[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"anemia-sickle-cell\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:anemia-sickle-cell":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,43,74],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":16,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100553070","phase-1-a-study-to-evaluate-bms-986470-in-healthy-volunteers-and-participants-with-sickle-cell-disease-100553070",false,"NCT06481306","A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease","A Phase 1\u002F2a, First-in-human, Randomized, Double-blinded, Placebo-controlled, Dose-finding Study in Healthy Volunteers and Participants With Sickle Cell Disease to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics, pH and Food Effect, and Preliminary Efficacy of BMS-986470","Inclusion Criteria:\n\nCohort A:\n\n* Healthy male and female (who are not of childbearing potential) participants, as determined by the investigator based on medical history and other determinations. Females not of childbearing potential must have been amenorrhoeic for at least 12 months without an alternative medical cause and have follicle-stimulating hormone (FSH) levels of at least 40 IU\u002FL or have undergone a hysterectomy, bilateral oophorectomy, or bilateral salpingectomy.\n* Body mass index (BMI) of 18.0 to 32.0 kg\u002Fm2, inclusive. BMI = weight (kg)\u002F\\[height (m)\\]2 as measured at screening.\n* No evidence of organ dysfunction or any clinically significant deviation from normal in physical examination, vital signs, ECG, or clinical laboratory assessments beyond what is consistent with the target population.\n\nCohort B:\n\n* Participants with a documented diagnosis of sickle cell disease (SCD) with genotype HbSS, HbSβ0-thal, or HbSβ+-thal.\n* For Cohort B Part 1 only: Participants with ≥ 4 vaso-occlusive crises (VOCs) within the previous 12 months or ≥ 2 VOCs within the previous 6 months. For Cohort B Part 2 only: Participants with ≥ 2 VOCs and ≤ 15 VOCs within the previous 12 months.\n* Participant has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.\n* Participants must have the following laboratory values:\n\n  i) Hemoglobin ≥ 5.5 and ≤ 12 g\u002FdL (males) or ≥ 5.5 and ≤ 10.6 g\u002FdL (females). ii) Absolute neutrophil count ≥ 1500\u002FμL. iii) Platelet count ≥ 100 × 10\\^3\u002FμL. iv) Absolute reticulocyte count \\> 100 × 10\\^3\u002FμL or \\> 50 × 10\\^3\u002FμL if taking hydroxyurea.\n\nExclusion Criteria:\n\nCohort A:\n\n* Any significant medical condition or any condition that confounds the ability to interpret data from the study.\n* Participant has any condition, including the presence of laboratory abnormalities, that places the participant at unacceptable risk if the participant was to participate in the study.\n* Any major surgery or planned surgery (except GI surgery) within 12 weeks of the first study intervention administration.\n\nCohort B:\n\n* Participants with any condition, including significant acute or chronic medical illness, active or uncontrolled infection, or the presence of laboratory abnormalities, that places participants at unacceptable risk if participating in this study.\n* For Cohort B Part 1 only: participants with more than 6 severe VOCs defined as VOCs requiring ≥ 24 hours of hospital admission within 12 months prior to the first dose of study intervention.\n* For Cohort B Part 1 only: participants with any episode of acute chest syndrome within the last 6 months prior to the first dose of study intervention.\n* Creatinine clearance (CrCl) \\\u003C 60 mL\u002Fmin\u002F1.72m2 using Chronic Kidney Disease Epidemiology (CKD-EPI) equation.\n\nCohort A and B:\n\n* Participant is receiving regularly scheduled RBC or platelet transfusions or has received a RBC transfusion within 28 days and a platelet transfusion within 14 days prior to starting treatment with BMS-986470.\n* Other protocol-defined Inclusion\u002FExclusion criteria apply.",true,"ALL","18 Years",{"count":20,"type":21},224,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell disease.",[28,29],"Anemia, Sickle Cell","Healthy Volunteers","RECRUITING","2026-08-17",{"date":33,"type":34},"2026-08-18","ACTUAL",{"date":36,"type":34},"2024-07-17",{"date":38,"type":21},"2027-11-16",{"name":40,"class":41},"Bristol-Myers Squibb","INDUSTRY",32,{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":47,"acronym":48,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":17,"minAge":50,"maxAge":51,"enrollmentInfo":52,"targetDuration":4,"studyType":22,"phases":54,"briefSummary":56,"conditions":57,"keywords":58,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":63,"lastUpdatePostDateStruct":64,"startDateStruct":66,"completionDateStruct":68,"leadSponsor":70,"locationsCount":73},"100538405","a-socio-ecological-approach-for-improving-self-management-in-adolescents-with-scd-100538405","NCT06290401","A Socio-ecological Approach for Improving Self-management in Adolescents With SCD","SC-Thrive","Inclusion Criteria:\n\n* Patient of a participating SCD Clinic\n* Confirmed diagnosis of SCD\n* 13-21 years of age\n\nExclusion Criteria:\n\n* Another chronic disease (which would complicate measurement of patient activation)\n* Non-English-speaking\n* Cognitive or psychiatric disorder that the physician or study therapists believe would impair study participation.","13 Years","21 Years",{"count":53,"type":21},310,[55],"NA","The goal of this clinical trial is to evaluate the impact of SCThrive (a behavioral self-management intervention) on patient activation, self-management behaviors, daily functioning, and emergency room visits in 260 adolescents and young adults with sickle cell disease (SCD) ages 13-21 receiving care at 1 of 4 pediatric SCD clinics.\n\nThe main question\\[s\\]it aims to answer are:\n\n* Does SCThrive improve patient activation?\n* Does SCThrive improve self-management behaviors, daily functioning, and decrease emergency room visits?\n* Are any improvements maintained 3 months after treatment?\n\nParticipants will complete self-management related surveys before, after, and 3 months following their participation in an 8- week, virtual group intervention with an accompanying mobile app (SCThrive).\n\nResearchers will compare outcomes for participants who receive SCThrive and participants who receive uniform standard care (SCHealthED which = standard of care plus SCD educational text messages) to see if there are differences in patient activation, self-management behaviors, daily functioning, and emergency room visits.",[28],[59,60,61,62],"adolescent and young adult (AYA)","self-management","behavioral intervention","sickle cell disease","2026-05-06",{"date":65,"type":34},"2026-05-11",{"date":67,"type":34},"2025-02-03",{"date":69,"type":21},"2028-06-30",{"name":71,"class":72},"Children's Hospital Medical Center, Cincinnati","OTHER",4,{"id":75,"slug":76,"hasResults":11,"nctId":77,"briefTitle":78,"officialTitle":78,"acronym":79,"eligibilityCriteria":80,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":81,"enrollmentInfo":82,"targetDuration":84,"studyType":85,"phases":4,"briefSummary":86,"conditions":87,"keywords":99,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":107,"lastUpdatePostDateStruct":108,"startDateStruct":110,"completionDateStruct":112,"leadSponsor":114,"locationsCount":116},"100535343","european-rare-blood-disorders-platform-enrol-100535343","NCT06250595","European Rare Blood Disorders Platform (ENROL)","ENROL","Inclusion Criteria:\n\n* Patients must meet all of the following criteria to be included in the ENROL Registry\n* Age from 0-100, both female and male\n* Diagnosed as RHDs according to ORPHANET classification\n* Able and willing to provide written informed consent (patient or legal representative for minors) if needed according to national legislation.\n\nExclusion Criteria:\n\n* Patients diagnosed as traits or trait conditions for other recessive RHDs","100 Years",{"count":83,"type":21},37090,"15 Years","OBSERVATIONAL","ENROL, the European Rare Blood Disorders Platform has been conceived in the core of ERN-EuroBloodNet as an umbrella for both new and already existing registries on Rare Hematological Diseases (RHDs). ENROL aims at avoiding fragmentation of data by promoting the standards for patient registries' interoperability released by the EU RD platform.\n\nENROL's principle is to maximize public benefit from data on RHDs opened up through the platform with the only restriction needed to guarantee patient rights and confidentiality, in agreement with EU regulations for cross-border sharing of personal data.\n\nAccordingly, ENROL will map the EU-level demographics, survival rates, diagnosis methods, genetic information, main clinical manifestations, and treatments in order to obtain epidemiological figures and identify trial cohorts for basic and clinical research. To this aim, ENROL will connect and facilitate the upgrading of existing RHD registries, while promoting the building of new ones when \u002F where lacking. Target-driven actions will be carried out in collaboration with EURORDIS for educating patients and families about the benefits of enrolment in such registries, including different cultural and linguistic strategies.\n\nThe standardized collection and monitoring of disease-specific healthcare outcomes through the ENROL user-friendly platform will determine how specialized care is delivered, where are the gaps in diagnosis, care, or treatment and where best to allocate financial, technical, or human resources.\n\nMoreover, it will allow for promoting research, especially for those issues that remain unanswered or sub-optimally addressed by the scientific community; furthermore, it will allow promoting clinical trials for new drugs. ENROL will enable the generation of evidence for better healthcare for RHD patients in the EU as the ultimate goal.\n\nENROL officially started on 1st June 2020 with a duration of 36 months. ENROL is co-funded by the Health Programme of the European Union under the call for proposals HP-PJ-2019 on Rare disease registries for the European Reference Networks. GA number 947670",[88,89,90,91,92,93,94,95,28,96,97,98],"Anemia","Bone Marrow Failure","Bleeding Disorder","Iron Metabolism Disorders","Myeloma","Lymphoid Neoplasm","Myeloma, Malignant","Leukemia","Thalassemia","Blood Cancer","Red Cell Membrane and Enzyme Abnormalities",[88,89,100,101,102,103,104,95,105,96,106],"Bleeding disorder","Iron metabolism disorder","Myeloid","Lymphoid","Blood cancer","Red Cell membrane and Enzyme Abnormalities","Sickle Cell Disease","2024-02-06",{"date":109,"type":34},"2024-02-09",{"date":111,"type":34},"2022-07-01",{"date":113,"type":21},"2037-07",{"name":115,"class":72},"Hospital Universitari Vall d'Hebron Research Institute",1]