A PHASE 2, TWO-PART, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF SUBCUTANEOUSLY ADMINISTERED REGN7999 (AN INHIBITOR OF TMPRSS6) IN PARTICIPANTS WITH IRON OVERLOAD DUE TO NONTRANSFUSION DEPENDENT β-THALASSEMIA.
概览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 95
- 试验地点
- 7
- 主要终点
- 1. Change in Liver iron concentration (LIC) by R2 magnetic resonance imaging (MRI)
概览
简要总结
This study is researching an experimental drug called REGN7999 (called study drug). The study is focused on patients with non-transfusion dependent beta-thalassemia. The aim of the study is to see how safe and effective the study drug is.
The study is looking at several other research questions, including:
-Whether the study drug lowers extra iron levels in the body
-What side effects may happen from taking the study drug
-How much study drug is in the blood at different times
-Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)
研究设计
- 研究类型
- Interventional
- 分配方式
- Other
- 盲法
- Participant and Investigator Blinded
入排标准
- 年龄范围
- 18.00 Year(s) 至 65.00 Year(s)（—）
- 性别
- All
入选标准
- •Clinical diagnosis of Non-transfusion dependent beta-thalassemia (NTDT) as described in the protocol
- •Resultant Iron overload (IOL), defined as Liver iron concentration (LIC) greater than or equal to 5 mg Fe/g DW as measured by R2 magnetic resonance imaging (MRI) at screening
- •Serum ferritin greater than or equal to 300 ng/mL as described in the protocol.
排除标准
- •Hemoglobin less than or equal to 8 g/dL at screening
- •Any RBC transfusion within 12 weeks of visit 3
- •For Part A only: Any ICT use in approximately 12 weeks prior to screening as described in the protocol
- •For Part B only: If on ICT, any change in Iron chelation therapy (ICT) dose in approximately 12 weeks prior to screening as described in the protocol
- •Any use of luspatercept or mitapivat in 6 months prior to screening as described in the protocol
- •Absolute contraindication to MRI
- •Diagnosis of cirrhosis of the liver
- •Diagnosis of Chronic kidney disease (CKD) stage 4 or higher
- •Other protocol-defined Inclusion/ Exclusion Criteria apply.
结局指标
主要结局
1. Change in Liver iron concentration (LIC) by R2 magnetic resonance imaging (MRI)
时间窗: 1. Baseline to week 24 | 2. Up to week 72
2. Incidence and Severity of Treatment-emergent adverse events (TEAEs)
时间窗: 1. Baseline to week 24 | 2. Up to week 72
次要结局
- Achievement of greater than or equal to 20% reduction in LIC by R2 MRI(Baseline to week 52)
- Change in hemoglobin(Baseline to week 24)
- Change in LIC by R2 MRI(Baseline to week 52)
- Percent change in LIC by R2 MRI(Baseline to week 24 and week 52)
- Achievement of greater than or equal to 20% reduction in LIC by R2 MRI.(Baseline to week 24)
- Change in hemoglobin over time(Up to week 56)
- Achievement of greater than or equal to 1.5 g/dL increase in hemoglobin for two consecutive assessments in the absence of red blood cell (RBC) transfusions(Baseline to week 56)
- Number of RBC transfusions required(Baseline to week 72)
- Achievement of transfusion independence(Baseline to week 72)
- Change in RBC counts over time(Baseline to week 56)
- Concentrations of REGN7999 in serum over time(Up to week 56)
- Incidence of anti-drug antibody (ADA) to REGN7999 over time(Up to week 56)
- Magnitude of ADA to REGN7999 over time(Up to week 56)