An Outpatient Phase 3 Efficacy Study of Ecopipam (PSYRX 101) in the Symptomatic Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 9
- 试验地点
- 2
- 主要终点
- Behavior Problems Inventory - Self-Injurious Behavior Subscale
研究概览
简要总结
The purpose of this research study is to gather scientific information about the effectiveness and safety of the study drug, Ecopipam (PSYRX 101), for the treatment of self-injurious behaviors when compared with the effectiveness and safety of placebo (inactive substance) in subjects with Lesch-Nyhan Disease.
详细描述
This study will be done in approximately 6 centers in approximately 4 countries, and approximately 24 subjects will be included. This study is divided into two parts. The first is a double-blinded portion lasting up to 18 weeks in total. The second portion is an optional open-label extension and lasts up to 54 weeks total. The total duration of the study, if you choose to participate in both portions, is anticipated to be up to approximately 78 weeks.
The first portion of this study is double-blind and assignment to a treatment group is done randomly. In this study, there are two treatment groups. One group will receive Ecopipam for one 6-week period and placebo for two 6-week periods, and the other group will receive Ecopipam for two 6-week periods and placebo for one 6-week period.
Subjects who did not experience any clinically significant side effects during the blinded portion of the study may be eligible to participate in an open-label extension that may last up to 54 weeks.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects must have classic LND as defined by (a) characteristic clinical syndrome (evidence of overproduction of uric acid, severe generalized dystonia, frequent and persistent self-injurious behavior (SIB), and cognitive impairment) and (b) laboratory confirmation for mutation of the HPRT gene or severe deficiency of the associated enzyme.
- •Subjects must have a minimum combined score of 20 on the Behavior Problems Inventory (BPI) SIB subscales for frequency and severity as assessed by the caregiver.
- •Subjects must have a minimum score of 4 on the Physician's Global Impression (PGI) severity scale.
- •Subject must be ≥ 6 years old.
- •Subjects must weigh > 10 kg.
排除标准
- •Subjects who are currently treated with medications for seizures.
- •Subjects who are on neuroleptics or dopamine-depleting agents.
- •Subjects with impaired renal function as defined by a serum creatinine >1.5 mg/dL.
研究组 & 干预措施
Ecopipam
Active substance being tested, orally once a day at bedtime
干预措施: Ecopipam (Drug)
Placebo
Inactive substance being tested, orally once a day at bedtime
干预措施: Placebo (Drug)
结局指标
主要结局
Behavior Problems Inventory - Self-Injurious Behavior Subscale
时间窗: Baseline, end of period 1 (6 weeks), end of period 2 (12 weeks), end of period 3 (18 weeks),
The primary endpoint is the BPI (SIB subscales - total for frequency and severity) as assessed by the caregiver. BPI Self-Injurious Behavior Subscale ranges from 0 to 45, with higher scores indicating more self-injurious behavior.
次要结局
- Effect of Ecopipam Withdrawal and Maintenance(Baseline, 6 weeks, 12 weeks, 18 weeks)
- Safety Summary of Ecopipam in Patients With Lesch-Nyhan Disease: Total Number of Serious and Non-Serious Adverse Events Experienced During 3 Double-blind Crossover Periods(Total duration over which participants recieved double-blind ecopipam or placebo, up to 6 or 12 weeks)