Ignite Creation Date:
2024-05-05 @ 4:56 PM
Last Modification Date:
2024-10-26 @ 9:26 AM
Study NCT ID:
NCT00359684
Status:
RECRUITING
Last Update Posted:
2024-06-25
First Post:
2006-08-01
Brief Title:
Use of Cysteamine in the Treatment of Cystinosis
Sponsor:
National Human Genome Research Institute NHGRI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Use of Cysteamine in the Treatment of Cystinosis
Status:
RECRUITING
Status Verified Date:
2024-05-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 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 ProCysBi 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
1 Long-term surveillance of cysteamine treated patients
2 Detection of new non-kidney complications of cystinosis
3 Maintenance of a patient population for genetic testing mutational analysis of the cystinosis geneTAB
The drug cysteamine Cystagon ProCysBi 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
1 Long-term surveillance of cysteamine treated patients
2 Detection of new non-kidney complications of cystinosis
3 Maintenance of a patient population for genetic testing mutational analysis of the cystinosis geneTAB
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 parenchymal tissues of cystine Based largely upon data produced through this protocol the Food and Drug Administration approved cysteamine bitartrate for use in cystinosis patients on August 15 1994 Cysteamine is available as CystagonR through Mylan Pharmaceuticals in 50 mg and 150 mg capsules and as ProcysbiR in 75 mg capsules By virtue of the current protocol patients are admitted to the NIH Clinical Center for investigations every two years except for cases of great interest or urgency On each 1-3 day admission a battery of tests is performed and the adequacy of cystine depletion by cysteamine is monitored This protocol demonstrates the course of cystinosis patients treated with cysteamine describes new complications of the disorder in poorly treated adults and maintains NHGRI expertise in the field Its monitoring and followup of patients over the course of 3 decades represents an invaluable contribution to our understanding of the natural history of this rare disease
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 78-HG-0093 | None | None | None |