Status:
RECRUITING
Use of Cysteamine in the Treatment of Cystinosis
Lead Sponsor:
National Human Genome Research Institute (NHGRI)
Conditions:
Cystinosis
Eligibility:
All Genders
1-115 years
Brief Summary
Cystinosis is an inherited disease resulting in poor growth and kidney failure. There is no known cure for cystinosis, although kidney transplantation may help the renal failure and prolong survival. ...
Detailed Description
Patients with nephropathic cystinosis have been treated with the cystine-depleting agent cysteamine since 1978. This therapy prevents or delays renal deterioration, improves growth, and depletes paren...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Diagnosis of cystinosis, whether classical or one of the variants with later onset or no renal complications.
- Patients will be diagnosed as having cystinosis based upon a leucocyte cystine content greater than 1 nmol half-cystine/mg protein (normal, less than 0.2) and a typical clinical course.
- EXCLUSION CRITERIA:
- Inability to travel to the NIH.
- Age less than one week.
- Nonviable neonates and neonates of uncertain viability will be excluded.
Exclusion
Key Trial Info
Start Date :
January 4 1979
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
Estimated Enrollment :
330 Patients enrolled
Trial Details
Trial ID
NCT00359684
Start Date
January 4 1979
Last Update
January 8 2026
Active Locations (1)
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1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892