Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease - Full Text View

Purpose

The purpose of this study is to determine the effects of ecopipam in patients with Lesch-Nyhan Disease (LND). Ecopipam specifically blocks the actions of one chemical in the brain that helps nerves talk to one another; ecopipam does this by stopping the chemical (dopamine) from binding to one of its family of receptors (i.e, the D1 family). LND is a very rare genetic disease. The sponsor is doing this study to find out what side effects ecopipam causes in LND patients, and whether ecopipam may be able to relieve the self-injurious behaviors seen in these patients.

Condition	Intervention	Phase
Lesch-Nyhan Disease	Drug: Ecopipam	Phase 1

Study Type:	Interventional
Study Design:	Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	A Safety and Pilot Activity 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: Lesch-Nyhan syndrome MECP2 duplication syndrome PPM-X syndrome Renpenning syndrome succinic semialdehyde dehydrogenase deficiency

MedlinePlus related topics: Self-harm

Drug Information available for: Dopamine Dopamine hydrochloride

U.S. FDA Resources

Further study details as provided by Psyadon Pharma:

Primary Outcome Measures:

Number of Participants With Clinically Significant Changes in Standard Laboratory Tests [ Time Frame: Two weeks ] [ Designated as safety issue: Yes ]
This study's primary outcome is the safety of ecopipam in Lesch-Nyhan patients as measured by standard clinical laboratory tests. The patients will also be observed and questioned about other side effects, such as whether they feel more or less tired.Standard clinical laboratory tests for liver, kidney and blood function were conducted. The normal ranges for each of these tests were different and are too numerous to be individually listed here. However, if any individual value were to be either three-times greater or lesser than the upper or the lower limit of the test, then that value was considered to have been changed.

Secondary Outcome Measures:

Decrease in Self-injurious Behavior at End of Study (Two Weeks After Screening) Compared to Screening [ Time Frame: Screening visit and end of study (two weeks) ] [ Designated as safety issue: No ]
Change in the self-injurious subscale of the Behavior Problems Inventory (BPI). the BPI is a well-validate test to evaluate the frequency and severity of a patient's self-injurious behavior. Values range from 0 to 50, and a low score means few/less severe behaviors

Enrollment:	5
Study Start Date:	February 2010
Study Completion Date:	June 2011
Primary Completion Date:	January 2011 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Ecopipam 12.5 - 200 mg/day Patients were administered ecopipam on an escalated dosing schedule over 11 days starting at 12.5 mg/day and increasing to the maximal tolerated dose or to 200 mg/day.	Drug: Ecopipam Tablets, once daily, for two weeks up to 200 mg/day Other Name: PSYRX101, SCH 39166

Eligibility

Ages Eligible for Study:	6 Years to 65 Years
Genders Eligible for Study:	Male
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria

Diagnosis of Lesch-Nyhan disease with moderate to severe Self- Injurious Behavior and are routinely restrained
Male - 6 years of age and above
Body weight greater than or equal to 44 pounds

Exclusion Criteria:

Taking Neuroleptic or Dopamine Depleting medications
Currently treated with medications for depression or seizures
Impaired renal function
Medical conditions that may interfere with completing the study

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01065558

Locations

United States, California
University of California at San Diego
San Diego, California, United States, 92103
United States, Georgia
Emory Univesity
Atlanta, Georgia, United States, 30329

Sponsors and Collaborators

Psyadon Pharma

Investigators

Principal Investigator:	Hyder A Jinnah, MD	Emory University
Principal Investigator:	William L Nyhan, MD	University of California, San Diego

More Information

Publications:

Chipkin RE, Iorio LC, Coffin VL, McQuade RD, Berger JG, Barnett A. Pharmacological profile of SCH39166: a dopamine D1 selective benzonaphthazepine with potential antipsychotic activity. J Pharmacol Exp Ther. 1988 Dec;247(3):1093-102.

Responsible Party:	Psyadon Pharma
ClinicalTrials.gov Identifier:	NCT01065558 History of Changes
Other Study ID Numbers:	Ecopipam and Lesch-Nyhan
Study First Received:	February 6, 2010
Results First Received:	December 18, 2012
Last Updated:	April 17, 2013
Health Authority:	United States: Food and Drug Administration

Keywords provided by Psyadon Pharma:

Ecopipam
Lesch-Nyhan Disease
Safety
Self-Injurious Behavior

Additional relevant MeSH terms:

Lesch-Nyhan Syndrome
Self-Injurious Behavior
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Mental Retardation, X-Linked
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

Metabolism, Inborn Errors
Purine-Pyrimidine Metabolism, Inborn Errors
Metabolic Diseases
Behavioral Symptoms
Sch 39166
Dopamine Antagonists
Dopamine Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs
Antipsychotic Agents
Tranquilizing Agents
Central Nervous System Depressants
Central Nervous System Agents

ClinicalTrials.gov processed this record on May 19, 2013