Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
Stopped early · Phase 3 · Has a placebo group
Conditions studied: Lesch-Nyhan Disease, Self-injurious Behavior
In brief
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.
Key facts
- Study ID
- NCT01751802
- Run by
- Emalex Biosciences Inc.
- People needed
- 9
- Starts
- 2012-12-01
- Expected to finish
- 2014-12-01
- Last updated by the study team
- 2024-04-22
Who can join
Age: 6 and older, up to 70. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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.
Where it is running
- H.J. Jinnah — Atlanta, Georgia, United States
- Hospital Universitario La Paz — Madrid, Spain
Full record on ClinicalTrials.gov
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