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ClinicalTrials.gov public records Last synced Oct 14, 2026, 4:54 AM EDT

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Showing 145–168 of 206 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Mitochondrial Disease Clear all
Not yet recruiting Phase 2 Interventional

GV101 for Friedreich's Ataxia (FA) Treatment

NCT07870590
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
GV101, Placebo
Drug
Lead sponsor
Graviton Bioscience Corporation
Industry
Eligibility
16 Years to 40 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 14, 2026, 4:54 AM EDT
Completed Not applicable Interventional

An Open-Label Safety Study of Oral Ganciclovir Maintenance Treatment of CMV Retinitis in People With Limited Venous Access

NCT00002135
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 14, 2026, 4:54 AM EDT
Recruiting No phase listed Observational

Clinical Course Of Disease In Participants With FA-CM

NCT06865482
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathy
Interventions
Not listed
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
6 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
8
States / cities
La Jolla, California • Tampa, Florida • Indianapolis, Indiana + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 14, 2026, 4:54 AM EDT
Recruiting Phase 1 Interventional

Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels

NCT05241262
View directory record Official record
Conditions
Mitochondrial Disease
Interventions
N-Acetylcysteine
Drug
Lead sponsor
Michio Hirano, MD
Other
Eligibility
18 Years to 80 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 23, 2026 · Synced Oct 14, 2026, 4:54 AM EDT
Completed Phase 2 Interventional

Comparison of Two Methods in the Treatment of Cytomegalovirus of the Eyes in Patients With AIDS

NCT00001061
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Sevirumab, Foscarnet sodium, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
167 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1998
U.S. locations
20
States / cities
Birmingham, Alabama • Los Angeles, California • San Jose, California + 13 more
Source: ClinicalTrials.gov public record
Updated Nov 1, 2021 · Synced Oct 14, 2026, 4:54 AM EDT
Terminated No phase listed Observational

Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation

NCT06140329
View directory record Official record
Conditions
Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant, Optic Atrophies, Hereditary, Kjer Optic Atrophy
Interventions
Not listed
Lead sponsor
PYC Therapeutics
Industry
Eligibility
8 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
2
States / cities
Miami, Florida • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Mar 14, 2025 · Synced Oct 14, 2026, 4:54 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Oxygen Uptake Kinetics During Submaximal Exercise in Adults With Down Syndrome

NCT05995223
View directory record Official record
Conditions
Down Syndrome
Interventions
Submaximal exercise protocol
Other
Lead sponsor
University of Nevada, Las Vegas
Other
Eligibility
18 Years to 35 Years
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2024
U.S. locations
1
States / cities
Las Vegas, Nevada
Source: ClinicalTrials.gov public record
Updated Jul 1, 2024 · Synced Oct 14, 2026, 4:54 AM EDT
Completed Phase 2 Interventional

Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia

NCT01035671
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
alpha-tocopherolquinone (A0001), placebo
Drug
Lead sponsor
Penwest Pharmaceuticals Co.
Industry
Eligibility
18 Years to 60 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 22, 2011 · Synced Oct 14, 2026, 4:54 AM EDT
Completed Phase 1 Interventional

Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

NCT04519567
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
CTI-1601, Placebo
Biological
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Jun 30, 2021 · Synced Oct 14, 2026, 4:54 AM EDT
Terminated Phase 2 Interventional Results available

A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease

NCT02473445
View directory record Official record
Conditions
Mitochondrial Diseases
Interventions
Cysteamine Bitartrate
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
5
States / cities
San Diego, California • Stanford, California • Akron, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 14, 2026, 4:54 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 14, 2026, 4:54 AM EDT
Not listed Phase 2 Interventional

Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome

NCT00068913
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 24, 2005 · Synced Oct 14, 2026, 4:54 AM EDT
Completed Phase 2 Interventional

A Phase 2b Study of Zagociguat in Patients With MELAS

NCT06402123
View directory record Official record
Conditions
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
Interventions
zagociguat 15mg, zagociguat 30mg, Placebo
Drug
Lead sponsor
Tisento Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
10
States / cities
La Jolla, California • Aurora, Colorado • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 14, 2026, 4:54 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

NCT01694940
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 4, 2026 · Synced Oct 14, 2026, 4:54 AM EDT
Recruiting Phase 1 Interventional

Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

NCT06340685
View directory record Official record
Conditions
Pyruvate Dehydrogenase Complex Deficiency
Interventions
Triheptanoin
Drug
Lead sponsor
Jirair Krikor Bedoyan
Other
Eligibility
1 Year to 17 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced Oct 14, 2026, 4:54 AM EDT
Completed No phase listed Observational

Study of the Metabolism of Pyruvate and Related Problems in Patients With Lactic Acidemia

NCT00004353
View directory record Official record
Conditions
Mitochondrial Myopathy, MELAS Syndrome, Lactic Acidosis
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1978
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Jul 10, 2008 · Synced Oct 14, 2026, 4:54 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Role of p53 Gene in Metabolism Regulation in Patients With Li-Fraumeni Syndrome

NCT00406445
View directory record Official record
Conditions
Mitochondrial Disorders, Li-Fraumeni Syndrome, Carriers of p53 Mutation
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
82 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 22, 2021 · Synced Oct 14, 2026, 4:54 AM EDT
Completed Not applicable Interventional

Coronary Artery Disease in Patients With Friedreich's Ataxia

NCT04649866
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
plethysmography
Diagnostic Test
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years to 79 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 8, 2023 · Synced Oct 14, 2026, 4:54 AM EDT
Recruiting Phase 4 Interventional

Clinical Trial in Patients With Barth Syndrome- 4TAZPower

NCT07531251
View directory record Official record
Conditions
Barth Syndrome
Interventions
Elamipretide, Placebo
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
5 Years to 55 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Needham, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 14, 2026, 4:54 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 Oct 13, 2026 · Synced Oct 14, 2026, 4:54 AM EDT
Completed No phase listed Observational

Neonatal CMV-Ganciclovir Follow-up Study

NCT00031421
View directory record Official record
Conditions
Cytomegalovirus Infections
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
9 Years to 14 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2005
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Aug 12, 2011 · Synced Oct 14, 2026, 4:54 AM EDT
Recruiting No phase listed Observational

Hepatic Histopathology in Urea Cycle Disorders

NCT04908319
View directory record Official record
Conditions
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia, Carbamyl Phosphate Synthetase Deficiency, NAGS Deficiency
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 20, 2025 · Synced Oct 14, 2026, 4:54 AM EDT
Completed Phase 1 Interventional

A Phase I Pharmacokinetic and Tolerance Study of 28-Day Regimens of Oral Ganciclovir

NCT00000668
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years to 60 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1995
U.S. locations
5
States / cities
San Diego, California • San Francisco, California • New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 3, 2021 · Synced Oct 14, 2026, 4:54 AM EDT
Completed Not applicable Interventional

A Randomized Controlled Study of the Efficacy and Safety of Maintenance Treatment With Oral Ganciclovir for Newly Diagnosed Cytomegalovirus Retinitis in People With AIDS

NCT00002257
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
12
States / cities
Berkeley, California • San Francisco, California • Washington D.C., District of Columbia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 14, 2026, 4:54 AM EDT