A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients With Prader-Willi Syndrome (PWS)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 19
- 试验地点
- 4
- 主要终点
- Incidence of Treatment-Emergent Adverse Events (TEAE)
研究概览
简要总结
A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients with Prader-Willi Syndrome
详细描述
This is a Phase 2, open-label study to investigate the effects of ARD-101 in subjects with Prader-Willi Syndrome. The study will consist of a Screening Period (up to 28 days), a Treatment Period (28 days), and a Follow-up Period (End-of-Study Visit within 14 days after receiving the last dose of ARD-101). The screening procedures will be initiated upon completion of the informed consent process. Following completion of screening procedures and confirmation of eligibility, subjects will be enrolled to receive ARD-101 in an outpatient setting and will be instructed to visit the clinical center periodically for safety and efficacy assessments.
ARD-101 will be provided as a fixed dose of 200 mg BID for 28 days (Group 1) and then in a dose escalation of 1 week at 400 mg BID, 1 week at 600 mg BID, then 2 weeks at 800 mg BID (Group 2).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 17 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female subjects, 17-65 years of age
- •Provide voluntary, written informed consent (parent(s) / legal guardian(s) of participant); provide voluntary, written assent (participants, as appropriate)
- •PWS due to chromosome 15 micro-deletion, maternal uniparental disomy, or imprinting defect, confirmed by fluorescent in situ hybridization, chromosomal microarray, and/or methylation studies
- •BMI ≥ 18.5 kg/m²
- •Qualifying HQ-CT score
排除标准
- •Use of weight loss agents, including herbal medication, within 3 months prior to enrollment
- •Diagnosis of schizophrenia, bipolar disorder, personality disorder, or other DSM-III disorders which the investigator believes will interfere significantly with study compliance
- •Clinically significant illness in the 8 weeks prior to enrollment
- •Current, clinically significant liver, renal, pulmonary, cardiac, oncologic, or gastrointestinal (GI) disease
- •Diagnosis of type 1 diabetes mellitus or other active endocrine disorders (e.g., Cushing syndrome, or thyroid dysfunction except if on stable adequate thyroid or glucocorticoid replacement supplement)
- •Significant history of abuse of drugs within 1 year prior to enrollment or a positive Drugs of Abuse (DOA) test at screening
- •History of alcohol abuse within 1 year prior to enrollment or currently drinks in excess of 21 units per week (3 servings or units/day)
研究组 & 干预措施
ARD-101 (Fixed Dose)
ARD-101: 4 weeks dosing at 200 mg BID (twice daily)
干预措施: ARD-101 (Drug)
ARD-101 (Dose Escalation)
ARD-101: 1 week at 400 mg BID (twice daily), second week at 600 mg BID, third and fourth weeks at 800 mg BID. Oral administration.
干预措施: ARD-101 (Drug)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events (TEAE)
时间窗: Baseline to Day 28
The incidence of treatment-emergent adverse events (TEAE) during the treatment period
Incidence of Treatment Emergent Adverse Events (TEAE)
时间窗: Adverse Events were collected from time of informed consent through end of study; approximately 70 days (28 days of screening, 28 days of treatment, 14 days post treatment follow up).
The incidence of treatment emergent adverse events (TEAE) reported through the 28 days of treatment and the 14 days post treatment phase. TEAEs were those events occurring after treatment began.
次要结局
- Effect on Weight(Baseline to Day 28)
- Efficacy Evaluation of Hyperphagia in Prader-Willi Syndrome(Baseline, Day 15, Day 28)
- Change in Body Weight(Baseline to day 28)