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Use of Cysteamine in the Treatment of Cystinosis

StatusRecruiting
PhaseNot specified
Started
View on ClinicalTrials.gov ↗

Record History

The 50 most recent of 359 record versions are accounted for below. See every version on ClinicalTrials.gov ↗

  1. New version filed on ClinicalTrials.gov
  2. 49 versions Re-verified since Feb 8, 2025, no change to tracked fieldsv310-v358
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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\>
Trial Details
NCT Number NCT00359684
Lead Sponsor National Human Genome Research Institute (NHGRI)
Conditions Cystinosis
Enrollment 330 participants
Start Date
Updated on ClinicalTrials.gov