Mitochondrial disease

Tests elamipretide against placebo in people with genetically confirmed Barth syndrome.

Tests sonlicromanol against placebo for fatigue and physical ability in adults with mitochondrial disease.

Tests an oxygen nanosensor in muscle of people with mitochondrial myopathy and healthy volunteers.

Tests glycerol tributyrate taken by mouth in adults with MELAS or LHON-Plus.

Tests KL1333 over a long time in adults with primary mitochondrial disease.

Tests an eye imaging device in adults with retina or optic nerve conditions.

Tests KL1333 against placebo for fatigue and leg strength in adults with mitochondrial disease.

Tests coenzyme Q10 against placebo for symptoms in veterans with Gulf War illness.

Tests sirolimus for safety and effect in people with Leigh syndrome.

Tests a tiny oxygen sensor in muscle of adults with mitochondrial myopathy and healthy volunteers.

Tests N-acetylcysteine doses in people with m.3243A>G mitochondrial disease.

Tests gentle bed-based passive exercise in people with mitochondrial disease and healthy volunteers.

Follows people with mitochondrial disease to see how infection relates to worsening health.

Collects voluntary health information from people with rare or undiagnosed conditions for future research.

Studies genes and symptoms in pyruvate dehydrogenase complex deficiency.

Follows adults with mitochondrial disease and other fatigue conditions using an energy score.

Studies how primary mitochondrial myopathy changes over time, compared with healthy people.

Observes how POLG-related mitochondrial disease progresses in people over time.

Studies how genes relate to illness in people with mitochondrial disease and their relatives.

Tests own bone marrow stem cells in adults with retinal or optic nerve disease.