Condition (MeSH)
Mitochondrial Encephalomyopathies
ClinicalTrials.gov lists 42 trials for Mitochondrial Encephalomyopathies or a narrower condition beneath it. Khondrion BV has the most among industry and government lead sponsors, with 6.
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Trials per phase
1 more section with rows
9 of the 42 trials are tagged with Mitochondrial Encephalomyopathies itself; the rest are tagged with a narrower condition beneath it.
When did Mitochondrial Encephalomyopathies 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: 6
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 | 2 |
| 2023 | 6 |
| 2022 | 1 |
| 2021 | 6 |
| 2020 | 2 |
| 2019 | 2 |
| 2017 | 2 |
| 2016 | 1 |
Showing 10 of 18 earlier years.
No forecast is made for this condition: it averages 2.6 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 4 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 Mitochondrial Encephalomyopathies 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 | 15 |
| Phase 2/3 | 1 |
| Phase 3 | 2 |
| Phase 4 | 1 |
| Not applicable | 4 |
| Observational | 9 |
| Expanded access | 1 |
How many Mitochondrial Encephalomyopathies 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 | 8 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 1 |
| Completed | 22 |
Showing 5 of 9 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Mitochondrial Encephalomyopathies trials? (6)
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 |
| Amgen | 2 |
| Abliva AB | 1 |
Showing 5 of 6 sponsors.
Which drugs are being tested for Mitochondrial Encephalomyopathies? (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 |
| (Cystagon) Cysteamine Bitartrate | 2 |
| KL1333 | 2 |
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 Mitochondrial Encephalomyopathies 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 |
| Vomiting | 15 | 4 |
| Seizure | 14 | 2 |
| Acute respiratory failure | 12 | 2 |
| COVID-19 | 10 | 2 |
Showing 5 of 10 adverse events.
Which publications cover Mitochondrial Encephalomyopathies? (30)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| HPDL Variant Type Correlates With Clinical Disease Onset and Severity. | Annals of clinical and translational neurology | 2025 | Yes |
| MtDNA-maintenance defects: syndromes and genes. | Journal of inherited metabolic disease | 2017 | Yes |
| Clinical and biochemical improvements in a patient with MNGIE following enzyme replacement. | Neurology | 2013 | Yes |
| Course and management of allogeneic stem cell transplantation in patients with mitochondrial neurogastrointestinal encephalomyopathy. | Journal of neurology | 2012 | Yes |
| Limited dCTP availability accounts for mitochondrial DNA depletion in mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). | PLoS genetics | 2011 | Yes |
Showing 5 of 30 publications.
Sub-conditions in MeSH (2)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence and rare-disease identity.