Mitochondrial encephalomyopathy
MONDO:0004675A heterogenous group of disorders characterized by alterations of mitochondrial metabolism that result in muscle and nervous system dysfunction. These are often multisystemic and vary considerably in age at onset (usually in the first or second decade of life), distribution of affected muscles, severity, and course. (From Adams et al., Principles of Neurology, 6th ed, pp984-5)
27 clinical trials for this condition and its sub-types.
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Broader categories
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New pill hopes to ease rare mitochondrial disease
Disease control CompletedThis study tests an oral drug called TTI-0102 in 12 people with MELAS, a rare genetic disorder that causes muscle weakness, strokes, and fatigue. Participants receive either the drug or a placebo for 6 months. Researchers will measure walking ability, fatigue, and quality of life…
Phase: PHASE2 • Sponsor: Thiogenesis Therapeutics, Inc. • Aim: Disease control
Last updated Jul 26, 2026 00:00 UTC
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New pill aims to ease fatigue and brain fog in rare mitochondrial disease
Disease control CompletedThis Phase 2b trial tests an oral drug called zagociguat in 43 adults with MELAS syndrome, a rare genetic disorder that causes fatigue, muscle weakness, and stroke-like episodes. Participants take either 15 mg, 30 mg, or a placebo daily for 12 weeks. The study measures changes in…
Phase: PHASE2 • Sponsor: Tisento Therapeutics • Aim: Disease control
Last updated Jul 09, 2026 00:00 UTC
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New drug shows promise for rare energy disorder
Disease control CompletedThis study tested a new drug called OMT-28 in 28 people with primary mitochondrial disease, a condition that affects how cells produce energy. Participants took the drug once daily for 6 months, and researchers measured safety, blood markers of inflammation, and symptoms like fat…
Phase: PHASE2 • Sponsor: Omeicos Therapeutics GmbH • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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Long-Term safety of mitochondrial drug confirmed in 101 patients
Disease control CompletedThis study looked at the safety of vatiquinone in 101 people with inherited mitochondrial disease who had already taken the drug in a previous study or treatment plan. The goal was to track any side effects until the drug became commercially available or the program ended. Partic…
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC