Testing Cysteamine for cystinosis bone health
Treatments studied
Part of Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your child) have confirmed nephropathic cystinosis.
- You are currently taking cysteamine by mouth (oral cysteamine).
- You are older than 2 years.
- You (and/or your parent/guardian) agree to take part by giving written non-opposition/permission.
- You are able and willing to follow all study visits and instructions.
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 is an observational study that asks people with nephropathic cystinosis about their quality of life while taking cystine-depleting treatment (cysteamine). It may help researchers better understand how this condition and treatment affect daily living.
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.
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 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.
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