Condition (MeSH)
Mitochondrial Myopathies
ClinicalTrials.gov lists 78 trials for Mitochondrial Myopathies 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 (log scale)
1 more section with rows
41 of the 78 trials are tagged with Mitochondrial Myopathies itself; the rest are tagged with a narrower condition beneath it.
When did Mitochondrial Myopathies trials start? (21)
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: 12
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) | 4 |
| 2025 | 4 |
| 2024 | 3 |
| 2023 | 12 |
| 2022 | 6 |
| 2021 | 8 |
| 2020 | 4 |
| 2019 | 3 |
| 2018 | 2 |
| 2017 | 4 |
Showing 10 of 21 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 5 trials starting in 2026 and about 4 in 2027, the average of the 24 months through September, counted as registered and carried forward. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.
Prediction, not a registry value: median of 36 months from start to primary completion across 13 trials still running at the 2026-10-09 snapshot; registered median of 18 months across 46 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Mitochondrial Myopathies trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 11 |
| Phase 1/2 | 3 |
| Phase 2 | 21 |
| Phase 2/3 | 1 |
| Phase 3 | 3 |
| Phase 4 | 1 |
| Not applicable | 16 |
| Observational | 21 |
| Expanded access | 1 |
How many Mitochondrial Myopathies 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 | 18 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 2 |
| Completed | 44 |
Showing 5 of 9 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Mitochondrial Myopathies trials? (7)
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 |
| Stealth BioTherapeutics Inc. | 4 |
| PTC Therapeutics | 3 |
| Tisento Therapeutics | 3 |
| Amgen | 2 |
Showing 5 of 7 sponsors.
Which drugs are being tested for Mitochondrial Myopathies? (7)
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 |
| Elamipretide | 3 |
| IW-6463 | 3 |
| Nicotinamide Riboside | 2 |
Showing 5 of 7 drugs.
Interventions as named in the registry (8)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| ER Niacin | 2 |
| TTI-0102 | 1 |
| N Acetylcysteine | 1 |
| Lumason® contrast agent | 1 |
| Intra-arterial delivery of autologous MABs | 1 |
Showing 5 of 8 interventions.
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 Myopathies 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 Myopathies? (60)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| RePOWER: An International, Prospective, Non-Interventional Registry of Patients With Primary Mitochondrial Myopathy. | Clinical genetics | 2026 | Yes |
| Pyrimidine Nucleos(t)ide Therapy in Patients With Thymidine Kinase 2 Deficiency: A Multicenter Retrospective Chart Review Study. | Neurology | 2025 | Yes |
| Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial. | Orphanet journal of rare diseases | 2024 | Yes |
| Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial. | Neurology | 2023 | Yes |
| Advances in Thymidine Kinase 2 Deficiency: Clinical Aspects, Translational Progress, and Emerging Therapies. | Journal of neuromuscular diseases | 2022 | Yes |
Showing 5 of 60 publications.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Mitochondrial myopathy | 206966 | Exact | Orphadata Science knowledge files, Product 1 |
| Mitochondrial myopathy | 206966 | Exact | Orphanet Rare Disease Ontology (ORDO) |
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.