- 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
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 121–144
of 210
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Cytomegalovirus Infections, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
- Interventions
- triheptanoin
- Drug
- Lead sponsor
- Jerry Vockley, MD, PhD
- Other
- Eligibility
- 1 Month and older
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- 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
- 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
- Cytomegalovirus Infections
- Interventions
- Valganciclovir, Ganciclovir
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- Up to 30 Days
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2007
- U.S. locations
- 21
- States / cities
- Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 18 more
- Conditions
- Friedreich Ataxia
- Interventions
- Vatiquinone
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 130 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Gainesville, Florida • Tampa, Florida + 2 more
- 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
- Conditions
- Leigh Syndrome
- Interventions
- EPI-743
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 1 Year to 18 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2023
- U.S. locations
- 4
- States / cities
- Stanford University, California • Akron, Ohio • Houston, Texas + 1 more
- 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
- Conditions
- Friedreich Ataxia, Rare Diseases
- Interventions
- Geneticlly confirmed disease causing FXN mutatuion, Healthy Control
- Other
- Lead sponsor
- Friedreich's Ataxia Research Alliance
- Other
- Eligibility
- 4 Years to 21 Years
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2028
- U.S. locations
- 3
- States / cities
- Iowa City, Iowa • Philadelphia, Pennsylvania • Memphis, Tennessee
- Conditions
- Cytomegalovirus Infections, HIV Infections
- Interventions
- Valganciclovir
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 13 Years and older
- Enrollment
- 350 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2006
- U.S. locations
- 56
- States / cities
- Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 33 more
- Conditions
- Sickle Cell Disease
- Interventions
- MitoQ
- Dietary Supplement
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 18 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2025
- U.S. locations
- 5
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Iron-sulfur Cluster Deficiency, Pulmonary Hypertension, Friedreich Ataxia
- Interventions
- Right Heart Catheterization, Blood draw
- Procedure · Other
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2023
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Leber Hereditary Optic Neuropathy (LHON)
- Interventions
- NR082 Injection, Injection needle
- Drug · Device
- Lead sponsor
- Neurophth Therapeutics Inc
- Other
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2029
- U.S. locations
- 3
- States / cities
- Palo Alto, California • Aurora, Colorado • Philadelphia, Pennsylvania
- Conditions
- Dilated Cardiomyopathy, Hypertrophic Cardiomyopathy, Mitochondrial Cardiomyopathy, Noncompaction Cardiomyopathy, Restrictive Cardiomyopathy
- Interventions
- Not listed
- Lead sponsor
- University of California, Irvine
- Other
- Eligibility
- Not listed
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007
- U.S. locations
- 1
- States / cities
- Irvine, California
- Conditions
- Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
- Interventions
- Not listed
- Lead sponsor
- Foundation Fighting Blindness
- Other
- Eligibility
- Not listed
- Enrollment
- 20,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2037
- U.S. locations
- 1
- States / cities
- Columbia, Maryland
- Conditions
- MELAS Syndrome
- Interventions
- L-Citrulline
- Drug
- Lead sponsor
- Baylor College of Medicine
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Spinal Muscular Atrophy Type 3, Mitochondrial Myopathy
- Interventions
- Not listed
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 8 Years to 55 Years
- Enrollment
- 42 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2021
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Small Fiber Neuropathy, Fabry Disease, Ehlers Danlos Syndrome, Mitochondrial Disease
- Interventions
- Sudoscan, Skin biopsy, QSART
- Device · Procedure
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 8 Years to 70 Years
- Enrollment
- 102 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2020
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- 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
- MELAS Syndrome
- Interventions
- Arginine and citrulline supplementations
- Dietary Supplement
- Lead sponsor
- Baylor College of Medicine
- Other
- Eligibility
- 3 Years to 65 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009 – 2016
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Cidofovir, Probenecid, Ganciclovir
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 13 Years and older
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 12
- States / cities
- Los Angeles, California • Miami, Florida • Atlanta, Georgia + 7 more
- 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