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Showing 1–24 of 432 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Mitochondrial Disease Clear all
Completed No phase listed Observational Results available

Neurology Measures in FA Children

NCT03418740
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Years to 18 Years
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
3
States / cities
Los Angeles, California • Gainesville, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 15, 2025 · Synced Sep 14, 2026, 6:03 AM EDT
Completed Phase 1 Interventional Results available

Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia

NCT01898884
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
VP 20629, Placebo
Drug
Lead sponsor
Shire
Industry
Eligibility
18 Years to 45 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
5
States / cities
Los Angeles, California • Tampa, Florida • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 1, 2021 · Synced Sep 14, 2026, 6:03 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 14, 2026, 6:03 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Natural History Study of Mitochondrial Myopathy

NCT05250375
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 9, 2025 · Synced Sep 14, 2026, 6:03 AM EDT
Terminated Not applicable Interventional Accepts healthy volunteers

Mitochondrial Function in Patients With Severe Liver Disease

NCT02457702
View directory record Official record
Conditions
Hepatic Insufficiency, Hypoalbuminemia
Interventions
Orally administered labeled glycerol
Other
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
21 Years to 70 Years
Enrollment
2 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2021
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 28, 2022 · Synced Sep 14, 2026, 6:03 AM EDT
Not yet 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
Up to 75 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
6
States / cities
San Diego, California • Aurora, Colorado • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Sep 14, 2026, 6:03 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Responses to Influenza Vaccine in Patients With Mitochondrial Disorders (MELAS)

NCT01831934
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Fluzone®
Biological
Lead sponsor
Stanford University
Other
Eligibility
13 Years to 65 Years
Enrollment
22 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated May 29, 2017 · Synced Sep 14, 2026, 6:03 AM EDT
Completed Phase 2 Interventional Results available

Effects of Acipimox on Mitochondrial Function in Obesity

NCT01488409
View directory record Official record
Conditions
Abdominal Obesity, Insulin Resistance, Hypertriglyceridemia
Interventions
Acipimox, Placebo
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 55 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 29, 2016 · Synced Sep 14, 2026, 6:03 AM EDT
Completed Phase 2 Interventional Results available

Mitochondria and Chronic Kidney Disease

NCT03177798
View directory record Official record
Conditions
Hemodialysis-Induced Symptom, Mitochondrial Diseases
Interventions
Icatibant, Placebo
Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years to 78 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 28, 2019 · Synced Sep 14, 2026, 6:03 AM EDT
Completed Phase 2 Interventional Results available

(+) Epicatechin to Treat Friedreich's Ataxia

NCT02660112
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
(+)-Epicatechin
Drug
Lead sponsor
Ralitza Gavrilova
Other
Eligibility
10 Years to 50 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 4, 2019 · Synced Sep 14, 2026, 6:03 AM EDT
Completed Phase 2 Interventional Results available

A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy

NCT02693119
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
elamipretide (MTP-131) 1% topical ophthalmic solution, Vehicle topical ophthalmic solution
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Pasadena, California
Source: ClinicalTrials.gov public record
Updated Nov 29, 2021 · Synced Sep 14, 2026, 6:03 AM EDT
Completed Not applicable Interventional

Studies of the Ocular Complications of AIDS (SOCA) CMV Retinitis Trial: Foscarnet-Ganciclovir Component

NCT00000665
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Foscarnet sodium, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
9
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2011 · Synced Sep 14, 2026, 6:03 AM 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 2, 2012 · Synced Sep 14, 2026, 6:03 AM EDT
Recruiting No phase listed Observational

Natural History Study of Patients with HPDL Mutations

NCT05848271
View directory record Official record
Conditions
Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia, Spastic Paraplegia, White Matter Disease, Neonatal Encephalopathy, Mutation, Genetic Disease
Interventions
Patient Registry, Dry blood spots sampling
Other
Lead sponsor
University of California, San Diego
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 29, 2025 · Synced Sep 14, 2026, 6:03 AM EDT
Suspended No phase listed Observational

Longitudinal Study of Mitochondrial Hepatopathies

NCT01148550
View directory record Official record
Conditions
Acute Liver Failure, Mitochondrial Diseases, End Stage Liver Disease, Respiratory Chain Deficiencies, Mitochondrial, Disorder of Fatty Acid Oxidation
Interventions
Not listed
Lead sponsor
Arbor Research Collaborative for Health
Other
Eligibility
Up to 18 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2029
U.S. locations
15
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Sep 14, 2026, 6:03 AM EDT
Completed Phase 1 Interventional

Phase 1 Trial of Idebenone to Treat Patients With Friedreich's Ataxia

NCT00078481
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Idebenone
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 14, 2026, 6:03 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Biomarkers in Friedreich's Ataxia

NCT02497534
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
6 Years to 70 Years
Enrollment
203 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2030
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Sep 14, 2026, 6:03 AM EDT
Completed Phase 1Phase 2 Interventional Results available

FRDA Investigator Initiated Study (IIS) With Elamipretide

NCT05168774
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Elamipretide
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
16 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 11, 2025 · Synced Sep 14, 2026, 6:03 AM EDT
Not listed Phase 2 Interventional

The Synergy Trial: Methylphenidate Plus a CFS-Specific Nutrient Formula as a Treatment for Chronic Fatigue Syndrome

NCT01966276
View directory record Official record
Conditions
Chronic Fatigue Syndrome (CFS), Myalgic Encephalomyelitis (ME)
Interventions
Methyl-P plus Nutrient Formula, Methyl-P plus Nutrient matched placebos
Drug
Lead sponsor
K-PAX Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 59 Years
Enrollment
134 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
4
States / cities
Stanford, California • Fort Lauderdale, Florida • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2014 · Synced Sep 14, 2026, 6:03 AM EDT
Terminated Phase 2 Interventional Results available

The Effect of Nicotinamide Riboside on Skeletal Muscle Function in Heart Failure Subjects

NCT03565328
View directory record Official record
Conditions
Heart Failure
Interventions
Nicotinamide Riboside
Dietary Supplement
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 75 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 27, 2020 · Synced Sep 14, 2026, 6:03 AM EDT
Completed Not applicable Interventional Results available

High-Intensity Exercise to Attenuate Limitations and Train Habits in Older Adults With HIV

NCT04550676
View directory record Official record
Conditions
Hiv, Fatigue, Mobility Limitation
Interventions
HIIT (intervention), CME (active control), Coaching, Control text message
Behavioral
Lead sponsor
University of Colorado, Denver
Other
Eligibility
50 Years and older
Enrollment
118 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
3
States / cities
Aurora, Colorado • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Sep 14, 2026, 6:03 AM 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 15, 2026 · Synced Sep 14, 2026, 6:03 AM EDT
Completed Phase 2 Interventional Results available

Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder

NCT01379625
View directory record Official record
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency, Mitochondrial Trifunctional Protein (TFP) Deficiency, Long-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) Deficiency
Interventions
Triheptanoin
Drug
Lead sponsor
Oregon Health and Science University
Other
Eligibility
7 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
2
States / cities
Portland, Oregon • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 22, 2017 · Synced Sep 14, 2026, 6:03 AM EDT
No Longer Available No phase listed Expanded access

EPI-743 for Mitochondrial Respiratory Chain Diseases

NCT01370447
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 Sep 14, 2026, 6:03 AM EDT