NCT06828861终止3 期
A Phase 3, Randomized, Double-blind, Placebo-controlled Study of ARD-101 for the Treatment of Hyperphagia in Patients With Prader-Willi Syndrome
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 68
- 试验地点
- 68
- 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). It will also teach us about the safety of ARD-101.
The main questions it aims to answer are:
- Does ARD-101 improve the total score of the HQCT-9 (hyperphagia questionnaire for clinical trials, 9 questions)?
- What medical problems do participants have when taking ARD-101?
Researchers will compare ARD-101 to a placebo (a look-alike substance that contains no drug) to see if ARD-101 works to treat hyperphagia in PWS subjects.
Eligible participants will:
- Take ARD-101 or a placebo every day for 12 weeks.
- Visit the clinic or have a tele-visit once every 2 to 4 weeks during dosing and then have a tele-visit 4 weeks after stopping the ARD-101 or placebo.
- Patients/Caregivers will keep a daily diary.
Participants who complete the study may be eligible to enter an open-label extension study where everyone will receive ARD-101.
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
- 7 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Documented confirmation of Prader-Willi Syndrome (PWS)
- •Stable care setting with same, single designated caregiver for at least 6 months prior to Visit 1
- •At least 7 years of age or older in the US at the time of consent
- •At least 10 years of age or older in Australia
- •At least 13 years of age or older in countries outside of the US and Australia
排除标准
- •Diagnosis of schizophrenia, bipolar disorder, personality disorder or other severe mood, anxiety or eating disorder (other than hyperphagia).
- •Presence of any malignancy within 5 years with the exception of basal or squamous cell carcinoma of the skin, in situ carcinoma of the service, or in situations prostate cancer.
- •Presence of clinically relevant renal, hepatic, pancreatic, cardiovascular, neurological, psychiatric, hematological, pulmonary, or GI abnormality that, in the opinion of the investigator, may preclude the patient from safe completion of the study
- •Adults: systolic blood pressure >=160 mmHg and/or diastolic blood pressure >=100 mmHg
- •Children and Adolescents: systolic blood pressure >=140 mmHg and/or diastolic blood pressure >=90 mmHg.
- •Type 1 diabetes mellitus; HbA1c >8.5%
- •Use of agents to promote weight gain or loss, alter hunger or appetite within 30 days of Visit 1 and throughout the study.
- •Use of any commercially available medication for the treatment of hyperphagia (i.e., Vykat) within 60 days of randomization and throughout the study.
- •Very high doses of glucocorticoids in the previous 3 months of Visit 1 and throughout the study.
研究组 & 干预措施
Treatment Arm A
Experimental
ARD-101
干预措施: ARD-101 (Drug)
Treatment Arm B
Placebo Comparator
Placebo for ARD-101
干预措施: Placebo (Drug)
结局指标
主要结局
Change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Score
时间窗: Baseline to Week 12
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(Baseline to Week 12)
- Change in Clinical Global Impression of Severity (CGI-S) Score for Hyperphagia in Prader-Willi patients(Baseline to Week 12)
研究者
研究点 (68)
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