Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease

NCT01065558 | Completed Phase 1 Results

• 1 other identifier: Ecopipam and Lesch-Nyhan
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 2

Brief 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 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

Keywords

Ecopipam; Lesch-Nyhan Disease; Safety; Self-Injurious Behavior

Outcome Measures

Primary Outcomes (1)

• 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.

  Two weeks

Secondary Outcomes (1)

• Decrease in Self-injurious Behavior at End of Study (Two Weeks After Screening) Compared to Screening

  Screening visit and end of study (two weeks)

Study Arms (1)

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.

Drug: Ecopipam

Interventions

Ecopipam DRUG

Tablets, once daily, for two weeks up to 200 mg/day

Also known as: PSYRX101, SCH 39166

Ecopipam 12.5 - 200 mg/day

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Psyadon Pharma: lead

Study Sites (2)

University of California at San Diego

San Diego, California, 92103, United States

Location

Emory Univesity

Atlanta, Georgia, 30329, United States

Location

Related Publications (1)

• 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.

  PMID: 2905002 BACKGROUND

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

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.

Results Point of Contact

• Title: Dr. Richard Chipkin
• Organization: Psyadon Pharmaceuticals

rchipkin@psyadonrx.com

301-919-2020

Study Officials

• Hyder A Jinnah, MD

  Emory University

  PRINCIPAL INVESTIGATOR

• William L Nyhan, MD

  University of California, San Diego

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 6, 2010
• First Posted: February 9, 2010
• Study Start: February 1, 2010
• Primary Completion: January 1, 2011
• Study Completion: June 1, 2011
• Last Updated: April 18, 2013
• Results First Posted: April 18, 2013

Record last verified: 2013-04

Locations

US (2)