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ClinicalTrials.gov public records Last synced Oct 21, 2026, 1:35 AM EDT

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Showing 169–192 of 210 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Mitochondrial Genetic Disorders Clear all
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 21, 2026, 1:35 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 21, 2026, 1:35 AM EDT
Completed Not applicable Interventional

An Open Label Evaluation of the Safety and Pharmacokinetics of Ganciclovir in Children

NCT00002015
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Roche Global Development
Other
Eligibility
3 Months to 12 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
8
States / cities
Birmingham, Alabama • Los Angeles, California • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 21, 2026, 1:35 AM EDT
Completed Phase 2 Interventional Results available

Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia

NCT03214588
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
TAK-831, TAK-831 Placebo
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
18 Years to 55 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
6
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2021 · Synced Oct 21, 2026, 1:35 AM EDT
Completed Not applicable Interventional

A Study of Valganciclovir in the Treatment of Cytomegalovirus (CMV) Retinitis in Patients With AIDS

NCT00002222
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Valganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 21, 2026, 1:35 AM 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 21, 2026, 1:35 AM EDT
Completed Phase 3 Interventional Results available

Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)

NCT02652780
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
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
3
States / cities
Los Angeles, California • Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 21, 2026, 1:35 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

NCT05445323
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathy, Secondary
Interventions
Low dose LX2006, Mid Dose LX2006, High Dose LX2006
Genetic
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
18 Years to 50 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
3
States / cities
Los Angeles, California • Tampa, Florida • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 23, 2025 · Synced Oct 21, 2026, 1:35 AM EDT
Recruiting No phase listed Observational

A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

NCT06623890
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone
Drug
Lead sponsor
Biogen
Industry
Eligibility
16 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
4
States / cities
Los Angeles, California • Aurora, Colorado • Gainesville, Florida + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 28, 2026 · Synced Oct 21, 2026, 1:35 AM EDT
Terminated Phase 2Phase 3 Interventional

Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia

NCT00803868
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
varenicline, placebo
Drug
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 80 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
2
States / cities
Tampa, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 24, 2012 · Synced Oct 21, 2026, 1:35 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Assessment of Longitudinal Changes in Endothelial Function and Oxidative Stress in Normotensive Patients With ADPKD

NCT03493802
View directory record Official record
Conditions
Autosomal Dominant Polycystic Kidney Disease
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 40 Years
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2020
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated May 6, 2024 · Synced Oct 21, 2026, 1:35 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 21, 2026, 1:35 AM EDT
Completed Phase 1 Interventional

A Phase I Pharmacokinetic Study in HIV-Positive Subjects of Oral Ganciclovir and Concomitant Antiretroviral Zidovudine and Didanosine

NCT00002096
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Zidovudine, Didanosine, Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
3
States / cities
Irvine, California • Washington D.C., District of Columbia • Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 21, 2026, 1:35 AM EDT
Recruiting Not applicable Interventional

Cardiac Output and Fatigue in Friedreich's Ataxia

NCT07444333
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Aerobic Exercise
Behavioral
Lead sponsor
Scott Barbuto
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 21, 2026, 1:35 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

NCT06016946
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Not listed
Lead sponsor
Friedreich's Ataxia Research Alliance
Other
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2048
U.S. locations
9
States / cities
Los Angeles, California • Denver, Colorado • Gainesville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Oct 21, 2026, 1:35 AM 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 21, 2026, 1:35 AM 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 21, 2026, 1:35 AM EDT
Completed No phase listed Observational

Natural History Study of FDXR Mutation-related Mitochondriopathy

NCT04580979
View directory record Official record
Conditions
Neurodegenerative Disease, Hereditary, Mitochondrial Diseases, Optic Atrophy
Interventions
Mutation analysis
Genetic
Lead sponsor
State University of New York at Buffalo
Other
Eligibility
0 Years to 65 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Mar 8, 2024 · Synced Oct 21, 2026, 1:35 AM EDT
Completed Not applicable Interventional

A Study of Ganciclovir in the Treatment of Cytomegalovirus of the Large Intestine in Patients With AIDS

NCT00002273
View directory record Official record
Conditions
Colitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
21 Years and older · Male only
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 21, 2026, 1:35 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

New Non-invasive Modalities for Assessing Retinal Structure and Function

NCT03475173
View directory record Official record
Conditions
Ischemic Optic Neuropathy, Branch Retinal Artery Occlusion, Hemianopia, Leber Hereditary Optic Neuropathy, Acute Zonal Occult Outer Retinopathy
Interventions
LSFG-NAVI
Device
Lead sponsor
Randy Kardon
Other
Eligibility
18 Years to 99 Years
Enrollment
500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2028
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 21, 2026, 1:35 AM EDT
Completed No phase listed Observational

A Study to Assess Variation in Potential Biomarkers in Friedreich Ataxia

NCT04255680
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Buccal Swabs and Blood Draws
Diagnostic Test
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
20 participants
Timeline
2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 18, 2020 · Synced Oct 21, 2026, 1:35 AM EDT
Completed Phase 3 Interventional Results available

Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year

NCT03293524
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy
Interventions
GS010, Placebo
Genetic · Drug
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
7
States / cities
Pasadena, California • Aurora, Colorado • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 21, 2026, 1:35 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Acute Infection in Mitochondrial Disease: Metabolism, Infection and Immunity

NCT04419870
View directory record Official record
Conditions
Mitochondrial Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Months to 115 Years
Enrollment
400 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2099
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 10, 2026 · Synced Oct 21, 2026, 1:35 AM EDT
Completed Phase 2 Interventional Results available

Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes

NCT00887562
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Idebenone, Placebo
Drug · Other
Lead sponsor
Michio Hirano
Other
Eligibility
8 Years to 65 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 26, 2016 · Synced Oct 21, 2026, 1:35 AM EDT