NCT06772597进行中（未招募）2 期
A Phase 2 Open-label Study of Setmelanotide in Patients With Prader-Willi Syndrome
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- Frequency and severity of adverse events (AEs)
研究概览
简要总结
This is a Phase 2, open-label study designed to assess the safety and efficacy of setmelanotide in patients with obesity due to Prader-Willi Syndrome (PWS) who are 6 to 65 years of age. Up to 20 patients are planned to be enrolled. Patients will take a daily subcutaneous dose of open-label setmelanotide for up to 52 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of Prader-Willi Syndrome (PWS)
- •Age 6 to 65
- •BMI ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th Percentile for age and sex
- •Agree to use a highly effective form of contraception and follow the study contraception requirements throughout study duration and for 90 days after.
- •Patient and/or guardian is able to communicate well with the Investigator, understand and comply with the requirements of the study, and understand English and sign the written informed consent.
排除标准
- •Use of weight modulating medications
- •Abnormal eGFR, ALT, AST, and bilirubin values
- •Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease considered severe enough to interfere with the trial and/or confound the results.
- •Participation in any clinical trial with an investigational drug/device within 3 months or 5 half-lives, whichever is longer, prior to the first trial dose
- •Hypersensitivity to setmelanotide
- •Diagnosis of severe psychiatric disorders
- •Pregnant and/or breastfeeding
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Setmelanotide (Open-label)
Experimental
干预措施: Setmelanotide (Drug)
结局指标
主要结局
Frequency and severity of adverse events (AEs)
时间窗: Baseline to Week 52
次要结局
- Proportion of patients who achieve 5% reduction in BMI(Baseline to Week 52)
- Mean percent change in BMI(Baseline to Week 52)
- Mean percent change in BMI and weight in patients ≥18 years of age(Baseline to Week 52)
- Mean change in BMI Z-Score and BMI % of 95th percentile(Baseline to Week 52)
- Proportion of patients who achieved 5% reduction in weight from Baseline in patients ≥18 years of age and who achieved ≥0.2 reduction in BMI Z-score in patients <18 years of age(Baseline to Week 52)
- Mean change in the weekly average of the Prader-Willi Syndrome Food Problem Diary (PWS-FPD) total score(Baseline to Week 52)
研究者
研究点 (1)
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