An outpatient phase 3 efficacy study of Ecopipam (PSYRX 101) in the symptomatic treatment of self-injurious behavior in subjects with Lesch-Nyhan disease.
- Conditions
- zelfverwondend gedragHPRT gene mutationLesch-Nyhan disease1008362410028037
- Registration Number
- NL-OMON37257
- Lead Sponsor
- Psyadon Pharmaceuticals Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Withdrawn
- Sex
- Not specified
- Target Recruitment
- 5
* 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.
* 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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary endpoint is the Behavior Problems Inventory (self-injurious<br /><br>behavior subscales for severity and frequency) as assessed by the caregiver.</p><br>
- Secondary Outcome Measures
Name Time Method <p> Secondary parameters that will be analyzed are the total and other subscales<br /><br>of the Behavior Problems Inventory, and the withdrawal and maintenance effects<br /><br>of Ecopipam. Safety data that will be evaluated include adverse events,<br /><br>clinical laboratory results, vital signs, and other results.</p><br>