A Safety and Pilot Activity Study of Ecopipam (PSYRX 101) in the Symptomatic Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
| Registry | NCT01065558 |
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| Status | Completed |
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| Phase | Phase 1 |
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| Study type | Interventional; na, single group, masking: none, treatment |
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| Enrollment | 5 (Actual) |
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| Sponsor | Psyadon Pharma (Industry) |
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| Conditions | Lesch-Nyhan Disease |
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| Interventions | Ecopipam (Drug) |
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| Dates | start 2010-02-01, primary completion 2011-01-01, completion 2011-06-01, first posted 2010-02-09, last updated 2013-04-18 |
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| Locations | 2 sites in United States |
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Related Papers Related Patents Related Grants Related Experts
All trials for Lesch-Nyhan Disease All trials of Ecopipam
-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.
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+Arms and interventions
| Arm | Type | Interventions | Description |
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| Ecopipam 12.5 - 200 mg/day | Experimental | Drug: Ecopipam | 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. |
- Ecopipam (Drug), also known as PSYRX101, SCH 39166: Tablets, once daily, for two weeks up to 200 mg/day
+Outcome measures
Primary outcomes
- Number of Participants With Clinically Significant Changes in Standard Laboratory Tests [Two weeks]: 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 outcomes
- Decrease in Self-injurious Behavior at End of Study (Two Weeks After Screening) Compared to Screening [Screening visit and end of study (two weeks)]: 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
+Eligibility
Sex: Male, ages: 6 Years to 65 Years
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
+Posted results
Participant flow
| Milestone | Ecopipam (12.5- 200 mg/Day) |
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| Started | 5 |
| Completed | 5 |
| Not completed | 0 |
Outcome results
- Number of Participants With Clinically Significant Changes in Standard Laboratory Tests (Primary), Number, Participants: Ecopipam Treated Patients 0
- Decrease in Self-injurious Behavior at End of Study (Two Weeks After Screening) Compared to Screening (Secondary), Mean, Change in BPI score: Ecopipam Treated Patients 22.5 +/- 13.4
Adverse events
Time frame: 12 days of active treatment and 28 days follow-up
| Arm | Deaths | Serious adverse events | Other adverse events |
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| Ecopipam Treated Patients | 0 | 2/5 (40.0%) | 5/5 (100.0%) |
Serious adverse events by term
| Term | Organ system | Ecopipam Treated Patients |
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| Flank Pain | Renal and urinary disorders | 1/5 |
| Respiratory Infection | Infections and infestations | 1/5 |
Other adverse events by term
| Term | Organ system | Ecopipam Treated Patients |
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| Sedation | Nervous system disorders | 3/5 |
| Dystonia | Nervous system disorders | 2/5 |
| Nausea | Gastrointestinal disorders | 2/5 |
Limitations and caveats: This was an open label trial of oral ecopipam in pediatric, adolescent and adult patients with Lesch-Nyhan Disease.In this population, ecopipam's safety and PK was similar to non-LND subjects.
+Locations (2)
| Facility | City | Country | Status |
|---|
| University of California at San Diego | San Diego, California | United States | |
| Emory Univesity | Atlanta, Georgia | United States | |
+Publications
- 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. PMID 2905002
+Investigators