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 ]
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) ]
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