An Open-Label Extension Study to Evaluate the Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 154
- 试验地点
- 96
- 主要终点
- Number (%) of subjects with treatment emergent adverse events (TEAEs) over the study.
研究概览
简要总结
This is an open label extension study in subjects with Sickle Cell Disease (SCD) who have completed the double blind Phase 3 study (B5201002).
详细描述
This is an open label extension study in subjects who are 6 years of age or older with Sickle Cell Disease (SCD) who have completed the double blind Phase 3 study (B5201002). This study is designed to evaluate the safety and describe the efficacy of rivipansel as treatment for one or more vaso-occlusive crisis (VOC) events in hospitalized subjects with SCD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completion of Study B
- •Documented diagnosis of SCD.
- •At least 6 years of age.
- •Male and female subjects of childbearing potential and at risk for pregnancy must agree to use a highly effective method of contraception throughout the study.
- •Diagnosis of VOC necessitating IV opioids and admission to the hospital.
- •Able to receive the first dose of rivipansel within 24 hours from administration of the first dose of IV opioids for this hospitalization.
排除标准
- •Non-compliance with study procedures in the double blind study (B5201002).
- •Occurrence of any severe and/or generalized cutaneous manifestation or any other adverse event during participation in Study B5201002 that was related to study drug and which would therefore make it inappropriate for the subject to receive rivipansel in the current study.
- •Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or study drug administration or may interfere with the interpretation of study results.
- •Clinically significant deterioration in renal function in Study B
- •Pregnant female subjects, breastfeeding female subjects and male and female subjects of childbearing potential who are unwilling or unable to use a highly effective method of contraception.
- •Active use of illicit drugs and/or alcohol dependence.
研究组 & 干预措施
Cohort 1
Includes one adult stratum (>18 years old) and one pediatric stratum (12-17 years old). Subjects aged 12 and over who weigh >40 kg, will receive a loading dose of 1680 mg followed by a maintenance dose of 840 mg.
干预措施: Rivipansel (Drug)
Cohort 2
Includes one pediatric stratum (6-11 years old). Subjects 6 to 11 years of age or subjects who weigh 40 kg, will receive a loading dose of 40 mg/kg (maximum of 1680 mg) followed by a maintenance dose of 20 mg/kg (maximum of 840 mg).
干预措施: Rivipansel (Drug)
结局指标
主要结局
Number (%) of subjects with treatment emergent adverse events (TEAEs) over the study.
时间窗: 18 months
Number (%) of subjects with treatment emergent adverse events (TEAEs) over the study, number of TEAEs over the study and rate of TEAEs per subject per Vaso-Occlusive Crisis (VOC) will be summarized overall, by system organ class and by preferred term.
Number (%) of subjects with adjudicated Acute Chest Syndrome (ACS) over the study.
时间窗: 18 months
Number (%) of subjects with adjudicated Acute Chest Syndrome (ACS) over the study, number of events of adjudicated ACS and the rates of these events per subject per Vaso-Occlusive Crisis (VOC) will be summarized.
Number (%) of subjects with adjudicated severe and/or generalized cutaneous manifestations over the study
时间窗: 18 months
Number (%) of subjects with adjudicated severe and/or generalized cutaneous manifestations over the study, number of events of adjudicated severe and/or generalized cutaneous manifestations and the rates of these events per subject per Vaso-Occlusive Crisis (VOC) will be summarized.
Number (%) of subjects with serious adverse events (SAEs) over the study.
时间窗: 18 months
Number (%) of subjects with serious adverse events (SAEs) over the study, number of SAEs over the study and rate of SAEs per subject per VOC will be summarized overall, by system organ class and by preferred term.
次要结局
- Subject re hospitalization(18 months)