Trial · NCT01065558
Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease
NCT01065558, Phase 1, Completed, Psyadon Pharma; results first posted 2013-04-18
3 sourcestrials-2026-11-12-0.4.0
Cite this record
ConductScience Library. Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease. Trials, record NCT01065558. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01065558
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease
- NCT number
- NCT01065558
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 5 participants, actual
- Lead sponsor
- Psyadon Pharma
- Conditions
- Lesch-Nyhan Disease
- Interventions
- Ecopipam
- First posted
- 2010-02-09
- Last updated
- 2013-04-18
- Results first posted
- 2013-04-18
- 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.
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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 | 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 |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Clinically Significant Changes in Standard Laboratory Tests | Two weeks | All patients were analyzed | Participants | NUMBER |
| SECONDARY | Decrease in Self-injurious Behavior at End of Study (Two Weeks After Screening) Compared to Screening | Screening visit and end of study (two weeks) | All patients who received any dose of ecopipam were in the analysis population | Change in BPI score | MEAN |
Source ClinicalTrials.gov
Results by group2
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Clinically Significant Changes in Standard Laboratory Tests | Ecopipam Treated Patients | 0 | Participants | ||
| Decrease in Self-injurious Behavior at End of Study (Two Weeks After Screening) Compared to Screening | Ecopipam Treated Patients | 22.5 | Standard Deviation 13.4 | Change in BPI score |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ecopipam (12.5- 200 mg/Day) | 5 | |
| Overall Study | COMPLETED | Ecopipam (12.5- 200 mg/Day) | 5 | |
| Overall Study | NOT COMPLETED | Ecopipam (12.5- 200 mg/Day) | 0 |
Source ClinicalTrials.gov
Adverse events5
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Sedation | Nervous system disorders | Ecopipam Treated Patients | 3 | 5 | 3 |
| other | Dystonia | Nervous system disorders | Ecopipam Treated Patients | 2 | 5 | 2 |
| other | Nausea | Gastrointestinal disorders | Ecopipam Treated Patients | 2 | 5 | 2 |
| serious | Flank Pain | Renal and urinary disorders | Ecopipam Treated Patients | 1 | 5 | 1 |
| serious | Respiratory Infection | Infections and infestations | Ecopipam Treated Patients | 1 | 5 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ecopipam Treated Patients | ||
| Total, other adverse events | Ecopipam Treated Patients | 5 | 5 |
| Total, serious adverse events | Ecopipam Treated Patients | 2 | 5 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 2905002 | BACKGROUND |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01065558
GET https://conductscience.org/api/trials/v1/NCT01065558/registered