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Showing 97–120 of 162 matching trials from the live ClinicalTrials.gov search.
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
3
States / cities
Phoenix, Arizona • Cincinnati, Ohio • Dallas, Texas
Conditions
Stargardt's Macular Dystrophy
Interventions
MA09-hRPE
Biological
Lead sponsor
Astellas Institute for Regenerative Medicine
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
3
States / cities
Los Angeles, California • Miami, Florida • Philadelphia, Pennsylvania
Conditions
Retinitis Pigmentosa
Interventions
Hydroxychloroquine lower dose, Hydroxychloroquine higher dose
Drug
Lead sponsor
University of Michigan
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
Ann Arbor, Michigan
Conditions
Stargardt's Macular Dystrophy
Interventions
avacincaptad pegol, Sham
Drug
Lead sponsor
Astellas Pharma Global Development, Inc.
Industry
Eligibility
18 Years to 60 Years
Enrollment
121 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
17
States / cities
Phoenix, Arizona • Los Angeles, California • Gainesville, Florida + 14 more
Conditions
Coats' Disease, Idiopathic Retinal Telangiectasia, Retinal Angiomatous Proliferation, Polypoidal Choroidal Vasculopathy, Pseudoxanthoma Elasticum, Pathological Myopia, Multi-focal Choroiditis, Rubeosis Iridis, Von Hippel Lindau Disease, BEST VITELLIFORM MACULAR DYSTROPHY, MULTIFOCAL (Disorder)
Interventions
ranibizumab injection (0.5 mg)
Drug
Lead sponsor
Manhattan Eye, Ear & Throat Hospital
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
2
States / cities
New York, New York
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
Recruiting No phase listed Observational Accepts healthy volunteers

High Resolution Retinal Imaging

NCT01866371
Conditions
Stargardts, Retinitis Pigmentosa, Age-related Macular Degeneration, Choroideremia, Geographic Atrophy
Interventions
Retinal imaging
Procedure
Lead sponsor
University of Pennsylvania
Other
Eligibility
7 Years and older
Enrollment
600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2031
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Retinitis Pigmentosa
Interventions
DSP-3077 Retinal Sheet Cohort 1, DSP-3077 Retinal Sheet Cohort 2, DSP-3077 Retinal Sheet Cohort 3
Drug
Lead sponsor
Sumitomo Pharma America, Inc.
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
2
States / cities
Boston, Massachusetts • Minneapolis, Minnesota
Active, not recruiting Phase 1 Interventional

FIGHT-RP 1 Extension Study

NCT03999021
Conditions
Retinitis Pigmentosa
Interventions
NAC effervescent tablets
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2030
U.S. locations
1
States / cities
Baltimore, Maryland
Conditions
Retinitis Pigmentosa
Interventions
CNTO 2476
Drug
Lead sponsor
Centocor, Inc.
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
3
States / cities
Miami, Florida • Southwest Portland, Oregon • Dallas, Texas
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
Completed Phase 1 Interventional Accepts healthy volunteers

Phase 1 Pilot Study of 4-MP to Treat Stargardt Macular Dystrophy

NCT00346853
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
Completed Not applicable Interventional Accepts healthy volunteers Results available

Environmental Localization Mapping and Guidance for Visual Prosthesis Users

NCT04359108
Conditions
Retinitis Pigmentosa, Visual Impairment, Visual Prosthesis
Interventions
Navigation system mode: Argus Vision, Navigation system mode: Depth Vision, Navigation system mode: Depth Vision with Haptic / Audio, Navigation system mode: Haptic / Audio, Navigation system mode: High Field-of-View Depth Vision, Distance test vision mode: Low Resolution / Low Field-of-View, Distance test vision mode: Low Resolution / High Field-of-View, Distance test vision mode: High Resolution / Low Field-of-View, Distance test vision mode: High Resolution / High Field-of-View
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
26 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2024
U.S. locations
2
States / cities
Baltimore, Maryland • Laurel, Maryland
Completed Not applicable Interventional Accepts healthy volunteers Results available

The Perceptual Experience of Argus II Users

NCT05285618
Conditions
Retinitis Pigmentosa
Interventions
Argus II
Device
Lead sponsor
University of California, Santa Barbara
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2023
U.S. locations
3
States / cities
Santa Barbara, California • Baltimore, Maryland • Ann Arbor, Michigan
Active, not recruiting Phase 3 Interventional Results available

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

NCT00999609
Conditions
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Interventions
AAV2-hRPE65v2,voretigene neparvovec-rzyl
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
3 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2030
U.S. locations
2
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania
Conditions
Retinitis Pigmentosa, Leber Congenital Amaurosis
Interventions
OCU400 Low Dose, OCU400 Med Dose, OCU400 High Dose, OCU400 Second Eye Dosing
Drug
Lead sponsor
Ocugen
Industry
Eligibility
6 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
7
States / cities
Phoenix, Arizona • La Jolla, California • Miami, Florida + 4 more
Conditions
Stargardt Disease
Interventions
Not listed
Lead sponsor
University of Michigan
Other
Eligibility
5 Years to 18 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Ann Arbor, Michigan
Conditions
LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)
Interventions
QLT091001
Drug
Lead sponsor
QLT Inc.
Industry
Eligibility
5 Years to 65 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
2
States / cities
Chicago, Illinois • Baltimore, Maryland
Conditions
Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease, Retinal Degeneration, Vision Disorders, Eye Disorders Congenital
Interventions
EDIT-101
Drug
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
3 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
5
States / cities
Miami, Florida • Boston, Massachusetts • Ann Arbor, Michigan + 2 more
Conditions
Non-syndromic Retinitis Pigmentosa
Interventions
Gene therapy: GS030-DP AND Medical device: GS030-MD
Combination Product
Lead sponsor
GenSight Biologics
Industry
Eligibility
18 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Cystoid Macular Edema, Fuchs Dystrophy
Interventions
Optical Coherence Tomography
Diagnostic Test
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Early Check: Expanded Screening in Newborns

NCT03655223
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
Advanced Retinitis Pigmentosa
Interventions
AGN-151597
Drug
Lead sponsor
AbbVie
Industry
Eligibility
18 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
4
States / cities
San Francisco, California • Edgewood, Kentucky • Durham, North Carolina + 1 more
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