Conditions: Lesch-Nyhan Disease
Keywords: Ecopipam Lesch-Nyhan Disease Safety Self-Injurious Behavior
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 5
Healthy volunteers: no

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 description:

Arms:
arm 1: 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.

Interventions: intervention 1: Ecopipam
Intervention descriptions: intervention 1: Tablets, once daily, for two weeks up to 200 mg/day

Protocol document: