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
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
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
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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