Mitochondrial Myopathies clinical trials
The registry lists 46 studies for Mitochondrial Myopathies across more than 10 countries. 14 are recruiting or about to start. 32 of them are mainly about Mitochondrial Myopathies (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-06. Counting its narrower condition MELAS Syndrome too: 75 studies, 20 open.
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Breast cancer · Recruiting: 2 came in, 3 changed
What changed in your saved searches, Oct 31 – Nov 6:
A study of drug A after surgery for early breast cancer
Came in · now Recruiting
Drug B with hormone therapy in advanced breast cancer
Changed
· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
Exercise during chemotherapy
Now Active, not recruiting
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Who runs them
Children's Hospital of Philadelphia (5) · Stealth BioTherapeutics Inc. (4) · Rigshospitalet, Denmark (4) · Maastricht University (3) · Reneo Pharma Ltd (3) · Khondrion BV (2) · Cristina Domínguez González (2) · Columbia University (2)
What is being tested here
Drugs under test in Mitochondrial Myopathies studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Elamipretide | 0 | 1 | 3 |
| Nicotinamide-beta-riboside | 0 | 0 | 2 |
| Niacin | 0 | 0 | 2 |
| Trolox | 0 | 0 | 2 |
| Napazimone | 0 | 0 | 1 |
| Resveratrol | 0 | 0 | 1 |
| Thioctic Acid | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 8
- Nov 2026A Study to Evaluate Vitamin B3 Derivative to Treat Mitochondrial Myopathy · Ralitza Gavrilova
- Dec 2026North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC) · Columbia University
- Jan 2027Doxecitin and Doxribthymine in Adult Subjects With Thymidine Kinase 2 (TK2) Deficiency · Cristina Domínguez González
- Feb 2027Role of AMPKalpha2 in Myonuclear Accretion in Mitochondrial Myopathies · Hospices Civils de Lyon
- Jun 2027Home Based Personalized Training and Video Consultation in Mitochondrial Myopathies: Study of Efficacy and Tolerance. · Centre Hospitalier Universitaire de Nice
- Aug 2027Validation of Oxygen Nanosensor in Mitochondrial Myopathy · Children's Hospital of Philadelphia
- Sep 2027Validation of Nanosensor Oxygen Measurement · Children's Hospital of Philadelphia
- Sep 2027Applying pGz in Mitochondrial Disease · Children's Hospital of Philadelphia
Who these studies take
Of the 13 Mitochondrial Myopathies studies recruiting now, 8 (62%) set no upper age limit and 4 (31%) stop at 75 or younger. 6 (46%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 6 (46%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 5 (38%) | 5 (38%) |
| 18 | 13 (100%) | 13 (100%) |
| 30 | 13 (100%) | 13 (100%) |
| 40 | 13 (100%) | 13 (100%) |
| 50 | 13 (100%) | 13 (100%) |
| 65 | 11 (85%) | 11 (85%) |
| 75 | 9 (69%) | 9 (69%) |
| 85 | 9 (69%) | 9 (69%) |
Across all 46 studies, recruiting or not: 35% stop at 75 or younger and 17% at 65; 39% take children; 0% are for women only and 2% for men only; 35% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 13, 2026.
Newly registered1
- Role of AMPKalpha2 in Myonuclear Accretion in Mitochondrial MyopathiesNov 5, 2026NCT07920562
Studies
Recruiting first, then most recently updated. Showing 46 of 46. search them.
- Role of AMPKalpha2 in Myonuclear Accretion in Mitochondrial MyopathiesNot yet recruitingNCT079205621 country
- Validation of Nanosensor Oxygen MeasurementRecruitingNCT06819683Phase 11 country
- Natural History Study of Mitochondrial MyopathyRecruitingNCT052503751 country
- Doxecitin and Doxribthymine in Adult Subjects With Thymidine Kinase 2 (TK2) DeficiencyRecruitingNCT06754098Phase 21 country
- Prospective Natural History Study of POLG DiseaseRecruitingNCT077758729 countries
- Global Registry and Natural History Study for Mitochondrial DisordersRecruitingNCT055548353 countries
- Home Based Personalized Training and Video Consultation in Mitochondrial Myopathies: Study of Efficacy and Tolerance.RecruitingNCT05346627Not applicable1 country
- Exercise Training Effects on Muscle Function in Adults With Mitochondrial MyopathyRecruitingNCT07450690Not applicable1 country
- Validation of Oxygen Nanosensor in Mitochondrial MyopathyRecruitingNCT04086329Phase 11 country
- North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)RecruitingNCT016949402 countries
- Applying pGz in Mitochondrial DiseaseRecruitingNCT05569122Phase 11 country
- Natural History in Primary Mitochondrial MyopathiesRecruitingNCT056535441 country
- Effect and Safety MABs Administration m.3243A>G Mutation CarriersRecruitingNCT05962333Phase 21 country
- mtDNA Mutation Load Analysis in MesoangioblastsRecruitingNCT051997401 country
- Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular DiseaseActive, not recruitingNCT07478172Not applicable1 country
- Assessment of Safety and Acute Effects of a Lower-limb Powered Dermoskeleton in Patients With Neuromuscular DisordersCompletedNCT05199246Not applicable1 country
- Assessment of Safety and Acute Effects of a Knee-hip Powered Soft Exoskeleton in Patients With Neuromuscular DisordersCompletedNCT05200702Not applicable1 country
- A Study to Evaluate Vitamin B3 Derivative to Treat Mitochondrial MyopathyActive, not recruitingNCT05590468Phase 21 country
- Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)CompletedNCT05162768Phase 310 countries
- Exercise-mediated Rescue of Mitochondrial Dysfunctions Driving Insulin ResistanceCompletedNCT06080594Not applicable1 country
- Human Mitochondrial Stress-driven Obesity ResistanceCompletedNCT060805681 country
- Mitochondrial Dysfunctions Driving Insulin ResistanceCompletedNCT060805811 country
- RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTORCompletedNCT02255422Phase 22 countries
- Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).CompletedNCT06051448Phase 1/21 country
- Genomic Profiling of Mitochondrial Disease - Imaging Analysis for Precise Mitochondrial MedicineCompletedNCT050123581 country
- An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)TerminatedNCT05267574Phase 2/312 countries
- An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy PatientsCompletedNCT04535609Phase 215 countries
- MABs Therapy m.3243A>G Mutation CarriersCompletedNCT05063721Phase 11 country
- Nicotinamide Riboside and Mitochondrial BiogenesisCompletedNCT03432871Not applicable1 country
- Niacin Supplementation in Healthy Controls and Mitochondrial Myopathy PatientsCompletedNCT03973203Not applicable
- Diagnostic Screening Tests and Potential Biomarkers in Mitochondrial MyopathiesCompletedNCT035138351 country
- A Single Case Study of HypermetabolismCompletedNCT04805268Not applicable1 country
- The KHENERGYZE StudyCompletedNCT04165239Phase 24 countries
- Oxidative Capacity and Exercise Tolerance in Ambulatory SMACompletedNCT028957891 country
- A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial DiseaseCompletedNCT03888716Phase 11 country
- NiaMIT Continuation With Early-stage Mitochondrial Myopathy PatientsCompletedNCT04538521Not applicable1 country
- Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER StudyCompletedNCT02805790Phase 21 country
- A Study of the Safety of REN001 in Patients With Primary Mitochondrial MyopathyTerminatedNCT03862846Phase 11 country
- Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial MyopathyCompletedNCT02367014Phase 1/21 country
- Resveratrol Supplementation in Patients With Mitochondrial Myopathies and Skeletal Muscle Fatty Acid Oxidation DisordersCompletedNCT03728777Not applicable1 country
- An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)CompletedNCT030486179 countries
- The KHENERGY StudyCompletedNCT02909400Phase 21 country
- Nutritional Assessment in Mitochondrial CytopathyCompletedNCT023754381 country
- The Effects of Exercise Versus Inactivity on People With Mitochondrial Muscle DiseaseStatus unknownNCT00457314Phase 21 country
- Study of the Metabolism of Pyruvate and Related Problems in Patients With Lactic AcidemiaCompletedNCT000043531 country
- Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial MyopathyCompletedNCT00004770Not applicable