Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Sep 30, 2026, 11:13 AM EDT

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Showing 49–71 of 71 matching trials from the live ClinicalTrials.gov search.
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
Conditions
Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes, Deletion and Duplication of Mitochondrial DNA
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2026
U.S. locations
16
States / cities
San Diego, California • Stanford, California • Aurora, Colorado + 12 more
Completed Phase 2 Interventional Results available

(-)- Epicatechin Becker Muscular Dystrophy

NCT03236662
Conditions
Becker Muscular Dystrophy
Interventions
(-)-Epicatechin
Drug
Lead sponsor
Craig McDonald, MD
Other
Eligibility
18 Years to 70 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Sacramento, California
Completed Early Phase 1 Interventional Accepts healthy volunteers

Nitric Oxide Production in MELAS Syndrome

NCT01339494
Conditions
MELAS Syndrome
Interventions
Arginine and citrulline supplementations
Dietary Supplement
Lead sponsor
Baylor College of Medicine
Other
Eligibility
3 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2016
U.S. locations
1
States / cities
Houston, Texas
Conditions
Thymidine Kinase 2 Deficiency
Interventions
doxecitine and doxribtimine
Drug
Lead sponsor
UCB BIOSCIENCES, Inc.
Industry
Eligibility
Not listed
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
New York, New York
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
Conditions
Mitochondrial Myopathies, Mitochondrial DNA Mutation, Mitochondrial Diseases, Chronic Progressive External Ophthalmoplegia With Myopathy, Kearns-Sayre Syndrome
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
15 Years to 80 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
Myopathy, HMG COA Reductase Inhibitor Adverse Reaction, Hypercholesterolemia
Interventions
ubiquinol, placebo, statin
Dietary Supplement · Drug
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
18 Years to 80 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Boston, Massachusetts
Recruiting No phase listed Observational Accepts healthy volunteers

Natural History Study of Mitochondrial Myopathy

NCT05250375
Conditions
Primary Mitochondrial Disease
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
0 Years to 100 Years
Enrollment
1,300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Primary Mitochondrial Myopathy
Interventions
elamipretide, placebo comparator, elamipretide open label treatment
Combination Product
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
16 Years to 80 Years
Enrollment
218 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
13
States / cities
La Jolla, California • Palo Alto, California • Aurora, Colorado + 9 more
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
Conditions
Blepharoptosis, Ptosis, Eyelid, Myasthenia Gravis, Stroke, Traumatic Brain Injury, Chronic Progressive External Ophthalmoplegia
Interventions
Magnetic Levator Prosthesis (MLP), Kinesiotape Frontalis Sling (KTFS)
Device
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
5 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Primary Mitochondrial Myopathy
Interventions
Bocidelpar, Placebo
Drug
Lead sponsor
Astellas Pharma Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
11
States / cities
La Jolla, California • Stanford, California • Aurora, Colorado + 7 more
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
Completed No phase listed Observational Accepts healthy volunteers

Oxidative Capacity and Exercise Tolerance in Ambulatory SMA

NCT02895789
Conditions
Spinal Muscular Atrophy Type 3, Mitochondrial Myopathy
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
8 Years to 55 Years
Enrollment
42 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2021
U.S. locations
1
States / cities
New York, New York
Conditions
MELAS Syndrome
Interventions
Idebenone, Placebo
Drug · Other
Lead sponsor
Michio Hirano
Other
Eligibility
8 Years to 65 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
New York, New York
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
Conditions
Mitochondrial Disease, Chronic Kidney Disease
Interventions
Probucol
Drug
Lead sponsor
RiboNova Inc.
Industry
Eligibility
18 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
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
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
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
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
Completed Early Phase 1 Interventional

Ketones & Mitochondrial Heteroplasmy

NCT01252979
Conditions
MELAS Syndrome, Mitochondrial Diseases
Interventions
Medium-Chain Triglycerides
Dietary Supplement
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
Not listed
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Houston, Texas