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ClinicalTrials.gov public records Last synced Sep 16, 2026, 10:44 AM EDT

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Showing 1–24 of 71 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Mitochondrial Myopathies Clear all
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 16, 2020 · Synced Sep 16, 2026, 10:44 AM EDT
Recruiting No phase listed Observational

Systemic Biomarkers of Brain Injury From Hyperammonemia

NCT04602325
View directory record Official record
Conditions
Urea Cycle Disorder, Organic Acidemia, Maple Syrup Urine Disease, Glutaric Acidemia I, Fatty Acid Oxidation Disorder, Hypoxic-Ischemic Encephalopathy
Interventions
Not listed
Lead sponsor
Children's National Research Institute
Other
Eligibility
7 Years to 18 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 6, 2024 · Synced Sep 16, 2026, 10:44 AM 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 7, 2020 · Synced Sep 16, 2026, 10:44 AM 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 22, 2026 · Synced Sep 16, 2026, 10:44 AM EDT
Completed No phase listed Observational

Mitochondrial Video Assessment Source Material Study

NCT06180421
View directory record Official record
Conditions
PolG, Primary Mitochondrial Myopathy
Interventions
Not listed
Lead sponsor
The Emmes Company, LLC
Industry
Eligibility
16 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Kingston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 13, 2024 · Synced Sep 16, 2026, 10:44 AM EDT
Active, not recruiting Phase 2 Interventional

A Study to Evaluate Vitamin B3 Derivative to Treat Mitochondrial Myopathy

NCT05590468
View directory record Official record
Conditions
Mitochondrial Myopathy
Interventions
Nicotinamide Riboside, Placebo
Dietary Supplement · Drug
Lead sponsor
Ralitza Gavrilova
Other
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced Sep 16, 2026, 10:44 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Tissue Sample Study for Mitochondrial Disorders

NCT01803906
View directory record Official record
Conditions
Mitochondrial Disorders, Mitochondrial Disease, Melas, Kearns Sayer, NARP, MNGIE, LHON, Mitochondrial Depletion Syndrome, Leigh's Disease
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
6,900 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced Sep 16, 2026, 10:44 AM EDT
Active, not recruiting Phase 2 Interventional

An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)

NCT06961344
View directory record Official record
Conditions
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
Interventions
zagociguat 15mg
Drug
Lead sponsor
Tisento Therapeutics
Industry
Eligibility
Not listed
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
9
States / cities
La Jolla, California • Aurora, Colorado • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Sep 16, 2026, 10:44 AM 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
96 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 16, 2026 · Synced Sep 16, 2026, 10:44 AM EDT
Completed Phase 1Phase 2 Interventional

Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).

NCT06051448
View directory record Official record
Conditions
Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS, MERRF
Interventions
Promoting Resilience in Stress Management (PRISM), Clinical-focused narrative (CFN)
Behavioral
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 28, 2024 · Synced Sep 16, 2026, 10:44 AM EDT
Withdrawn No phase listed Observational

Therapy for Chronic Fatigue Syndrome: A Presumptive Mitochondrial Disorder

NCT01872351
View directory record Official record
Conditions
Chronic Fatigue Syndrome
Interventions
Nutraceuticals
Drug
Lead sponsor
Columbia University
Other
Eligibility
20 Years to 70 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 13, 2016 · Synced Sep 16, 2026, 10:44 AM 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 17, 2026 · Synced Sep 16, 2026, 10:44 AM EDT
Completed Phase 2 Interventional Results available

An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients

NCT04535609
View directory record Official record
Conditions
Primary Mitochondrial Myopathy
Interventions
Mavodelpar, Placebo
Drug
Lead sponsor
Reneo Pharma Ltd
Industry
Eligibility
18 Years and older
Enrollment
213 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
9
States / cities
La Jolla, California • Gainesville, Florida • Boston, Massachusetts + 6 more
Source: ClinicalTrials.gov public record
Updated May 7, 2024 · Synced Sep 16, 2026, 10:44 AM 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 30, 2026 · Synced Sep 16, 2026, 10:44 AM 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 29, 2012 · Synced Sep 16, 2026, 10:44 AM EDT
Completed Phase 1 Interventional

L-Citrulline Dose Finding Safety Study in MELAS

NCT03952234
View directory record Official record
Conditions
MELAS Syndrome
Interventions
L-Citrulline
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 25, 2024 · Synced Sep 16, 2026, 10:44 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Effects of Epicatechin on Statins for Mitochondrial Dysfunction and Impaired Exercise Capacity

NCT02490527
View directory record Official record
Conditions
Statin Intolerance
Interventions
Pure epicatechin capsules (50mg), Simvastatin (40mg), Placebo
Dietary Supplement · Drug · Other
Lead sponsor
University of California, San Diego
Other
Eligibility
30 Years to 75 Years
Enrollment
19 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2021
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Jul 30, 2026 · Synced Sep 16, 2026, 10:44 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Inherited Retinal Degenerative Disease Registry

NCT02435940
View directory record Official record
Conditions
Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
Interventions
Not listed
Lead sponsor
Foundation Fighting Blindness
Other
Eligibility
Not listed
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2037
U.S. locations
1
States / cities
Columbia, Maryland
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced Sep 16, 2026, 10:44 AM EDT
Completed Phase 2 Interventional Results available

Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease

NCT02023866
View directory record Official record
Conditions
Inherited Mitochondrial Disease, Including Leigh Syndrome
Interventions
Cysteamine Bitartrate
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
5
States / cities
San Diego, California • Stanford, California • Akron, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 26, 2024 · Synced Sep 16, 2026, 10:44 AM EDT
Terminated Phase 2 Interventional Results available

Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)

NCT02976038
View directory record Official record
Conditions
Primary Mitochondrial Disease
Interventions
elamipretide
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
16 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
4
States / cities
San Diego, California • Boston, Massachusetts • Akron, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 16, 2021 · Synced Sep 16, 2026, 10:44 AM 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 20, 2015 · Synced Sep 16, 2026, 10:44 AM 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 21, 2025 · Synced Sep 16, 2026, 10:44 AM EDT
Recruiting No phase listed Observational

Prospective Natural History Study of POLG Disease

NCT07775872
View directory record Official record
Conditions
PolG, Primary Mitochondrial Myopathy
Interventions
Not listed
Lead sponsor
The POLG Foundation
Other
Eligibility
Not listed
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
7
States / cities
San Diego, California • Aurora, Colorado • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Sep 16, 2026, 10:44 AM 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 26, 2019 · Synced Sep 16, 2026, 10:44 AM EDT