Cysteamine treatment for cystinosis
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial looks at the use of cysteamine to treat cystinosis, a rare inherited condition. It may help lower harmful cystine buildup in the body and improve outcomes, especially early in life.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a diagnosis of cystinosis (classical or a later-onset variant, even without kidney complications).
- Your diagnosis is based on a blood test showing high cystine in white blood cells.
- You can travel to the National Institutes of Health (NIH) for study visits.
- You are at least 1 week old.
- You are considered viable (not nonviable or of uncertain viability).
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study looks at how cystinosis affects the energy centers (mitochondria) in your cells. It may help doctors understand how cysteamine treatment works in patients with kidney cystinosis.
This study gathers information from people with cystinosis to learn more about the condition. You may be able to join if you have a confirmed diagnosis.
This study follows people with confirmed cystinosis to better understand the condition. It mainly checks that you have the right diagnosis and can join voluntarily.
This study offers genetic screening to newborns to look for two rare metabolic conditions. It helps doctors find these conditions earlier, so treatment can start sooner if needed.
This study checks how people with cystinosis are doing long-term after finishing an earlier study medicine called CTNS-RD-04. It may help researchers understand the lasting effects and safety of that treatment over time.
This study is testing a health-and-quality-of-life questionnaire for children and young adults with cystinosis. If you join, you may complete surveys and take part in interviews to help make the questions clearer and more useful.
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