A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 241
- 试验地点
- 58
- 主要终点
- Rate of Vaso-occlusive Crises (VOCs) [Adjudicated] Through Week 48
研究概览
简要总结
This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age) with sickle cell disease (SCD). Participants will be randomized to receive inclacumab or placebo.
详细描述
Eligible participants will be administered inclacumab or placebo intravenous (IV) every 12 weeks.
The total duration of treatment for each participant will be 48 weeks.
Participants that complete the study through Week 48 will be provided the opportunity to enroll in an open-label extension (OLE) study.
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)
盲法说明
Double blind study
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant has a confirmed diagnosis of SCD (HbSS, HbSC, HbSB0 thalassemia, or HbSB+ thalassemia genotype).
- •Documentation of SCD genotype is required and may be based on documented history of laboratory testing or confirmed by laboratory testing during Screening.
- •Participant is male or female, ≥ 12 years of age at the time of informed consent.
- •Participant has experienced between 2 and 10 VOCs within the 12 months prior to the Screening Visit as determined by documented medical history. A prior VOC is defined as an acute episode of pain which:
- •Has no medically determined cause other than a vaso-occlusive event, and
- •Results in a visit to a medical facility (hospital, emergency department, urgent care center, outpatient clinic, or infusion center) or results in a remote contact with a healthcare provider; and
- •Requires parenteral narcotic agents, parenteral nonsteroidal anti- inflammatory drugs (NSAIDs), or an increase in treatment with oral narcotics.
- •Participants receiving erythropoiesis-stimulating agents (ESA, e.g., erythropoietin [EPO]) must be on a stable dose for at least 90 days prior to the Screening Visit and expected to continue with the stabilized regimen throughout the course of the study.
- •Participants receiving hydroxyurea (HU), L-glutamine, or voxelotor (Oxbryta®) must be on a stable dose for at least 30 days prior to the Screening Visit and expected to continue with the stabilized regimen throughout the course of the study.
排除标准
- •Participant is receiving regularly scheduled red blood cell (RBC) transfusion therapy (also termed chronic, prophylactic, or preventative transfusion).
- •Participant is taking or has received crizanlizumab (ADAKVEO®) within 90 days prior to the Screening Visit
- •Participant weighs > 133 kg (292 lbs.).
- •Other protocol-defined Inclusion/Exclusion may apply.
研究组 & 干预措施
inclacumab, 30 mg/kg
Participants will receive inclacumab 30 mg/kg administered IV every 12 weeks
干预措施: Inclacumab (Drug)
placebo
Participants will receive placebo administered IV every 12 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Rate of Vaso-occlusive Crises (VOCs) [Adjudicated] Through Week 48
时间窗: Randomization (Day 1) up to Week 48
A VOC was defined as an acute episode of pain that: had no medically determined cause other than a vaso-occlusive event; resulted in a visit to a medical facility (hospitalization, emergency department, urgent care center, outpatient clinic, or infusion center), or resulted in a remote contact with a healthcare provider and required parenteral narcotic agents, parenteral nonsteroidal anti-inflammatory drugs (NSAIDs), or an increase in treatment with oral narcotics. The rate of VOC was defined as number of VOC events per 48 weeks and presented in this outcome measure.
次要结局
- Time to First VOC Through Week 48(Randomization (Day 1) up to Week 48)
- Time to Second VOC Through Week 48(Randomization (Day 1) up to Week 48)
- Percentage of Participants With no VOCs Through Week 48(Randomization (Day 1) up to Week 48)
- Rate of VOCs Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication [Adjudicated] Through Week 48(Randomization (Day 1) up to Week 48)
- Rate of Inpatient Hospitalization Days for a VOC Through Week 48(Randomization (Day 1) up to Week 48)
- Number of Participants With Treatment Emergent Adverse Events (TEAEs)(Day 1 up to Week 60 (12 week of follow-up post Week 48))