- Conditions
- Primary Mitochondrial Myopathy
- Interventions
- Bocidelpar, Placebo
- Drug
- Lead sponsor
- Astellas Pharma Inc
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 34 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 11
- States / cities
- La Jolla, California • Stanford, California • Aurora, Colorado + 7 more
Search Results
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Showing 1–24
of 71
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Mitochondrial Disease, Neurology, Myopathy
- Interventions
- EPI-743, Placebo
- Drug
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 2 Years to 11 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2019
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Mitochondrial Disease
- Interventions
- Not listed
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- 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
- Urea Cycle Disorder, Organic Acidemia, Maple Syrup Urine Disease, Glutaric Acidemia I, Fatty Acid Oxidation Disorder, Hypoxic-Ischemic Encephalopathy
- Interventions
- Not listed
- Lead sponsor
- Children's National Research Institute
- Other
- Eligibility
- 7 Years to 18 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2027
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Mitochondrial Diseases
- Interventions
- EPI-743
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 1 Year and older
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 16
- States / cities
- Los Angeles, California • Orange, California • Palo Alto, California + 12 more
- 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
- Conditions
- Fibromyalgia, Mitochondrial Diseases, Movement Disorders, Diabetes Mellitus, Type 2
- Interventions
- Metformin
- Drug
- Lead sponsor
- Woodinville Psychiatric Associates
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2016
- U.S. locations
- 1
- States / cities
- Woodinville, Washington
- Conditions
- Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
- Interventions
- zagociguat 15mg
- Drug
- Lead sponsor
- Tisento Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 9
- States / cities
- La Jolla, California • Aurora, Colorado • Atlanta, Georgia + 5 more
- 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
- Conditions
- Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS), Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
- Interventions
- Sonlicromanol, Placebo
- Drug
- Lead sponsor
- Khondrion BV
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 220 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Cleveland, Ohio • Houston, Texas
- Conditions
- Mitochondrial Myopathy
- Interventions
- Exercise
- Behavioral
- Lead sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- NIH
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2012
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- 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
- Conditions
- PolG, Primary Mitochondrial Myopathy
- Interventions
- Not listed
- Lead sponsor
- The Emmes Company, LLC
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 1
- States / cities
- Kingston, Massachusetts
- Conditions
- Primary Mitochondrial Disease
- Interventions
- Not listed
- Lead sponsor
- Stealth BioTherapeutics Inc.
- Industry
- Eligibility
- 16 Years to 80 Years
- Enrollment
- 215 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 13
- States / cities
- La Jolla, California • Palo Alto, California • Aurora, Colorado + 9 more
- Conditions
- MItochondrial Myopathies
- Interventions
- Omaveloxolone capsules, 2.5 mg, omaveloxolone capsules, 5 mg, omaveloxolone capsules, 10 mg, Placebo capsules, omaveloxolone capsules, 20 mg, omaveloxolone capsules, 40 mg, omaveloxolone capsules, 80 mg, omaveloxolone capsules, 160 mg
- Drug
- Lead sponsor
- Biogen
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 53 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 8
- States / cities
- Los Angeles, California • Boston, Massachusetts • Akron, Ohio + 4 more
- Conditions
- Mitochondrial Myopathy
- Interventions
- Nicotinamide Riboside, Placebo
- Dietary Supplement · Drug
- Lead sponsor
- Ralitza Gavrilova
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 34 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- MELAS
- Interventions
- IW-6463 Tablets
- Drug
- Lead sponsor
- Tisento Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2022
- U.S. locations
- 5
- States / cities
- Washington D.C., District of Columbia • Baltimore, Maryland • Boston, Massachusetts + 2 more
- Conditions
- Primary Mitochondrial Disease
- Interventions
- KL1333, Placebo
- Drug
- Lead sponsor
- Pharming Technologies B.V.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 180 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 20
- States / cities
- Orange, California • San Diego, California • San Francisco, California + 15 more
- Conditions
- Mitochondrial Myopathies, Mitochondrial Diseases
- Interventions
- Nanosensor
- Device
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2027
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Thymidine Kinase 2 Deficiency
- Interventions
- Observational retrospective data
- Other
- Lead sponsor
- Zogenix MDS, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Indianapolis, Indiana • Akron, Ohio + 1 more
- 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
- 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
- Conditions
- Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia, Spastic Paraplegia, White Matter Disease, Neonatal Encephalopathy, Mutation, Genetic Disease
- Interventions
- Patient Registry, Dry blood spots sampling
- Other
- Lead sponsor
- University of California, San Diego
- Other
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 1
- States / cities
- San Diego, California