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ClinicalTrials.gov public records Last synced Oct 12, 2026, 1:19 PM EDT

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Showing 97–120 of 205 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Mitochondrial Disease Clear all
Terminated No phase listed Observational Accepts healthy volunteers

Biomechanics of Optic Neuropathy

NCT02982499
View directory record Official record
Conditions
Optic Neuropathy
Interventions
magnetic resonance image (MRI), Optical Coherence Tomography (OCT)
Device
Lead sponsor
University of Miami
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2018
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Oct 23, 2020 · Synced Oct 12, 2026, 1:19 PM EDT
Withdrawn Phase 1 Interventional Accepts healthy volunteers

O2 Transport and Utilization in Health and Lung Disease

NCT04014712
View directory record Official record
Conditions
COPD, Tetrahydrobiopterin Deficiency, Oxidative Stress
Interventions
Tetrahydrobiopterin, Placebo oral tablet
Drug
Lead sponsor
University of Massachusetts, Amherst
Other
Eligibility
18 Years to 85 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Amherst, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 19, 2023 · Synced Oct 12, 2026, 1:19 PM EDT
Completed Phase 2 Interventional

CMV Retinitis Retreatment Trial

NCT00000766
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
18 Years and older
Enrollment
300 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 1, 2011 · Synced Oct 12, 2026, 1:19 PM EDT
Completed Phase 2 Interventional Results available

NAD+ Precursor Supplementation in Friedreich's Ataxia

NCT04817111
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
MIB-626
Drug
Lead sponsor
Metro International Biotech, LLC
Industry
Eligibility
18 Years to 64 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 17, 2023 · Synced Oct 12, 2026, 1:19 PM EDT
Completed Phase 3 Interventional

A Randomized, Controlled Study of Intravenous Ganciclovir Therapy for Peripheral Cytomegalovirus Retinitis in Patients With AIDS

NCT00000688
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 and older
Enrollment
180 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
22
States / cities
San Diego, California • San Francisco, California • San Mateo, California + 14 more
Source: ClinicalTrials.gov public record
Updated Mar 14, 2011 · Synced Oct 12, 2026, 1:19 PM 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 12, 2026, 1:19 PM EDT
Recruiting No phase listed Observational

Natural History of MADD

NCT07734090
View directory record Official record
Conditions
Multiple Acyl-CoA Dehydrogenase Deficiency
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
Aurora, Colorado • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 12, 2026, 1:19 PM 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 12, 2026, 1:19 PM EDT
Completed Phase 2 Interventional

Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants

NCT01624311
View directory record Official record
Conditions
Urea Cycle Disorders, Organic Acidemias
Interventions
Therapeutic Hypothermia, Standard of Care Therapy
Other
Lead sponsor
Uta Lichter-Konecki
Other
Eligibility
1 Day to 30 Days
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2015
U.S. locations
3
States / cities
Washington D.C., District of Columbia • New York, New York • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jun 1, 2015 · Synced Oct 12, 2026, 1:19 PM EDT
Completed Not applicable Interventional

A Study of the Safety and Tolerance of Long-Term Therapy With Intravenous Cytovene (Ganciclovir Sodium) for Cytomegalovirus Retinitis in Persons With AIDS

NCT00002034
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Zidovudine, Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
8
States / cities
Birmingham, Alabama • Oakland, California • San Francisco, California + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 12, 2026, 1:19 PM 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 12, 2026, 1:19 PM 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 30, 2021 · Synced Oct 12, 2026, 1:19 PM EDT
Recruiting Phase 2 Interventional

Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

NCT07721025
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathy, Secondary
Interventions
LX2006, Usual Care
Genetic · Other
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
6 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 12, 2026, 1:19 PM EDT
Completed Phase 1 Interventional Results available

Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy

NCT02161380
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low),, injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med), injection of scAAV2-P1ND4v2 2.4 X10e10vg (High), injection of scAAV2-P1ND4v2 1.0 X10e11vg (Higher)
Drug
Lead sponsor
Byron Lam
Other
Eligibility
15 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2025
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Jun 5, 2025 · Synced Oct 12, 2026, 1:19 PM 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 12, 2026, 1:19 PM EDT
Completed Phase 2 Interventional Results available

(-)- Epicatechin Becker Muscular Dystrophy

NCT03236662
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 24, 2021 · Synced Oct 12, 2026, 1:19 PM EDT
Terminated Phase 1Phase 2 Interventional

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations

NCT05820152
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy (LHON)
Interventions
NFS-02 Injection
Drug
Lead sponsor
Neurophth Therapeutics Inc
Other
Eligibility
18 Years to 75 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Sep 20, 2024 · Synced Oct 12, 2026, 1:19 PM EDT
Completed Phase 2 Interventional Results available

A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia

NCT02255435
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg, Placebo, Omaveloxolone Capsules, 20 mg, Omaveloxolone Capsules, 40 mg, Omaveloxolone Capsules, 80 mg, Omaveloxolone Capsules, 160 mg, Omaveloxolone Capsules, 300 mg, Omaveloxolone Capsules, 150 mg
Drug
Lead sponsor
Biogen
Industry
Eligibility
16 Years to 40 Years
Enrollment
172 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2025
U.S. locations
7
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 12, 2026, 1:19 PM EDT
Terminated Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Near-infrared Light-emitting Diode (NIR-LED) Therapy for Leber's Hereditary Optic Neuropathy (LHON)

NCT01389817
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy (LHON)
Interventions
Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.))
Device
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
3
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 29, 2014 · Synced Oct 12, 2026, 1:19 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism

NCT01780168
View directory record Official record
Conditions
Oxidative Phosphorylation Deficiencies, Electron Transport Chain Disorders, Mitochondrial, Mitochondrial Disorders, Leigh Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
4 Weeks to 115 Years
Enrollment
500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 12, 2026, 1:19 PM EDT
Recruiting Not applicable Interventional

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

NCT07478172
View directory record Official record
Conditions
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
Interventions
Whole-body Electrical Muscle Stimulation Exercise
Device
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Oct 12, 2026, 1:19 PM EDT
Completed Phase 2 Interventional

EPI-743 in Friedreich's Ataxia Point Mutations

NCT01962363
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
EPI-743
Drug
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jun 29, 2016 · Synced Oct 12, 2026, 1:19 PM EDT
Completed Phase 3 Interventional Results available

A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia

NCT04102501
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
RT001, Placebo
Drug
Lead sponsor
Biojiva LLC
Industry
Eligibility
12 Years to 50 Years
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
6
States / cities
Long Beach, California • Los Angeles, California • Gainesville, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated May 27, 2022 · Synced Oct 12, 2026, 1:19 PM EDT
Active, not recruiting Phase 3 Interventional

Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:

NCT02616484
View directory record Official record
Conditions
Pyruvate Dehydrogenase Complex Deficiency
Interventions
Dichloroacetate (DCA), Placebo, Genotype
Drug · Other · Genetic
Lead sponsor
Saol Therapeutics Inc
Industry
Eligibility
6 Months to 17 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
10
States / cities
Orange, California • Stanford, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated May 4, 2025 · Synced Oct 12, 2026, 1:19 PM EDT