Back to resultsPhase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease
Primary Purpose
Lesch-Nyhan Syndrome
Status
Completed
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
aminomidazole carboxamide riboside
Sponsored by
About this trial
This is an interventional treatment trial for Lesch-Nyhan Syndrome focused on measuring Lesch-Nyhan syndrome, inborn errors of metabolism, neurologic and psychiatric disorders, rare disease
Eligibility Criteria
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Men with Lesch-Nyhan syndrome Moderate erythrocyte macrocytosis No active urinary tract disease or impaired renal function
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT00004314
First Posted
October 18, 1999
Last Updated
June 23, 2005
Sponsor
National Center for Research Resources (NCRR)
Collaborators
University of California, San Diego
1. Study Identification
Unique Protocol Identification Number
NCT00004314
Brief Title
Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease
Study Type
Interventional
2. Study Status
Record Verification Date
January 2000
Overall Recruitment Status
Completed
Study Start Date
February 1996 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Name of the Sponsor
National Center for Research Resources (NCRR)
Collaborators
University of California, San Diego
4. Oversight
5. Study Description
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Lesch-Nyhan Syndrome
Keywords
Lesch-Nyhan syndrome, inborn errors of metabolism, neurologic and psychiatric disorders, rare disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Enrollment
2 (false)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
aminomidazole carboxamide riboside
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
0 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics-- Men with Lesch-Nyhan syndrome Moderate erythrocyte macrocytosis No active urinary tract disease or impaired renal function
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bruce Barshop
Organizational Affiliation
University of California, San Diego
Official's Role
Study Chair
12. IPD Sharing Statement
Citations:
Citation
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.
Results Reference
background
PubMed Identifier
7660927
Citation
Page T, Barshop B, Yu AL, Nyhan WL. Treatment of Lesch-Nyhan syndrome with AICAR. Adv Exp Med Biol. 1994;370:353-6. doi: 10.1007/978-1-4615-2584-4_76. No abstract available.
Results Reference
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Learn more about this trial
Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease
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