Use of Cysteamine in the Treatment of Cystinosis
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. Both the kidney damage and growth failure are thought to be due to the accumulation of the amino acid cystine within the cells of the body. The cystine storage later damages other organs besides the kidneys, including the thyroid gland, pancreas, eyes, and muscle.
The drug cysteamine (Cystagon) is an oral medication given to patients with cystinosis prior to kidney transplantation. The drug works by reducing the level of cystine in the white blood cells and muscle tissue. The drug may also decrease levels of cystine in the kidneys and other tissues.
This study has several goals:
- <TAB>Long-term surveillance of cysteamine (Cystagon) treated patients.
- <TAB>Detection of new non-kidney complications of cystinosis.
- <TAB>Maintenance of a patient population for genetic testing (mutational analysis) of the cystinosis gene.<TAB>
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Natural History Study of the Use of Cysteamine in the Treatment of Cystinosis|
- Survival [ Time Frame: Lifetime ] [ Designated as safety issue: Yes ]
- Renal function, secondary complications of disease [ Time Frame: Decades ] [ Designated as safety issue: Yes ]
|Study Start Date:||July 1978|
|Estimated Study Completion Date:||December 2018|
|Estimated Primary Completion Date:||December 2018 (Final data collection date for primary outcome measure)|
Please refer to this study by its ClinicalTrials.gov identifier: NCT00359684
|Contact: William A Gahl, M.D.||(301) email@example.com|
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL) 800-411-1222 ext TTY8664111010 firstname.lastname@example.org|
|Principal Investigator:||William A Gahl, M.D.||National Human Genome Research Institute (NHGRI)|