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

This study has been completed.
Information provided by (Responsible Party):
Psyadon Pharma Identifier:
First received: February 6, 2010
Last updated: April 17, 2013
Last verified: April 2013
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.

Condition Intervention Phase
Lesch-Nyhan Disease
Drug: Ecopipam
Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by Psyadon Pharma:

Primary Outcome Measures:
  • 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.

Secondary Outcome Measures:
  • 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) ]
    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

Enrollment: 5
Study Start Date: February 2010
Study Completion Date: June 2011
Primary Completion Date: January 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Ecopipam 12.5 - 200 mg/day
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
Tablets, once daily, for two weeks up to 200 mg/day
Other Name: PSYRX101, SCH 39166


Ages Eligible for Study:   6 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria

  • 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

Exclusion Criteria:

  • 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01065558

United States, California
University of California at San Diego
San Diego, California, United States, 92103
United States, Georgia
Emory Univesity
Atlanta, Georgia, United States, 30329
Sponsors and Collaborators
Psyadon Pharma
Principal Investigator: Hyder A Jinnah, MD Emory University
Principal Investigator: William L Nyhan, MD University of California, San Diego
  More Information

Responsible Party: Psyadon Pharma Identifier: NCT01065558     History of Changes
Other Study ID Numbers: Ecopipam and Lesch-Nyhan
Study First Received: February 6, 2010
Results First Received: December 18, 2012
Last Updated: April 17, 2013

Keywords provided by Psyadon Pharma:
Lesch-Nyhan Disease
Self-Injurious Behavior

Additional relevant MeSH terms:
Self-Injurious Behavior
Lesch-Nyhan Syndrome
Behavioral Symptoms
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Purine-Pyrimidine Metabolism, Inborn Errors
Metabolic Diseases
Dopamine Antagonists
Dopamine Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Antipsychotic Agents
Tranquilizing Agents
Central Nervous System Depressants
Psychotropic Drugs processed this record on April 21, 2017