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ClinicalTrials.gov public records Last synced Nov 6, 2026, 1:51 PM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 438 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Mitochondrial Disease Clear all

Conditions: Heart Failure，Congestive, Heart Failure New York Heart Association Class IV, Mitochondrial Alteration
Interventions: Nicotinamide riboside; Dietary Supplement
Lead sponsor: University of Washington; Other
Eligibility: 18 Years to 75 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Friedreich Ataxia, Cardiomyopathies, Central Nervous System Diseases, Neuromuscular Diseases, Mitochondrial Diseases, Mitochondrial Dysfunction, Genetic Disease
Interventions: ATI-105, Placebo; Drug
Lead sponsor: Adjuvia Therapeutics, Inc.; Industry
Eligibility: 18 Years to 60 Years
Enrollment: 60 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026 – 2027
U.S. locations: 1
States / cities: Miami, Florida

Conditions: Diabetes Mellitus, Type 2
Interventions: Pioglitazone; Drug
Lead sponsor: Pennington Biomedical Research Center; Other
Eligibility: 18 Years to 70 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2007
U.S. locations: 1
States / cities: Baton Rouge, Louisiana

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

Conditions: Autism Spectrum Disorders
Interventions: Suramin, Saline; Drug
Lead sponsor: University of California, San Diego; Other
Eligibility: 4 Years to 17 Years · Male only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2016
U.S. locations: 1
States / cities: La Jolla, California

Conditions: Cerebral Ischemia
Interventions: Endovascular autologous mitochondrial transplantation; Other
Lead sponsor: University of Washington; Other
Eligibility: 18 Years to 85 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2026
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Friedreich Ataxia
Interventions: Spinal tap for CSF collection, Venipuncture for blood sample; Other
Lead sponsor: University of Florida; Other
Eligibility: 18 Years to 65 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: Gainesville, Florida

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

Conditions: Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
Interventions: QR-110; Drug
Lead sponsor: Laboratoires Thea; Industry
Eligibility: 6 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 2
States / cities: Iowa City, Iowa • Philadelphia, Pennsylvania

Conditions: Friedreich's Ataxia
Interventions: Acetyl-L-Carnitine; Drug
Lead sponsor: University of South Florida; Other
Eligibility: 18 Years to 80 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2017
U.S. locations: 1
States / cities: Tampa, Florida

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

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

Conditions: Diabetes Mellitus, Type 2, Overweight
Interventions: Aerobic exercise program - 8 weeks; Behavioral
Lead sponsor: University of Pittsburgh; Other
Eligibility: 28 Years to 55 Years
Enrollment: 29 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2006 – 2010
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Retinal Disease, Age-Related Macular Degeneration, Retinitis Pigmentosa, Stargardt Disease, Optic Neuropathy, Nonarteritic Ischemic Optic Neuropathy, Optic Atrophy, Optic Nerve Disease, Glaucoma, Leber Hereditary Optic Neuropathy, Blindness, Vision Loss Night, Vision Loss Partial, Vision, Low, Retinopathy, Maculopathy, Macular Degeneration, Retina Atrophy
Interventions: Arm 1; Procedure
Lead sponsor: MD Stem Cells; Industry
Eligibility: 18 Years and older
Enrollment: 500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2028
U.S. locations: 2
States / cities: Westport, Connecticut • Coral Springs, Florida

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

Conditions: Breast Neoplasms
Interventions: Formal study using hyperpolarized 13C-pyruvate injection, Feasible study using hyperpolarized 13C-pyruvate injection; Drug
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: 18 Years and older
Enrollment: 79 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2025
U.S. locations: 1
States / cities: Dallas, Texas

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

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Zidovudine, Sargramostim, Ganciclovir; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 13 Years to 65 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Ends 1992
U.S. locations: 4
States / cities: Los Angeles, California • Boston, Massachusetts • New York, New York + 1 more

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

Conditions: MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
Interventions: Glycerol Tributyrate; Drug
Lead sponsor: George Washington University; Other
Eligibility: 18 Years to 65 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2027 – 2028
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Type 2 Diabetes
Interventions: Glycemic clamping; Other
Lead sponsor: Medical College of Wisconsin; Other
Eligibility: 21 Years and older
Enrollment: 5 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2012 – 2017
U.S. locations: 1
States / cities: Milwaukee, Wisconsin

Conditions: HIV, Aging, Sarcopenia, Angiotensin Receptor Antagonists
Interventions: valsartan, Placebo; Drug · Other
Lead sponsor: Wake Forest University Health Sciences; Other
Eligibility: 40 Years to 60 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2016
U.S. locations: 1
States / cities: Winston-Salem, North Carolina

Conditions: Friedreich Ataxia
Interventions: Interferon Gamma-1b; Drug
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: 5 Years to 17 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2014
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: MItochondrial Myopathies, Mitochondrial Disease
Interventions: Nanosensor; Device
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: 18 Years to 65 Years
Enrollment: 70 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2027
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Search by objective public record fields.

Nicotinamide Riboside in LVAD Recipients

A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults

"TAKE TIME" Pioglitazone Reverses Defects in Mitochondrial Biogenesis in Patients With T2DM

Palliative Care Needs of Children With Rare Diseases and Their Families

University of California, San Diego (UCSD) Suramin Autism Treatment-1 (SAT1) Trial

Autologous Mitochondrial Transplant for Cerebral Ischemia

Measurement of Frataxin mRNA in Biofluids

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

Extension Study to Study PQ-110-001 (NCT03140969)

An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia

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

NAD+ Precursor Supplementation in Friedreich's Ataxia

Mitochondrial Dysfunction in Type 2 Diabetes Mellitus and Capacity for Fat Oxidation During Exercise

Stem Cell Ophthalmology Treatment Study II

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

Using Hyperpolarized [1-13C]Pyruvate to Detect Cardiotoxicity

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

The Safety and Effectiveness of Ganciclovir Used Alone or in Combination With Granulocyte-Macrophage Colony Stimulating Factor in the Treatment of Cytomegalovirus (CMV) of the Eye in Patients With AIDS

Natural History Study of SLC25A46 Mutation-related Mitochondriopathy

A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)

Adverse Effects of Systemic Hypoglycemia Exposure on Endothelial Function in Humans

Angiotensin Receptors and Age Related Mitochondrial Decline in HIV Patients

Interferon Gamma-1b in Friedreich Ataxia (FRDA)

Validation of Nanosensor Oxygen Measurement