Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Mitochondrial Diseases
Open-data reference.
8 US clinical trials · 5 currently recruiting
Active & Recent Trials
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
Columbia University
NCT01694940
Transgenerational Metabolic-Immune Biomarkers of Neurological and Neurodevelopmental Disorders
Southwest Autism Research & Resource Center
NCT04322734
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
Khondrion
NCT06451757
Applying pGz in Mitochondrial Disease
Children's Hospital of Philadelphia
NCT05569122
Validation of Oxygen Nanosensor in Mitochondrial Myopathy
Children's Hospital of Philadelphia
NCT04086329
Heart Rate Variability in Response to Metformin Challenge
Woodinville Psychiatric Associates
NCT02500628
Phase III Trial of Coenzyme Q10 in Mitochondrial Disease
University of Florida
NCT00432744
Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).
Children's Hospital of Philadelphia
NCT06051448
Phase Distribution
| Phase | Trial count |
|---|---|
| Early Phase 1 | 3 |
| Phase 2 | 1 |
| Phase 3 | 2 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Mitochondrial Diseases Trial Landscape
ClinicalTrials.gov lists 8 US studies indexed under Mitochondrial Diseases, and 5 of those are currently open to recruitment — roughly 63% 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 Diseases shows 2 late-stage studies (Phase 3 and Phase 4 combined) alongside 4 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 Diseases is led by Children's Hospital of Philadelphia with 3 indexed trials, alongside 5 other organizations in the top contributor list. The list on this page surfaces up to 8 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 Diseases?
PlainTrial tracks 8 US clinical trials for Mitochondrial Diseases, of which 5 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Mitochondrial Diseases?
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 · 2024 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.