Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease

Emalex Biosciences

Status and phase

Terminated

Phase 3

Conditions

Self-injurious Behavior

Lesch-Nyhan Disease

Treatments

Drug: Placebo

Drug: Ecopipam

Study type

Interventional

Funder types

Industry

Identifiers

NCT01751802

PSY102

Details and patient eligibility

About

The purpose of this research study is to gather scientific information about the effectiveness and safety of the study drug, Ecopipam (PSYRX 101), for the treatment of self-injurious behaviors when compared with the effectiveness and safety of placebo (inactive substance) in subjects with Lesch-Nyhan Disease.

Full description

This study will be done in approximately 6 centers in approximately 4 countries, and approximately 24 subjects will be included. This study is divided into two parts. The first is a double-blinded portion lasting up to 18 weeks in total. The second portion is an optional open-label extension and lasts up to 54 weeks total. The total duration of the study, if you choose to participate in both portions, is anticipated to be up to approximately 78 weeks.

The first portion of this study is double-blind and assignment to a treatment group is done randomly. In this study, there are two treatment groups. One group will receive Ecopipam for one 6-week period and placebo for two 6-week periods, and the other group will receive Ecopipam for two 6-week periods and placebo for one 6-week period.

Subjects who did not experience any clinically significant side effects during the blinded portion of the study may be eligible to participate in an open-label extension that may last up to 54 weeks.

Enrollment

9 patients

Sex

All

Ages

6 to 70 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Subjects must have classic LND as defined by (a) characteristic clinical syndrome (evidence of overproduction of uric acid, severe generalized dystonia, frequent and persistent self-injurious behavior (SIB), and cognitive impairment) and (b) laboratory confirmation for mutation of the HPRT gene or severe deficiency of the associated enzyme.
Subjects must have a minimum combined score of 20 on the Behavior Problems Inventory (BPI) SIB subscales for frequency and severity as assessed by the caregiver.
Subjects must have a minimum score of 4 on the Physician's Global Impression (PGI) severity scale.
Subject must be ≥ 6 years old.
Subjects must weigh > 10 kg.

Exclusion criteria

Subjects who are currently treated with medications for seizures.
Subjects who are on neuroleptics or dopamine-depleting agents.
Subjects with impaired renal function as defined by a serum creatinine >1.5 mg/dL.