Condition (MeSH)
MELAS Syndrome
ClinicalTrials.gov lists 37 trials for MELAS Syndrome or a narrower condition beneath it. Khondrion BV has the most among industry and government lead sponsors, with 6.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials per phase
1 more section with rows
37 of the 37 trials are tagged with MELAS Syndrome itself; the rest are tagged with a narrower condition beneath it.
When did MELAS Syndrome trials start? (18)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year. 1 trial is registered to start in 2027 or later, and not listed here.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- Not applicable
- Observational
Top of scale: 5
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 1 |
| 2025 | 2 |
| 2024 | 1 |
| 2023 | 5 |
| 2022 | 1 |
| 2021 | 5 |
| 2020 | 2 |
| 2019 | 2 |
| 2017 | 2 |
| 2016 | 1 |
Showing 10 of 18 earlier years.
No forecast is made for this condition: it averages 2.0 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 3 trials still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are MELAS Syndrome trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 7 |
| Phase 1/2 | 2 |
| Phase 2 | 12 |
| Phase 2/3 | 1 |
| Phase 3 | 2 |
| Phase 4 | 1 |
| Not applicable | 4 |
| Observational | 7 |
| Expanded access | 1 |
How many MELAS Syndrome trials are recruiting? (9)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 2 |
| Recruiting | 5 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 1 |
| Completed | 21 |
Showing 5 of 9 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most MELAS Syndrome trials? (5)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| Khondrion BV | 6 |
| PTC Therapeutics | 3 |
| Tisento Therapeutics | 3 |
| Abliva AB | 1 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 1 |
Which drugs are being tested for MELAS Syndrome? (10)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| KH176 | 6 |
| EPI-743 | 3 |
| IW-6463 | 3 |
| KL1333 | 2 |
| Nicotinamide Riboside | 1 |
Showing 5 of 10 drugs.
Interventions as named in the registry (5)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| TTI-0102 | 1 |
| N Acetylcysteine | 1 |
| Arginine | 1 |
| Ketogenic diet | 1 |
| Fluzone | 1 |
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do MELAS Syndrome trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Pneumonia | 20 | 2 |
| Seizure | 14 | 2 |
| Acute respiratory failure | 11 | 1 |
| COVID-19 | 10 | 2 |
| Vomiting | 10 | 2 |
Showing 5 of 10 adverse events.
Which publications cover MELAS Syndrome? (22)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Magnetic resonance spectroscopy in MELAS syndrome: correlation with CSF and plasma metabolite levels and change after glutamine supplementation. | Neuroradiology | 2024 | Yes |
| High-dose oral glutamine supplementation reduces elevated glutamate levels in cerebrospinal fluid in patients with mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes syndrome. | European journal of neurology | 2023 | Yes |
| Renal manifestations in adults with mitochondrial disease from the mtDNA m.3243A>G pathogenic variant. | Nefrologia | 2023 | Yes |
| L-arginine effects on cerebrovascular reactivity, perfusion and neurovascular coupling in MELAS (mitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes) syndrome. | PloS one | 2020 | Yes |
| Taurine supplementation for prevention of stroke-like episodes in MELAS: a multicentre, open-label, 52-week phase III trial. | Journal of neurology, neurosurgery, and psychiatry | 2019 | Yes |
Showing 5 of 22 publications.
Prevalence (15)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| MELAS | Point prevalence | 1-9 / 100 000 | Finland | Not stated | Unspecified |
| MELAS | Point prevalence | Unknown | Worldwide | Not stated | Exact |
| MELAS | Point prevalence | 1-9 / 100 000 | Finland | Not stated | Exact |
| MELAS | Point prevalence | 1-9 / 1 000 000 | Europe | Not stated | Exact |
| MELAS | Point prevalence | 1-9 / 1 000 000 | Europe | Not stated | Exact |
Showing 5 of 15 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| MELAS | 550 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| MELAS | 550 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.