Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Mitochondrial Disease clinical trials
Every US clinical trial registered for Mitochondrial Disease — phase mix, recruiting status, and the sponsors running them, straight from the NIH ClinicalTrials.gov registry.
6 US clinical trials · 3 currently recruiting
The research picture
Mitochondrial Disease has 6 registered US clinical trials, 3 of them open to new participants right now — about 50% of the total.
- 3
- recruiting participants now
- 50%
- of trials open to enrollment
- 0
- in Phase 3–4 (later-stage)
- 1
- top sponsor: Medical Neurogenetics
Counts reflect the public ClinicalTrials.gov registry as last mirrored by PlainTrial. Status and phase are reported by each study's sponsor. This is reference information, not medical advice.
Active & Recent Trials
Acute Infection in Mitochondrial Disease: Metabolism, Infection and Immunity
National Human Genome Research Institute (NHGRI)
NCT04419870
Validation of Nanosensor Oxygen Measurement
Children's Hospital of Philadelphia
NCT06819683
Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels
Michio Hirano, MD
NCT05241262
Mitochondrial Dysfunction in Autism Spectrum Disorder
University of Arkansas
NCT02000284
Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan
Massachusetts General Hospital
NCT02985710
Mechanisms of Mitochondrial Defects in Gulf War Syndrome
Medical Neurogenetics
NCT01264471
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 2 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Mitochondrial Disease Trial Landscape
ClinicalTrials.gov lists 6 US studies indexed under Mitochondrial Disease, and 3 of those are currently open to recruitment — roughly 50% of the total volume on the registry. That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
The phase distribution for Mitochondrial Disease shows 0 late-stage studies (Phase 3 and Phase 4 combined) alongside 2 earlier-phase entries (Phase 1 through Phase 2). Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
Top sponsor activity for Mitochondrial Disease is led by Medical Neurogenetics with 1 indexed trial, alongside 5 other organizations in the top contributor list. The list on this page surfaces up to 6 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation — eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for Mitochondrial Disease?
PlainTrial tracks 6 US clinical trials for Mitochondrial Disease, of which 3 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Mitochondrial Disease?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology — how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.