MELAS Syndrome clinical trials
The registry lists 37 studies for MELAS Syndrome across more than 10 countries. 8 are recruiting or about to start. 26 of them are mainly about MELAS Syndrome (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-06.
Get an email when these change. Save the MELAS Syndrome studies recruiting or about to start — once a week, free: studies that open, close, add sites or post results.
See an example email
Example · made-up studies
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
One email a week for all your saved searches. Stop any time.
Who runs them
Khondrion BV (6) · Tisento Therapeutics (3) · PTC Therapeutics (3) · Columbia University (3) · Baylor College of Medicine (2) · National Taiwan University Hospital (1) · The Hospital for Sick Children (1) · LMU Klinikum (1)
What is being tested here
Drugs under test in MELAS Syndrome studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
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 2
- Dec 2026Tissue Sample Study for Mitochondrial Disorders · Columbia University
- Dec 2026North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC) · Columbia University
Who these studies take
Of the 5 MELAS Syndrome studies recruiting now, 4 (80%) set no upper age limit and 0 (0%) stop at 75 or younger. 3 (60%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 2 (40%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 3 (60%) | 3 (60%) |
| 18 | 5 (100%) | 5 (100%) |
| 30 | 5 (100%) | 5 (100%) |
| 40 | 5 (100%) | 5 (100%) |
| 50 | 5 (100%) | 5 (100%) |
| 65 | 5 (100%) | 5 (100%) |
| 75 | 5 (100%) | 5 (100%) |
| 85 | 4 (80%) | 4 (80%) |
Across all 37 studies, recruiting or not: 41% stop at 75 or younger and 32% at 65; 54% take children; 0% are for women only and 5% for men only; 24% 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.
Stopped1
- An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)TerminatedOct 30, 2026NCT06961344
Studies
Recruiting first, then most recently updated. Showing 37 of 37. search them.
- KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial DiseasesRecruitingNCT06451757Phase 37 countries
- A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)Not yet recruitingNCT06792500Phase 1/21 country
- Global Registry and Natural History Study for Mitochondrial DisordersRecruitingNCT055548353 countries
- North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)RecruitingNCT016949402 countries
- Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione LevelsRecruitingNCT05241262Phase 11 country
- Natural History Study - Mitochondrial DiseaseRecruitingNCT015327911 country
- Tissue Sample Study for Mitochondrial DisordersEnrolling by invitationNCT018039061 country
- Effectiveness of Ketogenic Diet in MELAS SyndromeNot yet recruitingNCT06013397Not applicable1 country
- An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)TerminatedNCT06961344Phase 25 countries
- A Phase 2b Study of Zagociguat in Patients With MELASCompletedNCT06402123Phase 26 countries
- A Study to Assess TTI-0102 vs Placebo in MELAS PatientsCompletedNCT06644534Phase 22 countries
- A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory EpilepsyTerminatedNCT04378075Phase 2/39 countries
- OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION)CompletedNCT05972954Phase 23 countries
- A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial DiseaseCompletedNCT05218655Phase 37 countries
- The KHENERGYC StudySuspendedNCT04846036Phase 21 country
- Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).CompletedNCT06051448Phase 1/21 country
- Mitochondrial Video Assessment Source Material StudyCompletedNCT061804211 country
- Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)TerminatedNCT04475549Phase 21 country
- L-Citrulline Dose Finding Safety Study in MELASCompletedNCT03952234Phase 11 country
- The KHENEREXT StudyCompletedNCT04604548Phase 24 countries
- EPI-743 for Mitochondrial Respiratory Chain DiseasesNo longer availableNCT013704471 country
- Clinical Long Term Evaluation of Glutamine Supplement in MELAS SyndromeActive, not recruitingNCT05255328Not applicable1 country
- Nicotinamide Riboside and Mitochondrial BiogenesisCompletedNCT03432871Not applicable1 country
- The KHENERGYZE StudyCompletedNCT04165239Phase 24 countries
- Glutamine Supplement in MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like Episodes) SyndromeCompletedNCT04948138Not applicable1 country
- A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial DiseaseCompletedNCT03888716Phase 11 country
- A Dose-escalating Clinical Trial With KH176CompletedNCT02544217Phase 11 country
- Safety, Tolerability and Pharmacokinetic Study of KL1333 in Healthy Male VolunteersCompletedNCT03056209Phase 11 country
- The KHENERGY StudyCompletedNCT02909400Phase 21 country
- Responses to Influenza Vaccine in Patients With Mitochondrial Disorders (MELAS)CompletedNCT01831934Phase 41 country
- Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like EpisodesCompletedNCT00887562Phase 21 country
- Nitric Oxide Production in MELAS SyndromeCompletedNCT01339494Early Phase 11 country
- Mitochondrial nt3243 A>G Mutation in TaiwanStatus unknownNCT021145541 country
- L-arginine Therapy on Endothelium-dependent Vasodilation & Mitochondrial Metabolism in MELAS SyndromeCompletedNCT01603446Phase 21 country
- Ketones & Mitochondrial HeteroplasmyCompletedNCT01252979Early Phase 11 country
- Study of the Metabolism of Pyruvate and Related Problems in Patients With Lactic AcidemiaCompletedNCT000043531 country
- Evaluating the Effectiveness of a Dichloroacetate in MELAS SyndromeStatus unknownNCT00068913Phase 21 country