Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease

• Status: Terminated
• Trial end date: 2014-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Psyadon Pharma
• Treatments: Ecopipam

Criteria

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.