EUCTR2021-004928-15-GR进行中（未招募）1 期
A Phase 2, Double-blind, Randomized, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of Luspatercept (BMS-986346/ACE-536) for the Treatment of Anemia in Adults with Alpha (a)-thalassemia - Phase 2 Study of Luspatercept in Adults with Alpha (a)-thalassemia
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 176
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •-Participant has documented diagnosis of a-thalassemia HbH disease (electrophoresis? or high-performance liquid chromatography [HPLC]?based methods for Hb variant analyses are accepted), with or without transfusion dependence; compounded combination with ß-thalassemia is allowed if at least 1 non-mutated ß-chain gene is present
- •-Transfusion dependence:
- •TD participant = 6 RBC units/24 weeks AND no transfusion-free period for > 56 days during the 24 weeks prior randomization
- •NTD participant: < 6 RBC units during the 24 weeks prior to randomization, RBC transfusion-free during at least 8 weeks prior to randomization
- •AND Mean baseline Hb = 10 g/dL
- •- Participant must be = 18 years of age at the time of signing the informed consent
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 172
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 4
排除标准
- •- 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 (eg, severe G6PD deficiency, pyruvate kinase deficiency, etc)
- •- Bleeding disorders manifested by frequent bleeding episodes (eg, menorrhagia, epistaxis, clotting disorders)
- •- Undergone episodes of hemolysis not related to a-thalassemia, eg, after use of hemolysis predisposing drugs (eg, anti-malarial, nonsteroidal anti-inflammatory drug [NSAID]), within the 8 weeks prior to randomization
- •- Women who are pregnant, plan to get pregnant during the study, or who are breastfeeding
- •- Physical and Laboratory Test Findings
- •a) Platelet count > 1,000 × 109 /L b)
- •b) Thrombocytopenia with < 70 × 109 /L if not associated with hypersplenism
- •c)No concurrent or history of severe hepatic disease or histopathological evidence of liver cirrhosis/fibrosis on liver biopsy: i) Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3× the upper limit of normal (ULN) ii) Albumin < 3 g/dL
- •d) Heart disease, heart failure as classified by the New York Heart Association (NYHA; see Appendix 6) classification 3 or higher, or significant arrhythmia requiring treatment, or recent myocardial infarction within 6 months of randomization
- •e)Lung disease, including pulmonary fibrosis or pulmonary hypertension, which are clinically significant (ie, = Grade 3 per NCI-CTCAE version 5.0 [current active minor version])
- •f)Creatinine clearance < 60 mL/min (per Cockroft-Gault formula) or estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73m2 (per modification of diet in renal disease [MDRD] study)
- •g)Proteinuria = Grade 3 according to NCI-CTCAE version 5.0 (current active minor version) or protein/creatinine ratio > 350 mg/mmol, or albumin/creatinine ratio > 220 mg/mmol
- •h)Active hepatitis C virus (HCV) infection, as demonstrated by a positive HCV-RNA test of sufficient sensitivity, or active infectious hepatitis B virus (HBV) as demonstrated by the presence of HBsAg and/or HBV DNA-positive, or known positive human immunodeficiency virus (HIV)
- •- Prior/Concomitant Therapy:
- •Treatment with another investigational drug or device = 30 days (or 5 half-lives, whichever is longer) prior to randomization
- •Previous exposure to sotatercept (ACE-011) or luspatercept (BMS-986346/ACE-536)
- •Use of an erythropoiesis-stimulating agent (ESA) = 24 weeks prior to randomization
- •Iron chelation therapy initiated = 24 weeks prior to randomization
- •Use of hydroxyurea treatment = 24 weeks prior to randomization
- •Prior exposure to gene therapy
- •Undergone HSCT
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