Cystinosis and Mitochondrial Energy Study
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be at least 2 years old.
- You must have a confirmed diagnosis of nephropathic cystinosis (a kidney form of cystinosis).
- You must be taking cysteamine (a medicine for cystinosis).
- You must not be on dialysis or have had a kidney transplant.
- You must not be pregnant or breastfeeding.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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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 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.
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This trial tests a new gene therapy called DFT383 for children aged 2 to 5 with nephropathic cystinosis. It aims to see if the treatment is safe and can help improve kidney function.
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