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Mitochondrial Diseases clinical trials recruiting in the U.S.

14 studies for Mitochondrial Diseases are recruiting participants at sites across the United States.

Mitochondria make energy for your cells. Diseases can affect mitochondria, often causing nerve and muscle problems. Find out more.

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Recently updated studies

The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism

RecruitingObservational studyHealthy volunteers welcome
4 Weeks – 115 Years · All sexes · Updated Sep 29, 2026 · NCT01780168

Natural History Study of Mitochondrial Myopathy

RecruitingObservational studyHealthy volunteers welcome
Ages 0 to 100 · All sexes · Updated Sep 17, 2026 · NCT05250375

Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease

RecruitingPhase 2
Ages 18 and older · All sexes · Updated Sep 16, 2026 · NCT07514338

Acute Infection in Mitochondrial Disease: Metabolism, Infection and Immunity

RecruitingObservational studyHealthy volunteers welcome
2 Months – 115 Years · All sexes · Updated Sep 10, 2026 · NCT04419870

KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases

RecruitingPhase 3
Ages 18 and older · All sexes · Updated Jun 29, 2026 · NCT06451757

Coenzyme Q10 for Gulf War Illness: A Replication Study

RecruitingPhase 3
Ages 50 and older · All sexes · Updated May 1, 2026 · NCT06515184

Transgenerational Metabolic-Immune Biomarkers of Neurological and Neurodevelopmental Disorders

RecruitingObservational studyHealthy volunteers welcome
Up to age 18 · All sexes · Updated Apr 23, 2026 · NCT04322734

Validation of Oxygen Nanosensor in Mitochondrial Myopathy

RecruitingPhase 1Healthy volunteers welcome
Ages 18 to 65 · All sexes · Updated Feb 18, 2026 · NCT04086329

Validation of Nanosensor Oxygen Measurement

RecruitingPhase 1Healthy volunteers welcome
Ages 18 to 65 · All sexes · Updated Feb 17, 2026 · NCT06819683

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

RecruitingObservational studyHealthy volunteers welcome
All ages · All sexes · Updated Feb 4, 2026 · NCT01694940

Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels

RecruitingPhase 1
Ages 18 to 80 · All sexes · Updated Jan 23, 2026 · NCT05241262

CoQ10 and Exercise for Mitochondrial Dysfunction in Advance Kidney Disease

RecruitingPhase 3
Ages 18 to 75 · All sexes · Updated Jan 2, 2026 · NCT05422534

Applying pGz in Mitochondrial Disease

RecruitingPhase 1Healthy volunteers welcome
Ages 10 to 60 · All sexes · Updated Nov 10, 2025 · NCT05569122

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

RecruitingObservational study
All ages · All sexes · Updated May 29, 2025 · NCT01793168

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.