- Conditions
- Leber's Congenital Amaurosis
- Interventions
- QR-110
- Drug
- Lead sponsor
- Laboratoires Thea
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 2
- States / cities
- Iowa City, Iowa • Philadelphia, Pennsylvania
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 49–72
of 79
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Ischemic Optic Neuropathy, Branch Retinal Artery Occlusion, Hemianopia, Leber Hereditary Optic Neuropathy, Acute Zonal Occult Outer Retinopathy
- Interventions
- LSFG-NAVI
- Device
- Lead sponsor
- Randy Kardon
- Other
- Eligibility
- 18 Years to 99 Years
- Enrollment
- 500 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2028
- U.S. locations
- 1
- States / cities
- Iowa City, Iowa
- Conditions
- Age Related Macular Degeneration (AMD)
- Interventions
- GT005
- Genetic
- Lead sponsor
- Gyroscope Therapeutics Limited
- Industry
- Eligibility
- 55 Years and older
- Enrollment
- 225 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2028
- U.S. locations
- 22
- States / cities
- Poway, California • Durango, Colorado • Gainesville, Florida + 18 more
- Conditions
- Gyrate Atrophy
- Interventions
- Not listed
- Lead sponsor
- National Eye Institute (NEI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 65 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1978 – 2004
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Stargardt Disease
- Interventions
- Not listed
- Lead sponsor
- Foundation Fighting Blindness
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 259 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 6
- States / cities
- Baltimore, Maryland • Cleveland, Ohio • Philadelphia, Pennsylvania + 2 more
- Conditions
- Optic, Atrophy, Hereditary, Leber
- Interventions
- GS010, Sham Intravitreal Injection
- Biological · Device
- Lead sponsor
- GenSight Biologics
- Industry
- Eligibility
- 15 Years and older
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Atlanta, Georgia • Philadelphia, Pennsylvania
- 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
- Gyrate Atrophy
- Interventions
- Gene therapy
- Procedure
- Lead sponsor
- National Eye Institute (NEI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2000
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Dry Age-related Macular Degeneration, Macular Degeneration, Retinal Disease, Retinal Degeneration, Geographic Atrophy, Macular Atrophy
- Interventions
- Not listed
- Lead sponsor
- Gyroscope Therapeutics Limited
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 83 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2024
- U.S. locations
- 33
- States / cities
- Phoenix, Arizona • Tucson, Arizona • Beverly Hills, California + 29 more
- Conditions
- Macular Degeneration, Geographic Atrophy, Stargardt Disease
- Interventions
- Observational
- Other
- Lead sponsor
- Nanoscope Therapeutics Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Optic Atrophy, Non-arteritic Anterior Ischemic Optic Neuropathy
- Interventions
- QPI-1007 at various doses
- Drug
- Lead sponsor
- Quark Pharmaceuticals
- Industry
- Eligibility
- 50 Years and older
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 22
- States / cities
- Phoenix, Arizona • Beverly Hills, California • Los Angeles, California + 19 more
- Conditions
- Healthy
- Interventions
- Whole grain oats and barley
- Other
- Lead sponsor
- Tufts University
- Other
- Eligibility
- 40 Years to 70 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2010 – 2013
- U.S. locations
- 1
- States / cities
- Boston, 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
- sepofarsen, Sham
- Drug · Other
- Lead sponsor
- ProQR Therapeutics
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 1
- States / cities
- Iowa City, Iowa
- Conditions
- Stroke, Ischemic, Stroke Hemorrhagic, Stroke (CVA) or TIA, Stroke, Acute, Traumatic Brain Injury, SCI - Spinal Cord Injury, Ehlers-Danlos Syndromes (EDS), Lupus Erythematosus, Guillain-Barre Syndrome, Multiple Sclerosis, Myasthenia Gravis, Amyotrophic Lateral Sclerosis, Graves Disease, Muscular Dystrophies
- Interventions
- Use of Rare Beauty makeup products
- Other
- Lead sponsor
- Casa Colina Hospital and Centers for Healthcare
- Other
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 57 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023
- U.S. locations
- 1
- States / cities
- Pomona, California
- Conditions
- Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes, Deletion and Duplication of Mitochondrial DNA
- Interventions
- Not listed
- Lead sponsor
- Columbia University
- Other
- Eligibility
- Not listed
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2026
- U.S. locations
- 16
- States / cities
- San Diego, California • Stanford, California • Aurora, Colorado + 12 more
- Conditions
- LGMD1B, LGMD1C, LGMD1D, LGMD1E, LGMD1F, LGMD1G, LGMD1H, LGMD2A, LGMD2B, LGMD2C, LGMD2D, LGMD2E, LGMD2F, LGMD2G, LGMD2I, LGMD2J, LGMD2K, LGMD2L, LGMD2M, LGMD2N, LGMD2O, LGMD2P, LGMD2Q, LGMD2S, LGMD2T, LGMD2U, LGMD2W, LGMD2X, LGMD2Y
- Interventions
- Not listed
- Lead sponsor
- Virginia Commonwealth University
- Other
- Eligibility
- 6 Years to 50 Years
- Enrollment
- 1,000 participants
- Timeline
- 2023 – 2029
- U.S. locations
- 1
- States / cities
- Richmond, Virginia
- Conditions
- Leber Hereditary Optic Neuropathy
- Interventions
- GS010, Placebo
- Genetic · Drug
- Lead sponsor
- GenSight Biologics
- Industry
- Eligibility
- 15 Years and older
- Enrollment
- 98 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2024
- U.S. locations
- 7
- States / cities
- Pasadena, California • Aurora, Colorado • Atlanta, Georgia + 4 more
- Conditions
- Leber's Hereditary Optic Neuropathy (LHON)
- Interventions
- Idebenone
- Drug
- Lead sponsor
- Santhera Pharmaceuticals
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 199 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2021
- U.S. locations
- 9
- States / cities
- Phoenix, Arizona • Palo Alto, California • Stanford, California + 6 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
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 1
- States / cities
- Buffalo, New York
- Conditions
- Age-Related Maculopathy, Age-Related Maculopathies, Eye Diseases, Retinal Degeneration, Macular Degeneration
- Interventions
- RN6G, Placebo
- Biological
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 60 Years to 85 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 24
- States / cities
- Campbell, California • Los Gatos, California • San Jose, California + 7 more
- Conditions
- Stargardt Disease
- Interventions
- Not listed
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 2
- States / cities
- Baltimore, Maryland • Dallas, Texas
- 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