NCT03036813已完成3 期
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 449
- 主要终点
- Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24
研究概览
简要总结
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
详细描述
This is a randomized, placebo-controlled, double blind, parallel group, multicenter study of participants, age 12 to 65 years, with SCD. The key purpose for the study is to establish efficacy and safety of voxelotor as 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
- Triple (Participant, Care Provider, Investigator)
盲法说明
This study is a double-blind study.
入排标准
- 年龄范围
- 12 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female study participants with sickle cell disease
- •Participants have had at least 1 episode of vaso-occlusive crisis (VOC) in the past 12 months.
- •Age 12 to 65 years
- •Hemoglobin (Hb) ≥5.5 and ≤10.5 g/dL during screening
- •For participants taking hydroxyurea (HU), the dose of HU (mg/kg) must be stable for at least 3 months prior to signing the ICF.
排除标准
- •More than 10 VOCs within the past 12 months that required a hospital, emergency room or clinic visit
- •Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or have received a RBC transfusion for any reason within 60 days of signing the ICF
- •Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF (i.e., a vaso-occlusive event cannot be within 14 days prior to signing the ICF)
- •Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × upper limit of normal
- •Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the central laboratory) <30 mL/min/1.73 m^2 or on chronic dialysis
研究组 & 干预措施
Dose 1
Active Comparator
voxelotor
干预措施: voxelotor (Drug)
Dose 2
Active Comparator
voxelotor
干预措施: voxelotor (Drug)
Placebo
Placebo Comparator
Placebo
干预措施: Placebo (Other)
结局指标
主要结局
Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24
时间窗: Baseline to Week 24
Number of participants with increase in Hb \>1 g/dL from Baseline to Week 24
次要结局
- Annualized Vaso-Occlusive Crisis (VOC) Incidence Rate(Baseline to Week 72)
- Percentage Change From Baseline in Hemolysis Measures(Baseline to Week 24)
研究者
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