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

Primary Purpose

Lesch-Nyhan Disease

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Ecopipam
Sponsored by
Psyadon Pharma
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Lesch-Nyhan Disease focused on measuring Ecopipam, Lesch-Nyhan Disease, Safety, Self-Injurious Behavior

Eligibility Criteria

6 Years - 65 Years (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • University of California at San Diego
  • Emory Univesity

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Ecopipam 12.5 - 200 mg/day

Arm Description

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.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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

Full Information

First Posted
February 6, 2010
Last Updated
April 17, 2013
Sponsor
Psyadon Pharma
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1. Study Identification

Unique Protocol Identification Number
NCT01065558
Brief Title
Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease
Official Title
A Safety and Pilot Activity 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
April 2013
Overall Recruitment Status
Completed
Study Start Date
February 2010 (undefined)
Primary Completion Date
January 2011 (Actual)
Study Completion Date
June 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Psyadon Pharma

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Lesch-Nyhan Disease
Keywords
Ecopipam, Lesch-Nyhan Disease, Safety, Self-Injurious Behavior

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Ecopipam 12.5 - 200 mg/day
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Ecopipam
Other Intervention Name(s)
PSYRX101, SCH 39166
Intervention Description
Tablets, once daily, for two weeks up to 200 mg/day
Primary Outcome Measure Information:
Title
Number of Participants With Clinically Significant Changes in Standard Laboratory Tests
Description
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 Outcome Measure Information:
Title
Decrease in Self-injurious Behavior at End of Study (Two Weeks After Screening) Compared to Screening
Description
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)

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Hyder A Jinnah, MD
Organizational Affiliation
Emory University
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
William L Nyhan, MD
Organizational Affiliation
University of California, San Diego
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of California at San Diego
City
San Diego
State/Province
California
ZIP/Postal Code
92103
Country
United States
Facility Name
Emory Univesity
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
2905002
Citation
Chipkin RE, Iorio LC, Coffin VL, McQuade RD, Berger JG, Barnett A. Pharmacological profile of SCH39166: a dopamine D1 selective benzonaphthazepine with potential antipsychotic activity. J Pharmacol Exp Ther. 1988 Dec;247(3):1093-102.
Results Reference
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Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease

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