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, 3 tagged with Mitochondrial encephalomyopathy itself.
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Browse by category →Sub-types of Mitochondrial encephalomyopathy
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MELAS syndrome 13 trials · 14 incl. sub-types
10 sub-types
- MELAS syndrome caused by mutation in MTTL1 1 trial
- MELAS syndrome caused by mutation in MTND1 0 trials
- MELAS syndrome caused by mutation in MTND5 0 trials
- MELAS syndrome caused by mutation in MTND6 0 trials
- MELAS syndrome caused by mutation in MTTC 0 trials
- MELAS syndrome caused by mutation in MTTH 0 trials
- MELAS syndrome caused by mutation in MTTK 0 trials
- MELAS syndrome caused by mutation in MTTQ 0 trials
- MELAS syndrome caused by mutation in MTTS1 0 trials
- MELAS syndrome caused by mutation in MTTS2 0 trials
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MERRF syndrome 5 trials
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New hope for MELAS: experimental drug tested for Long-Term safety
Disease control OngoingThis study is testing the long-term safety of a daily oral drug called zagociguat in 44 adults with MELAS, a rare genetic disease that affects energy production in cells. All participants previously completed a lead-in study of the same drug. Researchers will monitor side effects…
Phase 2 • Sponsor: Tisento Therapeutics • Aim: Disease control
Last updated Jul 09, 2026 00:00 UTC
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Last chance access: vatiquinone for mitochondrial disease patients
Disease control Expanded access (ended)This program offered vatiquinone, an experimental liquid medication, to patients with inherited mitochondrial diseases like Leigh syndrome who had already completed a previous safety study. The goal was to continue treatment for those who might benefit, but enrollment is now clos…
Sponsor: Medical University of South Carolina • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC
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New study tests workplace coaching to keep Parkinson's patients on the job
Symptom relief OngoingThis study tests a personalized workplace intervention for 124 Dutch workers with Parkinson's disease, cerebellar ataxia, hereditary spastic paraparesis, or slowly progressive neuromuscular/mitochondrial disorders. A trained facilitator helps employees and their managers identify…
Sponsor: Radboud University Medical Center • Aim: Symptom relief
Last updated Aug 16, 2026 00:00 UTC
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MRI scans could unlock secrets of rare muscle disease
Knowledge-focused By invitation onlyThis study uses special MRI scans to measure how well muscles produce energy in people with mitochondrial disease. Researchers hope to learn more about the condition and develop a new tool to help diagnose and track it. The study involves 230 participants aged 7 to 75 with suspec…
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 UTC
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Massive gene hunt launched for mysterious mitochondrial diseases
Knowledge-focused By invitation onlyThis study aims to discover new genetic mutations that cause mitochondrial disorders by analyzing tissue samples from up to 6,900 participants. It includes people with suspected or known mitochondrial diseases, such as MELAS or Leigh's Disease, who lack a genetic diagnosis. The r…
Sponsor: Columbia University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:09 UTC
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Tiny power plants in cells may weaken bones, new study hints
Knowledge-focused OngoingThis study looks at how problems with mitochondria—the tiny power plants inside cells—might affect bone health. Researchers will compare 30 people with certain genetic changes that cause mitochondrial dysfunction to healthy volunteers. They will take blood, bone marrow, and bone …
Sponsor: Aalborg University Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:53 UTC