NCT05075824已完成2 期
A Randomized Double-Blind Phase IIA Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab as Adjunct Treatment in Prevention of Vaso-Occlusive Episodes (VOE) in Sickle Cell Disease (SCD)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 95
- 试验地点
- 36
- 主要终点
- Annualized rate of medical facility VOEs (AVR)
研究概览
简要总结
This study is designed to evaluate the efficacy, safety and pharmacokinetics of crovalimab compared with placebo as adjunct therapy in the prevention of VOEs in participants with SCD.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 12 Years 至 55 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Body weight >=40 kg.
- •Male or female with confirmed diagnosis of HbSS (SCD genotype of sickle cell anemia) or HbSβ0 (SCD genotype of sickle cell beta zero thalassemia).
- •Two or more (>=2) to <=10 documented VOEs in the 12 months prior to randomisation.
- •If receiving concurrent SCD-directed therapy, the participant must have been on a stable dose for a minimum of 3 months prior to study enrollment. There should be no plans to modify the participants' dosing throughout the study duration, other than for safety reasons.
- •If receiving erythropoietin, the participant must have been prescribed this medication for the preceding 3 months and be dose-stabilised for at least 3 months prior to study enrollment.
- •Vaccination against N. meningitides serotypes A, C, W, and Y and Vaccinations against H. influenza type B and S. pneumonia.
- •Participants who have been vaccinated (partially or in full) against SARS-CoV-2 with a locally approved vaccine are eligible to be enrolled in the study, 3 days or longer after inoculation.
- •Adequate hepatic and renal function.
- •For women of childbearing potential: agreement to remain abstinent or use contraception during the treatment period and for 10.5 months after the final dose of study treatment.
排除标准
- •History of hematopoietic stem cell transplant.
- •Participating in a chronic transfusion program and/or planning on undergoing an exchange transfusion during the duration of the study.
- •History of hypersensitivity, allergic, or anaphylactic reactions to any ingredient contained in the study treatment.
- •Received active treatment on another investigational trial within 28 days (or within five half-lives of that agent, whichever is greater) prior to screening visit, or plans to participate in another investigational drug trial.
- •Hemoglobin <6 g/dL.
- •Known or suspected hereditary complement deficiency.
- •Active systemic bacterial, viral, or fungal infection within 14 days before first drug administration.
- •Presence of fever (>=38 degrees Celsius) within 7 days before the first drug administration.
- •Immunised with a live attenuated vaccine within 1 month before first drug administration.
- •Pregnant or breastfeeding, or intending to become pregnant during the study or within 10.5 months after the final dose of study treatment.
- •Known HIV infection with documented CD4 count <200 cells/microliter within 24 weeks prior to screening.
- •History of N. meningitidis infection within the prior 6 months.
研究组 & 干预措施
Placebo
Placebo Comparator
Participants will receive matching Placebo administered by IV infusion and SC injection over the same duration as Crovalimab, for a total of 48 weeks of treatment.
干预措施: Placebo (Drug)
Crovalimab
Experimental
Participants will receive a loading series of Crovalimab comprised of an intravenous (IV) loading dose on Day 1, followed by weekly Crovalimab subcutaneous (SC) doses for 4 weeks on Week 1 Day 2, then on Weeks 2, 3 and 4. Maintenance SC dosing will begin at Week 5 and will continue every 4 weeks (Q4W) thereafter for a total of 48 weeks of treatment.
干预措施: Crovalimab (Drug)
结局指标
主要结局
Annualized rate of medical facility VOEs (AVR)
时间窗: Baseline up to Week 49
次要结局
- Percentage of Participants with TRV >2.5 m/s(Week 49)
- Change in Tricuspid Regurgitant Jet Velocity (TRV)(Baseline up to Week 49)
- Annualized rate of uncomplicated medical facility VOE(Baseline up to Week 49)
- Annualized rate of home VOE(Baseline up to Week 49)
- Annualized rate of Acute Chest Syndrome (ACS)(Baseline to up Week 49)
- Annualized rate of days hospitalized for medical facility VOE(Baseline up to Week 49)
- Annualized rate of days hospitalized for treatment of non-VOE complications of SCD(Baseline up to Week 49)
- Time to first medical facility VOE from randomization(Baseline up to Week 49)
- Change in urinary albumin-creatinine ratio(Baseline up to Week 49)
- Change in Patient-Reported Outcomes Measurement Information System (PROMIS)-Fatigue Score in Adults(Baseline up to Week 49)
- Percentage of Participants with Adverse Events (AEs)(Up to 91 weeks)
- Serum Concentrations of Crovalimab over time(Baseline up to Week 49)
- Percentage of Participants with Anti-Drug Antibodies to Crovalimab(Baseline up to Week 49)
研究者
研究点 (36)
Loading locations...
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