Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease

NCT01751802 | Terminated Phase 3 Results DMC

• 1 other identifier: PSY102
• Study Type: interventional
• Target: 9
• Locations: 2 countries
• Sites: 2

Brief 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.

Conditions

Lesch-Nyhan Disease; Self-injurious Behavior

Keywords

Lesch Nyhan Disease; Self-Injury

Outcome Measures

Primary Outcomes (1)

• 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.

  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

  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

  Total duration over which participants recieved double-blind ecopipam or placebo, up to 6 or 12 weeks

Study Arms (2)

Ecopipam

EXPERIMENTAL

Active substance being tested, orally once a day at bedtime

Drug: Ecopipam

Placebo

PLACEBO COMPARATOR

Inactive substance being tested, orally once a day at bedtime

Drug: Placebo

Interventions

Ecopipam DRUG

Antagonist of the dopamine D1 receptor

Also known as: PSYRX 101, SCH 39166

Ecopipam

Placebo DRUG

Placebo for Ecopipam

Placebo

Eligibility Criteria

• Age: 6 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Emalex Biosciences Inc.: lead
• Psyadon Pharma: collaborator

Study Sites (2)

H.J. Jinnah

Atlanta, Georgia, 30322, United States

Location

Hospital Universitario La Paz

Madrid, Spain

Location

Related Links

• Sponsor's website

MeSH Terms

Conditions

Lesch-Nyhan Syndrome; Self-Injurious Behavior

Interventions

ecopipam

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Metabolism, Inborn Errors; Purine-Pyrimidine Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Behavioral Symptoms; Behavior

Results Point of Contact

• Title: President/CEO
• Organization: Psyadon Pharmaceuticals

info@psyadonrx.com

301-919-2020

Study Officials

• H J Jinnah, MD

  Emory University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 14, 2012
• First Posted: December 18, 2012
• Study Start: December 1, 2012
• Primary Completion: December 1, 2013
• Study Completion: December 1, 2014
• Last Updated: April 22, 2024
• Results First Posted: October 8, 2015

Record last verified: 2015-10

Locations

US (1); ES (1)