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 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.
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 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.
This study looks at how cysteamine affects the bone health of people with nephropathic cystinosis, a rare genetic condition. It includes children and adults who are already taking cysteamine by mouth and will follow the study plan closely.
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