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ClinicalTrials.gov public records Last synced Oct 24, 2026, 4:03 PM EDT

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Showing 1–24 of 72 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Mitochondrial Myopathy Clear all
Recruiting Not applicable Interventional Accepts healthy volunteers

Effect of Dietary Nitrate on Immobilization-induced Changes in Skeletal Muscle in Young Healthy Men

NCT07161973
View directory record Official record
Conditions
Mitochondrial Energetics, Dietary Nitrate, Disuse Atrophy (Muscle) of Lower Leg
Interventions
Beetroot Juice - Active, Placebo Beetroot Juice Without Nitrate
Dietary Supplement
Lead sponsor
Indiana University
Other
Eligibility
18 Years to 44 Years · Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Feb 23, 2026 · Synced Oct 24, 2026, 4:03 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Natural History Study - Mitochondrial Disease

NCT01532791
View directory record Official record
Conditions
MELAS or m.3243 A>G Mitochondrial DNA Mutation Carrier
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
4 Years and older
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 23, 2026 · Synced Oct 24, 2026, 4:03 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)

NCT01856868
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
(-)-epicatechin
Drug
Lead sponsor
Craig McDonald, MD
Other
Eligibility
18 Years to 60 Years · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Dec 22, 2021 · Synced Oct 24, 2026, 4:03 PM EDT
Completed No phase listed Observational

Defining 31Phosphorous Magnetic Resonance Spectroscopy Characteristics in Patients With Mitochondrial Myopathy

NCT01301235
View directory record Official record
Conditions
Mitochondrial Disease
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 30, 2012 · Synced Oct 24, 2026, 4:03 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers

Coenzyme Q10 in Older Athletes Treated With Statin Medications

NCT01026311
View directory record Official record
Conditions
Muscle Weakness, Myalgia, Side Effects of Statins
Interventions
Coenzyme q 10, placebo
Drug
Lead sponsor
Richard Deichmann, MD
Other
Eligibility
50 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
1
States / cities
New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated May 3, 2012 · Synced Oct 24, 2026, 4:03 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

Validation of Nanosensor Oxygen Measurement

NCT06819683
View directory record Official record
Conditions
MItochondrial Myopathies, Mitochondrial Disease
Interventions
Nanosensor
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years to 65 Years
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 7, 2026 · Synced Oct 24, 2026, 4:03 PM EDT
Completed Phase 3 Interventional Results available

A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

NCT05218655
View directory record Official record
Conditions
Inherited Mitochondrial Disease
Interventions
Vatiquinone
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
101 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
14
States / cities
La Jolla, California • Stanford, California • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Dec 22, 2025 · Synced Oct 24, 2026, 4:03 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy

NCT02367014
View directory record Official record
Conditions
Mitochondrial Myopathy
Interventions
elamipretide (low dose), elamipretide (intermediate dose), elamipretide (high dose), Placebo
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
16 Years to 65 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
4
States / cities
San Diego, California • Boston, Massachusetts • Akron, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2019 · Synced Oct 24, 2026, 4:03 PM EDT
Terminated Phase 2Phase 3 Interventional Results available

A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

NCT04378075
View directory record Official record
Conditions
Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome, Mitochondrial Encephalopathy (MELAS), Pontocerebellar Hypoplasia Type 6 (PCH6), Alpers Disease, Alpers Syndrome
Interventions
Vatiquinone, Placebo
Drug · Other
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Up to 20 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
12
States / cities
San Diego, California • Stanford, California • New Haven, Connecticut + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 31, 2026 · Synced Oct 24, 2026, 4:03 PM EDT
Completed Phase 2 Interventional Results available

Heart Rate Variability in Response to Metformin Challenge

NCT02500628
View directory record Official record
Conditions
Fibromyalgia, Mitochondrial Diseases, Movement Disorders, Diabetes Mellitus, Type 2
Interventions
Metformin
Drug
Lead sponsor
Woodinville Psychiatric Associates
Other
Eligibility
18 Years and older
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Woodinville, Washington
Source: ClinicalTrials.gov public record
Updated Jan 3, 2018 · Synced Oct 24, 2026, 4:03 PM EDT
Terminated Phase 2 Interventional Results available

Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)

NCT04475549
View directory record Official record
Conditions
MELAS
Interventions
IW-6463 Tablets
Drug
Lead sponsor
Tisento Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Baltimore, Maryland • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 29, 2024 · Synced Oct 24, 2026, 4:03 PM EDT
Completed No phase listed Observational

An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)

NCT03048617
View directory record Official record
Conditions
Primary Mitochondrial Disease
Interventions
Not listed
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
16 Years to 80 Years
Enrollment
215 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
13
States / cities
La Jolla, California • Palo Alto, California • Aurora, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 11, 2019 · Synced Oct 24, 2026, 4:03 PM EDT
Recruiting No phase listed Observational

Patterns of Neurodevelopmental Disorders

NCT06213090
View directory record Official record
Conditions
Neurodevelopmental Disorders, Autism Spectrum Disorder, Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal Infection, Pediatric Acute-Onset Neuropsychiatric Syndrome, Down Syndrome, Epilepsy, Mitochondrial Encephalomyopathies, Cerebral Folate Deficiency
Interventions
Not listed
Lead sponsor
Richard Frye
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 24, 2026, 4:03 PM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study

NCT02805790
View directory record Official record
Conditions
Primary Mitochondrial Disease
Interventions
Elamipretide, Placebo
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
16 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
4
States / cities
San Diego, California • Boston, Massachusetts • Akron, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 17, 2020 · Synced Oct 24, 2026, 4:03 PM EDT
Completed No phase listed Observational

A Retrospective Study of Subjects With Thymidine Kinase 2 Deficiency

NCT05017818
View directory record Official record
Conditions
Thymidine Kinase 2 Deficiency
Interventions
Observational retrospective data
Other
Lead sponsor
Zogenix MDS, Inc.
Industry
Eligibility
Not listed
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
4
States / cities
Los Angeles, California • Indianapolis, Indiana • Akron, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2023 · Synced Oct 24, 2026, 4:03 PM EDT
Completed No phase listed Observational

Study of the Metabolism of Pyruvate and Related Problems in Patients With Lactic Acidemia

NCT00004353
View directory record Official record
Conditions
Mitochondrial Myopathy, MELAS Syndrome, Lactic Acidosis
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1978
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Jul 10, 2008 · Synced Oct 24, 2026, 4:03 PM EDT
Terminated Not applicable Interventional

Vitamin E Treatment for Long-Chain 3-Hydroxyacyl Coenzyme A (CoA) Dehydrogenase (LCHAD) Associated Neuropathy

NCT00840112
View directory record Official record
Conditions
Peripheral Neuropathy, Mitochondrial Trifunctional Protein Deficiency
Interventions
Vitamin E supplement
Dietary Supplement
Lead sponsor
Oregon Health and Science University
Other
Eligibility
7 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 21, 2015 · Synced Oct 24, 2026, 4:03 PM EDT
Not listed Phase 2 Interventional

Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome

NCT00068913
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Dichloroacetate
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
6 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 24, 2026, 4:03 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Nicotinamide Riboside on Mitochondrial Function in Li-Fraumeni Syndrome

NCT03789175
View directory record Official record
Conditions
Cancer, Skin Fibroblasts, Muscle Weakness
Interventions
Nicotinamide Riboside (NR)
Dietary Supplement
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 70 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 8, 2020 · Synced Oct 24, 2026, 4:03 PM EDT
Not listed Phase 2 Interventional

The Effects of Exercise Versus Inactivity on People With Mitochondrial Muscle Disease

NCT00457314
View directory record Official record
Conditions
Mitochondrial Myopathy
Interventions
Exercise
Behavioral
Lead sponsor
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
NIH
Eligibility
18 Years to 65 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated May 19, 2009 · Synced Oct 24, 2026, 4:03 PM EDT
Recruiting Phase 3 Interventional

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

NCT06451757
View directory record Official record
Conditions
Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS), Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
Interventions
Sonlicromanol, Placebo
Drug
Lead sponsor
Khondrion BV
Industry
Eligibility
18 Years and older
Enrollment
220 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
3
States / cities
Boston, Massachusetts • Cleveland, Ohio • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 14, 2026 · Synced Oct 24, 2026, 4:03 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

Validation of Oxygen Nanosensor in Mitochondrial Myopathy

NCT04086329
View directory record Official record
Conditions
Mitochondrial Myopathies, Mitochondrial Diseases
Interventions
Nanosensor
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 18, 2026 · Synced Oct 24, 2026, 4:03 PM EDT
Recruiting Not applicable Interventional

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

NCT07478172
View directory record Official record
Conditions
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
Interventions
Whole-body Electrical Muscle Stimulation Exercise
Device
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Oct 24, 2026, 4:03 PM EDT
Completed Phase 3 Interventional

Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)

NCT05162768
View directory record Official record
Conditions
Mitochondrial Myopathies, Mitochondrial Pathology, Mitochondrial DNA Mutation, Mitochondrial Diseases, Mitochondrial DNA Deletion, Mitochondrial DNA Depletion, Mitochondrial Metabolism Defect, Mitochondrial Complex I Deficiency
Interventions
Elamipretide, Placebo
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
102 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
9
States / cities
San Diego, California • Atlanta, Georgia • Boston, Massachusetts + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 24, 2025 · Synced Oct 24, 2026, 4:03 PM EDT