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Published and searchable clinical trials
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
NCT06891443
- Conditions
- Blindness, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +28 more
- Locations
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- UCSF Wayne and Gladys Valley Center for Vision San Francisco, California, United States
- University of Miami - Bascom Palmer Eye Institute Miami, Florida, United States
- University of Iowa Iowa City, Iowa, United States
- University of Minnesota Medical School Minneapolis, Minnesota, United States
NCT02435940
- Conditions
- Abetalipoproteinemia, Abnormalities, Multiple, Achromatopsia, +100 more
- Locations
-
- Foundation Fighting Blindness Columbia, Maryland, United States
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
NCT01793168
- Conditions
- 1p36 Deletion Syndrome, 3-Methylglutaconic Aciduria, Type V, 4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome, +698 more
- Locations
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- Sanford Health Sioux Falls, South Dakota, United States
- Online Patient Enrollment System Sydney, Australia
NCT05906953
- Conditions
- Eye Diseases, Eye Diseases, Hereditary, Inherited Retinal Diseases Caused by RPE65 Mutations, +2 more
- Locations
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- Research Site Sacramento, California, United States
- Research Site Houston, Texas, United States
- Research Site Shanghai, Shanghai Municipality, China
Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)
NCT07681778
- Conditions
- Eye Diseases, Eye Diseases, Hereditary, Leber Congenital Amaurosis, +2 more
- Locations
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- Associated Retina Consultants Phoenix, Arizona, United States
- Perelman School of Medicine, University of Pennsylvania Philadelphia, Pennsylvania, United States
- Retina Consultants of Texas & Retina Group Inc. Houston, Texas, United States
NCT00481546
- Conditions
- Amaurosis of Leber, Eye Diseases, Eye Diseases, Hereditary, +2 more
- Locations
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- Shands Children's Hospital, University of Florida Gainesville, Florida, United States
- Scheie Eye Institute, University of Pennsylvania Philadelphia, Pennsylvania, United States
NCT03597399
- Conditions
- Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, +7 more
- Locations
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- Children's Hospital of Los Angeles Los Angeles, California, United States
- Bascom Palmer Eye Institute Miami, Florida, United States
- University of Iowa Hospitals & Clinics Iowa City, Iowa, United States
- Massachusetts Eye and Ear Institute Boston, Massachusetts, United States
NCT02946879
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +4 more
- Locations
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- Kellogg Eye Center, University of Michigan Health Ann Arbor, Michigan, United States
- Moorfields Eye Hospital NHS Foundation Trust London, United Kingdom
Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65
NCT02714816
- Conditions
- Eye Diseases, Eye Diseases, Hereditary, Leber Congenital Amaurosis, +1 more
- Locations
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- Kellogg Eye Center Ann Arbor, Michigan, United States
- Moorfields Eye Hospital London, United Kingdom
NCT03396042
- Conditions
- Blindness, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +17 more
- Locations
-
- Bascom Palmer Eye Institute Miami, Florida, United States
- Massachusetts Eye and Ear Infirmary Boston, Massachusetts, United States
- W.K. Kellogg Eye Center Ann Arbor, Michigan, United States
- Casey Eye Institute - OHSU Portland, Oregon, United States
NCT02781480
- Conditions
- Eye Diseases, Eye Diseases, Hereditary, Leber Congenital Amaurosis, +1 more
- Locations
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- Kellogg Eye Centre, University of Michigan Ann Arbor, Michigan, United States
- Moorfields Eye Hospital NHS Foundation Trust London, United Kingdom
NCT00516477
- Conditions
- Eye Diseases, Eye Diseases, Hereditary, Leber Congenital Amaurosis, +1 more
- Locations
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- The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
NCT00749957
- Conditions
- Eye Diseases, Eye Diseases, Hereditary, Leber Congenital Amaurosis, +1 more
- Locations
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- University of Massachusetts Medical School Worcester, Massachusetts, United States
- Casey Eye Institue, Oregon Health & Science University Portland, Oregon, United States
Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
NCT02575430
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +8 more
- Locations
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- Wilmer Eye Institute - Johns Hopkins Hospital Baltimore, Maryland, United States
- Casey Eye Institute - Marquam Hill Portland, Oregon, United States
- The Hospital for Sick Children, Ophthalmology and Vision Sciences Toronto, Ontario, Canada
- Montreal Children's Hospital, McGill University Health Centre Montreal, Quebec, Canada
NCT01521793
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +8 more
- Locations
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- The Chicago Lighthouse (University of Illinois at Chicago, Pangere Center for Inherited Retinal Disease) Chicago, Illinois, United States
- Wilmer Eye Institute (Johns Hopkins University) Baltimore, Maryland, United States
- Montreal Children's Hospital, McGill University Health Centre Montreal, Quebec, Canada
- Institute for Ophthalmic Research, University of Tubingen Tübingen, Germany
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +8 more
- Locations
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- The Chicago Lighthouse for People Who Are Blind or Visually Impaired (The Pangere Center For Inherited Retinal Diseases) Chicago, Illinois, United States
- Wilmer Eye Institute (Johns Hopkins University) Baltimore, Maryland, United States
- Scheie Eye Institute Philadelphia, Pennsylvania, United States
- Montreal Children's Hospital, McGill University Health Centre Montreal, Quebec, Canada
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
NCT05203939
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Enhanced S-Cone Syndrome, Eye Diseases, +9 more
- Locations
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- Associated Retina Consultants Phoenix, Arizona, United States
- Ocugen Site 5 - University of California, San Diego (UCSD) - Shiley Eye Institute La Jolla, California, United States
- Ocugen Site 3 - Bascom Palmer Eye Institute Miami, Florida, United States
- Ocugen Site 6 - Emory University Atlanta, Georgia, United States
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
NCT01208389
- Conditions
- Eye Diseases, Eye Diseases, Hereditary, Leber Congenital Amaurosis, +1 more
- Locations
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- The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
NCT00999609
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +7 more
- Locations
-
- University of Iowa Iowa City, Iowa, United States
- Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
NCT03920007
- Conditions
- Eye Diseases, Eye Diseases, Hereditary, LCA, +4 more
- Locations
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- Casey Eye Institute - Oregon Health & Science University Portland, Oregon, United States
- Scheie Eye Institute, University of Pennsylvania Philadelphia, Pennsylvania, United States