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 tests a new medicine called TTI-0102 for cystinosis. It aims to see if it can help manage cystine levels in the body, which may reduce kidney and other organ damage.
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 is a follow-up registry for people with cystinosis to track their health over time. It helps doctors learn more about the condition and how it affects daily life, without giving any new treatments.
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 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 trial tests a new medicine called LNP.UCD.ABE that aims to fix a specific genetic change causing a severe urea cycle disorder. It uses a technique called base editing to correct the gene mutation, which may help your body break down ammonia better.
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