Open-Label Study to Assess the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Povetacicept in Subjects With Autoimmune Cytopenias (RUBY-4)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 30
- 试验地点
- 88
- 主要终点
- Safety and Tolerability as Assessed by Adverse events (AEs) and Serious adverse events (SAEs)
研究概览
简要总结
The goal of this clinical study is to evaluate povetacicept in adults with autoimmune cytopenias of immune thrombocytopenia, autoimmune hemolytic anemia, and cold agglutinin disease to determine if povetacicept is safe and potentially beneficial in treating these diseases. During the study treatment period participants will receive povetacicept approximately every 4 weeks for 6 months, with the possibility of participating in a 6-month study treatment extension period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Indication-specific Criteria
- •Immune Thrombocytopenia (ITP)
- •Documented persistent or chronic primary ITP of at least 12 weeks duration from diagnosis to Cycle 1 Day 1
- •History of failure or relapse to at least 2 treatment regimens for ITP
- •History of exposure to a TPO-RA unless otherwise contraindicated or unavailable
- •Documented history of platelets <30 × 10^9/L
- •Warm Autoimmune Hemolytic Anemia (wAIHA)
- •Diagnosis of primary wAIHA of at least 12 weeks duration documented with a current or prior positive direct antiglobulin test (DAT) for anti-IgG (±C3d)
- •Documented history of anemia with hemoglobin ≤10 g/dL
- •At least one of the following: (i) haptoglobin < lower limit of normal (LLN) (ii) indirect bilirubin > upper limit of normal (ULN) (iii) lactate dehydrogenase>ULN
- •History of failure or relapse to at least 2 treatment regimens for wAIHA
- •Cold Agglutinin Disease (CAD)
- •Diagnosis of primary CAD of at least 12 weeks duration with all of the following: (i) chronic hemolysis (ii) polyspecific DAT positive (iii) monospecific DAT strongly positive for C3d (iv) cold agglutinin titer ≥64 at 4°C (v) IgG DAT ≤1+ (vi) no overt malignant disease
- •Documented history of anemia with hemoglobin ≤10 g/dL
- •Evidence of hemolysis during screening: (i) indirect bilirubin
- •>ULN and (ii) lactate dehydrogenase>ULN or haptoglobin <LLN
- •History of failure or relapse to at least 1 treatment regimen for CAD
- •(All indications) If receiving protocol-specified standard-of-care medications, doses must be stable for protocol-specified durations
排除标准
- •Secondary AIHA, CAD, or ITP
- •Treatment with any of the following within the noted period prior to study entry
- •rituximab: <12 weeks
- •IVIg: <4 weeks
- •sutimlimab, any use after initiation of screening is exclusionary
- •plasmapheresis, plasma exchange, or double-filtration plasmapheresis: <8 weeks
- •transfusions with blood, blood products or other rescue medications: <2 weeks
- •splenectomy: <12 weeks
- •other immunomodulatory or investigational agents, except for investigational agents for COVID-19 that have been granted emergency use authorization or approved by the applicable national health authority: <5 half-lives and requires agreement of the Medical Monitor
- •Recent serious or ongoing infection; risk or history of serious infection Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Part 1: Povetacicept 240mg
Participants grouped in 3 cohorts by diagnosis will be administered Povetacicept.
干预措施: Povetacicept (Drug)
Part 2: Povetacicept Dose A
Based on the findings of Part 1, one or two dose levels may be assessed in Part 2; if two dose levels are assessed participants will be randomized to receive one of the two dose levels of Povetacicept.
干预措施: Povetacicept (Drug)
结局指标
主要结局
Safety and Tolerability as Assessed by Adverse events (AEs) and Serious adverse events (SAEs)
时间窗: Study Day 1 through 30 days after last dose of study drug
Type, incidence, severity, and seriousness of AEs.
Type, incidence, severity, and seriousness of AEs.
次要结局
- Assessment of efficacy, PK, and ADA