Cystinosis drug under Long-Term watch: could cysteamine be key?
NCT ID NCT00359684
First seen Jun 27, 2026 · Last updated Jul 01, 2026 · Updated 2 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.
Disclaimer
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States