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ClinicalTrials.gov public records Last synced Oct 11, 2026, 9:56 AM EDT

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Showing 145–168 of 437 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Mitochondrial Disease Clear all
Recruiting No phase listed Observational

The International Registry for Leigh Syndrome

NCT03137355
View directory record Official record
Conditions
Leigh Syndrome, Leigh Disease, Leigh's Necrotizing Encephalopathy, Subacute Necrotizing Encephalomyopathy, Subacute Necrotizing Encephalomyelopathy
Interventions
Not listed
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
0 Days to 100 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2030
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Dec 1, 2023 · Synced Oct 11, 2026, 9:56 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Alpha-lipoic Acid/L-acetyl Carnitine for Progressive Supranuclear Palsy

NCT01537549
View directory record Official record
Conditions
Progressive Supranuclear Palsy
Interventions
alpha-lipoic acid and L-acetyl carnitine
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
40 Years to 75 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 30, 2017 · Synced Oct 11, 2026, 9:56 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

FA Clinical Outcome Measures

NCT03090789
View directory record Official record
Conditions
Friedreich Ataxia, Neuro-Degenerative Disease
Interventions
Not listed
Lead sponsor
Friedreich's Ataxia Research Alliance
Other
Eligibility
4 Years to 80 Years
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2030
U.S. locations
9
States / cities
Los Angeles, California • Denver, Colorado • Gainesville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 4, 2024 · Synced Oct 11, 2026, 9:56 AM EDT
Completed Phase 3 Interventional Results available

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation

NCT02652767
View directory record Official record
Conditions
Optic, Atrophy, Hereditary, Leber
Interventions
GS010, Sham Intravitreal Injection
Biological · Device
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
3
States / cities
Los Angeles, California • Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 11, 2026, 9:56 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 17, 2026 · Synced Oct 11, 2026, 9:56 AM EDT
Active, not recruiting Phase 2 Interventional

MIT-001 for Prevention of CCRT-Induced OM in HNSCC Patients

NCT04651634
View directory record Official record
Conditions
Head and Neck Squamous Cell Carcinoma, Oral Mucositis
Interventions
MIT-001 plus CCRT
Drug
Lead sponsor
MitoImmune Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
6
States / cities
Gilbert, Arizona • Los Angeles, California • Wichita, Kansas + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2025 · Synced Oct 11, 2026, 9:56 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

CO as a Stimulant for Mitochondrial Biogenesis in Human Cardiac Muscle

NCT01727167
View directory record Official record
Conditions
Cardiac Disease, Mitochondrial Biogenesis, Carbon Monoxide
Interventions
200ppm CO for one hour, Control
Drug · Other
Lead sponsor
John J Freiberger
Other
Eligibility
20 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 19, 2016 · Synced Oct 11, 2026, 9:56 AM EDT
Completed Phase 3 Interventional Results available

Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

NCT00697073
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
8 Years to 18 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
2
States / cities
Los Angeles, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 11, 2023 · Synced Oct 11, 2026, 9:56 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

NCT05515536
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Vatiquinone
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced Oct 11, 2026, 9:56 AM EDT
Withdrawn Phase 2 Interventional

Ph 2a Study of HU6 on Energy Metabolism, Muscle and Liver Substrate Metabolism, and Mitochondrial Function in Subjects Who Are Overweight or Obese With Type 2 Diabetes

NCT06104358
View directory record Official record
Conditions
Diabete Type 2, Obesity
Interventions
HU6
Drug
Lead sponsor
Rivus Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 70 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Jan 9, 2024 · Synced Oct 11, 2026, 9:56 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 29, 2019 · Synced Oct 11, 2026, 9:56 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Mitochondrial DNA Signatures of Poor Aerobic Exercise Trainability in Young Adults Born Preterm

NCT06334107
View directory record Official record
Conditions
Preterm Birth
Interventions
Exercise
Behavioral
Lead sponsor
Texas Tech University
Other
Eligibility
18 Years to 35 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2026
U.S. locations
1
States / cities
Lubbock, Texas
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Oct 11, 2026, 9:56 AM EDT
Completed No phase listed Observational Results available

Metabolic Effects of Non-Thymidine Analogue Anti-HIV Medications

NCT00433992
View directory record Official record
Conditions
HIV Infections
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 5, 2017 · Synced Oct 11, 2026, 9:56 AM 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 11, 2026, 9:56 AM EDT
Terminated Phase 1 Interventional

Hepatocyte Transplantation for Liver Based Metabolic Disorders

NCT01345578
View directory record Official record
Conditions
Metabolic Diseases
Interventions
human hepatocyte transplantation, Preparative Radiation Therapy
Drug · Radiation
Lead sponsor
Ira Fox
Other
Eligibility
Up to 21 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 7, 2022 · Synced Oct 11, 2026, 9:56 AM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia

NCT06560346
View directory record Official record
Conditions
Friedreich Ataxia, Rare Diseases
Interventions
Geneticlly confirmed disease causing FXN mutatuion, Healthy Control
Other
Lead sponsor
Friedreich's Ataxia Research Alliance
Other
Eligibility
4 Years to 21 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2028
U.S. locations
3
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 8, 2025 · Synced Oct 11, 2026, 9:56 AM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Effects of Roflumilast on Insulin and Blood Sugar Levels in Prediabetic Overweight and Obese Individuals

NCT01862029
View directory record Official record
Conditions
Obesity
Interventions
Roflumilast
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
30 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 4, 2018 · Synced Oct 11, 2026, 9:56 AM EDT
Terminated Not applicable Interventional Accepts healthy volunteers

Insulin and Muscle Fat Metabolism

NCT04759872
View directory record Official record
Conditions
Overweight and Obesity, Insulin Resistance, Metabolic Disease, Mitochondrial Metabolism, Sedentary Lifestyle
Interventions
Hyperinsulinemic-euglycemic Clamp
Other
Lead sponsor
Oregon State University
Other
Eligibility
30 Years to 55 Years
Enrollment
9 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Corvallis, Oregon
Source: ClinicalTrials.gov public record
Updated Jun 10, 2022 · Synced Oct 11, 2026, 9:56 AM EDT
Completed Phase 2 Interventional

A Phase II Dose-Ranging, Open-Labelled Trial of Foscarnet Salvage Therapy for AIDS Patients With Sight-Threatening CMV Retinitis Who Cannot Be Treated With Ganciclovir Due To Myelosuppression or Treatment Failure

NCT00000691
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Foscarnet sodium
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years to 65 Years
Enrollment
156 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1992
U.S. locations
11
States / cities
Los Angeles, California • San Francisco, California • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 3, 2021 · Synced Oct 11, 2026, 9:56 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 16, 2026 · Synced Oct 11, 2026, 9:56 AM EDT
Recruiting Early Phase 1 Interventional Accepts healthy volunteers

Imaging Mitochondrial Complex-I in Cocaine Use Disorder: a [F-18]BCPP-EF PET Study

NCT07773649
View directory record Official record
Conditions
Cocaine Use Disorder, Healthy Controls Group - Age and Sex-matched
Interventions
[F-18]BCPP-EF
Drug
Lead sponsor
Rajesh Narendran
Other
Eligibility
18 Years to 45 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2029
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Oct 11, 2026, 9:56 AM EDT
Completed No phase listed Observational

Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension

NCT02594917
View directory record Official record
Conditions
Iron-sulfur Cluster Deficiency, Pulmonary Hypertension, Friedreich Ataxia
Interventions
Right Heart Catheterization, Blood draw
Procedure · Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years to 60 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 22, 2023 · Synced Oct 11, 2026, 9:56 AM EDT
Recruiting Phase 1 Interventional

Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

NCT05302271
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
Interventions
AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
Biological · Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
12 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 11, 2026, 9:56 AM EDT
Completed Not applicable Interventional

A Pilot Study to Obtain Preliminary Information Regarding the Efficacy and Safety of the Combination of Immune Globulin and Ganciclovir as Compared to Ganciclovir Alone in the Treatment of Sight-Threatening CMV Retinitis in Patients With AIDS

NCT00001999
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Globulin, Immune, Ganciclovir
Drug
Lead sponsor
American National Red Cross
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 11, 2026, 9:56 AM EDT