Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease

Psyadon Pharma

Status and phase: Completed; Phase 1

Conditions: Lesch-Nyhan Disease

Treatments: Drug: Ecopipam

Study type: Interventional

Funder types: Industry

Identifiers

NCT01065558

Ecopipam and Lesch-Nyhan

Details and patient eligibility

About

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.

Enrollment: 5 patients

Sex: Male

Ages: 6 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

5 participants in 1 patient group

Ecopipam 12.5 - 200 mg/day

Experimental group

Description:

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.

Treatment:

Drug: Ecopipam