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

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 Drug: Placebo	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:

Genetics Home Reference related topics: CASK-related intellectual disability Lesch-Nyhan syndrome MECP2 duplication syndrome PPM-X syndrome Renpenning syndrome SYNGAP1-related intellectual disability succinic semialdehyde dehydrogenase deficiency

MedlinePlus related topics: Self-harm

Genetic and Rare Diseases Information Center resources: Lesch Nyhan Syndrome

U.S. FDA Resources

Further study details as provided by Psyadon Pharma:

Primary Outcome Measures:

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), ] [ Designated as safety issue: No ]
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:

Effect of Ecopipam Withdrawal and Maintenance [ Time Frame: Baseline, 6 weeks, 12 weeks, 18 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. 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 ] [ 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. Total number of serious and non-serious adverse events experienced by participants while receiving ecopipam or placebo. For additional detail, see Adverse Events


Enrollment:	9
Study Start Date:	December 2012
Study Completion Date:	December 2014
Primary Completion Date:	December 2013 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
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

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 (Child, Adult, Senior)
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:

Sponsor's website This link exits the ClinicalTrials.gov site


Responsible Party:	Psyadon Pharma
ClinicalTrials.gov Identifier:	NCT01751802 History of Changes
Other Study ID Numbers:	PSY102
Study First Received:	December 14, 2012
Results First Received:	October 5, 2015
Last Updated:	October 5, 2015
Health Authority:	United States: Food and Drug Administration

Keywords provided by Psyadon Pharma:


Lesch Nyhan Disease Self-Injury

Additional relevant MeSH terms:


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 Ecopipam 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

ClinicalTrials.gov processed this record on September 27, 2016