CTRI/2024/07/069743尚未招募2/3 期
A Phase 2/3 Randomized, Multicenter Study of Osivelotor Administered Orally to Participants with Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants with Sickle Cell Disease
Global Blood Therapeutics, Inc. a wholly owned subsidiary of Pfizer5 个研究点 分布在 1 个国家目标入组 332 人开始时间: 2024年7月12日最近更新:
试验速览
- 阶段
- 2/3 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 332
- 试验地点
- 5
- 主要终点
- To assess the effects of osivelotor adults 150 mg QD dose compared to placebo in adult and adolescent participants with SCD as measured by Hb response and rate of VOC events
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.
This study is three part, multicenter, phase 2/3 study for orally administered osivelotor in participants with sickle cell disease (SCD). India is participating in Part B of the study. Part B will evaluate the efficacy of osivelotor versus placebo in adult and adolescent participants with SCD for 48 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 盲法
- Participant, Investigator, Outcome Assessor and Date-entry Operator Blinded
入排标准
- 年龄范围
- 12.00 Year(s) 至 65.00 Year(s)（—）
- 性别
- All
入选标准
- •Key Inclusion Criteria:
- •Participant with SCD ages 12 years and older, inclusive at Screening.
- •Participants with stable Hb value as judged by the Investigator.
- •For participants taking hydroxyurea and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the ICF or assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator.
- •More than or equal to 2 and less than or equal to 10 VOCs within 12 months of Screening.
排除标准
- •Key Exclusion Criteria:
- •More than 10 VOCs within 12 months of Screening.
- •Female participant who is breastfeeding or pregnant.
- •Receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or has received an RBC or exchange transfusion for any reason within 90 days of Day
- •Hospitalized for sickle cell crisis or other vaso occlusive event within 14 days of signing the ICF.
- •Screening laboratory test of alanine aminotransferase (ALT) more than 4 x upper limit of normal (ULN) for age.
结局指标
主要结局
To assess the effects of osivelotor adults 150 mg QD dose compared to placebo in adult and adolescent participants with SCD as measured by Hb response and rate of VOC events
时间窗: Hb response (increase from baseline of more than 1 g per dL) at Week 48 (based on average of Hb levels at Week 40 and Week 48). Annualized rate of VOC through end of Week 48
次要结局
- To evaluate the effects of osivelotor (adults: 150 mg QD dose) compared to placebo on relevant clinical outcomes including time to first VOC, measures of hemolysis, healthrelated quality of life assessments, neurocognitive function, & total Hb. To evaluate the safety & tolerability of 48 weeks of daily osivelotor administration.(Key Secondary Efficacy Endpoints:)
- To evaluate the effects of osivelotor adults: 150 mg QD dose compared to placebo on relevant clinical outcomes including time to first VOC, measures of hemolysis, healthrelated quality of life assessments, neurocognitive function, & total Hb. To evaluate the safety & tolerability of 48 weeks of daily osivelotor administration.(4. Time from randomization to prohibited or restricted medications known to affect Hb or VOC)
- To evaluate the effects of osivelotor adults: 150 mg QD dose compared to placebo on relevant clinical outcomes including time to first VOC, measures of hemolysis, healthrelated quality of life assessments, neurocognitive function, & total Hb. To evaluate the safety & tolerability of 48 weeks of daily osivelotor administration(11. PGI-C of fatigue value from baseline through Week 48)
- To evaluate the effects of osivelotor adults 150 mg QD dose compared to placebo on relevant clinical outcomes including time to first VOC, measures of hemolysis, healthrelated quality of life assessments, neurocognitive function, & total Hb. To evaluate the safety & tolerability of 48 weeks of daily osivelotor administration(16. Change from baseline through Wk 48 in the Executive abilities composite score using Dimensional Change Card Sort Test, Flanker Inhibitory Control & Attention Test, & Pattern Comparison Processing Speed Test as assessed by the NIH Toolbox Cognition Module)
研究者
研究点 (5)
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