A Phase 3, Multicenter, Open-Label Extension Study to Assess the Safety and Efficacy of ARD-101 in Patients With Prader-Willi Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 19
- 试验地点
- 72
- 主要终点
- Change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Score
研究概览
简要总结
The goal of this clinical trial is to learn if ARD-101 works to treat hyperphagia-related behavior in patients with Prader-Willi syndrome (PWS) when used in a long term setting. It will also teach us about the safety of ARD-101.
The main questions it aims to answer are:
What medical problems do participants have when taking ARD-101 in a long term setting
Does ARD-101 improve the total score of the HQCT-9 (hyperphagia questionnaire for clinical trials, 9 questions)?
Eligible participants will:
Have completed treatment on the AVK-101-301 study through Week 12/End of Treatment
Take ARD-101 every day for up to 12 months.
Visit the clinic at Months 1, 3, 6 and 12 during dosing and then have tele-visits at Week 2, Months 3 and 9, then 4 weeks after stopping the ARD-101.
Patients/Caregivers will keep a daily diary.
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
- 13 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who have completed treatment on Aardvark Therapeutics clinical study AVK-101-301
排除标准
- •Any complications that makes participation unsafe in the Investigator's opinion
研究组 & 干预措施
ARD-101
(Open-label)
干预措施: ARD-101 (Drug)
结局指标
主要结局
Change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Score
时间窗: During treatment period (approximately 12 months)
The HQ-CT score is a 9 question, 5-point scale to describe the PWS patient's hyperphagia food-related problem behaviors. It is completed by the patient's caregiver. Each question is scored from 0 to 4. The minimum total score is 0 (hyperphagia related behavior symptoms not exhibited) and the maximum total score is 36 (hyperphagia related behavior symptoms are observed).
次要结局
- Change in Caregiver Global Impression of Severity (CaGI-S) for Hyperphagia in Prader-Willi patients(During treatment period (approximately 12 months))