Phase 2a, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- CSL Behring
- 入组人数
- 63
- 试验地点
- 16
- 主要终点
- Number of participants with Treatment Emergent Adverse Events (TEAEs), Overall and by severity, seriousness and relationship to Investigational Product (IP)
研究概览
简要总结
This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD.
The primary aim of the study is to assess the safety of anumigilimab in participants with SCD. Participants will be treated for 64 weeks: for 12 weeks in the dose escalation period, where the dose will be escalated to each participant's individual MTD; and for 52 weeks at the MTD in the maintenance period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Adults aged greater than or equal to (>=) 18 years on the day of signing the informed consent form.
- •Confirmed diagnosis of SCD of any genotype.
- •Experienced 1 to 12 VOCs requiring a visit to a medical facility and treatment with parenteral opioids or a parenteral nonsteroidal anti-inflammatory drug within the 12 months before Screening.
- •HU Regimen:
- •a. On stable and well-tolerated Hydroxyurea (HU) regimen for at least 30 days before Screening.
- •b. HU was discontinued or refused (eg, due to concern of side effects or lack of effect).
排除标准
- •Absolute neutrophil count less than (<) 2.5 ×10^9 cells/Litre at Screening or Baseline (Week 1 Day 1).
- •If on SCD preventive medication, dose is not stable in the 30 days before Screening.
研究组 & 干预措施
Placebo
Participants will receive matching volume of placebo.
干预措施: Placebo (Drug)
Anumigilimab
Safety of Anumigilimab (CSL324) in Adults with Sickle Cell Disease (SCD)
干预措施: Anumigilimab (Biological)
结局指标
主要结局
Number of participants with Treatment Emergent Adverse Events (TEAEs), Overall and by severity, seriousness and relationship to Investigational Product (IP)
时间窗: From baseline up to Week 75
Percentage of participants with TEAEs Overall and by severity, seriousness and relationship to IP
时间窗: From baseline up to Week 75
Number of participants with Adverse Events of Special Interest (AESI)
时间窗: From baseline up to Week 75
The following AEs are to be reported as AESI: neutropenia (Common Terminology Criteria for Adverse Events \[CTCAE\] Grade 3 or 4), severe infection and neutropenic fever.
Percentage of participants with AESI
时间窗: From baseline up to Week 75
The following AEs are to be reported as AESI: neutropenia (CTCAE Grade 3 or 4), severe infection and neutropenic fever.
Number of participants with clinically relevant changes from baseline in laboratory assessments and vital signs
时间窗: From baseline up to Week 75
Percentage of participants with clinically relevant changes from baseline in laboratory assessments and vital signs
时间窗: From baseline up to Week 75
次要结局
- Serum concentration of Anumigilimab(From the first dose of anumigilimab to Week 65)
- Annualized rate of Vaso-occlusive crisis (VOC) (medical facility)(During the maintenance period from Week 13 to Week 65)