A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study of RAD011 (Cannabidiol Oral Solution) for the Treatment of Patients With Prader- Willi Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 4
- 试验地点
- 8
- 主要终点
- Change From Baseline to Week 34 in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Questionnaire
研究概览
简要总结
This was a study investigating RAD011 in participants diagnosed with Prader-Willi Syndrome (PWS). The primary objective of the Phase 2 part of this study was to assess the safety and tolerability of multiple dose levels of RAD011 in order to select 1 or 2 dose level(s) for further evaluation in the Phase 3 part of the study. In Phase 3, the primary objective was to assess the effect of RAD011 on hyperphagia-related behavior in participants 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
- 8 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females between 8 and 65 years of age (inclusive) at Screening.
- •Genetically confirmed diagnosis of PWS. Documentation of genetically confirmed diagnosis of PWS is acceptable.
- •The same caregiver is available to complete the questionnaire throughout the duration of the study.
- •After completion of the Tolerability period, participants will have a mean Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score ≥13 and a decrease of HQ-CT score no more than 7 during Tolerability (run-in) period.
- •If receiving growth hormone, psychotropic therapy, metabolic treatments that could affect appetite (including metformin), and other treatment including thyroid hormone, must be on the same medication and dose for at least 90 days prior to consent/assent
排除标准
- •Known use of cannabis or cannabinoid containing products (including topical products) within 90 days prior to consent/ assent.
- •Use of prescription or over-the-counter weight loss agents within 90 days prior to consent/assent.
- •Implementation of new food or environmental restrictions within 90 days of consent/ assent.
- •If living in a group home, participant spends less than 25 waking hours with their caregiver per week.
- •Uncontrolled chronic conditions (diabetes, sleep apnea, etc.) as determined by the Investigator.
研究组 & 干预措施
RAD011 40 milligrams per kilogram (mg/kg)
Participants were administered 40 mg/kg of RAD011 orally daily with food.
干预措施: RAD011 (Drug)
RAD011 20 mg/kg
Participants were administered 20 mg/kg of RAD011 orally daily with food.
干预措施: RAD011 (Drug)
RAD011 10 mg/kg
Participants were administered 10 mg/kg of RAD011 orally daily with food.
干预措施: RAD011 (Drug)
Placebo
Participants were administered a placebo matching to RAD011, orally daily with food.
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline to Week 34 in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Questionnaire
时间窗: Baseline, Week 34
The HQ-CT measures hyperphagia by Prader-Willi syndrome (PWS)-specialized clinicians. The HQ-CT generates a score ranging from 0 to 36, where a higher score represents more severe abnormal food related behaviors. The change from baseline was calculated from two time points as the value at the later time point minus the value at the earlier time point.
次要结局
- Change From Baseline to Week 34 in Prader-Willi Syndrome (PWS)- Associated Irritability Using the Aberrant Behavior Checklist (ABC) Questionnaire - Irritability Subscale (ABC-I)(Baseline, Week 34)
- Change From Baseline to Week 34 in Hyperphagia as Measured by the Clinician Global Impression of Change (CGI-C)(Baseline, Week 34)
- Change From Baseline to Week 34 in Hyperphagia as Measured by the Clinician Global Impression of Severity (CGI-S)(Baseline, Week 34)