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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. 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
Information provided by (Responsible Party):
Psyadon Pharma

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.

Condition or disease Intervention/treatment Phase
Lesch-Nyhan Disease Self-injurious Behavior Drug: Ecopipam Drug: Placebo Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 9 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, 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
Study Start Date : December 2012
Actual Primary Completion Date : December 2013
Actual Study Completion Date : December 2014

Arm Intervention/treatment
Experimental: Ecopipam
Active substance being tested, orally once a day at bedtime
Drug: Ecopipam
Antagonist of the dopamine D1 receptor
Other Names:
  • PSYRX 101
  • SCH 39166

Placebo Comparator: Placebo
Inactive substance being tested, orally once a day at bedtime
Drug: Placebo
Placebo for Ecopipam

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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

  2. 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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   6 Years to 70 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01751802

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United States, Georgia
H.J. Jinnah
Atlanta, Georgia, United States, 30322
Hospital Universitario La Paz
Madrid, Spain
Sponsors and Collaborators
Psyadon Pharma
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Principal Investigator: H J Jinnah, MD Emory University

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Responsible Party: Psyadon Pharma Identifier: NCT01751802     History of Changes
Other Study ID Numbers: PSY102
First Posted: December 18, 2012    Key Record Dates
Results First Posted: October 8, 2015
Last Update Posted: October 8, 2015
Last Verified: October 2015

Keywords provided by Psyadon Pharma:
Lesch Nyhan Disease

Additional relevant MeSH terms:
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Self-Injurious Behavior
Lesch-Nyhan Syndrome
Behavioral Symptoms
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Purine-Pyrimidine Metabolism, Inborn Errors
Metabolic Diseases
Antipsychotic Agents
Tranquilizing Agents
Central Nervous System Depressants
Physiological Effects of Drugs
Psychotropic Drugs
Dopamine Antagonists
Dopamine Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action