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ClinicalTrials.gov public records Last synced May 21, 2026, 11:10 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Mitochondrial Myopathy Clear all
Terminated Phase 2 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 2, 2025 · Synced May 21, 2026, 11:10 PM EDT
Completed Phase 1 Interventional
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 May 21, 2026, 11:10 PM EDT
Recruiting Phase 3 Interventional
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 – 2028
U.S. locations
3
States / cities
Boston, Massachusetts • Cleveland, Ohio • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced May 21, 2026, 11:10 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 3, 2026 · Synced May 21, 2026, 11:10 PM EDT
Completed No phase listed Observational
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 May 21, 2026, 11:10 PM EDT
Completed No phase listed Observational
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 11, 2024 · Synced May 21, 2026, 11:10 PM EDT
No Longer Available No phase listed Expanded access
View directory record Official record
Conditions
Mitochondrial Diseases
Interventions
EPI-743
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
1 Year and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
16
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 12 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2024 · Synced May 21, 2026, 11:10 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers
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 May 21, 2026, 11:10 PM EDT
Completed No phase listed Observational
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 10, 2019 · Synced May 21, 2026, 11:10 PM EDT
Terminated Phase 2Phase 3 Interventional Results available
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 May 21, 2026, 11:10 PM EDT
Terminated Phase 2 Interventional Results available
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 May 21, 2026, 11:10 PM EDT
No Longer Available No phase listed Expanded access
View directory record Official record
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
1 Month and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 9, 2021 · Synced May 21, 2026, 11:10 PM EDT
Completed No phase listed Observational
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 May 21, 2026, 11:10 PM EDT
Active, not recruiting Phase 2 Interventional
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 May 21, 2026, 11:10 PM EDT
Completed Phase 2 Interventional Results available
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 May 21, 2026, 11:10 PM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
Interventions
Glycerol Tributyrate
Drug
Lead sponsor
George Washington University
Other
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 8, 2026 · Synced May 21, 2026, 11:10 PM EDT
Not listed Phase 2 Interventional
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 23, 2005 · Synced May 21, 2026, 11:10 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers
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 May 21, 2026, 11:10 PM EDT
Completed Phase 3 Interventional Results available
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 May 21, 2026, 11:10 PM EDT
Completed Phase 2 Interventional Results available
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 May 21, 2026, 11:10 PM EDT
Completed Phase 3 Interventional
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 23, 2025 · Synced May 21, 2026, 11:10 PM EDT
Withdrawn No phase listed Observational
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 May 21, 2026, 11:10 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Mitochondrial Disease, Neurology, Myopathy
Interventions
EPI-743, Placebo
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 11 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 13, 2021 · Synced May 21, 2026, 11:10 PM EDT
Withdrawn Phase 2 Interventional
View directory record Official record
Conditions
Chronic Fatigue Syndrome
Interventions
Nutraceutical supplements, Placebo
Drug
Lead sponsor
Columbia University
Other
Eligibility
25 Years to 55 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 12, 2015 · Synced May 21, 2026, 11:10 PM EDT