- Conditions
- Optic, Atrophy, Hereditary, Leber
- Interventions
- GS010, Sham Intravitreal Injection
- Biological · Device
- Lead sponsor
- GenSight Biologics
- Industry
- Eligibility
- 15 Years and older
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Atlanta, Georgia • Philadelphia, Pennsylvania
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- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders, Vision Disorder, Neurological Manifestations, Eye Diseases, Hereditary, Eye Diseases, Eye Disorders Congenital, Retinal Disease
- Interventions
- sepofarsen, Placebo IVT
- Drug · Other
- Lead sponsor
- Laboratoires Thea
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 5
- States / cities
- San Francisco, California • Miami, Florida • Iowa City, Iowa + 2 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
- Type 1 Diabetes, Diabetes Insipidus, Diabetes Mellitus, Wolfram Syndrome
- Interventions
- Not listed
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- 1 Day and older
- Enrollment
- 101 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2017
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- 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
- Parkinson's Disease (PD), Parkinsonism, Progressive Supranuclear Palsy (PSP), Multiple System Atrophy (MSA)
- Interventions
- Not listed
- Lead sponsor
- Milton S. Hershey Medical Center
- Other
- Eligibility
- 21 Years to 90 Years
- Enrollment
- 290 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2019
- U.S. locations
- 1
- States / cities
- Hershey, 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
- Leber Hereditary Optic Neuropathy (LHON)
- Interventions
- NFS-02 Injection
- Drug
- Lead sponsor
- Neurophth Therapeutics Inc
- Other
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 1
- States / cities
- Aurora, Colorado
- Conditions
- Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Ataxia
- Interventions
- dantrolene sodium
- Drug
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- 5 Years to 60 Years
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant, Optic Atrophies, Hereditary, Kjer Optic Atrophy
- Interventions
- Not listed
- Lead sponsor
- PYC Therapeutics
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 2
- States / cities
- Miami, Florida • Seattle, Washington
- Conditions
- Frontotemporal Degeneration(FTD), Primary Progressive Aphasia(PPA), Familial Frontotemporal Lobar Degeneration (fFTLD), Amyotrophic Lateral Sclerosis(ALS), Lewy Body Disease(LBD), Progressive Supranuclear Palsy(PSP), Corticobasal Syndrome(CBS), Posterior Cortical Atrophy(PCA), Alzheimer's Disease(AD)
- Interventions
- No intervention
- Other
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2070
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Parkinson Disease, Multiple System Atrophy, Progressive Supranuclear Palsy
- Interventions
- Not listed
- Lead sponsor
- The Parkinson Study Group
- Network
- Eligibility
- 18 Years and older
- Enrollment
- 200 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- Started 2007
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Frontotemporal Degeneration (FTD), ALS (Amyotrophic Lateral Sclerosis), Primary Progressive Aphasia (PPA), Familial Frontotemporal Lobar Degeneration (fFTLD), Fronto-temporal Lobar Dementia, Dementia With Lewy Bodies (DLB), Cortical Basal Syndrome (CBS), Alzheimer's Disease (AD), Progressive Supranuclear Palsy(PSP), Parkinson's Disease (PD), Parkinson's Disease Dementia (PDD), Limbic-predominant Age-related TDP-43 Encephalopathy (LATE), Posterior Cortical Atrophy (PCA), Primary Lateral Sclerosis (PLS), Primary Muscular Atrophy (PMA)
- Interventions
- Not listed
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2070
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Parkinson Disease, Multiple System Atrophy, Progressive Supranuclear Palsy
- Interventions
- Droxidopa, Placebo Oral Tablet
- Drug
- Lead sponsor
- Loma Linda University
- Other
- Eligibility
- 50 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Loma Linda, California
- 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
- Wolfram Syndrome
- Interventions
- Not listed
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- 1 Day and older
- Enrollment
- 128 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2023
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Retinal Disease, Macular Degeneration, Hereditary Retinal Dystrophy, Optic Nerve Disease, Glaucoma
- Interventions
- RB (Retrobulbar), ST (Subtenon), IV (Intravenous), IVIT (Intravitreal), IO (Intraocular)
- Procedure
- Lead sponsor
- MD Stem Cells
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2020
- U.S. locations
- 1
- States / cities
- Westport, Connecticut
- Conditions
- Essential Tremor, Multiple System Atrophy, Corticobasal Degeneration, Supranuclear Palsy, Progressive, Parkinson Disease
- Interventions
- Not listed
- Lead sponsor
- University of Colorado, Denver
- Other
- Eligibility
- 40 Years and older
- Enrollment
- 81 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2014
- U.S. locations
- 1
- States / cities
- Aurora, Colorado
- Conditions
- Parkinson Disease, Multiple System Atrophy, Progressive Supranuclear Palsy
- Interventions
- Not listed
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- Not listed
- Enrollment
- 90 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2026
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Parkinson Disease, Progressive Supranuclear Palsy, Multiple System Atrophy, Dementia With Lewy Bodies
- Interventions
- Not listed
- Lead sponsor
- University of Illinois at Urbana-Champaign
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 485 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 2
- States / cities
- Denver, Colorado • Urbana, Illinois
- Conditions
- Leber Hereditary Optic Neuropathy
- Interventions
- Patient-reported outcomes (PROs)
- Other
- Lead sponsor
- GenSight Biologics
- Industry
- Eligibility
- Not listed
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 5
- States / cities
- Pasadena, California • Atlanta, Georgia • Boston, Massachusetts + 2 more
- Conditions
- Parkinson Disease, Parkinsonism, Dementia With Lewy Bodies, Multiple System Atrophy, Progressive Supranuclear Palsy, Corticobasal Degeneration
- Interventions
- punch skin biopsy
- Procedure
- Lead sponsor
- University Hospitals Cleveland Medical Center
- Other
- Eligibility
- 21 Years to 89 Years
- Enrollment
- 184 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2025
- U.S. locations
- 2
- States / cities
- Cleveland, Ohio • South Euclid, Ohio
- 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
- 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