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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

5 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-02-28

Study Completion Date

2011-06-30

Brief Summary

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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.

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Detailed Description

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Conditions

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Lesch-Nyhan Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Ecopipam

Intervention Type DRUG

Tablets, once daily, for two weeks up to 200 mg/day

Interventions

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Ecopipam

Tablets, once daily, for two weeks up to 200 mg/day

Intervention Type DRUG

Other Intervention Names

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PSYRX101, SCH 39166

Eligibility Criteria

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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

Minimum Eligible Age

6 Years

Maximum Eligible Age

65 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No