A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 170
- 试验地点
- 30
- 主要终点
- Change from Baseline at Week 12 in caregiver-rated Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score
研究概览
简要总结
12-week, randomized, double-blind, placebo-controlled, parallel-group study of carbetocin nasal spray for the treatment of hyperphagia in Prader-Willi syndrome (PWS)
详细描述
This is a 12-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group study comparing carbetocin nasal spray 3.2 mg TID with placebo (matched placebo nasal spray TID) in subjects with PWS.
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
- 5 Years 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Carbetocin
Carbetocin nasal spray 3.2 mg three times daily (TID)
干预措施: Carbetocin (Drug)
Placebo
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Change from Baseline at Week 12 in caregiver-rated Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score
时间窗: Baseline to Week 12
The HQ-CT is a nine-item questionnaire designed to be completed by caregivers of subjects with PWS. It is a revision of the 11-item HPWSQ-R and has been further validated. The Foundation for Prader-Willi Research has made the HQ-CT available for clinical studies in PWS, and it is the consensus instrument within the PWS research community for measuring observable behaviors that stem from subjects' excessive drive to eat. The HQ-CT should be completed by the same caregiver throughout the study. The HQ-CT will be administered to the caregiver by a rater using standardized prompts. The Food Safe Zone should be administered immediately before administration of the HQ-CT. A higher score on the HQ-CT indicates greater severity of hyperphagia.
次要结局
- Change from Baseline at Week 12 in caregiver-rated Clinical Global Impression-Severity (CGI-S) score for PWS(Baseline to Week 12)
- Clinical Global Impression-Change (CGI-C) for PWS score at Week 12(Score at Week 12)