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ClinicalTrials.gov public records Last synced May 22, 2026, 5:21 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Inherited Mitochondrial Disease Clear all
Completed No phase listed Observational
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 9, 2008 · Synced May 22, 2026, 5:21 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Pyruvate Dehydrogenase Complex Deficiency Disease
Interventions
No intervention
Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2026
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 12, 2026 · Synced May 22, 2026, 5:21 AM EDT
Completed No phase listed Observational
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 17, 2020 · Synced May 22, 2026, 5:21 AM EDT
Completed No phase listed Observational
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
9 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 7, 2024 · Synced May 22, 2026, 5:21 AM EDT
Recruiting No phase listed Observational
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 27, 2026 · Synced May 22, 2026, 5:21 AM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
(-)-epicatechin
Drug
Lead sponsor
Craig McDonald, MD
Other
Eligibility
18 Years to 60 Years · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Dec 21, 2021 · Synced May 22, 2026, 5:21 AM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS, MERRF
Interventions
Promoting Resilience in Stress Management (PRISM), Clinical-focused narrative (CFN)
Behavioral
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 28, 2024 · Synced May 22, 2026, 5:21 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Cardiac magnetic resonance imaging (CMR), Exercise-stress test, Echocardiogram (ECHO), Cardiac-related blood studies
Procedure
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
12 Years to 50 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 18, 2025 · Synced May 22, 2026, 5:21 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Spinocerebellar Ataxia - All Sub-types, Friedreich's Ataxia
Interventions
Ataxia rating scale
Other
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Nov 15, 2021 · Synced May 22, 2026, 5:21 AM EDT
Completed Phase 2 Interventional Results available
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 May 22, 2026, 5:21 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Natural history
Other
Lead sponsor
Monash University
Other
Eligibility
5 Years and older
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
3
States / cities
Gainesville, Florida • Minneapolis, Minnesota • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 26, 2024 · Synced May 22, 2026, 5:21 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
MELAS or m.3243 A>G Mitochondrial DNA Mutation Carrier
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
4 Years and older
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced May 22, 2026, 5:21 AM EDT
Completed Phase 1 Interventional Results available
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 May 22, 2026, 5:21 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Inherited Mitochondrial Disease, Including Leigh Syndrome
Interventions
Cysteamine Bitartrate
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
5
States / cities
San Diego, California • Stanford, California • Akron, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 26, 2024 · Synced May 22, 2026, 5:21 AM EDT
Completed Phase 1 Interventional
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
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 11, 2006 · Synced May 22, 2026, 5:21 AM EDT
Completed Phase 1 Interventional
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 23, 2005 · Synced May 22, 2026, 5:21 AM EDT
Completed Phase 1 Interventional
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 May 22, 2026, 5:21 AM EDT
Completed Phase 3 Interventional
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
3 Months and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 29, 2008 · Synced May 22, 2026, 5:21 AM EDT
Recruiting Phase 3 Interventional
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone, Placebo
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 15 Years
Enrollment
255 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
7
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 15, 2026 · Synced May 22, 2026, 5:21 AM EDT
Terminated Phase 1Phase 2 Interventional Accepts healthy volunteers Results available
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 28, 2014 · Synced May 22, 2026, 5:21 AM EDT
Completed No phase listed Observational
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 7, 2024 · Synced May 22, 2026, 5:21 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Autosomal Dominant Polycystic Kidney Disease
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
15 Years to 40 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 8, 2026 · Synced May 22, 2026, 5:21 AM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
Interventions
Family Centered pediatric palliative care for family caregivers of children with rare diseases.
Behavioral
Lead sponsor
Children's National Research Institute
Other
Eligibility
12 Months to 99 Years
Enrollment
480 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2029
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jan 27, 2026 · Synced May 22, 2026, 5:21 AM EDT
Completed Phase 1Phase 2 Interventional Results available
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 May 22, 2026, 5:21 AM EDT