Clinical trial NCT01751802: Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Lesch-Nyhan Disease
Self-injurious Behavior
Keywords: Lesch Nyhan Disease
Self-Injury
Lead sponsor: Emalex Biosciences Inc.
Sponsor class: INDUSTRY
Collaborators: name: Psyadon Pharma
First posted: 2012-12-18
Enrollment: 9
Sex: ALL
Minimum age (years): 6
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief 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.

Detailed description: 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.

Eligibility 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.

Primary outcomes:
measure: Behavior Problems Inventory - Self-Injurious Behavior Subscale
timeFrame: Baseline, end of period 1 (6 weeks), end of period 2 (12 weeks), end of period 3 (18 weeks),
Secondary outcomes:
measure: Effect of Ecopipam Withdrawal and Maintenance
timeFrame: Baseline, 6 weeks, 12 weeks, 18 weeks
measure: Safety Summary of Ecopipam in Patients With Lesch-Nyhan Disease: Total Number of Serious and Non-Serious Adverse Events Experienced During 3 Double-blind Crossover Periods
timeFrame: Total duration over which participants recieved double-blind ecopipam or placebo, up to 6 or 12 weeks
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: H.J. Jinnah
state: Georgia
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
Study officials:
affiliation: Emory University
name: H J Jinnah, MD
role: PRINCIPAL_INVESTIGATOR
References: