NL-OMON37257撤回3 期
An outpatient phase 3 efficacy study of Ecopipam (PSYRX 101) in the symptomatic treatment of self-injurious behavior in subjects with Lesch-Nyhan disease. - N/A
适应症
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 99（—）
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 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.
研究者
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