- 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
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 169–192
of 206
matching trials from the live ClinicalTrials.gov search.
- 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
- 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
- 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
- Friedreich Ataxia
- Interventions
- CTI-1601, Placebo
- Biological
- Lead sponsor
- Larimar Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Eatontown, New Jersey
- Conditions
- Sexual Assault, Substance Use, Risky Health Behavior, Ema
- Interventions
- Ecological Momentary Intervention
- Behavioral
- Lead sponsor
- University of New Mexico
- Other
- Eligibility
- 18 Years to 24 Years · Female only
- Enrollment
- 183 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2023
- U.S. locations
- 1
- States / cities
- Albuquerque, New Mexico
- 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
- 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
- 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 Infections, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 14 Days to 20 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Ends 1998
- U.S. locations
- 18
- States / cities
- Los Angeles, California • Oakland, California • San Diego, California + 10 more
- 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
- 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
- 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
- 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
- Multiple Acyl-CoA Dehydrogenase Deficiency
- Interventions
- Not listed
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Other
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2031
- U.S. locations
- 2
- States / cities
- Aurora, Colorado • New York, New York
- Conditions
- Pyruvate Dehydrogenase Complex Deficiency
- Interventions
- Dichloroacetate (DCA), Placebo, Genotype
- Drug · Other · Genetic
- Lead sponsor
- Saol Therapeutics Inc
- Industry
- Eligibility
- 6 Months to 17 Years
- Enrollment
- 34 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2025
- U.S. locations
- 10
- States / cities
- Orange, California • Stanford, California • Washington D.C., District of Columbia + 7 more
- Conditions
- Friedreich's Ataxia (FA)
- Interventions
- SGT-212
- Drug
- Lead sponsor
- Solid Biosciences Inc.
- Industry
- Eligibility
- 18 Years to 40 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Columbus, Ohio • Philadelphia, Pennsylvania
- Conditions
- Friedreich Ataxia, Friedreich's Ataxia
- Interventions
- Nomlabofusp, Placebo
- Drug
- Lead sponsor
- Larimar Therapeutics, Inc.
- Industry
- Eligibility
- 12 Years to 40 Years
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 4
- States / cities
- Tampa, Florida • Chicago, Illinois • Eatontown, New Jersey + 1 more
- Conditions
- Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6, Friedreich Ataxia
- Interventions
- Not listed
- Lead sponsor
- University of Chicago
- Other
- Eligibility
- 12 Years to 75 Years
- Enrollment
- 128 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2024
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 2 more
- 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
- 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
- Foscarnet sodium
- Drug
- Lead sponsor
- Astra USA
- Industry
- Eligibility
- 18 Years to 65 Years
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Rett Syndrome
- Interventions
- triheptanoin
- Drug
- Lead sponsor
- Center for Rare Neurological Diseases, Norcross, GA
- Other
- Eligibility
- 2 Years and older · Female only
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2022
- U.S. locations
- 1
- States / cities
- Norcross, Georgia
- Conditions
- Friedreich's Ataxia
- Interventions
- Aerobic Exercise
- Behavioral
- Lead sponsor
- Scott Barbuto
- Other
- Eligibility
- Not listed
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 1
- States / cities
- New York, New York