A Phase II, Multicenter, Randomized, Placebo-Controlled, Double-Blind, 12-Month Study to Assess Safety and Efficacy of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Sickle Cell-Related Pain Crises
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 198
- 主要终点
- Annual Rate of Sickle Cell-related Pain Crises (SCPC) Per Hodges-Lehmann Median
研究概览
简要总结
The purpose of this study was to determine whether the investigational drug SelG1 when given to sickle cell disease patients either taking or not taking hydroxyurea was effective in preventing or reducing the occurrence of pain crises. SelG1 prevents various cells in the bloodstream from sticking together. By stopping these cell-cell interactions, SelG1 may prevent small blood vessels from becoming blocked and therefore reduce the occurrence and severity of pain crises. Other effects of SelG1 was evaluated, as well as the safety of the drug and how long it stayed in the blood stream.
Funding Source - FDA Office of Orphan Products Development (OOPD)
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)
入排标准
- 年龄范围
- 16 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Sickle Cell Disease (HbSS, HbSC, HbSβ⁰-thalassemia, or HbSβ⁺-thalassemia)
- •If receiving hydroxyurea or erythropoietin, treatment must have been prescribed for at least 6 months, with the dose stable for at least 3 months
- •Between 2 and 10 sickle cell-related pain crises in the past 12 months
排除标准
- •On a chronic transfusion program or planning on exchange transfusion during the study
- •Hemoglobin <4.0 g/dL
- •Planned initiation, termination, or dose alteration of hydroxyurea during the study
- •Receiving chronic anticoagulation therapy (e.g. warfarin, heparin) other than aspirin
研究组 & 干预措施
High-dose SelG1 (Selg1 5.0 mg/kg)
IV Infusion, once every 4 weeks through Week 50
干预措施: SelG1 (Drug)
Low-dose SelG1 (Selg1 2.5 mg/kg)
IV Infusion, once every 4 weeks through Week 50
干预措施: SelG1 (Drug)
Placebo
IV Infusion, once every 4 weeks through Week 50
干预措施: Placebo (Drug)
结局指标
主要结局
Annual Rate of Sickle Cell-related Pain Crises (SCPC) Per Hodges-Lehmann Median
时间窗: One year
An SCPC is defined as an acute episode of pain with no other medically determined cause than a vasoocclusive event that requires a medical facility visit and treatment with oral or parenteral narcotics, or parenteral non-steroidal anti-inflammatory drugs. The annual rate of SCPC is defined as the total number of pain crises for a patient occurring from the date of randomization to the end date multiplied by 365 divided by the number of days during that same time period. End date is defined as the last dose date plus 14 days. For participants never dosed, the end date was the end of study date. This calculation accounts for early dropouts or lost to follow-up by extrapolating the SCPC rate of every participant to one year.
Annual Rate of Sickle Cell-related Pain Crises (SCPC) - Per Standard Median
时间窗: One year
An SCPC is defined as an acute episode of pain with no other medically determined cause than a vasoocclusive event that requires a medical facility visit and treatment with oral or parenteral narcotics, or parenteral non-steroidal anti-inflammatory drugs. The annual rate of SCPC is defined as the total number of pain crises for a patient occurring from the date of randomization to the end date multiplied by 365 divided by the number of days during that same time period. End date is defined as the last dose date plus 14 days. For participants never dosed, the end date was the end of study date. This calculation accounts for early dropouts or lost to follow-up by extrapolating the SCPC rate of every participant to one year.
次要结局
- Patient Reported Outcome: Change From Baseline in Pain Severity/Pain Interference Domain From Brief Pain Inventory (BPI) Questionnaire(Baseline, Day 15, Week 14, Week 26, Week 38, Week 52, and Week 58, up to 58 weeks)
- Annual Rate of Days Hospitalized (Key Secondary Endpoint) Per Hodges-Lehmann Median(One year)
- Time to First Sickle Cell-related Pain Crisis(Up to one year)
- Annual Rate of Uncomplicated Sickle Cell-related Pain Crisis Per Hodges-Lehmann Median(Up to one year)
- Time to Second Sickle Cell-related Pain Crisis(Up to one year)
- Annual Rate of Acute Chest Syndrome Per Hodges-Lehmann Median(One year)