ClinicalTrials.gov
ClinicalTrials.gov Menu

Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00004314
Recruitment Status : Completed
First Posted : October 19, 1999
Last Update Posted : June 24, 2005
Sponsor:
Collaborator:
University of California, San Diego
Information provided by:
Office of Rare Diseases (ORD)

Brief Summary:

OBJECTIVES: I. Determine the effects of aminoimidazole carboxamide riboside (AICAR) on hematologic manifestations of Lesch-Nyhan disease.

II. Assess the behavioral and neurological benefits of AICAR in patients with Lesch-Nyhan disease.

III. Examine the effect of AICAR on purine production.


Condition or disease Intervention/treatment Phase
Lesch-Nyhan Syndrome Drug: aminomidazole carboxamide riboside Phase 2

Detailed Description:

PROTOCOL OUTLINE:

After a 3-day baseline, patients are treated with aminoimidazole carboxamide riboside (AICAR) by continuous infusion for 12 days. Concurrent allopurinol and folate are allowed.


Study Type : Interventional  (Clinical Trial)
Enrollment : 2 participants
Primary Purpose: Treatment
Study Start Date : February 1996

Resource links provided by the National Library of Medicine






Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics-- Men with Lesch-Nyhan syndrome Moderate erythrocyte macrocytosis No active urinary tract disease or impaired renal function


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004314


Sponsors and Collaborators
National Center for Research Resources (NCRR)
University of California, San Diego
Investigators
Study Chair: Bruce Barshop University of California, San Diego

Publications:
Page T, Barshop BA, Yu A, et al.: Treatment of Lesch-Nyhan syndrome with AICAR. In: Sahota A, Tayor M, eds.: Purine and Pyrimidine Metabolism in Man VIII. Plenum Press, New York, NY: 1995, pp 353-356.

ClinicalTrials.gov Identifier: NCT00004314     History of Changes
Other Study ID Numbers: 199/11823
UCSD-733
First Posted: October 19, 1999    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: January 2000

Keywords provided by Office of Rare Diseases (ORD):
Lesch-Nyhan syndrome
inborn errors of metabolism
neurologic and psychiatric disorders
rare disease

Additional relevant MeSH terms:
Lesch-Nyhan Syndrome
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