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

NCT ID: NCT01751802

Last Updated: 2024-04-22

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-12-31
• Study Completion Date: 2014-12-31

Brief Summary

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.

Detailed 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.

Conditions

Lesch-Nyhan Disease; Self-injurious Behavior

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Ecopipam

Active substance being tested, orally once a day at bedtime

Group Type: EXPERIMENTAL

Ecopipam

Intervention Type: DRUG

Antagonist of the dopamine D1 receptor

Placebo

Inactive substance being tested, orally once a day at bedtime

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo for Ecopipam

Interventions

Ecopipam

Antagonist of the dopamine D1 receptor

Intervention Type: DRUG

Placebo

Placebo for Ecopipam

Intervention Type: DRUG

Other Intervention Names

PSYRX 101; SCH 39166

Eligibility Criteria

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.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Psyadon Pharma

INDUSTRY

Sponsor Role: collaborator

Emalex Biosciences Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

H J Jinnah, MD

Role: PRINCIPAL_INVESTIGATOR

Emory University

Locations

H.J. Jinnah

Atlanta, Georgia, United States

Hospital Universitario La Paz

Madrid, , Spain

Countries

United States; Spain

Related Links

http://www.psyadonrx.com

Sponsor's website

Other Identifiers

PSY102

Identifier Type: -

Identifier Source: org_study_id