Mitochondrial disease
MONDO:0044970132 clinical trials for this condition and its sub-types, 40 tagged with Mitochondrial disease itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Mitochondrial disease
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Inborn mitochondrial metabolism disorder 59 trials · 127 incl. sub-types
14 sub-types
- Mitochondrial oxidative phosphorylation disorder 3 trials · 58 incl. sub-types Sub-types →
- Inborn mitochondrial myopathy 18 trials · 45 incl. sub-types Sub-types →
- Pyruvate dehydrogenase deficiency 2 trials · 4 incl. sub-types Sub-types →
- Histiocytoid cardiomyopathy 3 trials Sub-types →
- Fumaric aciduria 2 trials
- OPA1-related optic atrophy with or without extraocular features 1 trial Sub-types →
- Inherited lipoic acid biosynthesis defect 0 trials · 1 incl. sub-types Sub-types →
- Multiple acyl-CoA dehydrogenase deficiency 1 trial Sub-types →
- 3-methylglutaconic aciduria with deafness, encephalopathy, and Leigh-like syndrome 0 trials
- HSD10 mitochondrial disease 0 trials Sub-types →
- Hypotonia-cystinuria syndrome 0 trials Sub-types →
- Mitochondrial membrane transport disorder 0 trials Sub-types →
- Mitochondrial pyruvate carrier deficiency 0 trials
- Oxoglutaricaciduria 0 trials
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1 sub-type
- Optic atrophy 9 0 trials
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Bjornstad syndrome 0 trials
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GRACILE syndrome 0 trials
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4 sub-types
- Cerebellar ataxia-areflexia-pes cavus-optic atrophy-sensorineural hearing loss syndrome 0 trials
- Optic atrophy 8 0 trials
- Optic atrophy with or without deafness, ophthalmoplegia, myopathy, ataxia, and neuropathy 0 trials
- Optic atrophy, hearing loss, and peripheral neuropathy, autosomal dominant 0 trials
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Ethylmalonic encephalopathy 0 trials
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Pure mitochondrial myopathy 0 trials
Most studied deeper sub-types
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Can a common supplement shrink fatty liver in a rare diabetes?
Disease control Not yet recruitingThis trial tests whether taking coenzyme Q10 (CoQ10) for 12 weeks can reduce fat buildup in the liver of people with mitochondrial diabetes, a rare form of diabetes caused by mitochondrial dysfunction. Participants with the m.3243A>G mutation will take 300 mg of CoQ10 daily, and …
Sponsor: The 95th Hospital of Putian，Putian, Fujian, China • Aim: Disease control
Last updated Aug 07, 2026 00:00 UTC
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Can a new oral drug help tame leigh syndrome?
Disease control Not yet recruitingThis trial tests an experimental oral drug, TTI-0102, in people aged 5 to 55 with Leigh syndrome spectrum, a rare genetic disorder that damages the brain and nerves. The study aims to find the right dose and check safety over 12 weeks of twice-daily treatment. Participants will h…
Phase 2 • Sponsor: Thiogenesis Therapeutics, Inc. • Aim: Disease control
Last updated Jul 26, 2026 00:00 UTC
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Experimental drug aims to boost energy in rare genetic disorders
Disease control Not yet recruitingThis study tests an oral drug called glycerol tributyrate in 24 adults with MELAS or LHON-Plus, two rare mitochondrial diseases that cause severe symptoms like strokes and vision loss. The trial is open-label (everyone gets the drug) and uses each person as their own control over…
Phase 1/2 • Sponsor: George Washington University • Aim: Disease control
Last updated Jun 27, 2026 13:02 UTC
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Lifestyle makeover tested as MS symptom fighter
Disease control Not yet recruitingThis study tests whether a structured 12-week program focusing on nutrition, exercise, sleep, and stress management can improve fatigue, physical function, and quality of life in people with relapsing-remitting multiple sclerosis. Thirty participants will first be observed for 12…
Sponsor: New York University Abu Dhabi • Aim: Disease control
Last updated Jun 27, 2026 12:34 UTC
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Could your own stem cells fight this rare brain disorder?
Disease control Not yet recruitingThis study tests whether a person's own stem cells, processed and given by IV, can safely help with multiple system atrophy (MSA) — a rare, worsening brain disease that affects movement and automatic body functions like blood pressure. Fifty adults aged 35 to 65 will receive eith…
Phase 2 • Sponsor: Biocells Medical • Aim: Disease control
Last updated Jun 27, 2026 11:01 UTC
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Could vitamin B3 save sight in rare genetic blindness?
Disease control Not yet recruitingThis early study tests whether high-dose vitamin B3 (nicotinamide) can help preserve or improve vision in people with Leber's hereditary optic neuropathy (LHON), a rare genetic disease that causes sudden vision loss. Researchers will give 13 participants 2 grams of vitamin B3 dai…
Phase 1 • Sponsor: University Hospital, Angers • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC
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New DNA test could end years of uncertainty for mitochondrial disease patients
Diagnosis Not yet recruitingThis pilot study aims to develop a new digital PCR technique to more accurately diagnose mitochondrial diseases. Researchers will test the method on blood, urine, saliva, and muscle fiber samples from 4 patients. If validated, the technique could be faster and cheaper than curren…
Sponsor: Centre Hospitalier Universitaire de Nice • Aim: Diagnosis
Last updated Jun 27, 2026 12:04 UTC
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Could a daily supplement recharge aging cells?
Prevention Not yet recruitingThis trial tests whether taking coenzyme Q10 (CoQ10) for 10 weeks can improve biological resilience in adults aged 65 and older who show early signs of frailty. Participants will be randomly assigned to receive either 200 mg of CoQ10 daily or a placebo. The study will measure cha…
Sponsor: University Medical Centre Ljubljana • Aim: Prevention
Last updated Aug 21, 2026 00:00 UTC
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Scientists probe cellular 'Power Plants' to unlock secrets of aging
Knowledge-focused Not yet recruitingThis study looks at how aging changes tiny parts of our cells called mitochondria, which produce energy. Researchers will take small skin samples and blood from 90 healthy adults aged 18-90 to measure inflammation and cell aging markers. The goal is to better understand why we ag…
Sponsor: Mario Negri Institute for Pharmacological Research • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:36 UTC
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New study aims to unravel Parkinson's protein mysteries
Knowledge-focused Not yet recruitingThis study looks at how abnormal proteins, like alpha-synuclein and tau, build up and affect brain function in people with Parkinson's disease. Researchers will use brain scans, blood tests, and skin biopsies to track these changes. The goal is to find better ways to diagnose and…
Sponsor: University of Pavia • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:28 UTC