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.
|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|
- 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.
- 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
|Study Start Date:||February 2010|
|Study Completion Date:||June 2011|
|Primary Completion Date:||January 2011 (Final data collection date for primary outcome measure)|
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.
Tablets, once daily, for two weeks up to 200 mg/day
Other Name: PSYRX101, SCH 39166
Please refer to this study by its ClinicalTrials.gov identifier: NCT01065558
|United States, California|
|University of California at San Diego|
|San Diego, California, United States, 92103|
|United States, Georgia|
|Atlanta, Georgia, United States, 30329|
|Principal Investigator:||Hyder A Jinnah, MD||Emory University|
|Principal Investigator:||William L Nyhan, MD||University of California, San Diego|