Search clinical trials for: RPE65-associated Leber congenital amaurosis
Total 23903 results
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University of PennsylvaniaNational Eye Institute (NEI)Active, not recruitingRetinal Diseases | Amaurosis of LeberUnited States
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Hadassah Medical OrganizationCompletedLeber Congenital AmaurosisIsrael
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Xinhua Hospital, Shanghai Jiao Tong University...HuidaGene Therapeutics Co., Ltd.RecruitingLeber Congenital AmaurosisChina
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HuidaGene Therapeutics Co., Ltd.Xinhua Hospital, Shanghai Jiao Tong University School of MedicineRecruitingLeber Congenital AmaurosisChina
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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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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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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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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
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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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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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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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Spark TherapeuticsActive, not recruitingInherited Retinal Dystrophy Due to RPE65 Mutations
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Atsena Therapeutics Inc.Active, not recruitingLeber Congenital Amaurosis | LCA | LCA1United States
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Spark TherapeuticsActive, not recruitingConfirmed Biallelic RPE65 Mutation-associated Retinal DystrophyUnited States
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University of Campania "Luigi Vanvitelli"Retina Italia OnlusCompletedLeber Congenital Amaurosis 2 | Retinitis Pigmentosa 20Italy
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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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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 TherapeuticsCompletedLeber's Congenital AmaurosisUnited States, Belgium
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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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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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MeiraGTx UK II LtdCompletedLeber Congenital AmaurosisUnited Kingdom, United States
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Spark TherapeuticsCompleted
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Eyecure Therapeutics Inc.Beijing Tongren HospitalUnknownLeber Congenital Amaurosis, Retinitis PigmentosaChina
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Applied Genetic Technologies CorpOregon Health and Science University; University of Massachusetts, WorcesterCompletedLeber Congenital AmaurosisUnited States
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Assistance Publique - Hôpitaux de ParisCompletedLeber Congenital AmaurosisFrance
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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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University College, LondonMoorfields Eye Hospital NHS Foundation Trust; Targeted Genetics CorporationCompletedRetinal DegenerationUnited Kingdom
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Nantes University HospitalCompletedLeber Congenital AmaurosisFrance
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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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Nantes University HospitalCompletedRetinal Diseases | AmaurosisFrance
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Children's Mercy Hospital Kansas CityIndiana Clinical and Translational Sciences InstituteRecruitingFontan CirculationUnited States
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Mirum Pharmaceuticals, Inc.RecruitingAlagille SyndromeUnited States
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McGill University Health Centre/Research Institute...Canadian Institutes of Health Research (CIHR)RecruitingZellweger Spectrum DisorderCanada
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Lifetech Scientific (Shenzhen) Co., Ltd.Not yet recruitingAneurysm | Portal Hypertension | Arteriovenous Fistula | Endoleak | Pulmonary Arteriovenous Malformation | Splenic Laceration
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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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King's College LondonRecruitingLiver Diseases | Heart Diseases | Heart Failure | Stroke | Cerebrovascular Disorders | Epilepsy | Pain | Multiple Sclerosis | Hypertension | Obesity | Diabetes | Cancer | Arthritis | Osteoarthritis | Rheumatoid Arthritis | Parkinson Disease | Migraine | Hepatitis | Atrial Fibrillation | Asthma | Endometriosis | Dementia | Fibromyalgia | Celiac... and other conditionsUnited 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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National Taiwan University HospitalInsight Medical SolutionsRecruiting
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Tao ZhangRecruitingHypospadias | Caudal Block | Pediatrics | Postoperative Analgesia | S-ketamineChina
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Weibing TangRecruiting
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Tianjin First Central HospitalNot yet recruitingDesflurane | Biliary Atresia | Pediatric Acute Respiratory Distress SyndromeChina
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Centre Hospitalier Universitaire de LiegeSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaboratorsRecruitingCongenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditionsBelgium
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Sanguine BiosciencesRecruitingHeart Diseases | Stroke | Lymphoma | Multiple Sclerosis | Kidney Diseases | Diabetes | Leukemia | Age-Related Macular Degeneration | Cancer | Arthritis | Schizophrenia | Thyroid Diseases | Parkinson Disease | Hepatitis | Vasculitis | Psoriasis | Asthma | Amyloidosis | Fibromyalgia | Graves Disease | Alzheimer Disease | Celiac Disease | Cirr... and other conditionsUnited States
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Radboud University Medical CenterRecruitingAutosomal Dominant Polycystic Kidney | Polycystic Liver Disease | Liver Cyst | Autosomal Dominant Polycystic Liver DiseaseNetherlands
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Children's Hospital of Fudan UniversityRecruitingAlagille Syndrome | Atorvastatin | XanthomaChina
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University Medical Center GroningenRadboud University Medical CenterRecruitingAutosomal Dominant Polycystic Kidney | Polycystic Liver DiseaseNetherlands