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Clinical Trials on Leber Congenital Amaurosis 2
Total 96 results
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Hospital Universitari Vall d'Hebron Research InstituteRecruitingNeural Tube Defects | Chiari Malformation Type 2 | Myelomeningocele | Surgery; Maternal, Uterus or Pelvic Organs, Affecting Fetus | Hydrocephalus | Congenital Abnormality | Fetal Disease | Spina Bifida, OpenSpain
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Stanford UniversityCompletedEpidermolysis Bullosa Simplex | Epidermolysis Bullosa Simplex Kobner | Weber-Cockayne SyndromeUnited States
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AORTICA GroupSalamanca University Hospital; Instituto de Investigación Biomédica de SalamancaCompletedCOVID-19 | SARS-CoV 2 | Myocarditis | Coronavirus | Cardiac Magnetic Resonance | Cardiac AnomalySpain
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Washington University School of MedicineActive, not recruitingHeart Diseases | Virus Diseases | Diabetes Mellitus, Type 2 | Diabetes Mellitus | Diabetes | Diabetes Mellitus, Type 1 | Enterovirus Infections | Pregnancy Complications | Heart Defects, Congenital | Viremia | Congenital Heart Disease | Pregnancy in Diabetic | Enterovirus | Prenatal Infection | PrenatalUnited States
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Vancouver General HospitalNovo Nordisk A/STerminated
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UMC UtrechtSanquin Research & Blood Bank Divisions; National Institute for Public Health... and other collaboratorsRecruitingDown Syndrome | SARS-CoV-2 VaccinationNetherlands
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Institut National de la Santé Et de la Recherche...RecruitingLaminopathies | Emery Dreifuss Muscular Dystrophy 2 | LMNA-Related Congenital Muscular Dystrophy | Dilated Cardiomyopathy-1AFrance
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University of California, IrvineAnn & Robert H Lurie Children's Hospital of Chicago; Children's Hospital Los... and other collaboratorsRecruitingCystic Fibrosis | Sickle Cell Disease | SARS CoV 2 InfectionUnited 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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Hospital Israelita Albert EinsteinCompletedChiari Malformation Type 2 | Myelomeningocele | Meningomyelocele | Open Spina Bifida | Chiari Malformation With Spina BifidaBrazil
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ProQR TherapeuticsCompletedEye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf BlindUnited States, Canada, France
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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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ProQR TherapeuticsTerminatedEye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf BlindUnited States, United Kingdom
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Ottawa Heart Institute Research CorporationCompletedPulmonary Arterial Hypertension | Pulmonary Hypertension, Primary, 4 | Chronic Right-Sided Heart Failure | Pulmonary Hypertension, Primary, 2 | Pulmonary Hypertension, Primary, 3 | Cardiomyopathy Right VentricularCanada
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ProQR TherapeuticsActive, not recruitingEye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf BlindUnited States, Netherlands, United Kingdom, Germany
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Regeneron PharmaceuticalsRecruitingCongenital Hearing Loss Secondary to Biallelic Mutations in the Otoferlin Gene (OTOF) | Biallelic Mutations in the Gap Junction Beta 2 (GJB2) Gene | Digenic Mutations in GJB2/Gap Junction Beta 6 (GJB6) GenesUnited States
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University Hospital, AngersUnknownType 2 Diabetes | Chronic Kidney Disease | Pseudoxanthoma ElasticumFrance
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University of ThessalyCompletedDiabetes Mellitus, Type 2 | Insulin Resistance | PreDiabetes | Beta-ThalassemiaGreece
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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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University of RochesterNational Institute of Neurological Disorders and Stroke (NINDS)RecruitingMuscular Dystrophy | Myotonic Dystrophy Type 2 | Myotonic Dystrophy Type 1 | Myotonic Dystrophy | Facioscapulohumeral Muscular Dystrophy | Steinert's Disease | Congenital Myotonic Dystrophy | PROMM (Proximal Myotonic Myopathy) | Myotonic Muscular DystrophyUnited States
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Medical University of ViennaUnknownPolycystic Kidney, Type 1 Autosomal Dominant Disease | Polycystic Kidney, Type 2 Autosomal Dominant DiseaseAustria
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University Hospital, AngersRecruitingCharcot-Marie-Tooth Disease Type 2AFrance
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ProQR TherapeuticsTerminatedRetinitis Pigmentosa | Usher Syndrome Type 2United States, Canada, France
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Neurotech PharmaceuticalsUniversity of California, San FranciscoCompletedRetinitis Pigmentosa | Usher Syndrome Type 2 | Usher Syndrome Type 3United States
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University Hospital ErlangenCompletedHypohidrotic Ectodermal DysplasiaGermany
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Radboud University Medical CenterUnknownMyotonic Dystrophy Type 2 | McArdle Disease | Nemaline Myopathy Type 6
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University College, LondonWashington University School of Medicine; University of Washington; Alzheimer... and other collaboratorsNot yet recruitingAlzheimer Disease | Autosomal Dominant Alzheimer Disease Due to Mutation of Presenilin 1 (Disorder) | Autosomal Dominant Alzheimer Disease Due to Mutation of Presenilin 2 (Disorder) | Autosomal Dominant Alzheimer Disease Due to Mutation of Amyloid Precursor Protein (Disorder)
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University Hospital, Clermont-FerrandUnknownDelirium | SARS-CoV-2 Infection | Encephalopathy | Intensive Care Unit Patient | Neurological AbnormalityFrance
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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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Kobe UniversityBoehringer IngelheimActive, not recruitingInsulin Resistance - Type A | Insulin Resistance - Type B | Lipoatrophic Diabetes Mellitus | Insulin Resistance SyndromeJapan
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University College, LondonUniversity of IowaUnknownCharcot-Marie-Tooth Disease, Type IA | Charcot-Marie-Tooth Disease Type 2A | Charcot-Marie-Tooth Disease, Type IB | Charcot-Marie-Tooth Disease, Type XUnited Kingdom
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Kobe UniversityBoehringer IngelheimActive, not recruitingInsulin Resistance - Type A | Insulin Resistance - Type B | Lipoatrophic Diabetes Mellitus | Insulin Resistance SyndromeJapan
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University of ChicagoJohns Hopkins University; Mayo Clinic; National Institute of Neurological Disorders... and other collaboratorsCompletedCerebral Cavernous Malformation | Cavernous Angioma | Cavernoma | CCM | Cerebral Cavernous Malformations 1 | Cerebral Cavernous Malformations 2 | Cerebral Cavernous Malformations 3United States
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Chinook Therapeutics U.S., Inc.Active, not recruitingDiabetic Kidney Disease | Focal Segmental Glomerulosclerosis | Immunoglobulin A Nephropathy | IgA Nephropathy | Diabetic Nephropathy Type 2 | Alport SyndromeUnited States, Spain, Korea, Republic of, Australia, Italy, United Kingdom
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McGill University Health Centre/Research Institute...RecruitingPeroxisome Biogenesis Disorder | Zellweger Spectrum Disorder | RCDP - Rhizomelic Chondrodysplasia Punctata | D-Bifunctional Protein Deficiency | Alpha-Methylacyl-CoA Racemase Deficiency | Peroxisomal Acyl-CoA Oxidase Deficiency | Peroxisomal Acyl-CoA Oxidase 2 Deficiency | ATP Binding Cassette Subfamily... and other conditionsCanada
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National Medical Research Center for Therapy and...Moscow State University of Medicine and DentistryRecruitingMedication Adherence | Adherence, Medication | Treatment Adherence | Familial Hypercholesterolemia | Motivational Interviewing | Adherence, Patient | Treatment Adherence and Compliance | Patient Compliance | Adherence | Hypercholesterolemia, Familial | Patient Adherence | Hypercholesterolemia, Autosomal Dominant and other conditionsRussian Federation
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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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Jesse NodoraNational Cancer Institute (NCI)TerminatedCoronary Artery Disease | Heart Failure | Obesity | Diabetes Mellitus, Type 2 | Cancer | Chronic Kidney Diseases | Sickle Cell Disease | COPDUnited States
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St. Jude Children's Research HospitalRecruitingPancreatic Cancer | Hodgkin Lymphoma | Lynch Syndrome | Tuberous Sclerosis | Fanconi Anemia | AML | Non Hodgkin Lymphoma | Familial Adenomatous Polyposis | Acute Leukemia | Nevoid Basal Cell Carcinoma Syndrome | Neurofibromatosis Type 1 | Neuroblastoma | Retinoblastoma | MDS | Rhabdomyosarcoma | Von Hippel-Lindau Disease | Adrenocortical Carcinoma and other conditionsUnited States
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McGill University Health Centre/Research Institute...RecruitingDiabetes Mellitus, Type 1 | Wolfram Syndrome | Neonatal Diabetes | Monogenic Diabetes | Maturity-onset Diabetes in the Young (MODY) | Wolcott-Rallison Syndrome | Mitochondrial DiabetesCanada
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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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Masonic Cancer Center, University of MinnesotaRecruitingHurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditionsUnited States
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Roswell Park Cancer InstituteNational Cancer Institute (NCI)CompletedPrimary Myelofibrosis | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent... and other conditionsUnited States
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Roswell Park Cancer InstituteCompletedPrimary Myelofibrosis | Polycythemia Vera | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma and other conditionsUnited States
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UK Kidney AssociationRecruitingVasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditionsUnited Kingdom
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Hereditary Neuropathy FoundationRecruitingCharcot-Marie-Tooth Disease | Charcot-Marie-Tooth Disease, Type IA | Charcot-Marie-Tooth Disease Type 2A | Charcot-Marie-Tooth | Charcot-Marie-Tooth Disease, Type IB | Charcot-Marie-Tooth Disease Type 2 | Charcot-Marie-Tooth Disease, Type 2C | Charcot-Marie-Tooth Disease Type 2A2B | Charcot-Marie-Tooth... and other conditionsUnited States