NCT04912869已完成1 期
A Phase IB Randomized, Placebo-Controlled Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Patients With Sickle Cell Disease (SCD)
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 30
- 试验地点
- 16
- 主要终点
- Percentage of Participants With Adverse Events (AEs)
研究概览
简要总结
The purpose of this study is to evaluate crovalimab for the treatment of a sickle cell pain crisis (also known as a VOE) that requires hospitalisation in adult and adolescent participants with SCD. The primary objective of this study is safety and will additionally evaluate pharmacokinetics (how crovalimab is processed by your body), pharmacodynamics (how your body reacts to crovalimab) and the preliminary efficacy of crovalimab compared with placebo.
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.
- •Confirmed diagnosis of HbSS (SCD genotype of sickle cell anemia) or HbSβ0 (SCD genotype of sickle cell beta zero thalassemia).
- •Vaccination against Neisseria Meningitidis serotypes A, C, W, and Y.
- •Vaccinations against H. influenzae type B and S. pneumoniae.
- •Participants vaccinated against SARS-CoV-2 are eligible, as long as it has been 3 days or more after inoculation with the vaccine.
- •Diagnosis of an acute uncomplicated VOE, that requires admission to a hospital/acute medical facility and treatment with parenteral opioid analgesics.
- •Adequate hepatic and renal function.
- •Hemoglobin >=5 grams/deciliter (g/dL)
- •Platelet count >=100,000/microliter (µL)
- •Participants receiving SCD-directed therapies must be on a stable dose for >=28 days.
- •For female participants of childbearing potential, an agreement to remain abstinent or use contraception for 322 days (approximately 10.5 months) after the dose of study treatment.
排除标准
- •More than 10 VOEs within the last 12 months prior to presentation, that have required a medical facility visit.
- •Pain related to the current VOE ongoing for >36 hours.
- •Acute pain related to avascular necrosis, hepatic or splenic sequestration, or priapism.
- •Pain atypical of an acute uncomplicated VOE.
- •Evidence of or suspicion of ACS.
- •Evidence or high suspicion of a severe systemic infection.
- •Major surgery and/or hospitalization for any reason within 30 days.
- •History of Neisseria meningitidis infection within 6 months prior.
- •Known HIV infection with a documented CD4 count <200 cells/µL.
- •Transfusion or receipt of blood products within 3 months or current participation in a chronic transfusion protocol.
- •Immunized with a live attenuated vaccine within 30 days.
- •History of hematopoietic stem cell transplant.
- •Known or suspected hereditary complement deficiency.
- •Pregnant or breastfeeding, or intending to become pregnant during the study or within 322 days (approximately 10.5 months) after the study drug administration.
- •Participation in another interventional treatment study with an investigational agent or use of any experimental therapy within the prior 28 days or within five half-lives of that investigational product, whichever was greater.
研究组 & 干预措施
Placebo
Placebo Comparator
Participants will receive a single IV infusion of matching Placebo.
干预措施: Placebo (Drug)
Crovalimab
Experimental
Participants will receive a single intravenous (IV) infusion of Crovalimab based on body weight.
干预措施: Crovalimab (Drug)
结局指标
主要结局
Percentage of Participants With Adverse Events (AEs)
时间窗: Baseline up to Day 84
Percentage of Participants with Infusion-Related Reactions and Hypersensitivity
时间窗: Baseline up to Day 84
次要结局
- Time to Improvement of the Primary Acute Uncomplicated VOE From Baseline(Baseline up to Day 84)
- Total Cumulative Opioid Dose From Baseline(Baseline up to Day 84)
- Time to Discontinuation of all Parenteral Opioids From Baseline(Baseline up to Day 84)
- Time to Readiness For Hospital Discharge From Baseline(Baseline up to Day 84)
- Time to Hospital Discharge From Baseline(Baseline up to Day 84)
- Time to a Confirmed Decrease in Pain Score of at Least 2 Points From the Maximal Pre-dose Pain Score(Baseline up to Day 84)
- Change in Pain Score From the Maximal Pre-dose Pain Score to the Score at Hospital Discharge(Baseline up to Day 84)
- Percentage of Participants who Develop Acute Chest Syndrome (ACS)(Baseline up to Day 28)
- Percentage of Participants Requiring Intensive Care Unit (ICU)/Critical Care Admission for SCD-related Complications(Baseline up to Day 84)
- Percentage of Participants Requiring Blood Transfusion for SCD-related Complications(Baseline up to Day 84)
- Readmission Rate for a VOE or VOE-related Event Within 28 days of Discharge of the Primary Acute Uncomplicated VOE(Baseline up to Day 84)
- Serum Concentrations of Crovalimab Over Time(Baseline up to Day 84)
- Change in PD Biomarkers Including Complement Activity (CH50) Over Time(Baseline up to Day 84)
- Change Over Time in Free C5 Concentration(Baseline up to Day 84)
- Change Over Time in Soluble Complement 5b 9 (sC5b-9) Concentration(Baseline up to Day 84)
- Percentage of Participants with Anti-Drug Antibodies to Crovalimab(Baseline up to Day 84)
研究者
研究点 (16)
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