Access to elamipretide for rare mitochondrial disease
Part of Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This trial provides access to an investigational drug called elamipretide for people with rare mitochondrial diseases. The drug aims to improve energy production in cells and may help with serious symptoms like heart or kidney problems.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 1 and 80 years old (or at least 12 years old if you have Barth syndrome).
- You have a confirmed rare mitochondrial disease with serious symptoms like heart problems, kidney issues, nerve damage, or vision problems — or you have serious signs of mitochondrial dysfunction without a genetic diagnosis.
- The disease is serious or life-threatening, and no other approved treatment works well enough for you.
- You or a caregiver can give you a daily injection under the skin.
- You are not allergic to elamipretide, not pregnant or breastfeeding, and have not taken another experimental drug within the last 30 days.
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 trial tests a medicine called elamipretide (4TAZ) in boys with Barth Syndrome, a genetic condition affecting the heart and muscles. The goal is to see if this medicine can help improve heart function and how the body uses energy.
This trial tests a new medicine, sonlicromanol, to see if it can ease chronic fatigue and improve muscle function in people with a specific genetic condition called primary mitochondrial disease. If effective, it could offer a new treatment option to improve daily energy and quality of life.
This trial tests a one-time infusion of your own blood cells mixed with donated mitochondria to see if it can help children with Pearson Syndrome grow better and improve their health. It is a new approach that aims to provide the energy your cells need.
This is a follow-up study for patients who completed a previous trial (FALCON) testing KL1333, an experimental medicine for primary mitochondrial disease—a condition where cells don't produce enough energy. The study tracks how safe and effective the treatment remains over time.
This study tests a drug called elamipretide to see if it can improve physical function in older adults with lower mitochondrial health. It may help you keep your energy and independence as you age.
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.
Hear when a new Mitochondrial Diseases trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.