Trial record 9 of 138 for:
Open Studies | "Brain Diseases, Metabolic, Inborn"
Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
This study is currently recruiting participants.
Verified January 2013 by Psyadon Pharma
Sponsor:
Psyadon Pharma
Information provided by (Responsible Party):
Psyadon Pharma
ClinicalTrials.gov Identifier:
NCT01751802
First received: December 14, 2012
Last updated: January 17, 2013
Last verified: January 2013
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Lesch-Nyhan Disease Self-injurious Behavior |
Drug: Ecopipam |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Efficacy Study Intervention Model: Crossover Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| 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 |
Resource links provided by NLM:
Genetics Home Reference related topics:
Lesch-Nyhan syndrome
MECP2 duplication syndrome
PPM-X syndrome
Renpenning syndrome
succinic semialdehyde dehydrogenase deficiency
MedlinePlus related topics:
Self-harm
U.S. FDA Resources
Further study details as provided by Psyadon Pharma:
Primary Outcome Measures:
- Behavior Problems Inventory - Self-Injurious Behavior Subscale [ Time Frame: 6 weeks ] [ Designated as safety issue: No ]The primary endpoint is the BPI (SIB subscales - total for frequency and severity) as assessed by the caregiver.
Secondary Outcome Measures:
- Behavior Problems Inventory [ Time Frame: 6 weeks ] [ Designated as safety issue: No ]The secondary objectives of this study are to assess the effect of withdrawal and maintenance of ecopipam's effects in subjects with LND.
Other Outcome Measures:
- Safety of ecopipam in patients with Lesch-Nyhan Disease [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]An additional objective of the study is to assess the safety of ecopipam in subjects with LND for up to 52 weeks.
| Estimated Enrollment: | 24 |
| Study Start Date: | December 2012 |
| Estimated Study Completion Date: | December 2014 |
| Estimated Primary Completion Date: | December 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Ecopipam
Active substance being tested
|
Drug: Ecopipam
Antagonist of the dopamine D1 receptor
Other Names:
|
|
Placebo Comparator: Placebo
Inactive substance being tested
|
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.
Eligibility| Ages Eligible for Study: | 6 Years to 70 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01751802
Contacts
| Contact: Aimee Fox | 301-556-0841 | Amelia.Fox@sgs.com |
| Contact: Eric Batten | (301) 556-0846 | Eric.Batten@sgs.com |
Locations
| United States, Georgia | |
| H.J. Jinnah | Recruiting |
| Atlanta, Georgia, United States, 30322 | |
| Contact: Barbara Sommerfeld 404-728-6944 bsommer@emory.edu | |
| Principal Investigator: H. J. Jinnah, MD | |
| Spain | |
| Hospital Universitario La Paz | Recruiting |
| Madrid, Spain | |
| Contact: Rosa Torres, MD +34 (917) 277343 rtorres.hulp@salud.madrid.org | |
| Principal Investigator: Rosa Torres, MD | |
Sponsors and Collaborators
Psyadon Pharma
Investigators
| Principal Investigator: | H J Jinnah, MD | Emory University |
More Information
Additional Information:
No publications provided
| Responsible Party: | Psyadon Pharma |
| ClinicalTrials.gov Identifier: | NCT01751802 History of Changes |
| Other Study ID Numbers: | PSY102 |
| Study First Received: | December 14, 2012 |
| Last Updated: | January 17, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Psyadon Pharma:
|
Lesch Nyhan Disease Self-Injury |
Additional relevant MeSH terms:
|
Brain Diseases, Metabolic, Inborn Lesch-Nyhan Syndrome Self-Injurious Behavior Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Mental Retardation, X-Linked Mental Retardation Neurobehavioral Manifestations Neurologic Manifestations Genetic Diseases, X-Linked Genetic Diseases, Inborn |
Metabolism, Inborn Errors Purine-Pyrimidine Metabolism, Inborn Errors Metabolic Diseases Behavioral Symptoms Sch 39166 Dopamine Antagonists Dopamine Agents Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action Pharmacologic Actions Physiological Effects of Drugs Antipsychotic Agents Tranquilizing Agents Central Nervous System Depressants Central Nervous System Agents |
ClinicalTrials.gov processed this record on May 22, 2013