Completed
Aminoimidazole Carboxamide Riboside (AICAR) for Lesch-Nyhan Disease Treatment
Study Aim
This phase 2 study aims to evaluate the effectiveness of Aminoimidazole Carboxamide Riboside (AICAR) as a treatment for individuals with Lesch-Nyhan Disease.
What is being tested
aminomidazole carboxamide riboside
Drug
Who is being recruted
Brain Diseases+21
+ Brain Diseases, Metabolic
+ Central Nervous System Diseases
How is the trial designed
Treatment Study
Phase 2
Interventional
Study Start: February 1996
Summary
Principal SponsorNational Center for Research Resources (NCRR)
Last updated: June 24, 2005
Sourced from a government-validated database.Claim as a partner
Study start date: February 1, 1996
Actual date on which the first participant was enrolled.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.
Principal SponsorNational Center for Research Resources (NCRR)
Last updated: June 24, 2005
Sourced from a government-validated database.Claim as a partner
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.Design Details
2 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Conditions
Criteria
Male
Biological sex of participants that are eligible to enroll.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Brain DiseasesBrain Diseases, MetabolicCentral Nervous System DiseasesIntellectual DisabilityMetabolic DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNutritional and Metabolic DiseasesPathologic ProcessesPurine-Pyrimidine Metabolism, Inborn ErrorsSigns and SymptomsPathological Conditions, Signs and SymptomsNeurobehavioral ManifestationsHeredodegenerative Disorders, Nervous SystemBrain Diseases, Metabolic, InbornDisease AttributesGenetic Diseases, InbornX-Linked Intellectual DisabilityGenetic Diseases, X-LinkedMental DisordersLesch-Nyhan SyndromeMetabolism, Inborn ErrorsNeurologic ManifestationsRare Diseases
Criteria
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Men with Lesch-Nyhan syndrome Moderate erythrocyte macrocytosis No active urinary tract disease or impaired renal function
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has no location dataSave this study to your profile to know when the location data is available.
CompletedNo study centers