A Phase 2 Study for the Treatment of Anemia with Alpha (a)-Thalassemia to Determine the Efficacy and Safety of Luspatercept (BMS-986346/ACE-536) in Adults and Evaluate the Safety and Pharmacokinetics in Adolescents - CA056-015

Celgene Corp.0 个研究点目标入组 177 人开始时间: 2023年4月20日最近更新: 2024年7月22日

适应症

试验速览

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•Adult participant = 18 years with documented diagnosis of a-thalassemia HbH disease with Transfusion dependence defined as: - TD participant: = 6 RBC units during the 24 weeks prior to randomization and no transfusion-free period for > 56 days during the 24 weeks prior to randomization - NTD participant: < 6 RBC units during the 24 weeks prior to randomization and, RBC transfusion-free during at least 8 weeks prior to randomization and, mean baseline Hb = 10 g/dL, based on a minimum of 2 measurements = 1 week apart within 4 weeks prior to randomization; hemoglobin values within 21 days post-transfusion will be excluded. Adolescent participant 12 years to < 18 years with documented diagnosis of a- thalassemia HbH disease with transfusion dependence defined as: - TD participant: = 4 RBC events during the 24 weeks prior to enrollment and, no transfusion-free period for > 56 days during the 24 weeks prior to enrollment. Participants must have a history of regular transfusions for at least 2 years - NTD participant: < 4 RBC events during the 24 weeks prior to enrollment and RBC transfusion-free during at least 8 weeks prior to enrollment and, mean baseline Hb = 10 g/dL, based on a minimum of 2 measurements = 1 week apart within 4 weeks prior to enrollment, hemoglobin values within 21 days post-transfusion will be excluded. - Participant has Karnofsky (age =16 years) or Lansky (age < 16 years) performance status score = 50 at screening.

•Key Exclusion Criteria - Adult and Adolescent participants: - Medical Conditions: Diagnosis of a-thalassemia Trait, Hb Bart hydrops, ATRx a-thalassemia, hemoglobin S/ß-thalassemia, myelodysplasia subtype anemia, or with HbE homozygous beta gene mutation. Anemia related to nutritional deficiency, anemia of chronic disease, autoimmune hemolytic anemia, or any other hemolytic anemias. Bleeding disorders manifested by frequent bleeding episodes. Undergone episodes of hemolysis not related to a-thalassemia within the 8 weeks prior to randomization. - Reproductive Status: Women who are pregnant, plan to get pregnant during the study, or who are breastfeeding. - Prior/Concomitant Therapy: Prior exposure to gene therapy to treat a-thalassemia. Undergone hematopoietic stem cell transplantation (HSCT)

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