Lesch-Nyhan DiseasePhase 3NCT01751802
Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
A Phase 3 study testing Ecopipam in people with Lesch-Nyhan Disease and Self-injurious Behavior, run by Emalex Biosciences Inc. at 2 sites in 2 countries.
Trial summary
The purpose of this research study is to gather scientific information about the effectiveness and safety of the study drug, Ecopipam (PSYRX 101), for the treatment of self-injurious behaviors when compared with the effectiveness and safety of placebo (inactive substance) in subjects with Lesch-Nyhan Disease. Read more about this study
- Who is it for?
- Ages 6 to 70 years
- Conditions the study is for
- Lesch-Nyhan Disease, Self-injurious Behavior
- What is being tested?
- Ecopipam
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 9 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Dec 2012to Dec 2014
- Phase
- Phase 3
- 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 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subjects must have classic LND as defined by (a) characteristic clinical syndrome (evidence of overproduction of uric acid, severe generalized dystonia, frequent and persistent self-injurious behavior (SIB), and cognitive impairment) and (b) laboratory confirmation for mutation of the HPRT gene or severe deficiency of the associated enzyme.
- Subjects must have a minimum combined score of 20 on the Behavior Problems Inventory (BPI) SIB subscales for frequency and severity as assessed by the caregiver.
- Subjects must have a minimum score of 4 on the Physician's Global Impression (PGI) severity scale.
- Subject must be ≥ 6 years old.
- Subjects must weigh > 10 kg.
Who cannot join
- Subjects who are currently treated with medications for seizures.
- Subjects who are on neuroleptics or dopamine-depleting agents.
- Subjects with impaired renal function as defined by a serum creatinine >1.5 mg/dL.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: An Outpatient Phase 3 Efficacy Study of Ecopipam (PSYRX 101) in the Symptomatic Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
The purpose of this research study is to gather scientific information about the effectiveness and safety of the study drug, Ecopipam (PSYRX 101), for the treatment of self-injurious behaviors when compared with the effectiveness and safety of placebo (inactive substance) in subjects with Lesch-Nyhan Disease.
Read the detailed description
This study will be done in approximately 6 centers in approximately 4 countries, and approximately 24 subjects will be included. This study is divided into two parts. The first is a double-blinded portion lasting up to 18 weeks in total. The second portion is an optional open-label extension and lasts up to 54 weeks total. The total duration of the study, if you choose to participate in both portions, is anticipated to be up to approximately 78 weeks.
The first portion of this study is double-blind and assignment to a treatment group is done randomly. In this study, there are two treatment groups. One group will receive Ecopipam for one 6-week period and placebo for two 6-week periods, and the other group will receive Ecopipam for two 6-week periods and placebo for one 6-week period.
Subjects who did not experience any clinically significant side effects during the blinded portion of the study may be eligible to participate in an open-label extension that may last up to 54 weeks.
- Conditions
- Lesch-Nyhan Disease, Self-injurious Behavior
- Being tested
- Ecopipam, Placebo
- Keywords
- Lesch Nyhan Disease, Self-Injury
- 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 PSYRX 101, SCH 39166
Antagonist of the dopamine D1 receptor
PlaceboDrug
Placebo for Ecopipam
| Study group | Role | What they receive |
|---|---|---|
| Ecopipam | Experimental | Active substance being tested, orally once a day at bedtime Receives: Drug: Ecopipam |
| Placebo | Placebo comparator | Inactive substance being tested, orally once a day at bedtime Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Behavior Problems Inventory - Self-Injurious Behavior Subscale
The primary endpoint is the BPI (SIB subscales - total for frequency and severity) as assessed by the caregiver. BPI Self-Injurious Behavior Subscale ranges from 0 to 45, with higher scores indicating more self-injurious behavior.
Measured: Baseline, end of period 1 (6 weeks), end of period 2 (12 weeks), end of period 3 (18 weeks),
Secondary outcomes (2)
Effect of Ecopipam Withdrawal and Maintenance
The secondary objectives of this study are to assess the effect of withdrawal and maintenance of ecopipam's effects in subjects with LND. Measured by the number of participants whose score changes significantly from baseline on ecopipam or placebo
Measured: Baseline, 6 weeks, 12 weeks, 18 weeks
Safety Summary of Ecopipam in Patients With Lesch-Nyhan Disease: Total Number of Serious and Non-Serious Adverse Events Experienced During 3 Double-blind Crossover Periods
An additional objective of the study is to assess the safety of ecopipam in subjects with LND for up to 52 weeks. Total number of serious and non-serious adverse events experienced by participants while receiving ecopipam or placebo. For additional detail, see Adverse Events
Measured: Total duration over which participants recieved double-blind ecopipam or placebo, up to 6 or 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Dec 2012 (estimated)
- Study startDec 2012
- Primary completionDec 2013
- Study completionDec 2014
Why it stopped: Study terminated for commercial reasons
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.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emalex Biosciences Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Psyadon Pharma
- Responsible party
- Sponsor
- Investigators
- H J Jinnah, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01751802
- Sponsor's protocol ID
- PSY102
- Status last verified
- Oct 2015
- First submitted
- 14 Dec 2012
- Last updated
- 22 Apr 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Outpatient Phase 3 Efficacy Study of Ecopipam (PSYRX 101) in the Symptomatic Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
- ClinicalTrials.gov ID
- NCT01751802
- Acronym / sponsor ID
- PSY102
- Conditions
- Lesch-Nyhan Disease, Self-injurious Behavior
- Keywords
- Lesch Nyhan Disease, Self-Injury
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study terminated for commercial reasons
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Emalex Biosciences Inc.Industry (pharma, biotech, devices)
- Collaborators
- Psyadon Pharma
- Study start
- Dec 2012
- Primary completion
- Dec 2013 (actual)
- Study completion
- Dec 2014 (actual)
- First posted
- 18 Dec 2012 (estimated)
- Results
- Posted 8 Oct 2015
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 research study is to gather scientific information about the effectiveness and safety of the study drug, Ecopipam (PSYRX 101), for the treatment of self-injurious behaviors when compared with the effectiveness and safety of placebo (inactive substance) in subjects with Lesch-Nyhan Disease.
Detailed description
This study will be done in approximately 6 centers in approximately 4 countries, and approximately 24 subjects will be included. This study is divided into two parts. The first is a double-blinded portion lasting up to 18 weeks in total. The second portion is an optional open-label extension and lasts up to 54 weeks total. The total duration of the study, if you choose to participate in both portions, is anticipated to be up to approximately 78 weeks.
The first portion of this study is double-blind and assignment to a treatment group is done randomly. In this study, there are two treatment groups. One group will receive Ecopipam for one 6-week period and placebo for two 6-week periods, and the other group will receive Ecopipam for two 6-week periods and placebo for one 6-week period.
Subjects who did not experience any clinically significant side effects during the blinded portion of the study may be eligible to participate in an open-label extension that may last up to 54 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ecopipam | Experimental | Drug: EcopipamActive substance being tested, orally once a day at bedtime |
| Placebo | Placebo comparator | Drug: PlaceboInactive substance being tested, orally once a day at bedtime |
Who may qualify
- Ages
- 6 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Subjects must have classic LND as defined by (a) characteristic clinical syndrome (evidence of overproduction of uric acid, severe generalized dystonia, frequent and persistent self-injurious behavior (SIB), and cognitive impairment) and (b) laboratory confirmation for mutation of the HPRT gene or severe deficiency of the associated enzyme.
- Subjects must have a minimum combined score of 20 on the Behavior Problems Inventory (BPI) SIB subscales for frequency and severity as assessed by the caregiver.
- Subjects must have a minimum score of 4 on the Physician's Global Impression (PGI) severity scale.
- Subject must be ≥ 6 years old.
- Subjects must weigh > 10 kg.
Key exclusion criteria (3 listed)
- Subjects who are currently treated with medications for seizures.
- Subjects who are on neuroleptics or dopamine-depleting agents.
- Subjects with impaired renal function as defined by a serum creatinine >1.5 mg/dL.
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: PSYRX 101, SCH 39166Antagonist of the dopamine D1 receptor | Drug | Ecopipam |
| PlaceboPlacebo for Ecopipam | Drug | Placebo |
Primary outcome
Behavior Problems Inventory - Self-Injurious Behavior Subscale
Time frame: Baseline, end of period 1 (6 weeks), end of period 2 (12 weeks), end of period 3 (18 weeks),
The primary endpoint is the BPI (SIB subscales - total for frequency and severity) as assessed by the caregiver. BPI Self-Injurious Behavior Subscale ranges from 0 to 45, with higher scores indicating more self-injurious behavior.
2 secondary outcomes
Effect of Ecopipam Withdrawal and Maintenance
Time frame: Baseline, 6 weeks, 12 weeks, 18 weeks
Safety Summary of Ecopipam in Patients With Lesch-Nyhan Disease: Total Number of Serious and Non-Serious Adverse Events Experienced During 3 Double-blind Crossover Periods
Time frame: Total duration over which participants recieved double-blind ecopipam or placebo, up to 6 or 12 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
H J Jinnah, MD principal investigator
Emory University
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ecopipam in people with Lesch-Nyhan Disease and Self-injurious Behavior, run by Emalex Biosciences Inc. at 2 sites in 2 countries.
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 5 inclusion and 3 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: stopped early, as of the sponsor's last update on 22 Apr 2024. 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, Spain. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 8 Oct 2015). 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?
Behavior Problems Inventory - Self-Injurious Behavior Subscale (time frame: Baseline, end of period 1 (6 weeks), end of period 2 (12 weeks), end of period 3 (18 weeks),). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 22 Apr 2024.