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: 0
• Participant gender: Male

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Psyadon Pharma
• Treatments: Dopamine Antagonists; Ecopipam

Criteria

Inclusion Criteria

• Diagnosis of Lesch-Nyhan disease with moderate to severe Self- Injurious Behavior and are routinely restrained

• Male - 6 years of age and above

• Body weight greater than or equal to 44 pounds

Exclusion Criteria:

• Taking Neuroleptic or Dopamine Depleting medications

• Currently treated with medications for depression or seizures

• Impaired renal function

• Medical conditions that may interfere with completing the study