A Randomized, Double-blind, Placebo-controlled, Multicenter Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- Pfizer
- 入组人数
- 72
- 试验地点
- 59
- 主要终点
- Percentage of Participants With at Least 1 VOC That Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication Within 90 Days of Randomization
研究概览
简要总结
This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD). Participants will be randomized to receive either inclacumab or placebo.
详细描述
The study will include approximately 280 adult and adolescent participants (≥ 12 years of age) with SCD.
Eligible participants will be administered inclacumab or placebo intravenous (IV) as a single dose.
Participants that complete the study through Day 90 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 an index VOC. The index VOC is any VOC that required admission to a healthcare facility and treatment with parenteral pain medication. An admission for the index VOC includes:
- •A hospital admission, or
- •An admission to an emergency room, observation unit, or infusion center for ≥ 12 hours, or
- •2 visits to an emergency room, observation unit, or infusion center over a 72-hour period
- •for an acute episode of pain with no other cause other than a vaso- occlusive event that includes the following:
- •Uncomplicated VOC,
- •Acute chest syndrome (ACS),
- •Acute hepatic sequestration,
- •Acute splenic sequestration, or
- •Priapism.
- •Participant has a confirmed diagnosis of SCD (any genotype). Documentation of SCD genotype is required and may be based on documented history of laboratory testing or confirmed by laboratory testing at Baseline.
- •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 Screening as determined by documented medical history. The index VOC is not to be considered as one of the 2 to 10 events. A prior VOC is defined as an acute episode of pain that:
- •Has no medically determined cause other than a vaso-occlusive event, and
- •Results in a visit to a healthcare 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 Screening 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 Screening 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 Screening.
- •Participant weighs > 133 kg (292 lbs.).
- •Other protocol-defined Inclusion/Exclusion may apply.
研究组 & 干预措施
inclacumab 30 mg/kg
Inclacumab 30 mg/kg administered intravenously (IV)
干预措施: Inclacumab (Drug)
placebo
Placebo administered IV
干预措施: Placebo (Drug)
结局指标
主要结局
Percentage of Participants With at Least 1 VOC That Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication Within 90 Days of Randomization
时间窗: Within 90 days of randomization (randomization happened on Day 1 [Day 1 to Day 91])
A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. An admission for a VOC included a hospital admission, or an admission to an emergency room, observation unit, or infusion center for \>= 12 hours, or 2 visits to an emergency room, observation unit, or infusion center over a 72-hour period. An acute episode of pain with no other cause other than a vaso-occlusive event included: uncomplicated VOC, acute chest syndrome, hepatic sequestration, splenic sequestration, or priapism. All on-study VOCs were adjudicated by an independent, blinded VOC Adjudication Committee comprised of experts in SCD.
次要结局
- Time to First VOC That Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication Within 90 Days of Randomization(Within 90 days of randomization (randomization happened on Day 1 [Day 1 to Day 91]) or censored day, whichever occurred earlier)
- Percentage of Participants With at Least 1 VOC That Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication Within 30 Days of Randomization(Within 30 days of randomization (randomization happened on Day 1 [Day 1 to Day 31]))
- Rate of VOCs Leading to a Healthcare Visit That Requires Parenteral Pain Medication or an Increase in Treatment With Oral Narcotics Within 90 Days Following Randomization(Within 90 days of randomization (randomization happened on Day 1 [Day 1 to Day 91]))