Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
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.
|Study Design:||Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||An Outpatient Phase 3 Efficacy Study of Ecopipam (PSYRX 101) in the Symptomatic Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease|
- Behavior Problems Inventory - Self-Injurious Behavior Subscale [ Time Frame: 6 weeks ] [ Designated as safety issue: No ]The primary endpoint is the BPI (SIB subscales - total for frequency and severity) as assessed by the caregiver.
- Behavior Problems Inventory [ Time Frame: 6 weeks ] [ Designated as safety issue: No ]The secondary objectives of this study are to assess the effect of withdrawal and maintenance of ecopipam's effects in subjects with LND.
- Safety of ecopipam in patients with Lesch-Nyhan Disease [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]An additional objective of the study is to assess the safety of ecopipam in subjects with LND for up to 52 weeks.
|Study Start Date:||December 2012|
|Estimated Study Completion Date:||December 2014|
|Estimated Primary Completion Date:||December 2013 (Final data collection date for primary outcome measure)|
Active substance being tested
Antagonist of the dopamine D1 receptor
Placebo Comparator: Placebo
Inactive substance being tested
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.
|United States, Georgia|
|Atlanta, Georgia, United States, 30322|
|Hospital Universitario La Paz|
|Principal Investigator:||H J Jinnah, MD||Emory University|