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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief Summary: 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.

Detailed Summary:
Sponsor: Psyadon Pharma

Current Primary Outcome: 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.


Original Primary Outcome: 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. [ Time Frame: Two weeks ]

Current Secondary Outcome: 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


Original Secondary Outcome: Decrease in self-injurious behavior [ Time Frame: Two weeks ]

Information By: Psyadon Pharma

Dates:
Date Received: February 6, 2010
Date Started: February 2010
Date Completion:
Last Updated: April 17, 2013
Last Verified: April 2013