Lesch-Nyhan Disease Clinical Trial
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
| Verified date | April 2013 |
| Source | Psyadon Pharma |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | United States: Food and Drug Administration |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 5 |
| Est. completion date | June 2011 |
| Est. primary completion date | January 2011 |
| Accepts healthy volunteers | No |
| Gender | Male |
| Age group | 6 Years to 65 Years |
| Eligibility |
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 |
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| United States | Emory Univesity | Atlanta | Georgia |
| United States | University of California at San Diego | San Diego | California |
| Lead Sponsor | Collaborator |
|---|---|
| Psyadon Pharma |
United States,
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. — View Citation
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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. | Two weeks | Yes |
| Secondary | 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 | Screening visit and end of study (two weeks) | No |
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Terminated |
NCT01751802 -
Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
|
Phase 3 |