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

Primary Purpose

Lesch-Nyhan Disease, Self-injurious Behavior

Status

Terminated

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Ecopipam

Placebo

About this trial

This is an interventional treatment trial for Lesch-Nyhan Disease focused on measuring Lesch Nyhan Disease, Self-Injury

Eligibility Criteria

6 Years - 70 Years (Child, Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

H.J. Jinnah
Hospital Universitario La Paz

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Ecopipam

Placebo

Arm Description

Active substance being tested, orally once a day at bedtime

Inactive substance being tested, orally once a day at bedtime

Outcomes

Primary Outcome Measures

Behavior Problems Inventory - Self-Injurious Behavior Subscale

The primary endpoint is the BPI (SIB subscales - total for frequency and severity) as assessed by the caregiver. BPI Self-Injurious Behavior Subscale ranges from 0 to 45, with higher scores indicating more self-injurious behavior.

Secondary Outcome Measures

Effect of Ecopipam Withdrawal and Maintenance

The secondary objectives of this study are to assess the effect of withdrawal and maintenance of ecopipam's effects in subjects with LND. Measured by the number of participants whose score changes significantly from baseline on ecopipam or placebo

Safety Summary of Ecopipam in Patients With Lesch-Nyhan Disease: Total Number of Serious and Non-Serious Adverse Events Experienced During 3 Double-blind Crossover Periods

An additional objective of the study is to assess the safety of ecopipam in subjects with LND for up to 52 weeks. Total number of serious and non-serious adverse events experienced by participants while receiving ecopipam or placebo. For additional detail, see Adverse Events

Full Information

NCT ID

NCT01751802

First Posted

December 14, 2012

Last Updated

October 5, 2015

Sponsor

Psyadon Pharma

1. Study Identification

Unique Protocol Identification Number

NCT01751802

Brief Title

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

Official Title

An Outpatient Phase 3 Efficacy Study of Ecopipam (PSYRX 101) in the Symptomatic Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease

Study Type

Interventional

2. Study Status

Record Verification Date

October 2015

Overall Recruitment Status

Terminated

Why Stopped

Study terminated for commercial reasons

Study Start Date

December 2012 (undefined)

Primary Completion Date

December 2013 (Actual)

Study Completion Date

December 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Psyadon Pharma

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Lesch-Nyhan Disease, Self-injurious Behavior

Keywords

Lesch Nyhan Disease, Self-Injury

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Crossover Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

9 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Ecopipam

Arm Type

Experimental

Arm Description

Active substance being tested, orally once a day at bedtime

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Inactive substance being tested, orally once a day at bedtime

Intervention Type

Drug

Intervention Name(s)

Ecopipam

Other Intervention Name(s)

PSYRX 101, SCH 39166

Intervention Description

Antagonist of the dopamine D1 receptor

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Placebo for Ecopipam

Primary Outcome Measure Information:

Title

Behavior Problems Inventory - Self-Injurious Behavior Subscale

Description

Time Frame

Baseline, end of period 1 (6 weeks), end of period 2 (12 weeks), end of period 3 (18 weeks),

Secondary Outcome Measure Information:

Title

Effect of Ecopipam Withdrawal and Maintenance

Description

Time Frame

Baseline, 6 weeks, 12 weeks, 18 weeks

Title

Safety Summary of Ecopipam in Patients With Lesch-Nyhan Disease: Total Number of Serious and Non-Serious Adverse Events Experienced During 3 Double-blind Crossover Periods

Description

Time Frame

Total duration over which participants recieved double-blind ecopipam or placebo, up to 6 or 12 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Years

Maximum Age & Unit of Time

70 Years

Accepts Healthy Volunteers

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

H J Jinnah, MD

Organizational Affiliation

Emory University

Official's Role

Principal Investigator

Facility Information:

Facility Name

H.J. Jinnah

City

Atlanta

State/Province

Georgia

ZIP/Postal Code

30322

Country

United States

Facility Name

Hospital Universitario La Paz

City

Madrid

Country

Spain

12. IPD Sharing Statement

Links:

URL

http://www.psyadonrx.com

Description

Sponsor's website

Learn more about this trial

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

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