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mitochondrial diseases

Total Trials
5
Recruiting Now
5
Trial Phases
Phase 2, Phase 3, Phase 1

ClinicalMetric tracks all active clinical trials for mitochondrial diseases sourced from ClinicalTrials.gov. Research in this area spans early-phase safety investigations through large Phase 3 confirmatory trials, conducted by NIH-funded academic centers, independent investigators, and pharmaceutical sponsors worldwide. Each listing is updated daily as new studies open enrollment and existing ones reach capacity or complete follow-up.

Trial listings include the NCT identifier, current recruitment status, phase classification, primary and secondary endpoints, estimated enrollment size, participating countries, and direct links to the full protocol on ClinicalTrials.gov. Eligibility criteria — including age range, disease stage, and prior treatment requirements — are documented for every study to help patients and clinicians assess suitability before contacting a trial site.

Frequently Asked Questions — mitochondrial diseases Clinical Trials

How many clinical trials are currently recruiting for mitochondrial diseases?
ClinicalMetric currently tracks 5 actively recruiting clinical trials for mitochondrial diseases, sourced in real time from ClinicalTrials.gov. The total number of registered studies—including those not yet enrolling or in active follow-up—is 5. Trial availability changes daily as new studies open enrollment and existing ones reach capacity.
What trial phases are available for mitochondrial diseases?
mitochondrial diseases research spans Phase 1 (1 trial), Phase 2 (2 trials), Phase 3 (1 trial). Phase 1 studies evaluate safety and dosing in small groups, Phase 2 studies assess preliminary efficacy in 100–300 participants, and Phase 3 trials compare the new treatment against the standard of care in 300–3,000+ patients. Phase 4 post-approval studies monitor long-term outcomes in real-world populations.
How do I find out if I qualify for a mitochondrial diseases clinical trial?
Eligibility criteria for mitochondrial diseases trials vary by study and typically specify age range, disease stage or severity, prior treatment history, and specific diagnostic or laboratory parameters. Each listing on ClinicalMetric links to the full protocol on ClinicalTrials.gov, where inclusion and exclusion criteria are documented. Contact the sponsoring site's research coordinator directly to confirm your eligibility—your treating physician or specialist can also help identify the most appropriate trial based on your medical history and current treatment status.
Trial Phases
Phase 1
1
Phase 2
2
Phase 3
1
Top Sponsors
Minovia Therapeutics Ltd. 1 trial
Khondrion BV 1 trial
Heinrich-Heine University, Duesseldorf 1 trial
Children's Hospital of Philadelphia 1 trial
Kenneth Myers, MD 1 trial

Recruiting Clinical Trials

NCT06017869 Phase 2
Recruiting
Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS)
Enrollment
6 pts
Location
Israel
Sponsor
Minovia Therapeutics Ltd.
View Trial →
NCT06451757 Phase 3
Recruiting
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
Enrollment
220 pts
Location
United States, Denma...
Sponsor
Khondrion BV
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NCT05958706
Recruiting
Mitochondrial Substrate Utilization in the Diabetic Human Heart
Enrollment
500 pts
Location
Germany
Sponsor
Heinrich-Heine University, Due...
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NCT05569122 Phase 1
Recruiting
Applying pGz in Mitochondrial Disease
Enrollment
90 pts
Location
United States
Sponsor
Children's Hospital of Philade...
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NCT04802707 Phase 2
Recruiting
Deoxynucleosides Pyrimidines as Treatment for Mitochondrial Depletion Syndrome
Enrollment
200 pts
Location
Canada
Sponsor
Kenneth Myers, MD
View Trial →

Related Conditions

pearson syndrome (1) maternally inherited diabetes and deafness midd (1) mitochondrial encephalomyopathy lactic acidosis and stroke like episodes melas (1) mitochondrial dna trnaleu uur m 3243a g mutation (1) heart failure (1) type2diabetes (1) insulin resistance (1) diabetic cardiomyopathies (1) mitochondrial myopathies (1) mitochondrial encephalomyopathy (1) mitochondrial encephalopathy (1) mitochondrial dna depletion (1)
ClinicalMetric — Independent clinical trial intelligence platform. Not affiliated with NIH, ClinicalTrials.gov, the U.S. FDA, or any pharmaceutical company, hospital, or clinical research organization. Trial data is sourced from ClinicalTrials.gov for informational purposes only and does not constitute medical advice. Do not make any treatment, enrollment, or health decisions based solely on information found here — always consult a qualified healthcare professional. Full Disclaimer  ·  Last Reviewed: September 2026  ·  Data Methodology