[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Forma Therapeutics, Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":65},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,42],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":41},"100531354","phase-2-a-study-to-evaluate-the-pharmacokinetics-and-safety-of-etavopivat-in-pediatric-patients-with-sickle-cell-disease-100531354",false,"NCT06198712","A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease","A Single Arm, Open Label, Phase 1\u002F2 Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease","HIBISCUS KIDS","Inclusion Criteria:\n\n* Type of Participant and Disease Characteristics\n\n  1. Patient's parent, legal guardian, or legal representative has provided documented informed consent and patients have provided age-appropriate assent\n  2. Age greater than or equal to (≥) 6 months and lesser than (\\\u003C) 18 years of age at time of enrollment, according to the enrolling cohort:\n\n     * Cohort 1: age 12 to \\\u003C 18 years (adolescents)\n     * Cohort 2: age 6 to \\\u003C 12 years\n     * Cohort 3: age 2 to \\\u003C 6 years\n     * Cohort 4: age 6 months to \\\u003C 2 years\n  3. Patient has confirmed diagnosis of SCD\n\n     • Documentation of SCD genotype (HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing. Molecular genotyping is not required. SCD genotype may be determined from the results of Hb electrophoresis, high-performance liquid chromatography (HPLC), or similar testing. Note that Hb electrophoresis is performed by the local laboratory at Screening.\n  4. Hemoglobin ≥ 5.5 and lesser than or equal to (≤) 10.5 grams per deciliter (g\u002FdL)\n  5. Pediatric patients with severe SCD, as defined by at least 1 of the following:\n\n     * 2-15 episodes of documented VOC within the 12 months prior to screening. Documentation must exist in the patient's medical record prior to screening. Events based solely on patient recall without supporting documentation should not be counted towards eligibility.\n     * Hospitalization for any SCD-related complication in the last 12 months prior to starting study treatment\n     * Proteinuria, defined as an albumin:creatinine ratio (ACR) \\> 100 mg\u002Fg on 2 measures (separated by ≥ 1 month) as an indicator of early renal disease\n     * History of a conditional TCD in the last 12 months prior to starting study treatment, but not currently being treated with chronic transfusion therapy (applicable to participants \\> 2 years of age). Conditional TCD is defined as a TAMMV of 170-199 cm\u002Fs by TCD or 155-184 cm\u002Fs by imaging TCD (TCDi).\n  6. For participants taking hydroxyurea (HU), the dose of HU (mg\u002Fkg) must be stable (no more than a 20% change in dosing) for at least 90 days prior to start of study treatment with no anticipated need for dose adjustments during the study, in the opinion of the Investigator\n  7. Patients on crizanlizumab or L-glutamine treatment at the time of consent may be eligible if they:\n\n     * Have been on a stable dose for ≥ 12 months at the time of consent (ie, no changes to the dose except for changes to weight or for safety reasons)\n     * For patients on crizanlizumab, have been ≥ 80% compliant with the planned regimen during the 12 months prior to the time of consent\n  8. Female patients of childbearing potential who are using acceptable methods of contraception and agree not to donate ova from study start to 90 days after the last dose of study drug, and male patients who are willing to use acceptable methods of contraception and agree not to donate sperm, from study start to 90 days after the last dose of study drug.\n\nExclusion Criteria:\n\n* Medical Conditions\n\n  1. Female who is breastfeeding or pregnant\n  2. More than 15 VOCs within the 12 months prior to starting study treatment that required a hospital, emergency room (ER), or clinic visit\n  3. Hospitalized for sickle cell crisis or other vaso-occlusive event occurring in the 14 days prior to starting study treatment\n  4. Abnormal TCD in the 12 months prior to starting study treatment\n\n     Prior\u002FConcomitant Therapy\n  5. Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion)\n  6. Received any blood products within 30 days of starting study treatment\n  7. Receiving or use of concomitant medications that are strong inducers of cytochrome P450 (CYP) 3A4\u002F5 within 2 weeks of starting study treatment\n  8. Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent during the study\n  9. Receipt of erythropoietin or other hematopoietic growth factor treatment within 28 days of starting study treatment or anticipated need for such agents during the study\n  10. Receipt of prior cellular based therapy (eg, hematopoietic cell transplant, gene modification therapy)","ALL","6 Months","18 Years",{"count":21,"type":22},95,"ESTIMATED","INTERVENTIONAL",[25],"PHASE2","The purpose of this study is to evaluate the pharmacokinetics and safety of etavopivat in paediatric participants with sickle cell disease (SCD). Participants will receive etavopivat and will be enrolled in a staggered manner, starting with the oldest age group and followed sequentially by younger cohorts after review of pharmacokinetic and safety data from the preceding cohort. All participants will undergo a 24-week primary treatment period followed by a 72-week extension treatment period to further evaluate long-term safety and pharmacokinetics of etavopivat. The total duration of the study will be approximately 96 weeks.",[28],"Sickle Cell Disease","RECRUITING","2026-08-07",{"date":32,"type":33},"2026-08-12","ACTUAL",{"date":35,"type":33},"2023-01-12",{"date":37,"type":22},"2029-08-08",{"name":39,"class":40},"Forma Therapeutics, Inc.","INDUSTRY",18,{"id":43,"slug":44,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":46,"acronym":47,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":17,"minAge":49,"maxAge":50,"enrollmentInfo":51,"targetDuration":4,"studyType":23,"phases":53,"briefSummary":54,"conditions":55,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":56,"lastUpdatePostDateStruct":57,"startDateStruct":59,"completionDateStruct":61,"leadSponsor":63,"locationsCount":64},"100512525","phase-2-a-phase-2-open-label-study-to-evaluate-the-activity-of-etavopivat-on-transcranial-doppler-velocities-in-pediatric-patients-with-sickle-cell-disease-who-are-at-increased-risk-for-primary-stroke-100512525","NCT05953584","A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke","HIBISCUS 3","Inclusion Criteria:\n\n1. Patient's parent, legal guardian, or legal representative has provided documented informed consent and patients have provided age-appropriate assent\n\n   Age:\n2. 12 to 16 years of age (inclusive) at time of screening\n\n   Type of Participant and Disease Characteristics:\n3. Confirmed diagnosis of SCD\n\n   • Documentation of any SCD genotype (e.g. HbSS, HbSβ0 -thalassemia) based on prior history of laboratory testing. Molecular genotyping is not required. SCD genotype may be determined from the results of Hb electrophoresis, high-performance liquid chromatography, or similar testing.\n4. TAMMV greater than or equal to (≥) 170 cm\u002Fs in the ICA and\u002For MCA during the Screening Period and confirmed on 2 occasions and without history of primary ischemic or hemorrhagic stroke, transient ischemic attack, or severe central nervous system (CNS) vasculopathy on magnetic resonance angiography (MRA). This includes patients with cTCD (170-199 centimeter per second \\[cm\u002Fs\\]) or aTCD (≥ 200 cm\u002Fs). Patients with aTCD cohort must have refused transfusion therapy.\n5. Hb ≥ 6 grams per deciliter (g\u002FdL) and lesser than or equal to (≤) 9 g\u002FdL at screening\n6. For participants with aTCD and cTCD and already taking HU, the dose of HU milligram per kilogram (mg\u002Fkg) must be stable (no more than a 20% change in dosing except for weight-based changes) for at least 90 days prior to start of study treatment with no anticipated need for dose adjustments except for weight-based changes during the study, in the opinion of the Investigator.\n\n   Sex and Contraceptive Requirements\n7. Patients, who if female and of childbearing potential, are using acceptable methods of contraception and agree not to donate ova from study start to 90 days after the last dose of study drug, and who if male, are willing to use acceptable methods of contraception and agree not to donate sperm, from study start to 90 days after the last dose of study drug\n\nExclusion Criteria:\n\nMedical Conditions\n\n1. Female who is breast feeding or pregnant\n2. History of seizure disorder\n3. Prior overt stroke (a focal neurological deficit of acute onset) by history or significant concerns for history of overt stroke based on Screening MRL, history of transient ischemic attack, focal neurological deficit on standardized neurological examination, or concern for moderate or severe neurological deficit (which could be due to stroke) based on a positive \"10 questions\" screening. Patients with significant or suggestive severe CNS vasculopathy (ie, moya moya) of Grade 4 or higher based on MRA read locally.\n4. Significant cytopenias (absolute neutrophil count \\[ANC\\] \\\u003C 1.5 × 10\\^3\u002Fmicroliter (µL), platelets \\\u003C 150,000\u002FµL, reticulocytes \\\u003C 80,000\u002FµL)\n5. Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the local laboratory \\\u003C 30 mL\u002Fmin\u002F1.73 m\\^2) or on chronic dialysis\n6. Hepatic dysfunction characterized by alanine aminotransferase (ALT) \\> 4 × upper limit of normal (ULN) and\u002For direct bilirubin \\> 3 × ULN\n7. Patients with clinically significant bacterial, fungal, parasitic, or viral infection requiring systemic therapy or history of such infections leading to significant neurological impairment:\n\n   * Patients with acute bacterial, fungal, parasitic, or viral infection requiring systemic therapy should delay screening\u002Fenrollment until active therapy has been completed.\n   * Patients with acute viral infections (eg, coronavirus disease 2019 \\[COVID-19\\]) should delay screening\u002Fenrollment until the acute infection has resolved.\n   * Patients enrolled in areas where malaria is prevalent must be on malaria prophylaxis based on regional guidance and resistance results. Note: Infection prophylaxis is allowed (see concomitant medication restrictions).\n8. Known human immunodeficiency virus (HIV) positivity\n9. Known infection with hepatitis B virus (hepatitis B surface antigen \\[HepBsAg\\] and hepatitis B core antibody \\[HepBcAb\\] positive.","12 Years","16 Years",{"count":52,"type":22},27,[25],"The study will test a new medicine, etavopivat, for sickle cell disease and see if it is safe and help-ful for participants with sickle cell disease who are at an increased risk of stroke. Participants will be divided into two cohorts depending on their transcranial doppler (TCD) ultrasound results and whether or not they receive hydroxyurea (medication that they may already be taking). In one cohort, participants with conditional transcranial doppler (TCD) or participants with abnormal TCD who are not able to receive hydroxyurea will be included. The study doctor will determine if the TCD result is conditional or abnormal. In another cohort, participants with conditional TCD or participants with abnormal TCD who are receiving a stable dose of hydroxyurea will be included. The study doctor will determine if the TCD result is conditional or abnormal. The participant will start a 52-week (1 year) treatment period. The participant will take 400 milligrams (mg) of etavopivat once a day for the 52 weeks. The dose of 400 mg will be taken as 2 tablets by mouth, each containing 200 mg of etavopivat. Etavopivat may be taken with or without food. Each dose should be taken with a glass of water. As part of the study, the participants will be asked to visit the clinic frequently. The participant will have the opportunity to participate in a 48-week optional extension treatment period. The optional extension treatment period will allow continued as-sessment of safety of etavopivat in paediatric patients. At the end of the study, if deemed appro-priate the participant, the caregiver, and the study doctor, the participant may be offered the op-portunity to participate in a separate study to continue receiving etavopivat. If\u002Fwhen this separate study becomes available, the participant may only transfer to the new study after completion of the 52-week primary treatment period and at any time during the 48-week optional extension treatment period.",[28],"2025-09-17",{"date":58,"type":33},"2025-09-18",{"date":60,"type":33},"2023-06-20",{"date":62,"type":22},"2027-09-20",{"name":39,"class":40},9,""]