a partner able to become pregnant must agree to use one (1) highly effective form of contraception (e.g., intrauterine device \\[IUD\\], surgical) or two (2) effective forms of contraception (e.g., barrier method) while on study drug and for 30 days after the last dose of study drug\n* Nursing participants must discontinue breastfeeding and\u002For not begin breastfeeding until 2 weeks after the last dose of study drug\n* Ability of participant or parent\u002Fguardian (for participants 12-17 only) to understand and the willingness to sign a written informed consent document\n\nEXCLUSION CRITERIA:\n\n* Participants with platelet transfusion-refractory thrombocytopenia, with inability to keep platelet threshold above 10 K\u002FmcL with transfusions\n* Participants with evidence of ongoing hemorrhage, active signs\u002Fsymptoms of bleeding, or history of severe (grade \\>= 3) unprovoked bleeding complications in the one year prior to enrollment, or any unprovoked grade 2 bleeding complications in the 3 months prior\n\nto enrollment\n\n* Use of anti-platelet or anticoagulant medication other than low-dose aspirin (100 mg daily or less) in the 14 days prior to enrollment, or any ongoing requirement for these medications\n* Participants who are unwilling to accept blood transfusions\n* Participants with ANC \\\u003C 500 cells\u002FmcL AND hospitalization for a fungal infection in the 12 months prior to enrollment\n* History of allergic reactions attributed to compounds of similar chemical or biologic composition to pacritinib\n* Concomitant administration with sensitive substrates\u002Fnarrow therapeutic index drugs of CYP3A4, CYP1A2, P-gp BCRP, and OCT1 should be avoided. Concurrent use of strong inhibitors and inducers of CYP3A4 are not allowed. Prior use is allowed as long as medication is stopped two weeks prior to study drug initiation. Because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated medical reference. As part of the enrollment\u002Finformed consent procedures, the participant will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the participant is considering a new over-the-counter medicine or herbal product.\n* Concomitant administration of medications with significant potential to cause QTc prolongation.\n* Participants with the following cardiac conditions at screening:\n\n  * Symptomatic congestive heart failure\n  * Unstable angina pectoris\n  * Uncontrolled cardiac dysrhythmias\n  * QTc(F) prolongation \\>450 ms, or other factors that increase the risk for QT prolongation (i.e., heart failure, or a history of long QT interval syndrome)\n* Grade \\>= 3 cardiac complication in the 6 months prior to enrollment\n* Left ventricular ejection fraction \\\u003C= 50% by transthoracic echocardiogram (TTE) at screening\n* Participants with any active, uncontrolled viral, bacterial, or fungal infection, including active HIV-1, Hepatitis B (HBV) and\u002For Hepatitis C (HCV) infection (positive HBV or HCV viral load in the setting of positive HBV core antibody or surface antibody or HCV antibody); history of HIV, HBV, or HCV is allowed if there is no uncontrolled viral infection\n* Pregnancy\n* Presence of another known cause of cytopenia or dysplastic marrow that is untreated and may limit interpretation of results\n* Uncontrolled intercurrent illness or any significant disease, evaluated by history, physical exam and chemistries or social situations that may limit interpretation of results, limit compliance with study requirements, or that could increase risk to the participant","12 Years","120 Years",{"count":57,"type":21},160,[59,60],"PHASE1","PHASE2","Background:\n\nMyelodysplastic syndrome (MDS) and myelodysplastic\u002Fmyeloproliferative neoplasm (MDS\u002FMPN) are blood disorders that can cause serious complications in children and adults. MDS and MDS\u002FMPN can also progress to acute myeloid leukemia. Treatments for these disorders are risky and not always effective. Better treatments are needed.\n\nObjective:\n\nTo test a study drug (pacritinib) in adults and children with MDS or MDS\u002FMPN.\n\nEligibility:\n\nChildren (aged 12 to 17 years) and adults (aged 18 years and older) with MDS or MDS\u002FMPN.\n\nDesign:\n\nParticipants will be screened. They will have a physical exam with blood tests. They will have tests of their heart function. They may have a bone marrow biopsy: An area over the hip will be numbed; a needle will be inserted to remove a sample of soft tissue from inside the hipbone.\n\nPacritinib is a capsule taken by mouth. All participants will take the study drug 2 times a day, every day, in 28-day cycles. They will write down the date and time they take each capsule. Doctors will assign varying dosages of the drug to different participants.\n\nParticipants will have clinic visits each week during cycle 1; every 2 weeks during cycle 2; and gradually increasing to every 3 months after cycle 13. Treatment will continue for up to 8 years.\n\nBone marrow biopsies, heart tests, and other tests will be repeated at intervals throughout the study. Participants will also fill out questionnaires about their quality of life, the symptoms of their disease, and other topics.",[27],[64,65,66,67,68,69,70,71,72],"Myelodysplastic Syndrome","bone marrow disorder","Myelodysplasia","myeloproliferative disorders","pediatric bone marrow failure","5q deletion","multi-lineage dysplasia","Clonal Hematopoiesis","Aplastic Anemia",{"date":37,"type":38},{"date":75,"type":21},"2026-08-26",{"date":77,"type":21},"2035-01-01",{"name":79,"class":80},"National Cancer Institute (NCI)","NIH",1,{"id":83,"slug":84,"hasResults":12,"nctId":85,"briefTitle":86,"officialTitle":87,"acronym":4,"eligibilityCriteria":88,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":89,"targetDuration":4,"studyType":22,"phases":90,"briefSummary":91,"conditions":92,"keywords":94,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":98,"startDateStruct":99,"completionDateStruct":101,"leadSponsor":103,"locationsCount":104},"100394713","phase-2-a-study-of-elritercept-to-treat-anemia-in-adults-with-very-low-low-or-intermediate-risk-myelodysplastic-syndromes-mds-100394713","NCT04419649","A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)","A Phase 2, Open-Label, Ascending Dose Study of KER-050 for the Treatment of Anemia in Patients With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)","Inclusion Criteria:\n\n1. Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local study participant privacy regulations.\n2. Male or female ≥ 18 years of age, at the time of signing informed consent.\n3. Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related to anemia).\n4. Females of childbearing potential and sexually active males must agree to use highly effective methods of contraception.\n5. In the opinion of the Investigator, the participant is able and willing to comply with the requirements of the protocol (e.g., all study procedures, return for follow-up visits).\n\nPart 1 Inclusion Criteria\n\nParticipants are eligible to be included in Part 1 of the study only if all the following criteria apply:\n\n1. Diagnosis of MDS according to WHO classification that meets International Prognostic Scoring System-Revised (IPSS-R) classification of very low, low, or intermediate risk disease.\n2. Less than (\\\u003C)5percent (%) blasts in bone marrow during the Pretreatment Period.\n3. Peripheral blood white blood cell (WBC) count \\\u003C13,000\u002Fmicroliter (μL) during the Pretreatment Period.\n4. Anemia defined as:\n\n   1. In non-transfused participants, having received no RBC transfusions within 8 weeks, Hgb concentration ≤ 10.0 g\u002FdL during the Pretreatment Period OR\n   2. In LTB participants, having received 1 to 3 units of RBCs for Hgb ≤ 9.0 g\u002FdL within 8 weeks of the Pretreatment Period.\n\n      OR\n   3. In HTB participants, having received ≥ 4 units of RBCs for Hgb ≤ 9.0 g\u002FdL within 8 weeks of the Pretreatment Period.\n\nPart 1 Extension - Abbreviated Inclusion Criteria\n\nParticipants from Part 1 are eligible to be included in Part 1 Extension of the study only if all the following criteria apply:\n\n1. Previously completed 4 cycles of elritercept in Part 1 with no dose-limiting toxicities (DLTs).\n2. Participant has the potential to benefit from administration of elritercept, in the opinion of the Investigator.\n3. \\\u003C 5% blasts in bone marrow.\n4. Peripheral WBC count \\\u003C 13,000\u002FμL during the 28 days prior to cycle 5 day 1 (C5D1).\n\nPart 2 Inclusion Criteria\n\nParticipants are eligible to be included in Part 2 of the study only if all the following criteria apply:\n\n1. Cohort A:\n\n   * Diagnosis of MDS according to WHO classification that meets IPSS-R classification of very low, low, or intermediate risk disease.\n   * ring sideroblast (RS)-positive as defined by WHO 2016 criteria.\n   * Requiring at least 2 units of RBC transfusions in the preceding 8 weeks before cycle 1 day 1 (C1D1).\n2. Cohort B:\n\n   * Diagnosis of MDS according to WHO classification that meets IPSS-R classification of very low, low, or intermediate risk disease.\n   * Non-RS as defined by WHO 2016 criteria.\n   * Requiring at least 2 units of RBC transfusions in the 8 weeks before C1D1.\n3. Cohort C:\n\n   * Diagnosis of MDS according to WHO classification that meets IPSS-R classification of very low, low, or intermediate risk disease.\n   * Has anemia, defined by Hgb ≤ 10 g\u002FdL during the Pretreatment Period, and received no RBC transfusion in the 8 weeks before C1D1.\n4. Cohort D:\n\n   * Diagnosis of CMML according to WHO classification.\n   * Has anemia, defined by Hgb ≤ 10 g\u002FdL during the Pretreatment Period, and received no RBC transfusion in the 8 weeks before C1D1.\n   * OR\n   * Received at least 2 units of RBC transfusions for anemia in the 8 weeks before C1D1.\n5. Cohort E:\n\n   * Diagnosis of MDS according to WHO classification that meets IPSS-R classification of very low, low, or intermediate risk disease.\n   * Requiring ≥ 2 units of RBC transfusions in the preceding 8 weeks before C1D1.\n   * Receipt of ≥ 20 units of RBC in transfusion over the participant's lifetime.\n   * Serum ferritin \\> 1000 nanograms per milliliter (ng\u002FmL) on ≥ 2 assessments in the preceding 8 weeks before C1D1.\n   * Treated with stable dose of iron chelation therapy for ≥ 8 weeks prior to C1D1.\n6. Cohort F:\n\n   * Diagnosis of MDS according to WHO classification that meets IPSS-R classification of very low, low, or intermediate risk disease.\n   * Requiring ≥ 2 units of RBC transfusions in the preceding 8 weeks before C1D1.\n   * Receipt of ≥ 20 units of RBC in transfusion over the participant's lifetime.\n   * Serum ferritin \\> 1000 ng\u002FmL on ≥ 2 assessments in the preceding 8 weeks before C1D1.\n   * Not treated with iron chelation therapy in the preceding 8 weeks before C1D1 and not eligible to initiate iron chelation therapy in the opinion of the Investigator and in accordance with local treatment guidelines for initiation of iron chelation therapy.\n7. Cohort G:\n\n   * Diagnosis of MDS according to WHO classification that meets IPSS-R classification of very low, low, or intermediate risk disease.\n   * RS-positive as defined by WHO 2016 criteria OR non-RS as defined by WHO 2016 criteria.\n   * Relapsed, refractory, or intolerant to frontline luspatercept treatment and have not received an interceding therapy (for example, erythropoiesis-stimulating agent \\[ESA\\])\n\n     * Relapsed is defined as documentation of response to luspatercept therapy and subsequent development of a need for transfusion(s).\n     * Refractory is defined as documentation of no response with luspatercept ≥ 1 mg\u002Fkg administered for ≥ 12 weeks duration.\n     * Intolerant is defined as documentation of discontinuation of luspatercept therapy due to intolerance or an AE at any time after introduction.\n   * Requiring ≥ 2 units of RBC transfusions over 8 weeks prior to C1D1.\n   * Erythropoietin (EPO) \\\u003C 500 international units per liter (U\u002FL) at Baseline.\n   * Last dose of luspatercept is ≥ 3 weeks and \\\u003C 12 months from C1D1.\n8. \\\u003C 5% blasts in bone marrow assessed by bone marrow aspirate during the Pretreatment Period.\n\nPart 1 Exclusion Criteria\n\nParticipants are excluded from Part 1 of the study if any of the following criteria apply.\n\nMedical History\n\n1. Diagnosis of MDS with deletion of chromosome 5q (Del5q).\n2. Active infection requiring parenteral antibiotic therapy within 28 days prior to C1D1 or oral antibiotics within 14 days of C1D1. Prophylactic antibiotics and\u002For antifungals for neutropenia are allowed.\n3. Presence of uncontrolled heart disease or New York Heart Association (NYHA) Class III or IV heart failure.\n4. History of drug or alcohol abuse (as defined by the Investigator) within the past 2 years.\n5. History of stroke, deep venous thrombosis (DVT), or arterial embolism within 6 months prior to C1D1.\n6. Major surgery within 28 days prior to C1D1. Participants must be completely recovered from any previous surgery prior to C1D1.\n7. Known positive for human immunodeficiency virus (HIV), active infectious hepatitis B virus (HBV), or active infectious hepatitis C virus (HCV). Participants without known positive history of HIV, HBV, and\u002For HCV do not require further testing, unless testing is mandated per local guidelines.\n8. Any malignancy other than MDS that has not been in remission and\u002For has required systemic therapy including radiation, chemotherapy, hormonal therapy, or surgery, within 1 year prior to C1D1. Diagnosis of secondary MDS (i.e., MDS known to have arisen as the result of chemical injury or treatment with chemotherapy and\u002For radiation for other diseases).\n9. History of solid organ or hematological transplantation.\n10. Presence of uncontrolled hypertension, defined as systolic blood pressure (BP) ≥ 150 mmHg or diastolic BP ≥ 100 mmHg despite adequate treatment.\n11. Body mass index (BMI) ≥ 40 kilograms per meter square (kg\u002Fm\\^2) during the Pretreatment Period.\n12. History of severe allergic or anaphylactic reaction(s) or hypersensitivity to recombinant proteins or excipients in the investigational medicinal product (IMP).\n\nTreatment History\n\n1. Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.\n2. Treatment with ESA within 56 days prior to C1D1.\n3. Prior or concurrent chronic treatment with granulocyte colony-stimulating factor (G-CSF) or granulocyte-macrophage colony-stimulating factor (GM-CSF).\n4. Iron chelation therapy if initiated within 8 weeks prior to C1D1.\n5. Vitamin B12 therapy initiated within 8 weeks prior to C1D1. Participants on stable replacement doses for ≥ 8 weeks and without concurrent vitamin B12 or folate deficiency are allowed.\n6. Treatment with another investigational drug or device or approved therapy for investigational use ≤ 28 days prior to C1D1, or, if the half-life of the previous product is known, within 5 times the half-life prior to C1D1, whichever is longer.\n\nLaboratory Exclusions (during Pretreatment Period)\n\n1. Platelet count \\> 450 ✕ 10\\^9\u002FL or \\\u003C 30 ✕ 10\\^9\u002FL.\n2. Transferrin saturation \\\u003C 15%.\n3. Ferritin \\\u003C 50 nanograms per milliliter (ng\u002FmL).\n4. Folate \\\u003C 4.5 nanomoles per liter (nmol\u002FL) (\\\u003C 2.0 ng\u002FmL).\n5. Vitamin B12 \\\u003C 148 picomoles per liter (pmol\u002FL) (\\\u003C 200 picograms per milliliter \\[pg\u002FmL\\]).\n6. Estimated glomerular filtration rate (GFR) \\\u003C 30 milliliter per minute per 1.73 meter square (mL\u002Fmin\u002F1.73 m\\^2), as determined by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation.\n7. Positive for HIV.\n\nMiscellaneous\n\n1. Pregnant or lactating females.\n2. Any other condition not specifically noted above which, in the opinion of the Investigator, would preclude the participant from participating in the study.\n3. Participants who are investigational site staff members directly involved in the conduct of the trial and their immediate family members, site staff members otherwise supervised by the Investigator, or participants who are Sponsor or contract research organization (CRO) employees directly involved in the conduct of the study. Immediate family is defined as a spouse, parent, child, or sibling, whether biological or legally adopted.\n\nPart 1 Extension - Exclusion Criteria\n\nParticipants from Part 1 are excluded from Part 1 Extension of the study if any of the following criteria apply.\n\nMedical History\n\n1. Discontinuation of IMP in Part 1 for any reason.\n2. Has not completed a study visit in the past 12 months.\n3. Active infection requiring parenteral antibiotic therapy within 28 days prior to C5D1 or oral antibiotics within 14 days of C5D1. Prophylactic antibiotics and\u002For antifungals for neutropenia are allowed.\n4. Presence of uncontrolled heart disease or NYHA Class III or IV heart failure.\n5. History of drug or alcohol abuse (as defined by the Investigator) within the past 2 years.\n6. History of stroke, DVT, or arterial embolism within 6 months prior to C5D1.\n7. Major surgery within 28 days prior to C5D1. Participants must be completely recovered from any previous surgery prior to C5D1.\n8. Known positive for HIV, active infectious HBV, or active infectious HCV. Participants without known positive history of HIV, HBV, and\u002For HCV do not require further testing, unless testing is mandated per local guidelines.\n9. Any malignancy other than MDS that has not been in remission and\u002For has required systemic therapy including radiation, chemotherapy, hormonal therapy, or surgery, within 1 year prior to C5D1.\n10. History of solid organ or hematological transplantation.\n11. Presence of uncontrolled hypertension, defined as systolic BP ≥ 150 mmHg or diastolic BP ≥ 100 mmHg despite adequate treatment.\n12. BMI ≥ 40 kg\u002Fm\\^2 during the 28 days prior to C5D1.\n\nTreatment History\n\n1. Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.\n2. Treatment with ESA within 56 days prior to C5D1.\n3. Prior or concurrent chronic treatment with G-CSF or GM-CSF.\n4. Iron chelation therapy if initiated within 8 weeks prior to C5D1.\n5. Vitamin B12 with treatment initiated within 8 weeks prior to C5D1. Participants on stable replacement doses for ≥ 8 weeks and without concurrent vitamin B12 or folate deficiency are allowed.\n6. Treatment with another investigational drug or device or approved therapy for investigational use ≤ 28 days prior to C5D1, or, if the half-life of the previous product is known, within 5 times the half-life prior to C5D1, whichever is longer. Previous treatment with elritercept is acceptable.\n\nLaboratory Exclusions (during Abbreviated Pretreatment Period)\n\n1. Platelet count \\> 450 × 10\\^9\u002FL or \\\u003C 30 × 10\\^9\u002FL.\n2. Transferrin saturation \\\u003C 15%.\n3. Ferritin \\\u003C 50 ng\u002FmL.\n4. Folate \\\u003C 4.5 nmol\u002FL (\\\u003C 2.0 ng\u002FmL).\n5. Vitamin B12 \\\u003C 148 pmol\u002FL (\\\u003C 200 pg\u002FmL).\n6. Estimated GFR \\\u003C 30 mL\u002Fmin\u002F1.73 m\\^2, as determined by the CKD-EPI equation.\n\nMiscellaneous\n\n1. Pregnant or lactating females.\n2. Any other condition not specifically noted above which, in the opinion of the Investigator, would preclude the participant from participating in the study.\n3. Participants who are investigational site staff members directly involved in the conduct of the trial and their immediate family members, site staff members otherwise supervised by the Investigator, or participants who are Sponsor or CRO employees directly involved in the conduct of the study. Immediate family is defined as a spouse, parent, child, or sibling, whether biological or legally adopted.\n\nPart 2 Exclusion Criteria\n\nParticipants are excluded from Part 2 of the study if any of the following criteria apply.\n\nMedical History\n\n1. Diagnosis of MDS with Del5q.\n2. Diagnosis of secondary MDS (i.e., MDS known to have arisen as the result of chemical injury or treatment with chemotherapy and\u002For radiation for other diseases).\n3. Active infection requiring parenteral antibiotic therapy within 28 days prior to C1D1 or oral antibiotics within 14 days of C1D1. Prophylactic antibiotics and\u002For antifungals for neutropenia are allowed.\n4. Presence of the following cardiac conditions:\n\n   1. Presence of uncontrolled heart disease or NYHA Class III or IV heart failure.\n   2. QTcF (QT interval corrected by Fridericia's formula) \\> 500 msec on the screening or C1D1 electrocardiogram (ECG; mean of 3 measurements).\n   3. Uncontrolled clinically significant arrhythmia (participants with rate-controlled atrial fibrillation are not excluded).\n   4. Acute myocardial infarction or unstable angina pectoris ≤ 6 months prior to C1D1.\n5. Presence of uncontrolled hypertension, defined as systolic BP ≥ 160 mmHg or diastolic BP ≥ 100 mmHg despite adequate treatment.\n6. History of stroke, DVT, or arterial embolism within 6 months prior to C1D1.\n7. History of drug or alcohol abuse (as defined by the Investigator) within the past 2 years.\n8. Major surgery within 28 days prior to C1D1. Participants must be completely recovered from any previous surgery prior to C1D1.\n9. Any malignancy other than MDS or CMML that has not been in remission and\u002For has required major surgery or systemic therapy including radiation, chemotherapy, targeted therapy, or hormonal therapy within 1 year prior to C1D1.\n10. History of solid organ or hematological transplantation.\n11. Diagnosis of hemolytic anemia, active bleeding, hemoglobinopathies, or congenital disorders as a cause of the participant's anemia.\n12. NCI-CTCAE Grade ≥ 2 bleeding events within the 3 months prior to C1D1.\n13. Receipt of an RBC or platelet transfusion for any reason(s) or combination of reasons other than underlying MDS within the 16 weeks prior to C1D1. If a participant requires a transfusion for an unanticipated reason during the Pretreatment Period, a prolonged screening period may be considered after discussion with the Medical Monitor.\n14. Known positive for HIV, active infectious HBV with positive viral load (HBV DNA), or active infectious HCV with positive viral load (HCV RNA). Participants without known positive history of HIV, HBV, and\u002For HCV do not require further testing, unless testing is mandated per local guidelines.\n15. BMI ≥ 40 kg\u002Fm\\^2.\n16. History of severe allergic or anaphylactic reaction(s) or hypersensitivity to recombinant proteins or excipients in the IMP.\n17. Diagnosis of cirrhosis, non-alcoholic steatohepatitis, alcoholic liver disease, hepatitis, or other liver disease (acute or chronic) meeting Child-Pugh C criteria for hepatic impairment. Participants with elevated liver enzymes are allowed if the liver enzyme elevation is suspected to be due to iron-overload or iron chelation, and other hepatic causes have been ruled out, in the opinion of the Investigator.\n\nTreatment History\n\n1. Prior treatment with azacitidine, decitabine, lenalidomide, or sotatercept.\n2. Prior treatment with luspatercept (Cohorts A, B, C, D, E, and F only).\n3. Treatment with ESA within 8 weeks prior to C1D1.\n4. Prior or concurrent chronic treatment with G-CSF or GM-CSF, for reasons other than for treatment of MDS.\n\n   a. Note: Previous treatment with G-CSF or GM-CSF for MDS, which has been discontinued ≥ 8 weeks prior to C1D1 is allowed.\n5. Iron chelation therapy if initiated within 8 weeks prior to C1D1. Participants on stable doses of iron chelation therapy for ≥ 8 weeks are allowed.\n6. Vitamin B12 and\u002For folate therapy initiated within 8 weeks prior to C1D1. Participants on stable replacement doses for ≥ 8 weeks and without concurrent vitamin B12 or folate deficiency are allowed.\n7. Any need to receive a prohibited medication.\n8. Treatment with another investigational drug or device or approved therapy for investigational use within 8 weeks prior to C1D1, or, if the half-life of the previous product is known, within 5 times the half-life prior to C1D1, whichever is longer.\n\nLaboratory Exclusions (during Pretreatment Period)\n\n1. Peripheral WBC count ≥ 13,000\u002FμL.\n2. Platelet count \\> 450 × 10\\^9\u002FL or \\\u003C 25 × 10\\^9\u002FL.\n3. Transferrin saturation \\\u003C 15%.\n4. Ferritin \\\u003C 50 ng\u002FmL.\n5. Folate \\\u003C 4.5 nmol\u002FL (\\\u003C 2.0 ng\u002FmL).\n6. Vitamin B12 \\\u003C 148 pmol\u002FL (\\\u003C 200 pg\u002FmL).\n7. Estimated GFR \\\u003C 30 mL\u002Fmin\u002F1.73 m\\^2 as determined by the CKD-EPI equation.\n\nMiscellaneous\n\n1. Pregnant or lactating females.\n2. Any other condition not specifically noted above which, in the opinion of the Investigator, would preclude the participant from participating in the study.\n3. Participants who are investigational site staff members directly involved in the conduct of the trial and their immediate family members, site staff members otherwise supervised by the Investigator, or participants who are Sponsor or CRO employees directly involved in the conduct of the study. Immediate family is defined as a spouse, parent, child, or sibling, whether biological or legally adopted.\n\nFor Cohort G ONLY:\n\n1. Any luspatercept related AE Grade ≥ 3 that has not resolved to baseline or Grade ≤ 1.\n2. No history of allergy\u002Fanaphylaxis\u002Fhypersensitivity to luspatercept.\n3. No prior treatment with imetelstat.",{"count":57,"type":21},[60],"The main aim of this study is to learn how safe elritercept is and how well adults with anemia associated with lower-risk MDS tolerate treatment with different doses of elritercept. Other aims are to learn how safe elritercept is by looking at how many participants have MDS that worsens during the study and learn about the effects of elritercept on anemia linked to MDS. The study will also look to learn how elritercept affects the production of healthy RBCs.",[27,93],"Cytopenia",[95,96,30,97,32],"Transfusion","Drug Therapy","TAK-226",{"date":37,"type":38},{"date":100,"type":38},"2020-08-19",{"date":102,"type":21},"2031-10-30",{"name":44,"class":45},53,{"id":106,"slug":107,"hasResults":12,"nctId":108,"briefTitle":109,"officialTitle":109,"acronym":4,"eligibilityCriteria":110,"healthyVolunteers":111,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":112,"targetDuration":4,"studyType":22,"phases":114,"briefSummary":115,"conditions":116,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":119,"lastUpdatePostDateStruct":120,"startDateStruct":122,"completionDateStruct":124,"leadSponsor":126,"locationsCount":129},"100559639","phase-2-a-phase-2-study-evaluating-olutasidenib-in-patients-with-idh1-mutated-clonal-cytopenia-of-undetermined-significance-and-lower-risk-myelodysplasticsyndromeschronic-myelomonocytic-leukemia-100559639","NCT06566742","A Phase 2 Study Evaluating Olutasidenib in Patients With IDH1-mutated Clonal Cytopenia of Undetermined Significance and Lower-risk Myelodysplastic\u002FSyndromes\u002FChronic Myelomonocytic Leukemia.","Inclusion Criteria:\n\nTo be considered eligible to participate in this study, a patient must meet ALL inclusion criteria as follows:\n\n1. Pathologically proven CCUS or lower-risk MDS\u002FCMML.\n\n   1. CCUS is defined as the presence of cytopenia (absolute neutrophil count \\\u003C 1.8 x 10\\^9\u002FL, hemoglobin \\\u003C 13 g\u002FdL in males or \\\u003C 12 g\u002FdL in females, and\u002For platelets \\\u003C 150 x 10\\^9\u002FL) for at least 30 days that are otherwise unexplained and with no diagnostic hematopathologic features of myeloid neoplasms. Patients with known Duffy-null phenotype must have absolute neutrophil counts less than their lower limit of normal.\n   2. Lower-risk MDS\u002FCMML includes patients with International Prognostic Scoring System (IPSS) low- or intermediate-1-risk disease and Revised IPSS (IPSS-R) score ≤ 3.5 and Molecular IPSS (IPSS-M) very low-, low-, or moderate low-risk categories.\n2. Patients must have a documented IDH1 mutation with variant allele frequency (VAF) ≥ 0.02.\n3. Patients ≥ 18 years old.\n4. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 (Appendix A)\n5. Acceptable liver function\n\n   1. Bilirubin ≤ 2 times upper limit of normal (ULN) or ≤ 3 times ULN in patients with Gilbert Syndrome.\n   2. Aspartate transaminase (AST), alanine transaminase (ALT), and alkaline phosphatase ≤ 3 times ULN.\n6. Acceptable renal function with serum creatinine ≤ 1.5 times ULN or calculated creatinine clearance ≥ 50 mL\u002Fmin (as assessed by Cockcroft-Gault, Modification of Diet in Renal Disease Formula \\[MDRD\\], or Chronic Kidney Disease Epidemiology \\[CKD-Epi\\] validated measures).\n7. Negative serum or urine pregnancy test if female of childbearing potential.\n8. For fertile men and women, agreement to use highly effective contraceptive methods for the duration of study participation and 90 days after the last dose of study medication. Appropriate highly effective method(s) of contraception include oral or injectable hormonal birth control, intrauterine device (IUD), and double barrier methods (for example a condom in combination with a spermicide).\n9. Agreement for male patients not to donate sperm and for female patients of childbearing potential not to donate ova during the study and for 90 days after the final dose of study drug.\n10. Ability and willingness to signed informed consent prior to beginning study and undergoing procedures.\n\nExclusion Criteria:\n\nTo be eligible for entry into the study, the patient must NOT meet any of the exclusion criteria listed below:\n\n1. Patients unable to swallow oral medications, or patients with gastrointestinal conditions (e.g., malabsorption, resection, etc.) deemed by the Investigator to jeopardize intestinal absorption.\n2. Patients with any concurrent uncontrolled clinically significant medical condition, including life-threatening severe infection or psychiatric illness, which could place the patient at unacceptable risk of study treatment.\n3. Known active hepatitis B (hepatitis B virus \\[HBV\\]) or hepatitis C (hepatitis C virus \\[HCV\\]) or HIV infection.\n4. Pregnant or nursing women or women of childbearing potential not using highly effective contraception; male patients not using highly effective contraception as defined in the inclusion criteria.\n5. Subject with white blood cell count \\> 25 x10\\^9\u002FL.\n\n   * Note: hydroxyurea use is permitted to meet this criterion with no washout required.\n6. Unwillingness or inability to comply with procedures either required in this protocol or considered standard of care.",true,{"count":113,"type":21},15,[60],"To learn if olutasidenib can help to control CCUS, MDS, and\u002For CMML. The safety of the drug will also be studied.",[27,117,118],"Chronic Myelomonocytic Leukemia","Clonal Cytopenia of Undetermined Significance","2026-08-17",{"date":121,"type":38},"2026-08-19",{"date":123,"type":38},"2024-12-10",{"date":125,"type":21},"2029-08-31",{"name":127,"class":128},"M.D. Anderson Cancer Center","OTHER",2,{"id":131,"slug":132,"hasResults":12,"nctId":133,"briefTitle":134,"officialTitle":135,"acronym":4,"eligibilityCriteria":136,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":137,"enrollmentInfo":138,"targetDuration":4,"studyType":22,"phases":140,"briefSummary":141,"conditions":142,"keywords":149,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":152,"lastUpdatePostDateStruct":153,"startDateStruct":154,"completionDateStruct":156,"leadSponsor":158,"locationsCount":81},"100616220","phase-1-tacrolimus-targeted-immunosuppression-cessation-in-allogeneic-hct-100616220","NCT07302776","TACrolimus Targeted Immunosuppression Cessation in ALlogeneic HCT","TACTICAL: TACrolimus Targeted Immunosuppression Cessation in ALlogeneic HCT","Inclusion Criteria:\n\n* Eligible diseases:\n\n  * Acute myeloid leukemia (AML) in complete remission (CR), CR with incomplete hematologic recovery (CRi), or MLFS.\n  * Myelodysplasic syndrome (MDS) myelodysplastic syndromes eligible for alloHSCT based on IPSS-M of intermediate or higher, or IPSS-R of intermediate or higher, or refractory disease to standard growth factor or hypomethylating agent-based therapy\n  * Myelofibrosis (MF)\n  * Chronic myeloid leukemia (CML) in chronic phase with a prior history of accelerated phase or blast crisis or CML in chronic phase refractory to standard TKI therapy\n  * Chronic myelomonocytic leukemia (CMML)\n* Age ≥ 18 and ≤ 80 years at the time of enrollment.\n* Planned for first myeloablative or reduced intensity allogenic transplant using a conditioning regimen listed in Appendix B.\n* Has a related or unrelated donor available who is 8\u002F8 HLA match at HLA-A, -B, -C, and -DRB1, all typed using DNA-based high-resolution methods.\n* Estimated glomerular filtration rate (eGFR) ≥ 50 mL\u002Fminute or creatinine \\\u003C 2 mg\u002FdL.\n\nCardiac ejection fraction at rest ≥ 45% or shortening fraction of ≥ 27% by echocardiogram or radionuclide scan (MUGA).\n\n* Diffusing capacity of the lung for carbon monoxide (DLCO) (adjusted for hemoglobin) ≥ 50%.\n* Total bilirubin \\\u003C 2 times upper limit of normal (ULN) (patients with Gilbert's syndrome may be included once hemolysis has been excluded).\n* Karnofsky Performance Score ≥70%\n* Negative serum or urine beta-HCG test in females of childbearing potential (FCBP) within 3 weeks of enrollment.\n\nA female of childbearing potential (FCBP) is a female who: 1) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months).\n\n-Ability to understand and the willingness to provide written informed consent.\n\nExclusion Criteria:\n\n* Prior allogeneic HCT.\n* Planned donor lymphocyte infusion (DLI).\n* Recipient positive anti-donor HLA antibodies against a mismatched allele in the selected donor determined by either:\n\n  1. Positive crossmatch test of any titer (by complement-dependent cytotoxicity or flow cytometric testing), or\n  2. Presence of anti-donor HLA antibody to any of the following HLA loci: HLA-A, -B, -C, -DRB1, -DQB1, -DQA1, -DPB1, or -DPA1, with mean fluorescence intensity (MFI) \\>1000 by solid phase immunoassay.\n* Uncontrolled bacterial, viral, or fungal infections at time of enrollment including known, active tuberculosis infection.\n* Seropositive for HIV-1 or -2, HTLV-1 or -2, Hepatitis B sAg, and\u002For Hepatitis C antibody.\n\n  \\*History of hepatitis B or hepatitis C is permitted if viral load is undetectable per quantitative PCR and\u002For NAT.\n\nKnown allergy or hypersensitivity to planned GVHD prophylactic medications including PTCy, tacrolimus\n\n* Any uncontrolled autoimmune disease requiring active immunosuppressive treatment.\n* Concurrent malignancy diagnosed within 12 months of enrollment, except non-melanoma skin cancers or other early-stage solid tumors that have been curatively resected or treated to curative intent. Patients with history of low grade concurrent blood cancers that are controlled will be eligible.\n* Females of childbearing potential (FCBP) or men who have sexual contact with FCBP unwilling to use effective forms of birth control or abstinence for one year after transplantation.\n\n(FCBP definition: A female of childbearing potential (FCBP) is a female who: 1) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months).\n\n-Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the recipient's safe participation in and completion of the study, or which could affect compliance with the protocol or interpretation of results.\n\n\\* All subject files must include supporting documentation to confirm subject eligibility.","80 Years",{"count":139,"type":21},50,[59],"The purpose of this study is to test the feasibility and safety of early cessation of tacrolimus following allogeneic hematopoietic cell transplantation (HCT). Post-HCT tacrolimus is given to prevent graft-vs-host-disease (GVHD), but with the use of post-transplant cyclophosphamide (PTCy), the modern approach to GVHD prevention, GVHD rates have reduced markedly.",[143,144,145,27,146,147,148],"GVHD","Hematopoietic Cell Transplantation (HCT)","Acute Myeloid Leukemia (AML)","Myelofibrosis (MF)","Chronic Myeloid Leukemia (CML)","Chronic Myelomonocytic Leukemia (CMML)",[150,151],"Post-Hematopoietic Cell Transplant (HCT) tacrolimus","hematopoietic cell transplantation (HCT)","2026-08-13",{"date":119,"type":38},{"date":155,"type":38},"2026-07-21",{"date":157,"type":21},"2028-02",{"name":159,"class":128},"Stanford University",{"id":161,"slug":162,"hasResults":12,"nctId":163,"briefTitle":164,"officialTitle":164,"acronym":4,"eligibilityCriteria":165,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":166,"targetDuration":4,"studyType":22,"phases":168,"briefSummary":170,"conditions":171,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":176,"lastUpdatePostDateStruct":177,"startDateStruct":178,"completionDateStruct":180,"leadSponsor":182,"locationsCount":81},"100629049","ehealth-mindfulness-based-music-therapy-intervention-for-patients-undergoing-stem-cell-transplantation-100629049","NCT07469592","eHealth Mindfulness-based Music Therapy Intervention for Patients Undergoing Stem Cell Transplantation","Inclusion Criteria:\n\n* ≥ 18 years of age\n* have a primary diagnosis of a hematologic malignancy (e.g., myelodysplastic syndrome \\[MDS\\], acute myeloid leukemia \\[AML\\], acute lymphoblastic leukemia \\[ALL\\], or non Hodgkin's Lymphoma \\[NHL\\])\n* have a treatment plan for a hematopoietic stem cell transplant\n* Speak English or Spanish\n\nExclusion Criteria:\n\n* history of severe psychiatric illness (e.g., psychosis, active suicidality, inpatient treatment in the past 12 months)\n* severe cognitive impairment (per the short portable mental status questionnaire)\n* hearing impairment\n* active alcohol or substance dependence within the past six months\n* participated in the prior pilot MBMT R61 phase\n* participated in music therapy or mindfulness programs in the past six months",{"count":167,"type":21},165,[169],"NA","The goal of this study is to test an electronic health (eHealth) mindfulness-based music therapy intervention to improve health-related quality of life and reduce symptom burden and disease activity in patients undergoing stem cell transplantation.",[172,27,173,174,175],"Stem Cell Transplantation","Leukemia, Myeloid, Acute","Precursor Cell Lymphoblastic Leukemia-Lymphoma","Lymphoma, Non-Hodgkin","2026-08-11",{"date":152,"type":38},{"date":179,"type":38},"2026-07-22",{"date":181,"type":21},"2028-06-30",{"name":183,"class":128},"University of Miami",{"id":185,"slug":186,"hasResults":12,"nctId":187,"briefTitle":188,"officialTitle":189,"acronym":190,"eligibilityCriteria":191,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":192,"targetDuration":4,"studyType":22,"phases":194,"briefSummary":195,"conditions":196,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":176,"lastUpdatePostDateStruct":200,"startDateStruct":202,"completionDateStruct":204,"leadSponsor":206,"locationsCount":208},"100473396","compassionate-communication-and-advance-care-planning-to-improve-end-of-life-care-in-treatment-of-hematological-disease-act-100473396","NCT05444348","Compassionate Communication and Advance Care Planning to Improve End of Life Care in Treatment of Hematological Disease (ACT)","Compassionate Communication and Advance Care Planning to Improve End of Life Care in Treatment of Hematological Disease (ACT)- a Cluster Randomized Controlled Study","ACT","Inclusion Criteria:\n\nPatients must:\n\n* Be at least 18 years of age\n* Have a diagnosis of one of the following:\n* High-risk myelodysplastic syndrome (MDS) or MDS with overlap of myeloproliferative neoplasms (high-risk MDS\u002FMPN),\n* Acute myeloid leukemia(AML): Age≥80 or in palliative treatment or relapse\n* Lymphoma: Age≥80 or relapse or refractory or palliative treatment\n* Multiple myeloma(MM): Age≥80 or relapsed or refractory\n\nHave limited treatment options. Provide informed consent. Have sufficient Danish skills to complete intervention sessions and data collection\n\nAn informal caregiver is identified by the patient as the primary provider of informal physical, practical or emotional support and must:\n\n* Be at least 18 years of age\n* Be able to accompany patients to intervention appointments\n* Provide informed consent\n* Have sufficient Danish skills to complete intervention sessions and data collection\n\nPhysicians:\n\n* specialized in hematology\n* treating patients with High risk myelodysplastic syndrome, acute myeloid leukemia, lymphoma, or multiple myeloma\n* work at the same department for the entire time of intervention.\n\nNurses:\n\n* treating patients with High-risk myelodysplastic syndrome, acute myeloid leukemia, lymphoma, or multiple myeloma\n* work at the same department for the entire time of intervention.\n\nExclusion Criteria:\n\nPatient and caregiver are excluded if one of them is:\n\n\\- Suffering from a severe psychiatric disorder\n\nPhysicians and nurses:\n\n\\- If they do not meet the inclusion criterion.",{"count":193,"type":21},920,[169],"Patients diagnosed with hematologic cancer are at substantial risk of dying, as 5-year survival among patients with acute myeloid leukemia is 20 % and only every second patient treated for incurable myeloma lives 5 years after date of diagnosis. Nevertheless, many overestimate their prognosis, and value of therapy. Patients with hematological cancers frequently have poor end of life outcomes, such as high treatment activity close to death, where clinical effects are doubtful, and low utilization of palliative care. Prognostic awareness and end of life (EOL) issues have urgency in the communication between patients, their caregiving relatives, and clinicians, in order to avoid futile treatments and suffering at EOL. Inspired by advanced care planning, the investigators developed the concept \"Advance Consultations Concerning participants Life and Treatment\" (ACT) in collaboration with a group consisting of hematologists, nurses, patients, and caregivers. The ACT concept consists of an 8-hour training day for clinicians, clinical tools, system changes, and preparation material for patients and caregivers prior to the consultation. ACT involves patients and caregivers earlier in preparation for life with chronic progressive disease and EOL-decisions, through an intervention based on compassionate communication and early planning of EOL-care. The aim of the study is to investigate the effect of the intervention on use of chemotherapy and quality of EOL-care in patients with hematological malignancy. Based on the results of the completed pilot study, the investigators are planning a nationwide 2-arm cluster randomized controlled trial where 40 physicians and 80 nurses across seven different hematological departments are randomized to either usual care or ACT training and completing ACT conversations. The investigators expect to include a total of 400 patients and their family caregivers. It is hypothesized that the ACT intervention will decrease use of futile chemotherapy, prepare patients and caregivers for difficult end-of-life-decisions, and improve quality of end-of-life care in hematology.",[197,27,198,199],"Multiple Myeloma","Lymphoma","Acute Myeloid Leukemia",{"date":201,"type":38},"2026-08-12",{"date":203,"type":38},"2022-08-01",{"date":205,"type":21},"2028-07-01",{"name":207,"class":128},"Christoffer Johansen",7,{"id":210,"slug":211,"hasResults":12,"nctId":212,"briefTitle":213,"officialTitle":214,"acronym":4,"eligibilityCriteria":215,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":216,"targetDuration":4,"studyType":22,"phases":218,"briefSummary":219,"conditions":220,"keywords":226,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":229,"lastUpdatePostDateStruct":230,"startDateStruct":231,"completionDateStruct":233,"leadSponsor":235,"locationsCount":237},"100463861","phase-1-study-of-disc-0974-rally-mf-in-participants-with-myelofibrosis-or-myelodysplastic-syndrome-and-anemia-100463861","NCT05320198","Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia","RALLY-MF: A Phase 1b\u002F2 Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Clinical Activity of DISC-0974 in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia","Inclusion Criteria for Participants with MF and Anemia:\n\nParticipants are eligible for the study if all of the following criteria apply:\n\n1. Age 18 years or older at the time of signing the informed consent form (ICF).\n2. For Phase 1b: Dynamic International Prognostic Scoring System (DIPSS) score of 3 to 4 (intermediate 2 risk) or ≥5 (high-risk) primary MF, post PV MF, and\u002For post ET MF, as confirmed in the most recent local bone marrow biopsy report, according to World Health Organization (WHO) 2016 criteria.\n\n   For Phase 2: In addition to the criteria above, DIPSS score of ≥2 (intermediate 1 risk) may also be included.\n3. Washout of at least 28 days prior to Screening of the following treatments:\n\n   1. Androgens\n   2. EPO\n   3. Cladribine\n   4. Immunomodulators (lenalidomide, thalidomide)\n   5. Luspatercept\u002Fsotatercept\n   6. Systemic corticosteroids are permitted for non-hematological conditions if stable or decreasing dose for ≥28 days prior to Screening and receiving an equivalent to ≤10 mg prednisone for the 28 days immediately prior to Screening.\n\n   Screening can begin before the 28 day washout is completed, but the washout period must be completed prior to collection of Screening blood samples.\n4. Anemia:\n\n   For Phase 1b: Hgb \\\u003C10 g\u002FdL on ≥3 assessments over 84 days prior to Screening, without RBC transfusion, or Hgb \\\u003C10 g\u002FdL and receiving RBC transfusions periodically but not meeting criteria for TD participant as defined for the TD cohort. The baseline Hgb value for these participants is the lowest Hgb level during the 84 days prior to Screening, or RBC transfusion dependence, defined as an RBC transfusion frequency of ≥6 units PRBC over the 84 days immediately prior to Screening. There must not be any consecutive 42-day period without an RBC transfusion in the 84-day period, and the last transfusion must be within 28 days prior to Screening.\n\n   For Phase 2:\n\n   TD high transfusion burden cohort: RBC transfusion dependence, defined as an RBC transfusion requirement of 3 to 12 PRBC units over the 84 days immediately prior to Screening TD low transfusion burden cohort: RBC transfusion dependence, defined as an RBC transfusion requirement of 1 to 2 PRBC units over the 84 days immediately prior to Screening nTD Cohort: Non-transfusion dependence, baseline Hgb \\\u003C10 g\u002FdL as defined on ≥3 assessments over 84 days prior to Screening, without RBC transfusion\n5. Stable dosing of MF-directed therapy:\n\n   1. Hydroxyurea, or, if taking any other treatment for MF, stable for at least 28 days prior to Screening.\n   2. Interferon alpha stable dosing for at least 12 weeks prior to Screening.\n   3. JAK inhibitors require 12 weeks of stable dosing prior to Screening. For the TD high, TD low, and nTD cohorts, JAK inhibitors allowed include momelotinib, pacritinib, fedratinib, and ruxolitinib.\n   4. If the participant discontinues JAK inhibitor (including momelotinib\u002Fpacritinib\u002Fruxolitinib\u002Ffedratinib) and\u002For hydroxyurea prior to Screening, a 60-day washout period is required.\n6. Eastern Cooperative Oncology Group (ECOG) performance score ≤2.\n7. Infusion of hematopoietic stem cell transplant not anticipated within 8 months after Screening.\n8. TSAT \\\u003C75% (local lab acceptable) at or within 2 weeks of Screening.\n9. Liver iron concentration by MRI \\\u003C7 mg\u002Fg dry weight within 3 months of eligibility confirmation by central review. Required for TD high participants only.\n10. Serum ferritin ≥50 µg\u002FL at Screening.\n11. Platelet count ≥25,000\u002FµL and \\\u003C1,000,000\u002FµL; neutrophils ≥1,000\u002FµL; and total white blood cell (WBC) count \\\u003C50,000\u002FµL at Screening.\n12. Estimated glomerular filtration rate (eGFR) ≥30 mL\u002Fmin\u002F1.73 m2 by the Chronic Kidney Disease-Epidemiology Collaboration (CKD-EPI) formula.\n13. Aspartate aminotransferase (AST) and ALT \\\u003C3.0x upper limit of normal (ULN) at Screening.\n14. Direct bilirubin \\\u003C2x ULN at Screening. Higher levels are acceptable if these can be attributed by the Investigator to ineffective erythropoiesis or Gilbert's syndrome, with approval from Sponsor.\n15. If male with female sexual partner(s) of childbearing potential, agrees to use 1 of the following highly effective methods of contraception during the study and for at least 8 weeks after the last study drug dose:\n\n    1. Stable hormonal contraceptive (≥3 months; female partner)\n    2. Intrauterine device in place for at least 3 months (female partner)\n    3. Surgically sterile hysterectomy, bilateral oophorectomy, or bilateral tubal ligation (female partner)\n    4. Confirmed successful vasectomy\n16. If female, then EITHER postmenopausal (defined as 12 months of spontaneous amenorrhea, 6 months of spontaneous amenorrhea with serum follicle-stimulating hormone (FSH) levels \\>40 mIU\u002Fml, or 6 weeks postsurgical bilateral oophorectomy with or without hysterectomy), surgically sterile, OR agreeable to use 1 of the following highly effective contraception methods (listed below) on Day 1 (or earlier) and for at least 8 weeks after the last dose of study drug:\n\n    1. Stable hormonal contraceptive (≥3 months)\n    2. Intrauterine device in place for at least 3 months\n    3. Tubal ligation or single male partner with vasectomy\n17. Negative urine pregnancy test (females of childbearing potential) at Screening (Days 28 to 2).\n18. Able to understand the study aims, procedures, and requirements, and provide written informed consent.\n19. Able to comply with all study procedures.\n\nInclusion Criteria for Exploratory Cohort of Participants with MDS and Anemia:\n\nParticipants are eligible for the MDS exploratory cohort if all of the following criteria apply:\n\n1. Age 18 years or older at the time of signing the ICF.\n2. Molecular International Prognostic Scoring System (IPSS-M) classification of very low, low, or intermediate (ie, lower risk) MDS-ringed sideroblasts (RS) negative, MDS\u002FMPN with ringed sideroblasts and thrombocytosis (RS-T), Chronic Myelomonocytic Leukemia (CMML), Atypical Chronic Myeloid Leukemia (aCML), or Myelodysplastic\u002FMyeloproliferative Neoplasms, Unclassifiable (MDS\u002FMPN-U) as confirmed in the most recent local bone marrow biopsy report according to WHO criteria.\n3. Washout of at least 28 days is required for prior anemia\u002Fneutropenia-directed therapies, including:\n\n   1. Androgens\n   2. EPO-stimulating agents\n   3. Luspatercept\n   4. Sotatercept (ACE-011)\n   5. Imetelstat\n   6. Granulocyte colony-stimulating factor (G CSF) OR granulocyte-macrophage CSF (GM CSF).\n   7. Systemic corticosteroids (except for participants on a stable or decreasing dose for ≥28 days prior to randomization for non-hematological conditions and receiving an equivalent to ≤10 mg prednisone for the 28 days immediately prior to Screening) Screening can begin before the 28-day washout is completed, but the washout period must be completed prior to collection of Screening blood samples.\n4. Anemia:\n\n   1. Baseline Hgb of \\\u003C10 g\u002FdL on ≥3 assessments over 84 days prior to Screening, without RBC transfusion, or Hgb \\\u003C10 g\u002FdL and receiving RBC transfusions periodically during the 84 days prior to Screening\n   2. Medical history of ≤24 units of PRBC for MDS and anemia\n5. ECOG performance score ≤2\n6. Infusion of hematopoietic stem cell transplant not anticipated within 8 months after Screening\n7. TSAT \\\u003C75% (local lab acceptable) at or within 2 weeks of Screening\n8. Liver iron concentration by MRI \\\u003C7 mg\u002Fg dry weight within 3 months of eligibility confirmation by central review\n9. Serum ferritin ≥50 μg\u002FL at Screening\n10. Platelet count ≥25,000\u002FμL and \\\u003C1,000,000\u002FμL, and total WBC count \\\u003C50,000\u002FμL at Screening or otherwise approved by Sponsor.\n11. eGFR ≥30 mL\u002Fmin\u002F1.73 m2 by the CKD-EPI formula\n12. AST and ALT \\\u003C3x ULN at Screening\n13. Direct bilirubin \\\u003C2x ULN at Screening. Higher levels are acceptable if these can be attributed by the Investigator to ineffective erythropoiesis.\n14. If male with female sexual partner(s) of childbearing potential, agrees to use 1 of the following highly effective methods of contraception during the study and for at least 8 weeks after the last study drug dose:\n\n    1. Stable hormonal contraceptive (≥3 months; female partner)\n    2. Intrauterine device in place for at least 3 months (female partner)\n    3. Surgically sterile hysterectomy, bilateral oophorectomy, or bilateral tubal ligation (female partner)\n    4. Confirmed successful vasectomy\n15. If female, then EITHER postmenopausal (defined as 12 months of spontaneous amenorrhea, 6 months of spontaneous amenorrhea with serum FSH levels \\>40 mIU\u002Fml, or 6 weeks postsurgical bilateral oophorectomy with or without hysterectomy), surgically sterile, OR agreeable to use 1 of the following highly effective contraception methods (listed below) on Day 1 (or earlier) and for at least 8 weeks after the last dose of study drug:\n\n    1. Stable hormonal contraceptive (≥3 months)\n    2. Intrauterine device in place for at least 3 months\n    3. Tubal ligation or single male partner with vasectomy\n16. Negative urine pregnancy test (females of childbearing potential) at Screening (Days 28 to 2).\n17. Able to understand the study aims, procedures, and requirements, and provide written informed consent.\n18. Able to comply with all study procedures.\n\nExclusion Criteria for Participants with MF and Anemia:\n\nParticipants are excluded from the study if any of the following criteria apply:\n\nMedical History, Participants with MF and Anemia\n\n1. Hereditary hemochromatosis\n2. Hemoglobinopathy or intrinsic RBC defect associated with anemia\n3. Total splenectomy\n4. Hematopoietic cell transplant within the past 2 years, or graft vs host disease requiring immunosuppression\n5. Current anemia from iron deficiency, vitamin B12 or folate deficiency, infection, or bleeding\n6. Active immune-mediated hemolytic anemia\n7. Symptomatic bleeding, unrelated to surgery, in a critical area or organ and\u002For bleeding causing a decrease in Hgb of ≥2 g\u002FdL or leading to transfusion of ≥2 units of RBCs in the 6 months prior to Screening\n8. Major surgery within 8 weeks prior to Screening or incomplete recovery from any previous surgery\n9. Malignancy within the past 3 years, other than primary MF, post ET, or post PV MF. The following history or concurrent conditions are allowed:\n\n   1. basal or squamous cell carcinoma of the skin\n   2. carcinoma in situ of the cervix or the breast\n   3. histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis \\[TNM\\] clinical staging system) A history of completed treatment (medical or surgical) of stage 1-2 cancers may be permitted with prior Sponsor agreement\n10. Stroke, deep vein thrombosis, or pulmonary or arterial embolism within 3 months prior to Screening\n11. Known allergic reaction to any study drug excipient\n12. A history of anti-drug antibody formation\n13. Inadequately controlled heart disease (New York Heart Association Classification 3 or 4) and\u002For known to have left ventricular ejection fraction \\\u003C35%\n14. Hepatitis B or C, or human immunodeficiency virus (HIV) with detectable viral load\n15. Uncontrolled fungal, bacterial, or viral infection (ongoing signs\u002Fsymptoms related to the infection, without improvement despite appropriate treatment)\n\n    Treatment History, Participants with MF and Anemia\n16. Iron chelation therapy in the 28 days prior to Screening\n17. Change in anticoagulant therapy regimen within 8 weeks prior to Screening\n\n    Laboratory Exclusions, Participants with MF and Anemia\n18. Peripheral blood myeloblasts ≥10% of WBC differential at most recent evaluation prior to Screening\n19. Positive direct antiglobulin test in conjunction with a reactive RBC eluate at Screening\n\n    Miscellaneous, Participants with MF and Anemia\n20. Pregnant or lactating\n21. Condition or concomitant medication that would confound the ability to interpret study data\n22. Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgment of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude the participant from participating in the study\n23. Participation in any other clinical protocol or investigational study that involves administration of experimental therapy and\u002For therapeutic devices within 30 days prior to Screening\n\nExclusion Criteria for Exploratory Cohort of Participants with MDS and Anemia:\n\nParticipants are excluded from the MDS exploratory cohort if any of the following criteria apply:\n\nMedical History, Participants with MDS and Anemia\n\n1. Secondary MDS, ie, MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and\u002For radiation from other diseases\n2. Peripheral blasts ≥5%\n3. Current treatment with hypomethylating agent or other acute myeloid leukemia (AML)-like combination chemotherapy or planned use within 6 months after Screening\n4. Prior treatment with \\>3 anemia-directed therapies (unless otherwise approved by Sponsor) including:\n\n   1. Luspatercept\n   2. Sotatercept (ACE-011)\n   3. EPO-stimulating agent\n   4. Imetelstat\n5. Hereditary hemochromatosis\n6. Hemoglobinopathy or intrinsic RBC defect associated with anemia\n7. Total splenectomy\n8. Hematopoietic cell transplant within the past 10 years\n9. Current anemia from iron deficiency, vitamin B12 or folate deficiency, infection, or bleeding\n10. Active immune-mediated hemolytic anemia\n11. Symptomatic bleeding, unrelated to surgery, in a critical area or organ and\u002For bleeding causing a decrease in Hgb of ≥2 g\u002FdL or leading to transfusion of ≥2 units of RBCs in the 6 months prior to Screening\n12. Major surgery within 8 weeks prior to Screening or incomplete recovery from any previous surgery\n13. Malignancy within the past 3 years, other than MDS or MDS\u002FMPN without excess blasts. The following history or concurrent conditions are allowed:\n\n    1. Basal or squamous cell carcinoma of the skin\n    2. Carcinoma in situ of the cervix or the breast\n    3. Histologic finding of prostate cancer (T1a or T1b using the TNM clinical staging system) A history of completed treatment (medical or surgical) of stage 1-2 cancers may be permitted with prior Sponsor agreement\n14. Stroke, deep vein thrombosis, or pulmonary or arterial embolism within 6 months prior to Screening\n15. Known allergic reaction to any study drug excipient\n16. A history of antidrug antibody formation\n17. Inadequately controlled heart disease (New York Heart Association Classification 3 or 4) and\u002For known to have left ventricular ejection fraction \\\u003C35%\n18. Active hepatitis B or C, or HIV with detectable viral load\n19. Uncontrolled fungal, bacterial, or viral infection (ongoing signs\u002Fsymptoms related to the infection, without improvement despite appropriate treatment)\n\n    Treatment History, Participants with MDS and Anemia\n20. Iron chelation therapy in the 28 days prior to Screening\n21. Change in anticoagulant therapy regimen within 8 weeks prior to Screening\n\n    Laboratory Exclusions, Participants with MDS and Anemia\n22. Positive direct antiglobulin test in conjunction with a reactive RBC eluate at Screening\n\n    Miscellaneous, Participants with MDS and Anemia\n23. Pregnant or lactating\n24. Condition or concomitant medication that would confound the ability to interpret study data\n25. Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgment of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude the participant from participating in the study\n26. Participation in any other clinical protocol or investigational study that involves administration of experimental therapy and\u002For therapeutic devices within 30 days prior to Screening",{"count":217,"type":21},150,[59,60],"This phase 1b\u002F2a open-label study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of DISC-0974 as well as categorize the effects on hematologic response in participants with myelofibrosis or myelodysplastic syndrome and anemia.",[221,29,222,223,224,225,27],"Myelofibrosis; Anemia","Myelofibrosis","Myelofibrosis Due to and Following Polycythemia Vera","Primary Myelofibrosis","Post-essential Thrombocythemia Myelofibrosis",[227,228],"Myeloproliferative Neoplasm","Myeloproliferative Disorders","2026-08-10",{"date":201,"type":38},{"date":232,"type":38},"2022-06-06",{"date":234,"type":21},"2027-06",{"name":236,"class":45},"Disc Medicine, Inc",30,{"id":239,"slug":240,"hasResults":12,"nctId":241,"briefTitle":242,"officialTitle":243,"acronym":4,"eligibilityCriteria":244,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":245,"targetDuration":4,"studyType":22,"phases":247,"briefSummary":248,"conditions":249,"keywords":250,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":254,"lastUpdatePostDateStruct":255,"startDateStruct":256,"completionDateStruct":258,"leadSponsor":260,"locationsCount":262},"100556793","phase-2-interferon--ifn--with-donor-leukocyte-infusion-to-treat-relapsed-acute-myeloid-leukemia-and-myelodysplastic-syndromes-post-allogeneic-hematopoietic-stem-cell-transplantation-100556793","NCT06529731","Interferon-γ (IFN-γ) With Donor Leukocyte Infusion to Treat Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndromes Post Allogeneic Hematopoietic Stem Cell Transplantation","A Phase 2 Trial of Interferon-γ (IFN-γ) in Combination With Donor Leukocyte Infusion (DLI) to Treat Relapsed Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS) After Allogeneic Hematopoietic Stem Cell Transplantation (alloSCT)","Inclusion Criteria:\n\n1. Age ≥ 18 years\n2. Recipients of an alloSCT for AML or MDS from a minimally 8\u002F8 HLA-matched donor (Cohort 1: HLA -matched alloSCT recipients) or recipients of a haploidentical donor SCT for AML or MDS from a minimally 4\u002F8 HLA-matched donor (Cohort 2: Haplo-SCT recipients)\n3. AML\u002FMDS relapsed post-alloSCT with measurable residual disease defined by either of the following criteria:\n\n   1. At least 5% or more myeloblasts based on bone marrow biopsy morphology by pathologist review. Abnormal myeloblasts cannot not exceed 30% overall 36\n   2. At least 0.1% of abnormal myeloblasts with a leukemia-associated immunophenotype (LAIP) by multiparameter flow cytometry. The abnormal cells with LAIP should not exceed 30% of nucleated cells.\n   3. Recurrent or persistent cytogenetic abnormalities detectable by FISH or karyotype analysis.\n   4. For patients with mutant NPM1, at least 1,000 mutant transcript copies per 106 ABL or equivalent housekeeping transcripts in bone marrow by qPCR or dPCR\n4. Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) 0-2\n5. A DLI is available, or the donor is available and agrees to undergo apheresis to collect lymphocytes for infusion\n6. If salvage therapy for post-alloSCT relapse was received, the therapy is limited to 1 line of the following:\n\n   1. For hypomethylating agents, venetoclax, and targeted therapies (e.g., tyrosine kinase inhibitors, IDH1\u002FIDH2 inhibitors, or FLT3 inhibitors), the last dose must be \\> 2 week prior to the initiation of IFN-γ\n   2. For cytotoxic chemotherapy agents, the last dose must be \\>2 weeks prior to start of treatment for the present study\n   3. For investigational agents, the last dose must be ≥ 4 weeks or 5 half-lives (whichever is longer) prior to the start of treatment for the present study\n7. Provision of signed and dated informed consent form\n8. Stated willingness to comply with all study procedures and availability for the duration of the study\n9. For female subject, who is \\\u003C 55 years old without hysterectomy, oophorectomy or documented menopause, willingness to use two forms of contraception including one form of highly effective contraception (i.e., long-acting reversible contraception, oral contraceptive pills) for the duration of the study\n10. For male subject, willingness to use highly effective contraception methods including male condoms by male subject and one form of highly effective contraception by his female partner (i.e., long-acting reversible contraception, oral contraceptive pills) for the duration of the study\n\nExclusion Criteria:\n\n1. Primary engraftment failure after alloSCT\n2. Evidence of mixed phenotype leukemia with lymphoid differentiation (Cohort 1: HLA -matched alloSCT recipients)\n3. Grade 3 or 4 aGVHD per Mount Sinai Acute GVHD International Consortium (MAGIC) at the time of planned enrollment\n4. History of grade 4 aGVHD per the MAGIC criteria\n5. Moderate or severe cGVHD per NIH Consensus Criteria at time of planned enrollment\n6. Any systemic immunosuppressive medications taken within 2 weeks before the enrollment\n7. Grade 3 or higher non-hematologic toxicity related to any prior therapy at the time of enrollment\n8. A contraindication to receive IFN-γ including a known hypersensitivity to IFN-γ, E. coli derived products or any other component of the product\n9. Positive pregnancy test or currently breastfeeding on Day 1 of study treatment 37\n10. Active cardiac arrhythmia not controlled by medical management or current NYHA class II or higher congestive heart failure within 2 months of enrollment unless it was due to a tachyarrhythmia which is under control at the time of enrollment\n11. Active ischemic heart disease not controlled with medications within 2 months of enrollment\n12. Acute or chronic pulmonary disease requiring continuous oxygen treatment\n13. Seizure disorder not controlled by medications within 2 months of enrollment\n14. AST or ALT \\> 5x ULN or total bilirubin \\>3x ULN at time of enrollment\n15. Renal function CrCl \\\u003C30 mL\u002Fmin at time of enrollment using modified Cockcroft-Gault formula\n16. Detection of HLA loss of heterozygosity (LOH) in relapsed malignant cells. Specifically, there has been a loss of the mismatched set of HLA alleles encoded on chromosome 6 (ie uniparental disomy of chromosome 6), within 30 days prior to enrollment (Cohort 2: Haplo-SCT recipients)",{"count":246,"type":21},57,[60],"This phase 2 study aims to confirm the efficacy observed in the prior phase 1 trial in Cohort 1 and to evaluate the safety of IFN-γ in combination with DLI in Cohort 2, the haploidentical donor alloSCT recipient cohort. The study will further contribute to this effort through the collection of leukemia cells pre- and post-in vivo IFN-γ therapy. As in the previously conducted phase 1 trial, this trial will assess whether leukemia blasts are responsive to IFN-γ in vitro and in vivo. Single-cell RNA sequencing (scRNAseq) will be performed to evaluate transcriptomic changes induced by IFN-γ in leukemia cell subsets, including those with stem cell characteristics.",[199,27],[251,252,253],"interferon-γ (IFN-γ)","donor leukocyte infusion (DLI)","allogeneic hematopoietic stem cell transplantation (alloSCT)","2026-08-06",{"date":176,"type":38},{"date":257,"type":38},"2024-09-23",{"date":259,"type":21},"2029-10-31",{"name":261,"class":128},"Sawa Ito, MD",3,{"id":264,"slug":265,"hasResults":12,"nctId":266,"briefTitle":267,"officialTitle":268,"acronym":4,"eligibilityCriteria":269,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":270,"targetDuration":4,"studyType":271,"phases":4,"briefSummary":272,"conditions":273,"keywords":4,"overallStatus":274,"whyStopped":4,"lastUpdateSubmitDate":275,"lastUpdatePostDateStruct":276,"startDateStruct":277,"completionDateStruct":279,"leadSponsor":281,"locationsCount":4},"100636774","use-of-electrical-bioimpedance-in-acute-myeloid-leukemia-aml-and-myelodysplastic-syndrome-mds-patients-bioimpedance-100636774","NCT07570056","Use of Electrical Bioimpedance in Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) Patients (Bioimpedance)","A Pilot Study for the Use of Electrical Bioimpedance in Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) Patients (Bioimpedance)","Inclusion Criteria:\n\n* Suspected or confirmed diagnosis of Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) and undergoing a bone marrow biopsy.\n* Age 18 or older.\n* Able to provide informed consent and willing to sign an approved consent form that conforms to federal and institutional guidelines.\n\nExclusion Criteria:\n\n* Study prospect that has an electronic implant (cardiac, neurological, sensory, prosthetic implants with an electronic component. Also monitoring and drug delivery systems.)\n* Medical, psychiatric, cognitive, or other conditions that may compromise the participant's ability to understand the participant information, give informed consent, comply with the study protocol or complete the study.\n* Pregnant women\n* Inability to understand and\u002For speak the English or Spanish language.",{"count":7,"type":21},"OBSERVATIONAL","The goal of this project is to test non-invasive, painless skin electrical bioimpedance (BioZ) measurements as an adjunctive biomarker to standard bone marrow biopsies.",[27,199],"NOT_YET_RECRUITING","2026-08-05",{"date":254,"type":38},{"date":278,"type":21},"2026-08",{"date":280,"type":21},"2027-07",{"name":282,"class":128},"University of Utah",{"id":284,"slug":285,"hasResults":12,"nctId":286,"briefTitle":287,"officialTitle":288,"acronym":4,"eligibilityCriteria":289,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":290,"targetDuration":4,"studyType":22,"phases":292,"briefSummary":293,"conditions":294,"keywords":295,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":296,"lastUpdatePostDateStruct":297,"startDateStruct":298,"completionDateStruct":300,"leadSponsor":302,"locationsCount":303},"100617532","phase-2-a-study-of-tak-226-for-anemia-in-japanese-patients-with-lower-risk-myelodysplastic-syndromes-100617532","NCT07319845","A Study of TAK-226 for Anemia in Japanese Patients With Lower-Risk Myelodysplastic Syndromes","A Phase 2, Multicenter, Open-Label, Single-arm Study to Evaluate the Efficacy and Safety of TAK-226 for Anemia in Japanese Patients With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes","Inclusion Criteria:\n\n1. Participants or their legally authorized representative must be willing and able to sign the ICF and to adhere to the protocol requirements.\n2. Japanese adult male or female participant \\>=18 years of age at the time of signing informed consent.\n3. Diagnosis of MDS with or without ring sideroblasts (RS) (as determined in an evaluable bone marrow aspirate collected at Screening to confirm diagnosis) according to WHO 2016 classification that meets the International Prognostic Scoring System-Revised (IPSS-R) classification of very low-, low-, or intermediate-risk MDS.\n\n   Note: Due to expected impacts of transfusion, hemoglobin (Hgb) values from blood samples collected within 14 days following a RBC transfusion and platelet count obtained within 7 days following a platelet transfusion cannot be used to evaluate IPSS-R for eligibility.\n4. TD\\[YY1.1\\] cohort: Transfusion dependence assessed in the 16 weeks immediately preceding enrollment in two 8-week blocks classified as either:\n\n   1. Low-transfusion burden (LTB), defined as 4 to 7 RBC units per 16 weeks; or\n   2. HTB\\[YY2.1\\], defined as \\>=8 RBC units per 16 weeks; and\n   3. For all participants: i. Only transfusion events for a pretransfusion Hgb \\\u003C10 g\u002FdL are counted toward eligibility; ii. At least 1 transfusion event in each 8-week block and a minimum of 2 transfusion events separated by \\>=7 days within the 16-week period immediately preceding enrollment; and iii. No consecutive 56-day period can be RBC transfusion-free during the 16 week period immediately preceding enrollment.\n\n   Note: Only transfusions for the disease under study will be counted towards classification for LTB or HTB participants. Transfusions for intercurrent diseases (bleeding, surgical procedure, infection, etc.) are not considered.\n\n   NTD\\[YY3.1\\] cohort: NTD, defined as 0 to 1 RBC units per 8 weeks immediately preceding enrollment.\n\n   Note: RBC transfusions administered when Hgb levels were \\\u003C9.0 g\u002FdL are counted for eligibility. RBC transfusions administered for other than MDS-related anemia (bleeding, surgical procedure, infection, etc.) will not be counted as a required transfusion for the purpose of meeting eligibility criteria.\n5. TD cohort: Refractory or intolerant to prior erythropoiesis-stimulating agent (ESA) treatment (discontinued \\>=4 weeks before enrollment), or unlikely to respond to ESA treatment, defined as follows:\n\n   a. Refractory to prior ESA treatment: documentation of nonresponse or a response that was no longer maintained with a prior ESA-containing regimen, either as a single agent or combination (eg, with granulocyte colony-stimulating factor \\[G-CSF\\]); ESA regimen must have been either: i. Recombinant human erythropoietin (EPO) \\>=40,000 IU\u002Fweek for \\>=8 doses or equivalent; or ii. Darbepoetin alpha \\>=500 mcrg every 3 weeks for \\>=4 doses or equivalent. b. Intolerant to prior ESA treatment: documentation of discontinuation of a prior ESA containing regimen, either as a single agent or combination (eg, with G-CSF), at any time after introduction due to intolerance or an AE.\n\n   c. Unlikely to respond to ESA treatment: low chance of response to ESA based on an endogenous serum EPO level \\>200 U\u002FL.\n\n   Note: Due to expected impacts of transfusion on EPO levels, blood samples collected within the 14 days following an RBC transfusion or within 7 days following a platelet transfusion cannot be used to evaluate serum EPO level for eligibility.\n\n   NTD cohort: Hgb \\\u003C10 g\u002FdL and exhibiting anemia-related clinical symptoms (eg, fatigue, shortness of breath, or others) during screening.\n6. Less than 5% blasts in an evaluable bone marrow aspirate collected at Screening.\n7. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.\n8. Women of childbearing potential (WOCBP), defined as a sexually mature woman who has not undergone surgical sterilization or who has not been naturally postmenopausal for at least 12 consecutive months must\n\n   1. Agree to use 1 highly effective method of contraception and 1 additional effective (barrier) method at the same time, from the time of signing the informed consent through 60 days after the last dose of study drug; or\n   2. Agree to practice true abstinence, when this is in line with the preferred and usual lifestyle of the participant. (Periodic abstinence \\[eg, calendar, ovulation, symptothermal, postovulation methods\\], withdrawal, spermicides only, and lactational amenorrhea are not acceptable methods of contraception.)\n9. Male participants must, even if he is surgically sterilized (ie, status postvasectomy),\n\n   1. Agree to practice effective barrier contraception the time of signing the informed consent through 60 days after the last dose of study drug; or\n   2. Agree to practice true abstinence, when this is in line with the preferred and usual lifestyle of the participant. (Periodic abstinence \\[eg, calendar, ovulation, symptothermal, postovulation methods\\], withdrawal, spermicides only, and lactational amenorrhea are not acceptable methods of contraception.)\n\nExclusion Criteria:\n\nMedical History\n\n1. Del(5q) MDS or therapy-related (secondary) MDS.\n2. Anemia due to any other known cause (eg, thalassemia, hemolytic anemia, bleeding events, or deficiency of iron, B12, and\u002For folate).\n3. Receipt of RBC transfusion for any reason(s) other than underlying MDS within 16 weeks in TD cohort or 8 weeks in NTD cohort before enrollment.\n4. Clinically significant cardiovascular disease defined as:\n\n   1. New York Heart Association heart disease class III or IV;\n   2. Fridericia corrected QT (QTcF) interval \\>500 milliseconds during Screening;\n   3. Presence of uncontrolled hypertension defined as mean systolic blood pressure \\>=160 mm Hg or diastolic blood pressure \\>=100 mm Hg during Screening; or\n   4. Uncontrolled arrhythmia, myocardial infarction, or unstable angina within 6 months before Screening.\n5. Known ejection fraction \\\u003C35%, confirmed by a local echocardiogram performed during Screening, or a previously performed echocardiogram if collected within 6 months before Screening.\n6. Stroke, deep vein thrombosis, or pulmonary embolism within 6 months before Screening.\n7. Any known history of acute myeloid leukemia (AML).\n8. Prior history of malignancies, other than MDS, unless the participant has been free of the disease (including completion of any treatment, including maintenance, for prior malignancy) for \\>=5 years. However, participants with a history or concurrent diagnosis of the following conditions are allowed if not requiring systemic therapy:\n\n   1. Basal or squamous cell carcinoma of the skin;\n   2. Carcinoma in situ of the cervix;\n   3. Carcinoma in situ of the breast; and\u002For\n   4. Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, node, metastasis \\[TNM\\] clinical staging system).\n9. History of solid organ or bone marrow transplantation.\n10. Active infection requiring intravenous antibiotics within 28 days or oral antibiotics within 14 days before enrollment.\n11. History of or known active or chronic infection with HIV, active infectious hepatitis B virus (HBV), or active infectious hepatitis C virus (HCV). Participants who are positive for hepatitis B surface antigen (HBsAg), hepatitis B core antibody (HBcAb), or hepatitis B surface antibody (HBsAb) may be eligible for enrollment if their hepatitis B viral load is below the limit of detection. Participants who are positive for hepatitis C virus antibodies (HCVAb) may be enrolled if their hepatitis C viral load is below the limit of detection.\n12. Body mass index \\>=40 kg\u002Fm\\^2.\n13. Major surgery within 28 days before enrollment.\n14. History of allergy\u002Fanaphylaxis to investigational medicinal product (IMP) excipients (refer to the current elritercept \\[TAK 226\\] IB for a list of excipients) or recombinant proteins.\n\n    Treatment History\n15. TD cohort: Prior use of TAK 226, luspatercept, imetelstat, or sotatercept. \\[YY4.1\\] NTD cohort: Prior use of TAK 226, luspatercept, imetelstat, sotatercept, or ESAs.\n\n    Note for NTD cohort: At the investigator's discretion in consultation with the medical monitor, may be allowed if received no more than 2 doses of ESAs \\>=8 weeks prior to enrollment.\n16. Prior use of hypomethylating agents (HMAs), isocitrate dehydrogenase inhibitor, lenalidomide, or immunosuppressive therapy given for treatment of MDS.\n17. Iron chelation therapy initiated within 8 weeks before enrollment. Participants on stable doses of iron chelation therapy for \\>=8 weeks are allowed.\n18. Vitamin B12 or folate therapy initiated within 4 weeks before enrollment. Participants on stable replacement doses for \\>=4 weeks and without ongoing concurrent vitamin B12 or folate deficiency are allowed.\n19. Androgen use within 8 weeks before enrollment. Participants on stable androgen dosing for hypogonadism for \\>=8 weeks are allowed.\n20. High-dose corticosteroid use within 4 weeks before enrollment. Participants on stable chronic steroid doses of prednisone\u002Fprednisolone \\\u003C=10 mg\u002Fday or corticosteroid equivalent for \\>=4 weeks are allowed.\n21. Treatment with any investigational drug within 28 days before Screening or, if the half-life of the product is known, within 5 times the half-life before Screening, whichever is longer.\n22. Ongoing participation in another interventional clinical study. Laboratory Exclusions (during Screening)\n23. Serum EPO level \\>500 U\u002FL. Note: Due to expected impacts of transfusion on EPO levels, laboratory results from blood samples collected within the 14 days following an RBC transfusion cannot be used to evaluate serum EPO level for eligibility.\n24. Platelet count \\>=450\\*10\\^3\u002FmcrL or \\\u003C=25\\*10\\^3\u002FmcrL. Note: Due to expected impacts of transfusion, laboratory results from blood samples collected within the 7 days following a platelet transfusion cannot be used to evaluate platelet count for eligibility.\n25. Absolute neutrophil count \\\u003C=500\u002FmcrL\n26. Serum AST or ALT \\>=3\\*the upper limit of normal (ULN).\n27. Total bilirubin \\>=2\\*ULN unless attributable to Gilbert syndrome.\n28. Ferritin \\\u003C=50 mcrg\u002FL.\n29. Folate \\\u003C=2.0 ng\u002FmL.\n30. Vitamin B12 \\\u003C=200 pg\u002FmL.\n31. Estimated glomerular filtration rate \\\u003C30 mL\u002Fmin\u002F1.73m\\^2 as determined by Japanese Society of Nephrology. Calculated by the correction formula for Japanese. a)\n\n    a) eGFR=194\\* (serum creatinine value)\\^-1.094\\* (age)\\^-0.287\\* (sex correction factor), Sex correction factor: 0.739 in female.\n\n    Miscellaneous\n32. Pregnant or lactating female\\[YY5.1\\]. Note: Participants who may be in the very early stage of pregnancy based on the doctor's interview with a negative pregnancy test are excluded from the study. Participants who are lactating will be eligible if they discontinue breastfeeding from before the first dose of study drug until 60 days after the last dose of study drug.\n33. Any other condition not specifically noted above that, in the opinion of the investigator, would preclude the participant from participating in the study or could confound interpretation of data from the study.\n34. Investigational site staff members directly involved in the conduct of the study and site staff members otherwise supervised by the investigator, employees of the Sponsor or contract research organization (CRO) directly involved in the conduct of the study, or immediate family members (defined as a spouse, parent, child, or sibling, whether biological or legally adopted).",{"count":291,"type":21},42,[60],"The main aim of the study is to evaluate how TAK-226 improves symptoms of transfusion-dependent anemia in Japanese patients with lower-risk myelodysplastic syndromes.\n\nThe study consists of Screening Period (up to 6 weeks), Treatment Period, Safety Follow-Up Period (8 weeks), and Long-Term Follow-Up Period (5 years from the first dose of the study drug or 3 years after the last dose, whichever is longer).\n\nParticipants of this study will be administered TAK-226 during Treatment Period. Subsequently, the participants will be monitored for side effects related to the study treatment during Safety Follow-Up Period and Long-Term Follow-Up Period. The approximate duration of participation for a participant is up to approximately 6 years.\n\nDuring the study period, participants will visit the study clinic\u002Fhospital multiple times as per the study schedule. During Treatment Period, the participants will come to the clinic\u002Fhospital approximately every two to four weeks.",[27],[96],"2026-08-04",{"date":275,"type":38},{"date":299,"type":38},"2026-04-22",{"date":301,"type":21},"2033-01-10",{"name":44,"class":45},20,{"id":305,"slug":306,"hasResults":12,"nctId":307,"briefTitle":308,"officialTitle":309,"acronym":310,"eligibilityCriteria":311,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":312,"targetDuration":4,"studyType":22,"phases":314,"briefSummary":315,"conditions":316,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":321,"lastUpdatePostDateStruct":322,"startDateStruct":324,"completionDateStruct":326,"leadSponsor":328,"locationsCount":262},"100589944","mosaic-trial-for-stem-cell-transplant-recipients-100589944","NCT06960993","Mosaic Trial for Stem Cell Transplant Recipients","Mosaic: RCT of a Digital Health Intervention for English- and Spanish-speaking Stem Cell Transplant Recipients","Mosaic","Inclusion Criteria:\n\n* Diagnosed with a hematologic cancer according to medical records\n* Scheduled for or preparing for scheduling of an allogeneic or autologous stem cell transplant at one of our study sites\n* Aged 18 or older (no upper limit)\n* English or Spanish Proficient\n* Interested in using a website to learn about stem cell transplant\n* Ability to understand and willingness to sign an informed consent document and comply with all study procedures\n\nExclusion Criteria:\n\n* Currently participating in a behavioral intervention targeting distress, health-related quality of life, or symptoms\n* Undergoing the first in a planned tandem stem cell transplant\n* Unable to provide meaningful consent (severe cognitive impairment or language difficulties)",{"count":313,"type":21},356,[169],"The goal of this clinical trial is to learn if using an intervention website (Mosaic) improves selected patient-reported outcomes in adult blood cancer patients undergoing allogeneic or autologous stem cell transplant, compared to using an educational website (control group). Patients will be recruited prior to their scheduled transplant, then randomized to use one of these two study websites throughout the study. They will complete five assessments during the study: one before transplant (baseline) and four after transplant (2, 4, 6, and 8 month follow-ups).\n\nThe main questions this trial aims to answer are:\n\n1. Compared to patients using the control group website, do patients using the intervention website report greater improvements in general psychological distress, cancer treatment-related distress, physical symptoms, and health-related quality of life?\n2. Are these benefits at least partially explained by improvements in perceived preparedness, self-efficacy, and approach coping and\u002For reductions in avoidant coping and perceived stress?\n3. Do some patients benefit more from using the intervention website than others? Specifically, we will examine whether patients' primary language (English\u002FSpanish) and their initial psychological distress are related to the benefit they get from using the intervention website. We will also explore effects of sex, race, ethnicity, and transplant type.",[317,318,319,320,198,197,27],"Hematologic Malignancy","Stem Cell Transplant","Bone Marrow Transplant","Leukemia","2026-07-31",{"date":323,"type":38},"2026-08-03",{"date":325,"type":38},"2025-04-28",{"date":327,"type":21},"2030-02-01",{"name":329,"class":128},"Northwestern University",{"id":331,"slug":332,"hasResults":12,"nctId":333,"briefTitle":334,"officialTitle":335,"acronym":4,"eligibilityCriteria":336,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":337,"targetDuration":4,"studyType":22,"phases":338,"briefSummary":339,"conditions":340,"keywords":341,"overallStatus":274,"whyStopped":4,"lastUpdateSubmitDate":343,"lastUpdatePostDateStruct":344,"startDateStruct":345,"completionDateStruct":347,"leadSponsor":349,"locationsCount":4},"100649804","phase-2-evaluate-ofirnoflast-in-adults-with-very-low--to-intermediate-risk-myelodysplastic-syndromes-requiring-transfusions-100649804","NCT07738510","Evaluate Ofirnoflast in Adults With Very Low- to Intermediate-risk Myelodysplastic Syndromes Requiring Transfusions","A Phase 2b, Multicenter, Open-label, Randomized, Dose Optimization Study of Ofirnoflast (HT-6184) for the Treatment of Anemia in Adults With Very Low- to Intermediate-Risk Myelodysplastic Syndromes Requiring Red Blood Cell Transfusions","Inclusion Criteria:\n\n1. At least 18 years of age at the time of signing informed consent.\n2. Capable of giving signed informed consent\n3. Documented diagnosis of very low-, low-, or intermediate-risk MDS\n4. Documented diagnosis of anemia\n5. Relapsed or refractory disease after 1 to 3 prior lines of therapy for lower-risk MDS\n6. Willing to provide a bone marrow aspirate at Screening.\n7. Life expectancy of more than 6 months at screening.\n8. Participants of childbearing potential must have a negative pregnancy test at screening (serum) and Day 1 (urine).\n9. Participants and partners must use contraception consistent with local regulations and protocol-defined criteria during the intervention period and for at least 30 days after the last dose; periodic abstinence and withdrawal are not acceptable methods.\n\nExclusion Criteria:\n\n1. Anemia due to other causes (e.g., iron deficiency).\n2. Known clinically significant anemia due to iron, vitamin B12, or folate deficiency; autoimmune or hereditary hemolytic anemia; or gastrointestinal bleeding.\n3. History of hemoglobinopathies, intrinsic RBC membrane\u002Fenzyme defects, or hemolytic anemia.\n4. Prior history of AML, secondary MDS, or other malignancy (except non-melanoma skin cancer or in situ cervical\u002Fbreast carcinoma) unless disease-free for \\>1 year.\n5. Diagnosis of MPN, CMML, or overlap MDS\u002FMPN per WHO classification.\n6. Any condition or concomitant treatment that may impair absorption of orally administered study intervention.\n7. Uncontrolled infection or severe organ dysfunction.\n8. Concomitant intercurrent illness or condition that, per investigator judgment, would compromise safe participation (e.g., uncontrolled hypertension, uncontrolled seizure, unstable angina, new-onset\u002Fexacerbated cardiac arrhythmia).\n9. Prior treatment with disease-modifying agents (e.g., hypomethylating agents) or immunosuppressive therapy, except prior lenalidomide (permitted).\n10. Treatment with cytotoxic chemotherapy or experimental agents within 4 weeks prior to first dose.\n11. History of stem cell, bone marrow, or solid organ transplant.\n12. Known hypersensitivity to ofirnoflast or its excipients.\n13. Severe renal or hepatic impairment\n14. Inability to swallow tablets.\n15. Participation in another interventional clinical study within 90 days prior to first dose.\n16. QTcF \\>480 ms.\n17. Prior treatment with ofirnoflast.",{"count":139,"type":21},[60],"The primary objective of this study is to evaluate the efficacy and safety of ofirnoflast administered orally once daily in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS) who are transfusion-dependent and have failed one to three prior therapies, in order to identify the optimal dose for continuation into a Phase 3 study.\n\nThe secondary objectives of this study are to evaluate the extended hematologic response to ofirnoflast, to assess the safety and tolerability of ofirnoflast during the dose-selection phase, and to evaluate hematologic improvement with ofirnoflast treatment.",[27,29],[342],"Ofirnoflast, HT-6184, Myelodysplastic syndromes, MDS","2026-07-29",{"date":321,"type":38},{"date":346,"type":21},"2026-10",{"date":348,"type":21},"2028-12",{"name":350,"class":45},"Halia Therapeutics, Inc.",{"id":352,"slug":353,"hasResults":12,"nctId":354,"briefTitle":355,"officialTitle":355,"acronym":4,"eligibilityCriteria":356,"healthyVolunteers":111,"sex":17,"minAge":18,"maxAge":55,"enrollmentInfo":357,"targetDuration":4,"studyType":271,"phases":4,"briefSummary":359,"conditions":360,"keywords":361,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":179,"lastUpdatePostDateStruct":367,"startDateStruct":369,"completionDateStruct":371,"leadSponsor":373,"locationsCount":81},"100484446","collection-of-blood-bone-marrow-skin-saliva-and-stool-samples-from-healthy-volunteers-used-for-comparative-analysis-of-myeloid-malignancies-100484446","NCT05588154","Collection of Blood, Bone Marrow, Skin, Saliva, and Stool Samples From Healthy Volunteers Used for Comparative Analysis of Myeloid Malignancies","* INCLUSION CRITERIA:\n* Age \\>= 18 years old\n* Healthy volunteers; the following confirmed by the Principal Investigator or designees based on recent (within 3 months before study intervention(s))\n* medical history\n* physical exam\n* complete blood count (CBC) within the normal reference range per the reporting clinical laboratory, established published literature and reports, or as deemed acceptable by the medical team based on the age and condition of the volunteer consistent with established clinical standards.\n* The ability of the participant to understand and the willingness to sign a written consent document.\n\nEXCLUSION CRITERIA:\n\n\\- Active illnesses, immunodeficiency, history of opportunistic infection, autoimmune disease, history of or active malignancy, prior organ, bone marrow, or peripheral blood stem cell transplant or antibiotic treatment within 3 months before study intervention(s).\n\nNote: participants with non-melanoma skin cancer or carcinoma in situ of the cervix or breast are eligible.\n\n* Current immunosuppressive medication.\n* Any one of the following symptoms as declared by the participant at least one day per week within 3 months before study intervention(s) (Rome IV criteria \\[36\\])\n* Diarrhea characterized as frequent (\\>2) loose stools\n* Constipation defined as \\\u003C 3 spontaneous bowel movements per week\n* Bloating and\u002For distention\n* Abdominal pain.\n* Participants with a history of the human immunodeficiency virus (HIV), hepatitis C (HCV), or hepatitis B (HBV) as confirmed by a seropositive blood test.\n* Pregnancy confirmed with beta-Human Chorionic Gonadotropin (Beta-HCG) serum or urine pregnancy test performed in women of childbearing potential at screening.\n* Breastfeeding participants.",{"count":358,"type":21},1000,"Background:\n\nMyelodysplastic syndromes (MDS) are disorders of blood stem cells that can develop into blood cancers. Treatment options are limited. To find better treatments, researchers need to better understand how MDS develops. To do that, they must be able to compare biospecimens from people with the disease to those of healthy people.\n\nObjective:\n\nThis study will create a database of biospecimens collected from healthy volunteers.\n\nEligibility:\n\nHealthy people aged 18 and older.\n\nDesign:\n\nParticipants will be screened. They will have a physical exam with blood and urine tests.\n\nUp to 5 types of samples will be collected on 1 or more days within 1 month of screening:\n\nBlood: Blood will be drawn by inserting a needle into a vein.\n\nSaliva: Participants will scrape the insides of their cheeks with a brush.\n\nStool: Participants will be given a container to collect stool at home. They will use a prepaid envelope to mail in the sample.\n\nBone marrow: A sample of the soft tissue inside the bones will be drawn out. The area to be biopsied, usually the lower back, will be numbed. A needle will be inserted through a small cut to remove the sample. Participants' pain will be monitored; additional numbing medicine may be used.\n\nSkin: A piece of skin about 1\u002F6 of an inch across will be cut away. Stitches may be used to close the wound. Participants will return to the clinic to have the stitches removed.\n\nParticipants do not have to provide all of the samples listed. They will give each sample only once.",[27],[362,199,363,364,365,366],"Hematopoietic Stem Cell Malignancies","DNA methyltransferase inhibitors","Treatment-Related Complications","Cytotoxic Therapy","Natural History",{"date":368,"type":38},"2026-07-23",{"date":370,"type":38},"2023-01-11",{"date":372,"type":21},"2052-12-01",{"name":79,"class":80},{"id":375,"slug":376,"hasResults":12,"nctId":377,"briefTitle":378,"officialTitle":378,"acronym":4,"eligibilityCriteria":379,"healthyVolunteers":111,"sex":17,"minAge":380,"maxAge":55,"enrollmentInfo":381,"targetDuration":4,"studyType":271,"phases":4,"briefSummary":383,"conditions":384,"keywords":385,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":179,"lastUpdatePostDateStruct":391,"startDateStruct":392,"completionDateStruct":394,"leadSponsor":396,"locationsCount":81},"100466209","comprehensive-molecular-and-clinical-evaluation-of-pediatric-and-adult-mds-100466209","NCT05350748","Comprehensive Molecular and Clinical Evaluation of Pediatric and Adult MDS","* INCLUSION CRITERIA - MDS Participants\n* Either sex, any age.\n* Histologically or cytologically suspected or confirmed myelodysplastic syndromes (MDS), myelodysplastic syndromes\u002Fmyeloproliferative neoplasms (MDS\u002FMPN), MDS\u002Fmyeloproliferative neoplasm with ringed sideroblasts and thrombocytosis (MDS\u002FMPN-RS-T), myelodysplastic syndromes\u002Fmyeloproliferative neoplasms unclassified (MDS\u002FMPN-U), chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (aCML), secondary acute myeloid leukemia (sAML) with antecedent MDS or MDS\u002FMPN, or participants who have precursor conditions that are associated with a risk of progression to MDS, including but not limited to clonal hematopoiesis of indeterminate potential (CHIP) and clonal cytopenia of unknown significance (CCUS).\n* Participants may have had any amount of prior therapy and may be receiving MDS-directed therapy at time of enrollment.\n* Participants must have an identified primary oncologist, hematologist or generalist outside of NIH who agrees to manage participant care and any diagnostic findings provided by this study.\n\nINCLUSION CRITERIA - Marrow Control Donor Participants\n\n* Either sex, and must be eligible for marrow donation per NIH Clinical Center requirements.\n* No history of hematological malignancies as listed as inclusion in 'Inclusion Criteria - MDS Participants' or current autoimmune disease.\n* Must be scheduled for bone marrow harvest for clinical application (e.g., marrow donation); or, if being evaluated for malignancy, have a clinical bone marrow aspirate scheduled (e.g., to rule out bone marrow involvement).\n\nINCLUSION CRITERIA - All Participants\n\n* Stated willingness to comply with all study procedures and availability for the duration of the study.\n* Ability of participant or parent\u002Fguardian to understand and the willingness to sign a written consent document.\n\nEXCLUSION CRITERIA - All Participants\n\n-Uncontrolled intercurrent illness, psychiatric illness, or other that would limit compliance with study requirements, or at the investigator s discretion.","1 Day",{"count":382,"type":21},1100,"Background:\n\nMyelodysplastic syndromes (MDS) occur when the cells that make blood cells are abnormal. There are limited treatment options for MDS. Researchers want to learn more through this natural history study so they can develop better treatments.\n\nObjective:\n\nTo study the natural course of MDS and MDS\u002Fmyeloproliferative neoplasms (MPN) and collect biological samples that can help researchers understand the disease.\n\nEligibility:\n\nPeople with suspected or confirmed MDS or MDS\u002FMPN. Healthy donors are also needed. They can be people who are scheduled to donate bone marrow at NIH for a relative, or they may be providing bone marrow in another study.\n\nDesign:\n\nParticipants will be screened with a medical history.\n\nParticipants will have a physical exam. They will give blood and urine samples. They will discuss their symptoms, medications, and ability to perform their normal activities. They will complete surveys about how they are feeling.\n\nParticipants will have a bone marrow biopsy. A needle will be inserted through a small cut. Bone marrow will be removed. A small piece of bone may be removed.\n\nParticipants may have an optional skin biopsy.\n\nParticipants may give optional saliva and stool samples. They may collect these samples at home and mail them to NIH.\n\nParticipants may undergo optional apheresis. One or two needles or intravenous (IV) lines will be placed in their arm, neck, or groin veins. Blood will be removed. A machine will separate out the white cells. The rest of the blood will be returned to the participant.\n\nParticipants will be contacted for follow-up once a year for up to 20 years.\n\nHealthy donors will have marrow collected for this study during their scheduled procedure with no follow-up.",[27],[386,387,388,389,390,199,366],"Heterogenous Stem Cell Disorders","Gene Mutations","Dysplasia","cytopenias","Malignancies",{"date":368,"type":38},{"date":393,"type":38},"2022-08-18",{"date":395,"type":21},"2042-05-01",{"name":79,"class":80},{"id":398,"slug":399,"hasResults":12,"nctId":400,"briefTitle":401,"officialTitle":402,"acronym":4,"eligibilityCriteria":403,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":404,"targetDuration":4,"studyType":22,"phases":406,"briefSummary":407,"conditions":408,"keywords":409,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":155,"lastUpdatePostDateStruct":413,"startDateStruct":414,"completionDateStruct":416,"leadSponsor":418,"locationsCount":81},"100476367","phase-2-statins-in-patients-with-clonal-cytopenia-of-undetermined-significance-ccus-and-myelodysplastic-syndromes-mds-100476367","NCT05483010","Statins in Patients With Clonal Cytopenia of Undetermined Significance (CCUS) and Myelodysplastic Syndromes (MDS)","Pilot Study of Statins in Patients With Clonal Cytopenia of Undetermined Significance (CCUS) and Myelodysplastic Syndromes (MDS)","Inclusion Criteria:\n\n* Diagnosis of CCUS or lower-risk MDS as defined below:\n\n  * CCUS is defined as the presence of somatic mutation(s) in recurrently mutated genes identified through the clinical MyeloSeq assay with a VAF ≥ 2% in the absence of bone marrow morphology\u002Fcytogenetic changes diagnostic of MDS PLUS unexplained persistent cytopenia in at least one lineage for at least 6 months:\n\n    * Hemoglobin \\\u003C 11.3 g\u002FdL in females or \\\u003C 13 g\u002FdL in males\n    * ANC \\\u003C 1.8 x 109\u002FL\n    * Platelets \\\u003C 150 x 109\u002FL\n  * MDS is defined using the WHO 2016 definition and classified into lower-risk if IPSS-R score is ≤ 3.5 . Lower-risk MDS will be required to have at least one mutation in a recurrent mutated gene with a VAF ≥ 2%.\n* Patient must be transfusion independent.\n* At least 18 years of age.\n* Ability to understand and willingness to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable).\n\nExclusion Criteria:\n\n* CCUS patients with cytogenetic change alone.\n* Current or prior use of disease-modifying therapy (e.g., lenalidomide, Luspatercept, Imitelstat, HMAs, venetoclax) with any dose within the last 3 months, with the exception of concurrent use of erythropoetin stimulating agents\n* Prior use of a statin within 1 year prior to start of treatment.\n* A history of other malignancy with the exception of malignancies for which all treatment was completed at least 2 years before registration and the patient has no evidence active of disease.\n* Currently receiving any investigational agent for CCUS\u002FMDS. The minimum interval between the last dose of investigational agent used for CCUS\u002FMDS and Day 1 of this trial should be 5 half-lives of the investigational agent.\n* A history of allergic reactions or intolerance attributed to compounds of similar chemical or biologic composition to atorvastatin, rosuvastatin, any other statin, or other agents used in the study.\n* Uncontrolled intercurrent illness including, but not limited to, symptomatic infection, sepsis, or active liver disease (acute liver failure, decompensated cirrhosis, or persistent elevation in ALT or AST \\> 3 x ULN), or any other comorbidity that would preclude statin use based on FDA recommendation.\n* Pregnant and\u002For breastfeeding. Women of childbearing potential must have a negative pregnancy test within 14 days of study entry.\n* Patients with HIV and HCV are not eligible for the trial if they are concomitantly receiving active treatment for HIV\u002FHCV given the concern for potential drug interactions. The minimum interval between the last dose of antiviral and enrollment into the study should be 28 days or 5 half-lives of the antiviral drug, whichever is longer. The liver function profile of eligible HIV\u002FHCV patients must be within the acceptable limits.",{"count":405,"type":21},16,[60],"Patients with clonal cytopenia of undetermined significance (CCUS) and lower-risk myelodysplastic syndromes (MDS) have a life expectancy of 5 to 10 years. Mortality in these patients results from progression of disease to higher-risk MDS or acute myeloid leukemia (AML) and cardiovascular events. Currently there are no FDA-approved treatments with the potential to improve survival of patients with CCUS and lower-risk MDS. Statins are an appealing class of drugs to consider in this situation as preclinical data support their potential to suppress progression of myeloid malignancy, and they have a well-established role in prevention of major cardiovascular events. This is a pilot study to explore the role of statins in treatment of patients with CCUS and lower-risk MDS. In this study, change in inflammatory biomarkers and variant allele frequency (VAF) of somatic mutations will be used as a surrogate marker of response to statin therapy. The hypothesis is that the use of statins at diagnosis of CCUS or lower-risk MDS will reduce inflammation and delay or prevent the expected increase in the VAF of somatic mutations over time.",[118,27],[410,33,411,412],"CCUS","Statins","Inflammation",{"date":368,"type":38},{"date":415,"type":38},"2024-02-19",{"date":417,"type":21},"2028-05-31",{"name":419,"class":128},"Washington University School of Medicine",{"id":421,"slug":422,"hasResults":12,"nctId":423,"briefTitle":424,"officialTitle":425,"acronym":4,"eligibilityCriteria":426,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":427,"targetDuration":4,"studyType":22,"phases":429,"briefSummary":430,"conditions":431,"keywords":432,"overallStatus":274,"whyStopped":4,"lastUpdateSubmitDate":447,"lastUpdatePostDateStruct":448,"startDateStruct":449,"completionDateStruct":451,"leadSponsor":453,"locationsCount":4},"100648595","phase-3-a-study-to-investigate-the-efficacy-and-safety-of-roxadustat-fg-4592-for-treating-anemia-in-participants-with-myelodysplastic-syndromes-mds-100648595","NCT07722988","A Study to Investigate the Efficacy and Safety of Roxadustat (FG-4592) for Treating Anemia in Participants With Myelodysplastic Syndromes (MDS)","A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Investigating the Efficacy and Safety of Roxadustat (FG-4592) for Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) in Participants With High Red Blood Cell Transfusion Burden","Key Inclusion Criteria:\n\n* Diagnosis of MDS according to World Health Organization (WHO) criteria confirmed by bone marrow aspirate and biopsy within 12 weeks before randomization.\n* Revised International Prognostic Scoring System (IPSS-R) very low, low, or intermediate risk MDS\n* HTB: Participants requiring ≥ 4 units of packed red blood cell (pRBC) in two consecutive 8-week periods prior to randomization\n* Refractory to, intolerant to, or ineligible for prior erythropoiesis-stimulating agents (ESAs).\n* Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2\n\nKey Exclusion Criteria:\n\n* Pregnant or breastfeeding females\n* Participant has any significant medical illness or is considered vulnerable by local regulations\n* Prior allogeneic or autologous stem cell transplant\n* Major surgery within 8 weeks prior to randomization.\n\nNote: Other protocol-defined inclusion and exclusion criteria may apply.",{"count":428,"type":21},201,[24],"The study is designed to determine the efficacy and safety of roxadustat for the treatment of anemia due to very low, low, or intermediate risk MDS in participants with high transfusion burden (HTB).",[27],[433,434,435,33,436,437,95,438,29,439,440,441,442,443,444,445,446],"Roxadustat","FG-4592","LR-MDS","Myelodysplastic syndromes","Oral","High transfusion burden","Red blood cell","Transfusion independence","Hemoglobin","Kyntra Bio","Kyntra","FibroGen","KYNB","ESA","2026-07-20",{"date":368,"type":38},{"date":450,"type":21},"2027-04",{"date":452,"type":21},"2032-12",{"name":442,"class":45},{"id":455,"slug":456,"hasResults":12,"nctId":457,"briefTitle":458,"officialTitle":459,"acronym":4,"eligibilityCriteria":460,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":461,"enrollmentInfo":462,"targetDuration":464,"studyType":271,"phases":4,"briefSummary":465,"conditions":466,"keywords":468,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":470,"lastUpdatePostDateStruct":471,"startDateStruct":473,"completionDateStruct":475,"leadSponsor":477,"locationsCount":479},"100436866","genomically-profiling-collecting-archiving-and-distributing-hematologic-malignancy-specimens-100436866","NCT04968834","Genomically Profiling, Collecting, Archiving and Distributing Hematologic Malignancy Specimens","Protocol For Genomically Profiling, Collecting, Archiving and Distributing Blood and Bone Marrow Specimens From Children and Young Adults With Hematologic Malignancy","Inclusion Criteria:\n\n* Age: birth to \\\u003C 30 years of age\n* Diagnosis:\n\n  \\-- Patient with acute leukemia, chronic leukemia, MDS\u002FAML, myelodysplastic syndrome or myeloproliferative syndromes. Disease can be newly diagnosed or relapsed\u002Frefractory.\n* Pathology Criteria:\n\n  \\-- Histologic confirmation of leukemia or myelodysplastic syndrome (MDS) or myeloproliferative syndrome (MPS) at the time of diagnosis or recurrence\n* Specimen Criteria:\n\n  * Sufficient sample available for genomic profiling OR bone marrow aspirate\u002Fblood draw planned for clinical care which is anticipated to allow collection of minimum specimen for testing (See Section 6.1 for description of specimen requirements)\n\nExclusion Criteria:\n\n\\- Insufficient leukemia or MDS specimen available for profiling from diagnosis or recurrence (See Section 6.1); or bone marrow evaluations NOT planned for clinical care; or peripheral blast percentage \\\u003C20%, or clinical blood draw not planned","30 Years",{"count":463,"type":21},300,"5 Years","This research study is a genomic profiling and repository study for children and young adults who have leukemia, myelodysplastic syndrome (MDS) or myeloproliferative syndrome (MPS). Genes are the part of cells that contain the instructions which tell cells how to make the right proteins to grow and work. Genes are composed of DNA letters that spell out these instructions. Genomic profiling helps investigators understand why the disease develops and the instructions that led to its development. Understanding the genetic factors of the disease can also help investigator understand why the disease of some people can respond to certain therapies differently than others.\n\nThe genomic profiling will be performed using bone marrow and blood samples that either have already been obtained during a previous clinical procedure or will be obtained at the time of a scheduled clinical procedure. Studying the genetic information in the cells of these samples will provide information about the origin, progression, and treatment of leukemia and myeloproliferative syndromes and myelodysplastic syndrome. Storing the bone marrow and blood samples will allow for additional research and genomic assessments to be performed in the future.",[320,27,467],"Myeloproliferative Syndrome",[320,27,469],"Myeloproliferative syndrome (MPS)","2026-07-15",{"date":472,"type":38},"2026-07-16",{"date":474,"type":38},"2021-06-11",{"date":476,"type":21},"2033-06",{"name":478,"class":128},"Dana-Farber Cancer Institute",8,{"id":481,"slug":482,"hasResults":12,"nctId":483,"briefTitle":484,"officialTitle":485,"acronym":4,"eligibilityCriteria":486,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":487,"targetDuration":4,"studyType":22,"phases":489,"briefSummary":490,"conditions":491,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":493,"lastUpdatePostDateStruct":494,"startDateStruct":496,"completionDateStruct":498,"leadSponsor":500,"locationsCount":81},"100561767","phase-1-hm2023-05-gtb-3650-trike-for-high-risk-mds-and-rr-aml-100561767","NCT06594445","HM2023-05: GTB-3650 Trike for High Risk MDS and R\u002FR AML","HM2023-05: GTB-3650 (Anti-CD16\u002FIL-15\u002FAnti-CD33) Tri-Specific Killer Engager (TriKE®) for the Treatment of High Risk Myelodysplastic Syndromes (MDS) and Refractory\u002FRelapsed Acute Myeloid Leukemia (AML)","Inclusion Criteria:\n\n* Diagnosis of a high risk myelodysplastic syndromes (MDS), treatment related MDS, OR relapsed\u002Frefractory acute myelogenous leukemia (AML).\n* Absolute lymphocyte count (ALC) ≥ 200 cells\u002FµL OR absolute circulating CD56+\u002FCD3- NK cell count \\>25 cells\u002FµL within the 14 days prior to Cycle 1 Day 1.\n* Peripheral blasts ≤20,000 at the time of treatment start. Hydroxyurea may be used up to Day 1 of the 1st cycle to achieve this threshold and continued for the 1st two weeks of Cycle 1 to maintain it.\n* Karnofsky performance status ≥ 70%\n* Adequate organ function within 14 days (30 days for cardiac) of Cycle 1 Day 1\n* Sexually active persons of childbearing potential or persons with partners of childbearing potential must agree to use a highly effective form of contraception during study treatment and for at least 4 months after the last dose of GTB-3650. Non-childbearing is defined as \\>1 year postmenopausal or surgically sterilized.\n\n-For the Dose Finding Component Only: Must agree to stay within a 60- minute drive of the Study Center through the Cycle 1 Day 29 visit (end of the Dose Limiting Toxicity period).\n\n* Provides voluntary written consent prior to the performance of any research related activity.\n* Pulmonary: room air 0 2 saturation at ≥ 95%\n\nExclusion Criteria:\n\n* Pregnant or breast-feeding. The effect of GTB-3650 TriKE on the fetus is unknown. Persons of childbearing potential must have a negative serum or urine test within 7 days prior to Cycle 1 Day 1 to rule out pregnancy.\n* A candidate for hematopoietic stem cell transplant (HSCT) or newly relapsed after HSCT (e.g. no post-HSCT therapy given).\n* Bi-phenotypic acute leukemia or mixed lineage leukemia.\n* Acute promyelocytic leukemia (APL).\n* No anticancer therapy within 14 days of Cycle 1 Day 1; any AEs from therapy given prior must have resolved to Grade 1 or baseline\n* New or progressive pulmonary infiltrates on screening chest x-ray or chest CT scan unless cleared for study by Pulmonary. Infiltrates attributed to infection must be stable\u002Fimproving with associated clinical improvement after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections).\n* Active systemic infection requiring parenteral antibiotic therapy. Any prior systemic infections must have resolved following optimal therapy.\n* Known history of HIV.\n* Active Hepatitis B or Hepatitis C (virus detectable by PCR) - chronic asymptomatic viral hepatitis is allowed.\n* Positive test results from chronic hepatitis B infection (defined as positive HBsAg serology) and\u002For positive test results for hepatitis C (HCV antibody serology test).\n* Prior malignancy except for the following: adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated Stage I or II cancer currently in complete remission, or any other cancer from which the patient has been disease-free for 1 year\n* Active central nervous system (CNS) malignancy or symptoms of CNS spread or administration of IT chemotherapy within 14 days prior to Day 1.\n* Extramedullary disease causing symptoms and\u002For involving the CNS or spinal canal - asymptomatic extramedullary disease outside the CNS and spinal canal is eligible provided the marrow has measurable disease.\n* Known autoimmune disease requiring active treatment with steroids or other immunosuppressive medications within 14 days before Cycle 1 Day. Inhaled or topical steroids, and adrenal replacement steroid doses ≤ 10 mg daily prednisone equivalent, are permitted in the absence of active autoimmune disease.\n* Persons with a condition requiring systemic treatment with steroids (\\> 10 mg daily prednisone equivalent) or other immunosuppressive medications within 14 days before Cycle 1 Day 1.\n* The potential risk of QT\u002FQTc prolongation is unknown in humans receiving\n\nTriKE therefore either of the following is an exclusion criteria:\n\n* QTc interval \\> 480 msec at screening\n* A family history of long QT syndrome\n* Psychiatric illness\u002Fsocial situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements.\n* Other illness or a medical issue that, in the judgement of the enrolling Investigator, would exclude the patient from participating in this study.",{"count":488,"type":21},45,[59],"This is a Phase I dose finding study of GTB-3650 (anti-CD16\u002FIL-15\u002Fanti-CD33) Tri-Specific Killer Engager (TriKE®) for the treatment of select CD33-expressing refractory\u002Frelapsed myeloid malignancies in adults ≥ 18 years of age who are not a candidate for potentially curative therapy, including hematopoietic stem cell transplantation, and are refractory to, intolerant of, or ineligible for therapy options that are known to provide clinical benefit. The hypothesis is GTB-3650 TriKE will induce natural killer (NK) cell function by targeting malignant cells, as well as, CD33+ myeloid derived suppressor cells (MDSC) which contribute to a tumor induced immunosuppression. Because CD16 is the most potent activating receptor on NK cells, this single agent may induce a targeted antiCD33+ tumor response",[492,199,27],"Myeloid Malignancy","2026-07-13",{"date":495,"type":38},"2026-07-14",{"date":497,"type":38},"2024-11-19",{"date":499,"type":21},"2027-10-30",{"name":501,"class":128},"Masonic Cancer Center, University of Minnesota",{"id":503,"slug":504,"hasResults":12,"nctId":505,"briefTitle":506,"officialTitle":507,"acronym":4,"eligibilityCriteria":508,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":509,"enrollmentInfo":510,"targetDuration":4,"studyType":22,"phases":512,"briefSummary":513,"conditions":514,"keywords":4,"overallStatus":274,"whyStopped":4,"lastUpdateSubmitDate":516,"lastUpdatePostDateStruct":517,"startDateStruct":518,"completionDateStruct":520,"leadSponsor":522,"locationsCount":4},"100645643","phase-2-dexamethasone-intravenous-injection-of-human-immunoglobulin-and-increased-infusion-of-mononuclear-cells-to-reduce-donor-specific-antibodies-in-haploidentical-hematopoietic-stem-cell-transplantation-100645643","NCT07698080","Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploidentical Hematopoietic Stem Cell Transplantation","Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploid Hematopoietic Stem Cell Transplantation: a Prospective, Multicenter Study","Inclusion Criteria:\n\n1. Diagnosis of benign or malignant hematological diseases (including leukemia, lymphoma, thalassemia, aplastic anemia, myelodysplastic syndromes, etc.) confirmed by NCCN guidelines, and determined by the investigator to require allogeneic hematopoietic stem cell transplantation.\n2. Eastern Cooperative Oncology Group (ECOG) performance status ≤ 3.\n3. Donor-specific antibody (DSA) mean fluorescence intensity (MFI) \\> 500.\n4. Age between 18 and 65 years (age limits are also captured separately in the eligibility module).\n5. Body weight between 40 kg and 100 kg.\n6. No other suitable donor available (i.e., no DSA-negative related donor or unrelated donor).\n\nExclusion Criteria:\n\n1. Patients unsuitable for transplantation or without willingness to undergo transplantation, or diagnosed with non-hematological diseases.\n2. Estimated life expectancy \\\u003C 1 month.\n3. Known allergy to any drug or intervention used in the study regimen.\n4. Pregnancy, lactation, active severe infection, or severe major organ dysfunction.\n5. Severe psychiatric or neurological disorders that may affect the ability to provide informed consent and\u002For to report adverse events or comply with observation.\n6. Refusal or inability to sign the informed consent form.","65 Years",{"count":511,"type":21},60,[60],"This study tests whether a combination of three treatments - dexamethasone (a steroid), intravenous immunoglobulin (IVIG, a protein that helps the immune system), and an extra dose of donor mononuclear cells - can safely lower harmful antibodies called donor-specific antibodies (DSA) in patients who need a stem cell transplant from a half-matched (haploidentical) family donor.\n\nIn these transplants, DSA are antibodies made by the patient's own body that attack the donor's stem cells. If DSA levels are high, the transplant is more likely to fail - the donor cells may not \"take\" (engraft). Currently, there is no single, simple, and reliable way to reduce DSA, and many existing methods have drawbacks.\n\nBased on the investigators' earlier experience in 11 patients, this three-part approach seemed to work well. All patients successfully engrafted, and DSA levels dropped quickly. Now the study team want to confirm these results in a larger, prospective, multicenter study.\n\nThe investigators plan to enroll 60 patients aged 18-65 with blood cancers or other blood disorders who need a haploidentical transplant, have DSA levels above 500 MFI (a measure of antibody strength), and have no other suitable donor available. Participants will receive:\n\n* Dexamethasone (25 mg\u002Fm²) for 4 days before transplant,\n* IVIG (1 g\u002Fkg) one day before transplant,\n* Extra mononuclear cells on transplant day - the extra amount depends on how high their DSA level is (low, medium, or high).\n\nThe main goal is to see how many patients have primary graft failure (when the donor cells never engraft). The study team will also measure how long it takes for blood counts to recover, rates of graft-versus-host disease, survival, and side effects. All participants will be followed for 1 year.\n\nThis study will help the investigators find out whether this combination is a safe, simple, and effective way to improve transplant success for patients with DSA who have no other donor options.",[320,198,515,72,27],"Thalassemia","2026-07-11",{"date":495,"type":38},{"date":519,"type":21},"2026-07-03",{"date":521,"type":21},"2028-07-03",{"name":523,"class":128},"Hematology department of the 920th hospital",{"id":525,"slug":526,"hasResults":12,"nctId":527,"briefTitle":528,"officialTitle":529,"acronym":4,"eligibilityCriteria":530,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":531,"targetDuration":4,"studyType":22,"phases":533,"briefSummary":534,"conditions":535,"keywords":537,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":543,"lastUpdatePostDateStruct":544,"startDateStruct":545,"completionDateStruct":547,"leadSponsor":549,"locationsCount":551},"100421694","phase-1-a-study-of-bgb-11417-in-participants-with-myeloid-malignancies-100421694","NCT04771130","A Study of BGB-11417 in Participants With Myeloid Malignancies","A Phase 1b\u002F2, Open-Label, Dose Finding, and Expansion Study of the Bcl-2 Inhibitor BGB-11417 in Patients With Myeloid Malignancies","Key Inclusion Criteria:\n\n1. Confirmed diagnosis of one of the following by 2016 World Health Organization criteria:\n\n   * AML, nonacute promyelocytic leukemia\n   * MDS\n   * MDS\u002FMPN\n2. Eastern Cooperative Oncology Group performance status of 0 to 2.\n3. Adequate organ function defined as:\n\n   * Creatinine clearance ≥ 50 milliliters\u002Fminute (mL\u002Fmin) (or between 30 and 49 mL\u002Fmin in unfit AML cohort)\n   * Adequate liver function\n4. Life expectancy of \\> 12 weeks.\n5. Ability to comply with the requirements of the study.\n\nKey Exclusion Criteria:\n\n1. A diagnosis of acute promyelocytic leukemia.\n2. History of prior malignancy, with the exception of either a history of MDS or MDS\u002FMPN that has transformed to AML, or other prior malignancy that was treated with a full curative intent and no evidence of recurrence within the past 2 years (eg, localized skin cancer, superficial bladder cancer, carcinoma in situ of the cervix or breast, or localized Gleason score ≤ 6 prostate cancer)\n3. Antecedent MPN including myelofibrosis, essential thrombocytosis, polycythemia vera, or chronic myelogenous leukemia with or without BCR-ABL1 translocation and AML with BCR-ABL1 translocation.\n4. Prior therapy with a B-cell lymphoma-2 inhibitor\n5. Known central nervous system involvement by leukemia.\n\nNote: Other protocol defined Inclusion\u002FExclusion criteria may apply.",{"count":532,"type":21},260,[59,60],"The study will determine the safety, tolerability, recommended Phase 2 dose (RP2D) and preliminary efficacy of BGB-11417 as monotherapy and in combination with azacitidine in participants with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)or MDS\u002Fmyeloproliferative neoplasm (MPN) .",[199,27,536],"Myelodysplastic\u002FMyeloproliferative Neoplasm",[538,539,540,541,33,542],"BGB-11417","Azacitidine","Posaconazole","AML","MDS\u002FMPN","2026-07-10",{"date":493,"type":38},{"date":546,"type":38},"2021-05-24",{"date":548,"type":21},"2028-02-08",{"name":550,"class":45},"BeOne Medicines",46,{"id":553,"slug":554,"hasResults":12,"nctId":555,"briefTitle":556,"officialTitle":557,"acronym":4,"eligibilityCriteria":558,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":559,"enrollmentInfo":560,"targetDuration":4,"studyType":22,"phases":561,"briefSummary":562,"conditions":563,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":570,"lastUpdatePostDateStruct":571,"startDateStruct":573,"completionDateStruct":575,"leadSponsor":577,"locationsCount":81},"100347932","cd45ra-depleted-peripheral-stem-cell-addback-for-viral-or-fungal-infections-post-tcrcd19-depleted-hsct-100347932","NCT03810196","CD45RA Depleted Peripheral Stem Cell Addback for Viral or Fungal Infections Post TCRαβ\u002FCD19 Depleted HSCT","CD45RA Depleted Peripheral Stem Cell Addback for Patients at Risk for Viral or Fungal Infections Post TCRαβ\u002FCD19 Depleted Hematopoietic Stem Cell Transplant","Inclusion Criteria:\n\n1. Age: Patients \\\u003C25 years.\n2. First allogeneic HSCT only.\n3. Disease eligibility: Acute leukemias at high risk for relapse including positive minimal residual disease at end consolidation, high risk cytogenetics, or relapse. Hematologic malignancies including: acute myeloid leukemia, myelodysplastic syndromes, acute lymphoblastic leukemia, mixed lineage or bi-phenotypic leukemia, lymphoblastic or Burkitts, juvenile myelomonocytic leukemia\n4. Evaluation of organ and infectious status as per our Bone Marrow Transplant standard operating procedure (BMT SOP).\n5. Signed consent by parent\u002Fguardian or able to give consent if \\>18 years.\n\nExclusion Criteria:\n\n1. Patients who do not meet institutional disease, organ or infectious criteria\n2. No suitable donor available for mobilized peripheral stem cells\n3. Patients with genetic disorders including Fanconi anemia, Kostmann syndrome, dyskeratosis congenital or other DNA repair defects.\n4. Patients with Hodgkin lymphoma or non-Burkitts, non-lymphoblastic lymphoma\n5. Pregnant Participants\n\nDonor selection and eligibility\n\n1. Unrelated donor meets National Marrow Donor Program criteria for donation\n2. HLA testing\u002Fmatching\n3. Donor must be willing to undergo granulocyte colony stimulating factor (GCSF) mobilization and peripheral blood stem cell collection","25 Years",{"count":139,"type":21},[169],"The major morbidities of allogeneic hematopoietic stem cell transplant with non-human leukocyte antigen (HLA) matched siblings are graft vs host disease (GVHD) and life threatening infections. T depletion of the donor hematopoietic stem cell graft is effective in preventing GVHD, but immune reconstitution is slow, increasing the risk of infections. An addback of donor CD45RA (naive T cells) depleted cells may improve immune reconstitution and help decrease the risk of infections.",[564,199,27,565,566,567,568,569],"Acute Leukemia","Acute Lymphoblastic Leukemia","Mixed Lineage Leukemia","Lymphoblastic Lymphoma","Burkitt Lymphoma","Juvenile Myelomonocytic Leukemia","2026-07-05",{"date":572,"type":38},"2026-07-08",{"date":574,"type":38},"2019-03-01",{"date":576,"type":21},"2028-07",{"name":578,"class":128},"Children's Hospital of Philadelphia",{"id":580,"slug":581,"hasResults":12,"nctId":582,"briefTitle":583,"officialTitle":584,"acronym":4,"eligibilityCriteria":585,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":586,"targetDuration":4,"studyType":22,"phases":588,"briefSummary":589,"conditions":590,"keywords":592,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":614,"lastUpdatePostDateStruct":615,"startDateStruct":617,"completionDateStruct":619,"leadSponsor":620,"locationsCount":622},"100609581","phase-2-trial-of-orca-t-following-reduced-intensity-or-nonmyeloablative-conditioning-in-patients-with-acute-myeloid-leukemia-or-myelodysplastic-syndrome-100609581","NCT07216443","Trial of Orca-T Following Reduced Intensity or Nonmyeloablative Conditioning in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome","A Phase 2 Trial of Orca-T Following Reduced Intensity or Nonmyeloablative Conditioning in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome","Inclusion Criteria:\n\n1. Age ≥18 years at the time of enrollment\n2. Diagnosed with 1 of the following diseases:\n\n   1. Acute myeloid, or mixed phenotype leukemia in complete remission (CR) or CR with incomplete hematologic recovery (CRi), with or without the presence of known minimal residual disease.\n   2. Myelodysplastic syndrome that is indicated for alloHCT per the 2017 International Expert Panel recommendations and\u002For therapy-related\u002Fsecondary MDS as defined by the World Health Organization (WHO) classification of myeloid malignancies, with ≤10% blast burden in the bone marrow.\n3. Planned to undergo 1 of the following preparative regimens as per Investigator discretion:\n\n   1. RIC cohort: Planned RIC-alloHCT including RIC regimen with TBI\u002Fthiotepa\u002Ffludarabine\n   2. NMA cohort: Planned NMA-alloHCT including NMA regimen with fludarabine\u002Fcyclophosphamide\u002FTBI\n4. Identified related or unrelated donor who is an 8\u002F8 match for HLA-A, -B, -C, and -DRB1\n5. Estimated glomerular filtration rate ≥30 mL\u002Fminute\n6. Cardiac ejection fraction at rest ≥40% or shortening fraction of ≥22% by echocardiogram or radionuclide scan (MUGA)\n7. Diffusing capacity of the lung for carbon monoxide (adjusted for hemoglobin) ≥40%\n8. Negative serum or urine β-HCG test in persons of childbearing potential\n9. Alanine transaminase (ALT)\u002Faspartate transaminase (AST) \\\u003C5 times the upper limit of normal (ULN)\n10. Total bilirubin \\\u003C3 × ULN\n11. Deemed ineligible for a fully myeloablative alloHCT per assessment of the principal investigator\n\nExclusion Criteria:\n\n1. Prior alloHCT\n2. Currently receiving corticosteroids or other immunosuppressive therapy. Topical corticosteroids or oral systemic corticosteroid doses less than or equal to 10 mg\u002Fday are allowed.\n3. Planned donor lymphocyte infusion (DLI)\n4. Planned pharmaceutical in vivo or ex vivo T-cell depletion\n5. Recipient-positive antidonor HLA antibodies against a mismatched allele in the selected donor\n6. Karnofsky performance score \\\u003C60%\n7. For RIC cohort only: HCT-Specific Comorbidity Index (HCT-CI) ≥6\n8. Uncontrolled bacterial, viral, or fungal infection (currently taking antimicrobial therapy and with progression or no clinical improvement) at the time of enrollment\n9. Seropositive for HIV-1 or -2, HTLV-1 or -2, hepatitis B surface antigen, or HCV antibody unless previously treated with curative therapy and are HCV NAT negative\n10. Known allergy or hypersensitivity to or intolerance of tacrolimus\n11. Documented allergy or hypersensitivity to iron dextran or bovine, murine, algal, or Streptomyces avidinii proteins\n12. Any uncontrolled autoimmune disease requiring active immunosuppressive treatment\n13. Concurrent malignancy within 1 year except nonmelanoma skin cancer that has been curatively resected\n14. Psychosocial circumstances that preclude the participant being able to go through transplantation or participate responsibly in follow-up care\n15. Persons who are pregnant or breastfeeding\n16. Person of childbearing potential (POCBP) or men who have sexual contact with POCBP who are unwilling to use effective forms of birth control or abstinence for 1 year after transplantation.\n17. Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's or medical monitor's judgment, precludes the recipient's safe participation in and completion of the trial or which could affect compliance with the protocol or interpretation of results",{"count":587,"type":21},80,[60],"This study will evaluate the safety, tolerability, and efficacy of Orca-T in participants undergoing reduced intensity or non-myeloablative allogeneic hematopoietic cell transplantation (alloHCT) for hematologic malignancies. Orca-T is an allogeneic stem cell and T-cell immunotherapy biologic manufactured for each patient (transplant recipient) from the mobilized peripheral blood of a specific, unique donor. It is composed of purified hematopoietic stem and progenitor cells (HSPCs), purified regulatory T cells (Tregs), and conventional T cells (Tcons).",[173,27,591],"Mixed Phenotype Acute Leukemia",[173,27,591,593,594,595,596,597,598,599,600,601,602,603,604,605,606,607,608,609,320,610,611,612,613],"Therapy-Related Myelodysplastic Syndrome","Hematopoietic Stem Cell Transplantation","Humans","Graft vs Host Disease","SERENE-T","ORCA-T","Disease","Pathologic Processes","Neoplasms by Histologic Type","Neoplasms","Hematologic Diseases","Bone Marrow Diseases","Precancerous Conditions","Neoplasms by Site","Disease Attributes","Immunoproliferative Disorders","Immune System Diseases","Preleukemia","Hematologic Neoplasms","Syndrome","Acute Disease","2026-07-02",{"date":616,"type":38},"2026-07-07",{"date":618,"type":38},"2025-12-09",{"date":348,"type":21},{"name":621,"class":45},"Orca Biosystems, Inc.",6,{"id":624,"slug":625,"hasResults":12,"nctId":626,"briefTitle":627,"officialTitle":628,"acronym":4,"eligibilityCriteria":629,"healthyVolunteers":12,"sex":17,"minAge":630,"maxAge":55,"enrollmentInfo":631,"targetDuration":4,"studyType":271,"phases":4,"briefSummary":633,"conditions":634,"keywords":636,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":614,"lastUpdatePostDateStruct":642,"startDateStruct":644,"completionDateStruct":646,"leadSponsor":648,"locationsCount":81},"100594416","study-of-individuals-and-families-with-aberrations-in-ddx41-or-similar-cancer-predisposition-variants-100594416","NCT07019155","Study of Individuals and Families With Aberrations in DDX41 or Similar Cancer Predisposition Variants","Longitudinal Study of Individuals and Families With Aberrations in DDX41 or Similar Cancer Predisposition Variants","* INCLUSION CRITERIA:\n* Age \\> 1 month old.\n* Participants with history of aberrations that affect the DDX41 gene, DDX41 RNA, or DDX41 protein (Cohorts 1-2)\n\nOR\n\nParticipants with history of aberrations in another HHM variant (Cohort 3)\n\nOR\n\nParticipants with history of absence of HHM variants, who have first or second degree relative with history of confirmed or suspected HHM variant(s) per participant report (Cohort 4).\n\n* Participants must have an identified healthcare provider outside of NIH who manages participant care, and any diagnostic clinical findings provided by this study.\n* Ability of participant or parent\u002Fguardian to understand and the willingness to sign a written consent document.\n\nEXCLUSION CRITERIA:\n\nNone.","1 Month",{"count":632,"type":21},510,"Background:\n\nHereditary hematopoietic malignancy (HHM) syndromes are a group of inherited disorders that raises the risk of blood cancers. Many people with HHMs have changes in a gene (DDX41) that makes it more likely that they will develop myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), or other cancers. This natural history study will explore the link between HHM syndromes and these diseases.\n\nObjective:\n\nTo study the link between HHM and MDS\u002FAML.\n\nEligibility:\n\nPeople aged 1 month and older with HHM. Relatives with HHM are also needed.\n\nDesign:\n\nParticipants aged 3 years and older will have 1 initial clinic visit with the option to follow-up annually. They will undergo these procedures:\n\nThey will have a physical exam with blood and urine tests.\n\nThey may give samples of saliva, stool, nails, and skin.\n\nTheir ability to do normal activities will be reviewed.\n\nSome may have a bone marrow biopsy: A tissue sample will be drawn from inside a bone.\n\nThey may answer questions about their health and family medical history.\n\nParticipants younger than 3 years, and those who cannot come to the clinic, will be contacted by phone or email. Their samples may be collected locally and sent to researchers.\n\nFor participants who have changes in their DDX41 gene: Researchers will contact them or their primary care provider once a year for 10 years. Researchers will check on participants health and collect any new test results. Some may be asked to send new samples.\n\nParticipants who do not have changes in their DDX41 gene may be contacted yearly, or less often, for 10 years.\n\nSome participants may be asked to return to the clinic if needed.",[635,27,199],"Germline Mutation",[637,638,33,541,639,640,641],"DEAD-box helicase 41 (DDX41)","germline mutations","Germline Predisposition Syndromes","Hereditary Hematopoietic Malignancy","Cancer Predisposition",{"date":643,"type":38},"2026-07-06",{"date":645,"type":38},"2025-07-24",{"date":647,"type":21},"2035-06-15",{"name":79,"class":80},{"id":650,"slug":651,"hasResults":12,"nctId":652,"briefTitle":653,"officialTitle":654,"acronym":4,"eligibilityCriteria":655,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":656,"enrollmentInfo":657,"targetDuration":4,"studyType":22,"phases":659,"briefSummary":660,"conditions":661,"keywords":676,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":614,"lastUpdatePostDateStruct":685,"startDateStruct":686,"completionDateStruct":688,"leadSponsor":690,"locationsCount":81},"100501157","phase-2-allo-hsct-using-ric-and-ptcy-for-hematological-diseases-100501157","NCT05805605","Allo HSCT Using RIC and PTCy for Hematological Diseases","Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning (RIC) With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases","Inclusion Criteria:\n\n* Age 0 to 75 years of age with Karnofsky score ≥ 70% (≥ 16 years) or Lansky score ≥ 50 (\\\u003C 16 years).\n* 5\u002F6 or 6\u002F6 related donor, OR a 7-8\u002F8 HLA-A, B, C, DRB1 allele match, OR a haplotype (at least 5\u002F10) matched related donor. Donors will be requested to provide PBSCs although bone marrow is acceptable according to donor preference.\n\nEligible Diseases Acute Leukemias: Must be in remission by morphology (≤5% blasts) AND without evidence of MRD by flow cytometry, FISH, or conventional cytogenetics. PCR based MRD detection is not an exclusion to proceed.\n\nAcute Myeloid Leukemia (AML) and related precursor neoplasms:\n\n2nd or greater complete remission (CR); first complete remission (CR1) in patients \\> 60 years old; CR1 in ≤ 60 years old that is NOT considered as favorable-risk.\n\nFavorable risk AML is defined as having one of the following:\n\n* t(8,21) without cKIT mutation\n* inv(16) or t(16;16) without cKIT mutation\n* Normal karyotype with mutated NPM1 and wild type FLT-ITD (unless persistently NPM1 positive by PCR following two cycles of chemotherapy)\n* Normal karyotype with double mutated CEBPA\n* Acute prolymphocytic leukemia (APL) in first molecular remission at the end of consolidation\n\nAcute lymphoblastic Leukemia (ALL) \u002Flymphoma:\n\nCR2 or greater, CR1 unable to tolerate consolidation chemotherapy due to chemotherapy-related toxicities; CR1 high-risk ALL.\n\nHigh risk ALL is defined as having one of the following:\n\n* Evidence of high risk cytogenetics, e.g. t(9;22), t(1;19), t(4;11), other MLL rearrangements, IKZF1\n* 30 years of age or older at diagnosis\n* White blood cell counts of greater than 30,000\u002FmcL (B-ALL) or greater than 100,000\u002FmcL (T-ALL) at diagnosis\n* CNS leukemia involvement during the course of disease\n* Slow cytologic response (\\>10% lymphoblasts in bone marrow on Day 14 of induction therapy)\n* Evidence of persistent immonophenotypic or molecular minimal residual disease (MRD) at the end of induction and consolidation therapy.\n\nVery high risk pediatric patients with ALL:\n\npatients \\\u003C21 years are also considered high risk CR1 if they had M2 or M3 marrow at day 42 from the initiation of induction or M3 marrow at the end of induction. They are eligible once they achieved a complete remission.\n\nBiphenotypic\u002FUndifferentiated\u002FProlymphocytic Leukemias in first or subsequent CR.\n\nChronic Myelogenous Leukemia in chronic or accelerated phase, or CML blast crisis in morphological remission (\\\u003C5% blasts) and with negative MRD by flow cytometry (a positive PCR for BCRABL is acceptable for BMT): Chronic phase patients must have failed at least two different TKIs, been intolerant to all available TKIs or have T315I mutation. Patients with CML blast crisis in CR are only eligible if there is an feasible TKI maintenance plan following BMT.\n\nPlasma Cell Leukemia after initial therapy, who achieved at least a partial remission; or relapsed and achieved subsequent remission (CR\u002FPR) Myelodysplastic Syndrome: IPSS INT-2 or High Risk; R-IPSS High or Very High; WHO classification: RAEB-1, RAEB-2; Severe Cytopenias: ANC \\\u003C 0.8, Anemia or thrombocytopenia requiring transfusion; Poor or very poor risk cytogenetics based on IPSS or R-IPSS definitions; therapy-related MDS. Blasts must be \\\u003C 5% by bone marrow aspirate morphology. If ≥5% blasts, patient requires chemotherapy for cytoreduction to \\\u003C5% blasts prior to transplantation Leukemia or MDS in aplasia. These patients may be taken to transplant if after induction therapy they remain with aplastic bone marrow and no morphological or flow-cytometry evidence of disease ≥ 28 days post-therapy. These high risk patients will be analyzed separately.\n\nBurkitt's Lymphoma in CR2 or subsequent CR. Relapsed T-Cell Lymphoma that is chemotherapy sensitive in CR\u002FPR that has failed or ineligible for an autologous transplantNatural Killer Cell Malignancies. Relapsed Chronic Lymphocytic Leukemia\u002FSmall Lymphocytic Lymphoma (CLL\u002FSLL), Marginal Zone B-Cell Lymphoma or Follicular Lymphoma which have progressed within 12 months of achieving a partial or complete remission. Patients who had remissions lasting \\> 12 months, are eligible after at least two prior therapies. Patients with bulky disease should be considered for de-bulking chemotherapy before transplant. Patients with refractory disease may be eligible, unless bulky disease and an estimated tumor doubling time of less than one month.\n\nLymphoplasmacytic Lymphoma, Mantle-Cell Lymphomais eligible after initial therapy if chemotherapy sensitive.\n\nLarge Cell and other high risk NHL \\> CR2\u002F\\> PR2: Patients in CR2\u002FPR2 with initial short remission (\\\u003C6 months) are eligible.\n\nRelapsed Multiple Myeloma: that is chemotherapy sensitive and has failed or ineligible for an autologous transplant.\n\nMyeloproliferative Neoplasms\u002FMyelofibrosis - with transfusion dependence or expected survival under 5 years by DIPSS, DIPSS-plus, or MPSS70 calculator.\n\nAcquired Bone Marrow Failure Syndromes except for Fanconi anemia Other Leukemia Subtypes: A major effort in the field of hematology is to identify patients who are of high risk for treatment failure so that patients can be appropriately stratified to either more (or less) intensive therapy. This effort is continually ongoing and retrospective studies identify new disease features or characteristics that are associated with treatment outcomes. Therefore, if new features are identified after the writing of this protocol, patients can be enrolled with the approval of two members of the study committee.\n\nAdditional Criteria for Bulky Disease (lymphomas) if stable disease is best response, the largest residual nodal mass must \\\u003C 5 cm (approximately) If response to previous therapy, the largest residual mass must represent a 50% reduction and be \\\u003C 7.5 cm (approximately)\n\nOrgan Function Criteria\n\nAdequate organ function is defined as:\n\nLiver: Transaminases ≤ 5 x upper limit of normal (ULN) and total bilirubin ≤ 2.5 mg\u002FdL except for patients with Gilbert's syndrome or hemolysis.\n\nRenal: A normal creatinine (adults) or creatinine clearance ≥ 40 mL\u002Fmin (pediatrics). Adults with a creatinine \\> 1.2 mg\u002Fdl or a history of renal dysfunction must have estimated GFR ≥ 40 ml\u002Fmin\u002F1.73m2.\n\nCardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction \\> 40%. For children that are not able to cooperate with MUGA and echocardiography, such should be clearly stated in the physician's note.\n\nPulmonary: DLCO, FEV1, FVC ≥ 40% predicted, and absence of O2 requirements. For children that are not able to cooperate with PFTs, a pulse oximetry with exercise should be attempted. If neither test can be obtained it should be clearly stated in the physician's note.\n\nIf recent confirmed mold infection (e.g. aspergillus) must have minimum of 30 days of therapy and responsive disease and be cleared by Infectious Disease HIV infection with undetectable viral load. All HIV+ patients must be evaluated by Infectious Disease (ID) and a HIV management plan establish prior to transplantation Sexually active females of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control during study treatment Voluntary written consent (adult or parent\u002Fguardian with presentation of the minor information sheet, if appropriate)\n\nRelated donors will be evaluated and collected according to UMN BMT program standard processes. Unrelated donors will be identified and collected through the National Marrow and Donor Program (NMDP) per usual steps.\n\nExclusion Criteria:\n\n* Pregnant or breast feeding. The agents used in this study include Pregnancy Category D: known to cause harm to a fetus. Females of childbearing potential must have a negative pregnancy test prior to starting therapy.\n* Untreated active infection\n* Active central nervous system malignancy\n* CML in blast crisis\n* Intermediate or high grade NHL, mantle cell NHL, and Hodgkin disease that is progressive on salvage therapy. Stable disease is acceptable to move forward provided it is non-bulky.\n* Less than 3 months since prior myeloablative transplant\n* Evidence of progressive disease by imaging modalities or biopsy - persistent PET activity, though possibly related to lymphoma, is not an exclusion criterion in the absence of CT changes indicating progression.","75 Years",{"count":658,"type":21},56,[60],"This is a Phase II study following subjects proceeding with our Institutional non-myeloablative cyclophosphamide\u002F fludarabine\u002Ftotal body irradiation (TBI) preparative regimen followed by a related, unrelated, or partially matched family donor stem cell infusion using post-transplant cyclophosphamide (PTCy), sirolimus and MMF GVHD prophylaxis.",[662,663,664,665,666,667,668,27,669,670,568,671,672,673,674,675,227,222],"Acute Myelogenous Leukemia","Acute Lymphocytic Leukemia","Biphenotypic Acute Leukemia","Undifferentiated Leukemia","Prolymphocytic Leukemia","Chronic Myelogenous Leukemia","Plasma Cell Leukemia","Leukemia, Myeloid","Myelodysplastic Syndrome With Excess Blasts-1","Relapsed T-Cell Lymphoma","Relapsed Chronic Lymphocytic Leukemia","Small Lymphocytic Lymphoma","Marginal Zone Lymphoma","Follicular Lymphoma",[33,677,678,541,679,680,681,143,682,683,684,17],"CLL","SLL","CML","PFS","TRM","MMF","TBI","PTCy",{"date":643,"type":38},{"date":687,"type":38},"2023-05-01",{"date":689,"type":21},"2028-10-22",{"name":501,"class":128}]