- Conditions
- Autosomal Dominant Polycystic Kidney Disease
- Interventions
- Not listed
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 15 Years to 70 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
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 73–96
of 210
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Leigh Syndrome, Leigh Disease, Leigh's Necrotizing Encephalopathy, Subacute Necrotizing Encephalomyopathy, Subacute Necrotizing Encephalomyelopathy
- Interventions
- Not listed
- Lead sponsor
- The University of Texas Health Science Center, Houston
- Other
- Eligibility
- 0 Days to 100 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2030
- U.S. locations
- 1
- States / cities
- Houston, Texas
- 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
- 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
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 13 Years and older
- Enrollment
- 225 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 19
- States / cities
- Berkeley, California • Los Angeles, California • San Diego, California + 12 more
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
- Interventions
- Confirmatory Testing
- Diagnostic Test
- Lead sponsor
- RTI International
- Other
- Eligibility
- 1 Day to 31 Days
- Enrollment
- 30,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2026
- U.S. locations
- 1
- States / cities
- Research Triangle Park, North Carolina
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 13 Years and older
- Enrollment
- 180 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 22
- States / cities
- San Diego, California • San Francisco, California • San Mateo, California + 14 more
- Conditions
- Huntington Disease
- Interventions
- lumbar puncture
- Diagnostic Test
- Lead sponsor
- University Hospitals Cleveland Medical Center
- Other
- Eligibility
- 20 Years to 85 Years
- Enrollment
- 27 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2022
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Fomivirsen sodium, Ganciclovir
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 194 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 18
- States / cities
- Los Angeles, California • Pasadena, California • San Diego, California + 14 more
- Conditions
- Barth Syndrome
- Interventions
- Elamipretide, Placebo
- Drug
- Lead sponsor
- Stealth BioTherapeutics Inc.
- Industry
- Eligibility
- 5 Years to 55 Years · Male only
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 1
- States / cities
- Needham, Massachusetts
- 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
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- Roche Global Development
- Other
- Eligibility
- 13 Years and older
- Enrollment
- 450 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 23
- States / cities
- Beverly Hills, California • Irvine, California • Los Angeles, California + 16 more
- Conditions
- Leigh Syndrome
- Interventions
- Placebo, EPI-743 15 mg/kg, EPI-743 5 mg/kg
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 6 Years to 17 Years
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 4
- States / cities
- Palo Alto, California • Akron, Ohio • Houston, Texas + 1 more
- Conditions
- Friedreich Ataxia, Healthy Volunteers
- Interventions
- Buccal Swabs, Blood Draws and Skin Punch Biopsy
- Diagnostic Test
- Lead sponsor
- Larimar Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Eatontown, New Jersey
- Conditions
- Friedreich Ataxia
- Interventions
- DT-216, Placebo
- Drug
- Lead sponsor
- Design Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2023
- U.S. locations
- 1
- States / cities
- Eatontown, New Jersey
- Conditions
- Friedreich Ataxia
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2004
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
- Interventions
- Whole-body Electrical Muscle Stimulation Exercise
- Device
- Lead sponsor
- University of Missouri-Columbia
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2031
- U.S. locations
- 1
- States / cities
- Columbia, Missouri
- 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
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Sevirumab, Foscarnet sodium, Ganciclovir
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 13 Years and older
- Enrollment
- 167 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Ends 1998
- U.S. locations
- 20
- States / cities
- Birmingham, Alabama • Los Angeles, California • San Jose, California + 13 more
- Conditions
- Friedreich's Ataxia
- Interventions
- interferon γ-1b
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 11 Years to 27 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2017
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more
- Conditions
- Friedreich's Ataxia
- Interventions
- Interferon γ-1b
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 10 Years to 26 Years
- Enrollment
- 86 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more
- Conditions
- Friedreich's Ataxia
- Interventions
- Vatiquinone
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 7 Years to 21 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Tampa, Florida • Iowa City, Iowa + 1 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
- Friedreich's Ataxia
- Interventions
- Interferon γ-1b, Placebo
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 10 Years to 25 Years
- Enrollment
- 92 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2016
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more