Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease
Status:
Completed
Trial end date:
2011-06-01
Target enrollment:
Participant gender:
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.