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NCT00359684 · ClinicalTrials.gov registry record
Use of Cysteamine in the Treatment of Cystinosis
A clinical trial of Cystinosis, sponsored by National Human Genome Research Institute (NHGRI).
- Recruiting
- Registry status
- 330
- Enrollment target
- 1
- Study location
NCT00359684: Recruiting study of Cystinosis, sponsored by National Human Genome Research Institute (NHGRI).
NCT00359684 is a study of Cystinosis that is actively recruiting participants, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 330 participants, above the 122-participant average among 4 other Cystinosis trials with a reported enrollment target (170% higher). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00359684, a study of Cystinosis, is actively recruiting participants, sponsored by National Human Genome Research Institute (NHGRI).
- RECRUITING
- Registry status
- 330 participants
- Enrollment target
- 1
- Study location
Study 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 gene.\<TAB\> ...
Primary Outcome
Serve as a source of knowledge and advice for individual cystinosis patients and for the community at large
Conditions Studied
Interventions
- DRUG Cysteamine
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 330 participants |
| Start Date | 1979-01-04 |
What NCT00359684 shows while recruiting
NCT00359684 is an observational study that tracks outcomes without assigning an intervention. The registered 330 participants enrollment target is mid-sized for trials with a published cap, above the 122-participant average among 4 other Cystinosis trials with a reported enrollment target (170% higher).
The record links to 1 condition, with Cystinosis appearing as the primary indexed condition, and to 1 intervention - of which Cysteamine is the first listed.
NCT00359684 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT00359684 about?
NCT00359684 is a clinical study titled "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 aci...
What is the current status of trial NCT00359684?
This trial is currently recruiting. The enrollment target is 330 participants. The study started on 1979-01-04.
What conditions does trial NCT00359684 study?
This clinical trial studies the following conditions: Cystinosis.
What interventions are being tested in trial NCT00359684?
The interventions under investigation include: Cysteamine (DRUG).
Who is sponsoring clinical trial NCT00359684?
This trial is sponsored by National Human Genome Research Institute (NHGRI), which has 185 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00359684 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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