This study was a clinical trial to determine the safety and efficacy of cysteamine in the treatment of nephropathic cystinosis, a rare inborn metabolic disease which usually leads to end-stage renal disease before 10 years of age. All children enrolled in the trial received oral cysteamine. Control information was provided by data collected on 64 patients who participated in a previous trial evaluating ascorbic acid for the treatment of this disease. The cysteamine trial enrolled 94 patients; analysis of data is completed, and major findings have been published.

Project Start
Project End
Budget Start
Budget End
Support Year
9
Fiscal Year
1987
Total Cost
Indirect Cost
Name
U.S. National Inst/Child Hlth/Human Dev
Department
Type
DUNS #
City
State
Country
United States
Zip Code