Use of Cysteamine in the Treatment of Cystinosis
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|ClinicalTrials.gov Identifier: NCT00359684|
Recruitment Status : Recruiting
First Posted : August 2, 2006
Last Update Posted : October 10, 2019
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>
|Condition or disease|
|Study Type :||Observational|
|Estimated Enrollment :||300 participants|
|Official Title:||Natural History Study of the Use of Cysteamine in the Treatment of Cystinosis|
|Actual Study Start Date :||January 4, 1979|
Patients with a diagnosis of cystinosis
- Serve as a source of knowledge and advice for individual cystinosis patients and for the community at large [ Time Frame: Follow-up can occur every two years ]Serve as a source of knowledge and advice for individual cystinosis patients and for the community at large
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00359684
|Contact: William A Gahl, M.D.||(301) firstname.lastname@example.org|
|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 Office of Patient Recruitment (OPR) 800-411-1222 ext TTY8664111010 email@example.com|
|Principal Investigator:||William A Gahl, M.D.||National Human Genome Research Institute (NHGRI)|