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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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ecopipam 12.5 - 200 mg/day | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ecopipam | Drug | Tablets, once daily, for two weeks up to 200 mg/day |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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) |
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Inclusion Criteria
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Hyder A Jinnah, MD | Emory University | Principal Investigator |
| William L Nyhan, MD | University of California, San Diego | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California at San Diego | San Diego | California | 92103 | United States | ||
| Emory Univesity |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 2905002 | Background | 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. |
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No preassignment
Patients were recruited by the investigators according to the inclusion and exclusion criteria
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| ID | Title | Description |
|---|---|---|
| FG000 | Ecopipam (12.5- 200 mg/Day) | Patients were given ecopipapm over an 11 day period as follows: day 1 12.5 mg/day day 2-3 25 mg/day day 4-5 50 mg/day day 6-9 100 mg/day day 9-11 200 mg/day Note: Doses were reduced as necessary to a previously tolerated dose if paitents reached doses that were not safely tolerable. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Lesch-Nyhan patients with moderate to severe self-injurious behaviors at the start of the study
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| ID | Title | Description |
|---|---|---|
| BG000 | Ecopipam |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | All patients were analyzed | Posted | Number | Participants | Two weeks |
|
12 days of active treatment and 28 days follow-up
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Ecopipam Treated Patients |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Flank Pain | Renal and urinary disorders | MedDRA (10.0) | Non-systematic Assessment | Patient reported flank pain which was caused by a kidney stone. Investigator defined as not drug related, and resolved without incident. Patient received all doses and was not removed from study. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Sedation | Nervous system disorders | MedDRA (10.0) | Non-systematic Assessment |
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Richard Chipkin | Psyadon Pharmaceuticals | 301-919-2020 | rchipkin@psyadonrx.com |
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| ID | Term |
|---|---|
| D007926 | Lesch-Nyhan Syndrome |
| D016728 | Self-Injurious Behavior |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| C058081 | ecopipam |
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| Atlanta |
| Georgia |
| 30329 |
| United States |
| Participants |
|
| Age Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
|
| 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 | All patients who received any dose of ecopipam were in the analysis population | Posted | Mean | Standard Deviation | Change in BPI score | Screening visit and end of study (two weeks) |
|
|
|
| 2 |
| 5 |
| 5 |
| 5 |
|
| Respiratory Infection | Infections and infestations | MedDRA (10.0) | Non-systematic Assessment | Patient developed respiratory infection (pneumonia)following the trial. Investigator reported this as not related to ecopipam administration. Patient was briefly hosptialized and infection resolved. |
|
| Dystonia | Nervous system disorders | MedDRA (10.0) | Non-systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA (10.0) | Non-systematic Assessment |
|
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| D009422 | Nervous System Diseases |
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D008661 | Metabolism, Inborn Errors |
| D011686 | Purine-Pyrimidine Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D001526 | Behavioral Symptoms |
| D001519 | Behavior |