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

Psyadon Pharma5 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Lesch-Nyhan Disease

Interventions

Ecopipam

Eligibility

Sex: MALEMin age: 6 YearsMax age: 65 Years

Medical Language ↔ Plain English

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

Locations (2)

University of California at San Diego

San Diego, California, United States

Emory Univesity

Atlanta, Georgia, United States

Outcomes

Primary Outcomes

Number of Participants With Clinically Significant Changes in Standard Laboratory Tests

This study's primary outcome is the safety of ecopipam in Lesch-Nyhan patients as measured by standard clinical laboratory tests. The patients will also be observed and questioned about other side effects, such as whether they feel more or less tired.Standard clinical laboratory tests for liver, kidney and blood function were conducted. The normal ranges for each of these tests were different and are too numerous to be individually listed here. However, if any individual value were to be either three-times greater or lesser than the upper or the lower limit of the test, then that value was considered to have been changed.

Time frame: Two weeks

Secondary Outcomes

Decrease in Self-injurious Behavior at End of Study (Two Weeks After Screening) Compared to Screening

Change in the self-injurious subscale of the Behavior Problems Inventory (BPI). the BPI is a well-validate test to evaluate the frequency and severity of a patient's self-injurious behavior. Values range from 0 to 50, and a low score means few/less severe behaviors

Time frame: Screening visit and end of study (two weeks)