Testing existing drugs for mitochondrial disorders
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial looks at whether drugs already approved for other conditions might help people with Leigh syndrome, a rare mitochondrial disorder. Researchers will study cells made from your skin to see how these drugs work.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a genetic mutation that causes Leigh syndrome.
- You must have brain MRI changes typical of Leigh syndrome.
- You must not have a bleeding disorder that makes a skin biopsy unsafe.
- You must be willing to provide a small skin sample.
- You must not withdraw your consent to participate.
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 tests a new drug, TTI-0102, for people with Leigh syndrome spectrum. The goal is to see if it helps with symptoms and to find the right dose.
This study looks for the cause of suspected mitochondrial diseases by combining multiple lab tests on muscle and skin samples, plus genetic testing results. If your current gene tests didn’t find an answer, this could help researchers learn what might be causing your condition.
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This trial tests if sirolimus can help people with Leigh syndrome. It aims to see if the drug can improve neurological symptoms and is looking for participants aged 6 months to 55 years.
This study looks at how metabolism, infections, and the immune system work together in people with mitochondrial disease. It aims to learn more about the condition and may help guide future treatments.
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