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

This study has been terminated.
(Study terminated for commercial reasons)
Sponsor:
Information provided by (Responsible Party):
Psyadon Pharma
ClinicalTrials.gov Identifier:
NCT01751802
First received: December 14, 2012
Last updated: November 6, 2013
Last verified: November 2013
  Purpose

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 Intervention Phase
Lesch-Nyhan Disease
Self-injurious Behavior
Drug: Ecopipam
Phase 3

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by Psyadon Pharma:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Other Outcome Measures:
  • 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.


Enrollment: 9
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)
Arms Assigned Interventions
Experimental: Ecopipam
Active substance being tested
Drug: Ecopipam
Antagonist of the dopamine D1 receptor
Other Names:
  • PSYRX 101
  • SCH 39166
Placebo Comparator: Placebo
Inactive substance being tested

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

Ages Eligible for Study:   6 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01751802

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

Additional Information:
No publications provided

Responsible Party: Psyadon Pharma
ClinicalTrials.gov Identifier: NCT01751802     History of Changes
Other Study ID Numbers: PSY102
Study First Received: December 14, 2012
Last Updated: November 6, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Psyadon Pharma:
Lesch Nyhan Disease
Self-Injury

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

ClinicalTrials.gov processed this record on October 21, 2014