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

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.

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Key information

Conditions

Lesch-Nyhan Disease Behavior Behavioral Symptoms Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Intellectual Disability Lesch-Nyhan Syndrome Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutritional and Metabolic Diseases Purine-Pyrimidine Metabolism, Inborn Errors Self-Injurious Behavior X-Linked Intellectual Disability

Age range

6 year–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1

Primary location

University of California at San Diego, San Diego, California, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Ecopipam

Drug

Tablets, once daily, for two weeks up to 200 mg/day

Other names: PSYRX101, SCH 39166

Primary outcomes

Number of Participants With Clinically Significant Changes in Standard Laboratory Tests

Time frame: Two weeks

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 outcomes

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)

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

Sponsors and collaborators

Lead sponsor

Psyadon Pharma

Industry

Registry information

Official study 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

Important dates

Study start: 2010
Primary completion: 2011
Study completion: 2011
First posted: Feb 9, 2010
Registry last updated: Apr 18, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

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