Use of Cysteamine in the Treatment of Cystinosis

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Human Genome Research Institute (NHGRI)
Study ID
NCT00359684
Status
Recruiting
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Conditions

  • Cystinosis

Eligibility Criteria

Sex
ALL
Age
1 Week - 115 Years
Healthy Volunteers
Not accepted

Interventions

  • Cysteamine — DRUG
    Cystine-depleting agent

Study Details

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\>

Key Dates

Start date
Jan 4, 1979
Status verified
Apr 2026

Study Design

Enrollment
330 participants (estimated)

Arms

  • Arm: Cystinosis
    Patients with a diagnosis of cystinosis

Primary Outcome Measure

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 ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical CenterBethesdaMaryland20892-

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National Institutes of Health Clinical Center· Bethesda, MD

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