Clinical Trials on Leber Congenital Amaurosis (LCA)
Total 49 results
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Atsena Therapeutics Inc.Active, not recruitingLeber Congenital Amaurosis | LCA | LCA1United States
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QLT Inc.CompletedLCA (Leber Congenital Amaurosis) | RP (Retinitis Pigmentosa)Canada, United States, Germany, Netherlands, United Kingdom
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QLT Inc.CompletedLCA (Leber Congenital Amaurosis) | RP (Retinitis Pigmentosa)United States, Canada, Germany, Netherlands, United Kingdom
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QLT Inc.CompletedLeber Congenital Amaurosis (LCA) | Retinitis Pigmentosa (RP)United States, Canada, Denmark, Germany, Netherlands, Switzerland, United Kingdom
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MeiraGTx UK II LtdSyne Qua Non LimitedCompletedEye Diseases | Retinal Diseases | Eye Diseases, Hereditary | Leber Congenital Amaurosis (LCA)United States, United Kingdom
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ProQR TherapeuticsActive, not recruitingEye Diseases | Neurologic Manifestations | Eye Diseases, Hereditary | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders CongenitalUnited States, Belgium, Brazil, Canada, France, Germany, Italy, Netherlands, United Kingdom
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University of PennsylvaniaNational Eye Institute (NEI)Active, not recruitingRetinal Diseases | Amaurosis of LeberUnited States
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ProQR TherapeuticsTerminatedEye Diseases | Neurologic Manifestations | Eye Diseases, Hereditary | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders CongenitalUnited States, Belgium
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ProQR TherapeuticsRecruitingEye Diseases | Neurologic Manifestations | Retinal Degeneration | Retinal Dystrophies | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders CongenitalBelgium, Brazil, Canada, Germany, Italy, Netherlands, United Kingdom
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Eyecure Therapeutics Inc.Beijing Tongren HospitalUnknownLeber Congenital Amaurosis, Retinitis PigmentosaChina
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ProQR TherapeuticsCompletedLeber's Congenital AmaurosisUnited States, Belgium
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University of Campania "Luigi Vanvitelli"Retina Italia OnlusCompletedLeber Congenital Amaurosis 2 | Retinitis Pigmentosa 20Italy
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Editas Medicine, Inc.Active, not recruitingRetinal Degeneration | Eye Diseases, Hereditary | Vision Disorders | Leber Congenital Amaurosis 10 | Inherited Retinal Dystrophies | Retinal Disease | Eye Disorders CongenitalUnited States
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Editas Medicine, Inc.CompletedEye Diseases | Retinal Degeneration | Eye Diseases, Hereditary | Vision Disorders | Blindness | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders CongenitalUnited States, Netherlands, France, Germany
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HuidaGene Therapeutics Co., Ltd.Xinhua Hospital, Shanghai Jiao Tong University School of MedicineRecruitingLeber Congenital AmaurosisChina
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Spark TherapeuticsActive, not recruitingLeber Congenital AmaurosisUnited States
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MeiraGTx UK II LtdCompletedNatural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65Leber Congenital AmaurosisUnited Kingdom, United States
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University College, LondonMoorfields Eye Hospital NHS Foundation Trust; Targeted Genetics CorporationCompletedRetinal DegenerationUnited Kingdom
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Shanghai General Hospital, Shanghai Jiao Tong University...Not yet recruitingTo Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)
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Xinhua Hospital, Shanghai Jiao Tong University...HuidaGene Therapeutics Co., Ltd.RecruitingLeber Congenital AmaurosisChina
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Assistance Publique - Hôpitaux de ParisCompletedLeber Congenital AmaurosisFrance
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Spark TherapeuticsCompleted
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Applied Genetic Technologies CorpOregon Health and Science University; University of Massachusetts, WorcesterCompletedLeber Congenital AmaurosisUnited States
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MeiraGTx UK II LtdCompletedLeber Congenital AmaurosisUnited Kingdom, United States
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Hadassah Medical OrganizationCompletedLeber Congenital AmaurosisIsrael
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Nantes University HospitalCompletedLeber Congenital AmaurosisFrance
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HuidaGene Therapeutics Co., Ltd.Cholgene Therapeutics, Inc.RecruitingLeber Congenital Amaurosis | Inherited Retinal Diseases Caused by RPE65 MutationsUnited States, China
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OcugenRecruitingRetinitis Pigmentosa | Leber Congenital AmaurosisUnited States
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Spark Therapeutics, Inc.Children's Hospital of Philadelphia; University of IowaActive, not recruitingLeber Congenital Amaurosis | Inherited Retinal Dystrophy Due to RPE65 MutationsUnited States
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Qlaris Bio, Inc.CompletedOcular Hypertension | Glaucoma | Glaucoma Congenital | Sturge-Weber Syndrome (SWS)United States
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University Hospital, ToursCompletedPort Wine Stain | Parkes Weber Syndrome | Klippel Trenaunay SyndromeFrance
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Fundacion para la Investigacion Biomedica del Hospital...Sociedad Española De Medicina InternaActive, not recruitingHereditary Hemorrhagic Telangiectasia | HHT | Rendu Osler Weber DiseaseSpain
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National Human Genome Research Institute (NHGRI)CompletedTelangiectasia, Hereditary Hemorrhagic | Osler-Rendu-Weber Disease | Osler-Rendu DiseaseUnited States
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Center for Vascular Pathology, MoscowCompletedVascular Diseases | Port-Wine Stain | Sturge-Weber Syndrome | Vascular Malformation | Capillary Malformation-Arteriovenous Malformation | Vascular TumorRussian Federation
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University of California, Los AngelesUniversité de MontréalWithdrawnStroke | Epilepsy | Tuberous Sclerosis | Sturge-Weber Syndrome | Focal Cortical Dysplasia | Hemimegalencephaly | Polymicrogyria | Rasmussen Encephalitis | Gliosis | Tumor, BrainUnited States, Canada
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Children's Hospital Medical Center, CincinnatiLymphangiomatosis and Gorham's Disease Alliance (LGDA); Klippel Trenaunay (KT)... and other collaboratorsRecruitingKaposiform Hemangioendothelioma | Kaposiform Lymphangiomatosis | Vascular Anomaly | Klippel Trenaunay Syndrome | Generalized Lymphatic Anomaly | Gorham-Stout Disease | Congenital Lipomatous Overgrowth, Vascular Malformations, and Epidermal NeviUnited States
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Mayo ClinicCompletedHemangioma | Arteriovenous Malformations | Vascular Malformations | Lymphatic Malformation | Venous Malformation | Vascular Anomaly | Klippel Trenaunay SyndromeUnited States
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Foundation Fighting BlindnessRecruitingRetinitis Pigmentosa | Choroideremia | Usher Syndrome | Batten Disease | Leber Congenital Amaurosis | Goldmann-Favre Syndrome | Kearns-Sayre Syndrome | Retinal Disease | Bardet-Biedl Syndrome | Stargardt Disease | Cone Dystrophy | Retinoschisis | Achromatopsia | Gyrate Atrophy | Eye Diseases Hereditary | Bassen-Kornzweig... and other conditionsUnited States
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University Hospital, BordeauxAMRO-HHT-France - Association Maladie de Rendu-OslerCompletedHereditary Hemorrhagic Telangiectasia | Osler Weber Rendu DiseaseFrance
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Premier Specialists, AustraliaUnknownEpidermolysis Bullosa Simplex | Epidermolysis Bullosa Simplex Kobner | Weber-Cockayne SyndromeAustralia
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Stanford UniversityCompletedEpidermolysis Bullosa Simplex | Epidermolysis Bullosa Simplex Kobner | Weber-Cockayne SyndromeUnited States
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Institut National de la Santé Et de la Recherche...RecruitingVascular Malformations | PIK3CA-related Overgrowth Spectrum | Megalencephaly | Lymphatic Malformation | CLOVES Syndrome | Venous Malformation | Klippel Trenaunay Syndrome | MCAP | MacrodactylyFrance
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Diane GeorgeActive, not recruitingSickle Cell Disease | Hemoglobinopathies | Severe Congenital Neutropenia | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Macrophage Activation Syndrome | Bone Marrow Failure Syndrome | Amegakaryocytic Thrombocytopenia | Schwachman Diamond Syndrome | Primary Immunodeficiency Syndromes | Acquired Immunodeficiency... and other conditionsUnited States
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Boston Children's HospitalLymphatic Malformation InstituteRecruitingLymphangiomatosis | Kaposiform Lymphangiomatosis | Lymphatic Malformation | Generalized Lymphatic Anomaly (GLA) | Central Conducting Lymphatic Anomaly | CLOVES Syndrome | Gorham-Stout Disease ("Disappearing Bone Disease") | Blue Rubber Bleb Nevus Syndrome | Kaposiform Hemangioendothelioma/Tufted Angioma and other conditionsUnited States
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National Human Genome Research Institute (NHGRI)RecruitingMetabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditionsUnited States
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States
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Children's Hospital of PhiladelphiaIllumina, Inc.Active, not recruitingMucopolysaccharidoses | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2 Gangliosidosis | Zellweger... and other conditionsUnited States
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Children's Hospital of PhiladelphiaEli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaboratorsRecruitingMucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... and other conditionsUnited States
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Sanford HealthNational Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaboratorsRecruitingMitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditionsUnited States, Australia