NCT03274856已完成2 期
A Phase 2 Study to Evaluate Efficacy, Safety, and Pharmacokinetics of GLWL-01 in the Treatment of 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
- 已完成
- 入组人数
- 19
- 试验地点
- 7
- 主要终点
- Post-treatment Total Score on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT)
研究概览
简要总结
The aim of this study is to evaluate efficacy, safety, and pharmacokinetics of GLWL-01 in the treatment of patients with Prader-Willi Syndrome (PWS).
详细描述
Participants will be assigned to one of two treatment sequences (GLWL-01/Placebo or Placebo/GLWL-01), with each sequence consisting of two treatment periods separated by a washout period
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 16 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 PWS based on genetic confirmation using DNA method
- •Body mass index (BMI) of 27 to 60 kg/m2
- •No evidence of weight excursion beyond 10% of baseline weight
- •Patients must provide assent and have a reliable caregiver (must have been caring for the patient for at least 6 months) who provides a separate written informed consent to participate. The caregiver is expected to be the primary caregiver throughout the study and must be in frequent contact with the patient (defined as at least 4 awake hours per day). The caregiver must be able to communicate with site personnel and in the investigator's opinion must have adequate literacy to complete questionnaires. If a caregiver cannot continue, 1 caregiver replacement is allowed
- •Are on a stable diet and exercise regimen for >2 months prior
排除标准
- •Current enrollment in or discontinuation within the last 30 days from a clinical trial involving any investigational drug or device
- •Are currently living in a group home for more than 50% of the time
- •A history or presence of other medical illness that indicates a medical problem that would preclude study participation
- •Have an estimated glomerular filtration rate <60 mL/minute/1.73 m
- •Have macroalbuminuria (defined as spot urine albumin to creatinine ratio of >300 μg/mg) or hematuria
- •Are hypertensive (defined as sitting systolic blood pressure (BP) greater than or equal to (≥)140 millimeters of mercury (mmHg) and diastolic BP ≥90 mmHg)
- •Patients on weight loss medications within 30 days of dosing, or with a history of bariatric surgery
- •Unable to refrain from or anticipates the use of:
- •Any drugs known to be significant inhibitors of Cytochrome P450, family 3, subfamily A (CYP)3A enzymes and/or P-glycoprotein (P-gp) including regular consumption of grapefruit or grapefruit juice for 14 days prior to the first dose. Acetaminophen (up to 2 grams per 24-hour period) may be permitted
- •Any drugs known to be significant inducers of Cytochrome P450, family 3, subfamily A (CYP3A) enzymes and/or P-gp, including St. John's Wort
- •Any medications that prolong the QT/QTc interval, unless the participant has been stable on the medication for at least 3 months and has a corrected QT interval (QTc) <450 msec
- •Currently taking simvastatin >10 mg per day, atorvastatin >20 mg per day, or lovastatin >20 mg per day, or have a history of statin-induced myopathy/rhabdomyolysis
- •Unsuitable for inclusion in the study in the opinion of the investigator
研究组 & 干预措施
Treatment Sequence 1
Other
GLWL-01 (450mg) twice a day/ Placebo
干预措施: Placebo (Drug)
Treatment Sequence 2
Other
Placebo / GLWL-01 (450mg), twice a day
干预措施: GLWL-01 (Drug)
Treatment Sequence 1
Other
GLWL-01 (450mg) twice a day/ Placebo
干预措施: GLWL-01 (Drug)
Treatment Sequence 2
Other
Placebo / GLWL-01 (450mg), twice a day
干预措施: Placebo (Drug)
结局指标
主要结局
Post-treatment Total Score on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT)
时间窗: Up to approximately 4 weeks of double-blind treatment
GLWL-01 compared with placebo on the post-treatment HQ-CT score. Total range of score of zero to 36, with higher score indicating a worse outcome.
次要结局
- Number of Participants With One or More Treatment Emergent Adverse Events (AEs) or Any Serious AEs(Baseline up to approximately 18 weeks)
- Caregiver Global Impression of Change (CGIC)(Up to approximately 4 weeks of double-blind treatment)
- Area Under the Concentration Versus Time Curve From Time Zero to 12 Hours (AUC0-12)(Day 14 and Day 42, pre-dose, and 0.5, 1, 2, 4, 6, and between 8 and 12 hours postdose)
- Maximum Observed Drug Concentration (Cmax)(Day 14 and Day 42, pre-dose, and 0.5, 1, 2, 4, 6, and between 8 and 12 hours postdose)
研究者
研究点 (7)
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