Lesch-Nyhan DiseasePhase 1NCT01065558
Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease
A Phase 1 study testing Ecopipam in people with Lesch-Nyhan Disease, run by Psyadon Pharma at 2 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 to 65 years, men only
- Conditions the study is for
- Lesch-Nyhan Disease
- What is being tested?
- Ecopipam
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 2 sitesUnited States
- Study dates
- Feb 2010to Jun 2011
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Emory Univesity
Atlanta, Georgia, 30329
University of California at San Diego
San Diego, California, 92103
About this study
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
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.
- Conditions
- Lesch-Nyhan Disease
- Being tested
- Ecopipam
- Keywords
- Ecopipam, Lesch-Nyhan Disease, Safety, Self-Injurious Behavior
- Medical subject headings
- Lesch-Nyhan Syndrome, Self-Injurious Behavior
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EcopipamDrug
Also called PSYRX101, SCH 39166
Tablets, once daily, for two weeks up to 200 mg/day
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Ecopipam |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Two weeks
Secondary outcomes (1)
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
Measured: Screening visit and end of study (two weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Feb 2010 (estimated)
- Study startFeb 2010
- Primary completionJan 2011
- Study completionJun 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Lesch-Nyhan DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Psyadon Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hyder A Jinnah, MD, Principal investigator, Emory UniversityWilliam L Nyhan, MD, Principal investigator, University of California, San Diego
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01065558
- Sponsor's protocol ID
- Ecopipam and Lesch-Nyhan
- Status last verified
- Apr 2013
- First submitted
- 6 Feb 2010
- Last updated
- 18 Apr 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
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.
PubMed 2905002
Study at a glance
From the registry record, last updated by the sponsor on 18 Apr 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01065558
- Acronym / sponsor ID
- Ecopipam and Lesch-Nyhan
- Conditions
- Lesch-Nyhan Disease
- Keywords
- Ecopipam, Lesch-Nyhan Disease, Safety, Self-Injurious Behavior
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Psyadon PharmaIndustry (pharma, biotech, devices)
- Study start
- Feb 2010
- Primary completion
- Jan 2011 (actual)
- Study completion
- Jun 2011 (actual)
- First posted
- 9 Feb 2010 (estimated)
- Results
- Posted 18 Apr 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ecopipam 12.5 - 200 mg/day | Experimental | Drug: EcopipamPatients 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. |
Who may qualify
- Ages
- 6 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EcopipamAlso: PSYRX101, SCH 39166Tablets, once daily, for two weeks up to 200 mg/day | Drug | Ecopipam 12.5 - 200 mg/day |
Primary outcome
Number of Participants With Clinically Significant Changes in Standard Laboratory Tests
Time frame: 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.
1 secondary outcome
Decrease in Self-injurious Behavior at End of Study (Two Weeks After Screening) Compared to Screening
Time frame: Screening visit and end of study (two weeks)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Apr 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 2905002Chipkin 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hyder A Jinnah, MD principal investigator
Emory University
William L Nyhan, MD principal investigator
University of California, San Diego
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Ecopipam in people with Lesch-Nyhan Disease, run by Psyadon Pharma at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Apr 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 18 Apr 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Clinically Significant Changes in Standard Laboratory Tests (time frame: Two weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01065558, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Apr 2013.