ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01751802
Recruitment Status : Terminated (Study terminated for commercial reasons)
First Posted : December 18, 2012
Results First Posted : October 8, 2015
Last Update Posted : October 8, 2015
Sponsor:
Information provided by (Responsible Party):
Psyadon Pharma

December 14, 2012
December 18, 2012
October 5, 2015
October 8, 2015
October 8, 2015
December 2012
December 2013   (Final data collection date for primary outcome measure)
Behavior Problems Inventory - Self-Injurious Behavior Subscale [ Time Frame: Baseline, end of period 1 (6 weeks), end of period 2 (12 weeks), end of period 3 (18 weeks), ]
The primary endpoint is the BPI (SIB subscales - total for frequency and severity) as assessed by the caregiver. BPI Self-Injurious Behavior Subscale ranges from 0 to 45, with higher scores indicating more self-injurious behavior.
Behavior Problems Inventory - Self-Injurious Behavior Subscale [ Time Frame: 6 weeks ]
The primary endpoint is the BPI (SIB subscales - total for frequency and severity) as assessed by the caregiver.
Complete list of historical versions of study NCT01751802 on ClinicalTrials.gov Archive Site
  • Effect of Ecopipam Withdrawal and Maintenance [ Time Frame: Baseline, 6 weeks, 12 weeks, 18 weeks ]
    The secondary objectives of this study are to assess the effect of withdrawal and maintenance of ecopipam's effects in subjects with LND. Measured by the number of participants whose score changes significantly from baseline on ecopipam or placebo
  • 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 [ Time Frame: Total duration over which participants recieved double-blind ecopipam or placebo, up to 6 or 12 weeks ]
    An additional objective of the study is to assess the safety of ecopipam in subjects with LND for up to 52 weeks. Total number of serious and non-serious adverse events experienced by participants while receiving ecopipam or placebo. For additional detail, see Adverse Events
Behavior Problems Inventory [ Time Frame: 6 weeks ]
The secondary objectives of this study are to assess the effect of withdrawal and maintenance of ecopipam's effects in subjects with LND.
Not Provided
Safety of ecopipam in patients with Lesch-Nyhan Disease [ Time Frame: 52 weeks ]
An additional objective of the study is to assess the safety of ecopipam in subjects with LND for up to 52 weeks.
 
Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
An Outpatient Phase 3 Efficacy Study of Ecopipam (PSYRX 101) in the Symptomatic 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.

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.

Interventional
Phase 3
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
  • Lesch-Nyhan Disease
  • Self-injurious Behavior
  • Drug: Ecopipam
    Antagonist of the dopamine D1 receptor
    Other Names:
    • PSYRX 101
    • SCH 39166
  • Drug: Placebo
    Placebo for Ecopipam
  • Experimental: Ecopipam
    Active substance being tested, orally once a day at bedtime
    Intervention: Drug: Ecopipam
  • Placebo Comparator: Placebo
    Inactive substance being tested, orally once a day at bedtime
    Intervention: Drug: Placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
9
24
December 2014
December 2013   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
6 Years to 70 Years   (Child, Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
Spain,   United States
 
 
NCT01751802
PSY102
Yes
Not Provided
Not Provided
Psyadon Pharma
Psyadon Pharma
Not Provided
Principal Investigator: H J Jinnah, MD Emory University
Psyadon Pharma
October 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP