Use of Cysteamine in the Treatment of Cystinosis
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00359684|
Recruitment Status : Recruiting
First Posted : August 2, 2006
Last Update Posted : December 7, 2017
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||Intervention/treatment||Phase|
|Cystinosis||Drug: Cysteamine||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||300 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Natural History Study of the Use of Cysteamine in the Treatment of Cystinosis|
|Study Start Date :||July 6, 1978|
|Estimated Primary Completion Date :||December 1, 2018|
|Estimated Study Completion Date :||December 1, 2018|
- Survival [ Time Frame: Lifetime ]
- Renal function, secondary complications of disease [ Time Frame: Decades ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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)|