- Conditions
- Retinitis Pigmentosa
- Interventions
- Ciliary Neurotrophic Factor Implant NT-501
- Drug
- Lead sponsor
- National Eye Institute (NEI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2006
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
Search Results
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Showing 25–48
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- Conditions
- Stargardt Disease, Stargardt Macular Dystrophy, Stargardt-like Macular Dystrophy
- Interventions
- ASP2020
- Drug
- Lead sponsor
- Astellas Institute for Regenerative Medicine
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 1
- States / cities
- Phoenix, Arizona
- Conditions
- Macular Dystrophy, Corneal
- Interventions
- 4-Methylpyrazole, saline
- Drug · Other
- Lead sponsor
- University of Utah
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2005 – 2006
- U.S. locations
- 1
- States / cities
- Salt Lake City, Utah
- Conditions
- Retinal Detachment, Epiretinal Membrane, Macular Hole, Fuchs Endothelial Dystrophy
- Interventions
- Not listed
- Lead sponsor
- The Cleveland Clinic
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 750 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2017
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio
- Conditions
- Usher's Syndrome
- Interventions
- Blood draw for the laboratory assessment
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2031
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- 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
- Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Degeneration
- Interventions
- Gene Therapy Product-MCO-010, Sham Injection
- Biological · Procedure
- Lead sponsor
- Nanoscope Therapeutics Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 5
- States / cities
- Beverly Hills, California • Pensacola, Florida • Fargo, North Dakota + 2 more
- Conditions
- Retinitis Pigmentosa
- Interventions
- N-Acetyl Cysteine (NAC)
- Drug
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Degeneration
- Interventions
- Gene Therapy product-MCO-010
- Biological
- Lead sponsor
- Nanoscope Therapeutics Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 5
- States / cities
- Beverly Hills, California • Pensacola, Florida • Fargo, North Dakota + 2 more
- Conditions
- Retinitis Pigmentosa
- Interventions
- Object recognition subsystem
- Device
- Lead sponsor
- Minnesota HealthSolutions
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2022
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- X-Linked Retinitis Pigmentosa
- Interventions
- BIIB112
- Biological
- Lead sponsor
- Biogen
- Industry
- Eligibility
- 10 Years and older · Male only
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2020
- U.S. locations
- 5
- States / cities
- Gainesville, Florida • Miami, Florida • Portland, Oregon + 2 more
- Conditions
- Autosomal Dominant Retinitis Pigmentosa, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Disease, Retinitis, Vision Tunnel, Vision Disorders
- Interventions
- QR-1123, Sham procedure
- Drug · Other
- Lead sponsor
- ProQR Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 5
- States / cities
- Aurora, Colorado • Gainesville, Florida • Lexington, Kentucky + 2 more
- Conditions
- Retinitis Pigmentosa
- Interventions
- OCU400
- Drug
- Lead sponsor
- Ocugen
- Industry
- Eligibility
- 18 Years to 99 Years
- U.S. locations
- 5
- States / cities
- Phoenix, Arizona • Deerfield Beach, Florida • Erie, Pennsylvania + 2 more
- Conditions
- Stargardt Disease, Stargardt Macular Dystrophy, Stargardt-like Macular Dystrophy
- Interventions
- No Intervention
- Other
- Lead sponsor
- Astellas Pharma Global Development, Inc.
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 90 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 15
- States / cities
- Phoenix, Arizona • Palo Alto, California • Sacramento, California + 12 more
- Conditions
- Retinal Degeneration, Age-Related Macular Degeneration, Retinitis Pigmentosa, Hydroxychloroquine Retinopathy, Usher Syndromes, Late-Onset Retinal Degeneration, Cone Dystrophy, Cone Rod Dystrophy, Rod Cone Dystrophy, Rod Dystrophy
- Interventions
- Adaptive optics imaging
- Device
- Lead sponsor
- Food and Drug Administration (FDA)
- Federal
- Eligibility
- 21 Years and older
- Enrollment
- 100 participants
- Timeline
- 2021 – 2028
- U.S. locations
- 2
- States / cities
- Bethesda, Maryland • Silver Spring, Maryland
- Conditions
- Retinitis Pigmentosa
- Interventions
- Ultrasound Stimulus
- Procedure
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 1
- States / cities
- New York, New York
- 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
- Smith-Lemli-Opitz Syndrome, Cone-Rod Dystrophy, Hearing Loss
- Interventions
- Antioxidants, Cholesterol
- Drug
- Lead sponsor
- University of Colorado, Denver
- Other
- Eligibility
- Up to 65 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2025
- U.S. locations
- 1
- States / cities
- Aurora, Colorado
- Conditions
- Mitochondrial Myopathies, Mitochondrial DNA Mutation, Mitochondrial Diseases, Chronic Progressive External Ophthalmoplegia With Myopathy, Kearns-Sayre Syndrome
- Interventions
- Not listed
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 15 Years to 80 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy
- Interventions
- OPGx-BEST1
- Genetic
- Lead sponsor
- Opus Genetics, Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Gainesville, Florida • Cincinnati, Ohio + 1 more
- Conditions
- Retinitis Pigmentosa
- Interventions
- CAREN system training
- Device
- Lead sponsor
- The Cleveland Clinic
- Other
- Eligibility
- 25 Years to 100 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio
- Conditions
- Retinal Dystrophy
- Interventions
- nL-PRPH2-001
- Drug
- Lead sponsor
- n-Lorem Foundation
- Other
- Eligibility
- Female only
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- San Diego, California
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
- Interventions
- QR-421a, Sham-procedure (dose cohort 1&2 only)
- Drug · Other
- Lead sponsor
- ProQR Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 4
- States / cities
- Boston, Massachusetts • Ann Arbor, Michigan • Portland, Oregon + 1 more
- Conditions
- Retinitis Pigmentosa
- Interventions
- Lutein (10 or 30 mg/day) capsules
- Drug
- Lead sponsor
- National Center for Complementary and Integrative Health (NCCIH)
- NIH
- Eligibility
- Not listed
- Timeline
- 2001 – 2002
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland