Cystinosis drug under Long-Term watch: could cysteamine be key?

NCT ID NCT00359684

Recruiting now Disease control Sponsor: National Human Genome Research Institute (NHGRI) Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 29, 2026 · Updated 6 times

Summary

Cystinosis is a rare inherited disease that causes kidney failure and poor growth due to cystine buildup in cells. This study follows 330 patients taking cysteamine, a drug that lowers cystine levels, to track long-term effects, detect new complications, and support genetic research. The goal is to better manage the disease and improve patient outcomes.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: cysteamine
What this could lead to: If successful, this study could confirm cysteamine's long-term safety and effectiveness in managing cystinosis, potentially improving growth and reducing organ damage.
What could go wrong: This is an observational follow-up, not a controlled trial, so it cannot prove cysteamine works. Cystinosis remains a serious disease with no cure, and long-term complications may still occur.

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Conditions

The condition(s) this trial relates to.

cystinosis metabolic disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CYSTINOSIS

Contacts and locations

Locations

National Institutes of Health Clinical Center
RECRUITING

Bethesda, Maryland, 20892, United States

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