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Clinical Trials on Leber Congenital Amaurosis in United States
Total 3906 results
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Simons SearchlightBoston Children's Hospital; Geisinger Clinic; Simons FoundationRecruitingSMARCA4 Gene Mutation | DDX3X | 16P11.2 Deletion Syndrome | 16p11.2 Duplications | 1Q21.1 Deletion | 1Q21.1 Microduplication Syndrome (Disorder) | ACTL6B | ADNP | AHDC1 | ANK2 | ANKRD11 | ARID1B | ASH1L | BCL11A | CHAMP1 | CHD2 | CHD8 | CSNK2A1 | CTBP1 | CTNNB1 Gene Mutation | CUL3 | DNMT3A | DSCAM | DYRK1A | FOXP1 | GRIN2A | GRIN2B | HIVEP2-Related Intellectual... and other conditionsUnited States
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Otsuka Pharmaceutical Development & Commercialization...CompletedAutosomal Dominant Polycystic Kidney Disease (ADPKD)United States, Australia, Belgium, Germany, Italy, Poland, Romania, United Kingdom, Argentina, Canada, France, Netherlands, Russian Federation
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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
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PellePharm, Inc.CompletedBasal Cell Nevus SyndromeUnited States, Spain, Germany, United Kingdom, Italy, France, Belgium, Canada, Denmark, Netherlands
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Heart of England NHS TrustUniversity of Warwick; University of BirminghamCompletedLung Neoplasms | Pulmonary Disease, Chronic Obstructive | Surgery | Cystic Fibrosis | Mesothelioma | Empyema, Pleural | Pectus Excavatum | Pectus CarinatumUnited Kingdom
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Marinus PharmaceuticalsActive, not recruitingTuberous Sclerosis ComplexUnited States, Spain, United Kingdom, Israel, Italy, Germany, France, Canada
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Otsuka Pharmaceutical Development & Commercialization...CompletedAutosomal Dominant Polycystic Kidney Disease (ADPKD)Germany, Italy, Belgium, United Kingdom
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Regulus Therapeutics Inc.RecruitingPolycystic Kidney, Autosomal Dominant | Autosomal Dominant Polycystic Kidney Disease | ADPKDUnited States
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University of California, San FranciscoNational Institute of Neurological Disorders and Stroke (NINDS)CompletedEpilepsy | Infantile Spasms | Lennox-Gastaut Syndrome | Polymicrogyria | Localization-related Epilepsy | Periventricular HeterotopiasUnited States, Argentina, Australia
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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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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsSaudi Arabia, Algeria, Turkey, Germany, Poland, Netherlands, Italy, Belgium, South Africa, United Kingdom, Sweden, Austria, Portugal, France, Czech Republic, Venezuela
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CelgeneRecruitingBeta-thalassemia | Myelodysplastic Syndromes (MDS) | Myeloproliferative Neoplasm(MPN)-Associated MyelofibrosisUnited States, Japan, United Kingdom, Turkey, Greece, France, Australia, Belgium, Bulgaria, Canada, Germany, Israel, Italy, Malaysia, Spain, Sweden, Taiwan, Thailand, Tunisia, Lebanon, China, Netherlands
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Hoffmann-La RocheCompletedBasal Cell CarcinomaUnited States, Spain, France, Germany, Netherlands, Russian Federation, Italy, Mexico, Austria, Canada
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Otsuka Pharmaceutical Development & Commercialization...Otsuka Pharmaceutical Co., Ltd.CompletedPolycystic Kidney Disease, Autosomal DominantUnited States, Belgium, United Kingdom, Russian Federation, France, Australia, Netherlands, Italy, Japan, Denmark, Romania, Poland, Canada, Argentina, Germany
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GRIN Therapeutics, Inc.RecruitingTuberous Sclerosis Complex | Focal Cortical DysplasiaSpain, United Kingdom, Australia, Belgium, Canada, Italy, Poland
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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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TScan Therapeutics, Inc.RecruitingMelanoma | Cervical Cancer | Head and Neck Cancer | HPV-Related Malignancy | Ovarian Cancer | HPV-Related Carcinoma | HPV-Related Cervical Carcinoma | HPV Positive Oropharyngeal Squamous Cell Carcinoma | Non-small Cell Carcinoma | HPV - Anogenital Human Papilloma Virus Infection | HPV-Related Adenocarcinoma | HPV-Related Adenosquamous Carcinoma and other conditionsUnited States
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Novartis PharmaceuticalsCompletedTuberous Sclerosis Complex-associated Refractory SeizuresUnited States, Colombia, Belgium, France, Japan, Taiwan, Turkey, Spain, Thailand, Korea, Republic of, Australia, Hungary, United Kingdom, Canada, Norway, Germany, Italy, Russian Federation, Denmark, Argentina, Netherlands, Greece, P... and more
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Novo Nordisk A/SCompletedChronic Kidney Disease | Turner Syndrome | Noonan Syndrome | Chronic Renal Insufficiency | Growth Hormone Disorder | Growth Hormone Deficiency in Children | Adult Growth Hormone Deficiency | Genetic Disorder | Foetal Growth Problem | Small for Gestational AgeIsrael, Saudi Arabia, Spain, Argentina, Russian Federation, Germany, Netherlands, Italy, Belgium, United Kingdom, Slovenia, Ireland, Sweden, Denmark, Hungary, Finland, Norway, France, Czechia, Lithuania, Luxembourg, Serbia, Switzerland
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Reata, a wholly owned subsidiary of BiogenTerminatedAutosomal Dominant Polycystic Kidney | ADPKDUnited States, Spain, United Kingdom, Australia, France, Japan, Germany, Belgium, Czechia, Italy
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Novartis PharmaceuticalsCompletedLymphangioleiomyomatosis (LAM) | Tuberous Sclerosis Complex (TSC)United States, United Kingdom, Germany, Italy, Russian Federation, Netherlands, Japan, Canada, Poland, France, Spain
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Laminate Medical TechnologiesCompletedKidney Diseases | Kidney Failure, Chronic | ESRD | Renal Failure Chronic | Arterio-Venous Fistula | Arteriovenous Fistula ThrombosisUnited Kingdom, Spain, Germany, France
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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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Children's Oncology GroupNational Cancer Institute (NCI)RecruitingDown Syndrome | Myeloid Leukemia Associated With Down Syndrome | B Acute Lymphoblastic Leukemia Associated With Down SyndromeUnited States
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Cure HHTUniversity of Colorado, Denver; Massachusetts General Hospital; Mayo Clinic; Children... and other collaboratorsRecruitingArteriovenous Malformations | Epistaxis | Hereditary Hemorrhagic Telangiectasia | GastroIntestinal Bleeding | Telangiectasia | Vascular Malformation | Cerebral Arteriovenous MalformationsUnited States
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Center for International Blood and Marrow Transplant...National Cancer Institute (NCI); National Heart, Lung, and Blood Institute... and other collaboratorsActive, not recruitingAcute Myeloid Leukemia | Myelodysplastic Syndromes | Hodgkin Lymphoma | Acute Lymphoblastic Leukemia | Sickle Cell Disease | Non-hodgkin Lymphoma | Acquired Aplastic AnemiaUnited 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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Otsuka Pharmaceutical Development & Commercialization...Otsuka Pharmaceutical Co., Ltd.CompletedAutosomal Dominant Polycystic Kidney Disease (ADPKD)United States, Argentina, Australia, Belgium, Brazil, Canada, Czech Republic, France, Germany, Italy, Japan, Netherlands, Norway, Poland, Romania, Spain, Sweden, Switzerland, Turkey, United Kingdom
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Novartis PharmaceuticalsWithdrawnBasal Cell CarcinomaGreece, Switzerland, Germany, United Kingdom, Belgium, Canada, Czechia, Finland, Italy, Spain
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National Cancer Institute (NCI)Childrens Oncology Group (COG); LLS PedAL LLCRecruitingDown Syndrome | Recurrent Acute Myeloid Leukemia | Recurrent Myelodysplastic Syndrome | Refractory Acute Myeloid Leukemia | Refractory Myelodysplastic Syndrome | Recurrent Mixed Phenotype Acute Leukemia | Refractory Mixed Phenotype Acute Leukemia | Acute Myeloid Leukemia Post Cytotoxic Therapy | Myelodysplastic...United States
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Otsuka Pharmaceutical Development & Commercialization...CompletedChronic Kidney Disease | Autosomal Dominant Polycystic Kidney DiseaseAustralia, Belgium, Italy, Spain, Sweden, United States, Canada, Netherlands, Czechia, Poland, Germany, Russian Federation, Hungary, Israel, Puerto Rico, Argentina, Denmark, South Africa, United Kingdom, Norway, France, Romania
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Children's Oncology GroupNational Cancer Institute (NCI)CompletedMyelodysplastic Syndromes | Leukemia | Myelodysplastic/Myeloproliferative NeoplasmsUnited States, Canada, Puerto Rico, Australia, Netherlands, New Zealand, Switzerland
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University of BirminghamActive, not recruitingWolfram SyndromeFrance, United Kingdom, Poland, Spain
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National Cancer Institute (NCI)SuspendedDown Syndrome | Recurrent B Acute Lymphoblastic LeukemiaUnited States, Canada, Puerto Rico, Australia
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Travere Therapeutics, Inc.RecruitingFocal Segmental Glomerulosclerosis | Immunoglobulin A Nephropathy | Minimal Change Disease | Alport Syndrome | IgA VasculitisUnited States, Italy, Netherlands, Poland, Spain, United Kingdom, Germany, Sweden
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National Cancer Institute (NCI)WithdrawnHematopoietic and Lymphoid Cell Neoplasm | Lynch Syndrome | Refractory Lymphoma | Refractory Malignant Solid Neoplasm | Recurrent Lymphoma | Recurrent Malignant Solid Neoplasm | Recurrent Neuroblastoma | Refractory Neuroblastoma | Recurrent Primary Central Nervous System Neoplasm | Refractory Primary Central... and other conditionsUnited States, Canada
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University of MiamiNational Institute of Neurological Disorders and Stroke (NINDS); St. Jude Children... and other collaboratorsEnrolling by invitationAmyotrophic Lateral Sclerosis | Primary Lateral Sclerosis | Frontotemporal Dementia | Progressive Muscular Atrophy | Hereditary Spastic Paraplegia | Multisystem ProteinopathyUnited States, South Africa, Germany
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National Cancer Institute (NCI)RecruitingDown Syndrome | B Acute Lymphoblastic Leukemia | B Lymphoblastic LymphomaUnited States, Canada, Puerto Rico, Australia, New Zealand
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Children's Oncology GroupNational Cancer Institute (NCI)Active, not recruitingAcute Myeloid Leukemia | Down Syndrome | Myelodysplastic Syndrome | Myeloproliferative Neoplasm | Myeloid Leukemia Associated With Down SyndromeUnited States, Canada, Saudi Arabia, Puerto Rico, Australia, New Zealand
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Atara BiotherapeuticsRecruitingLymphoproliferative Disorders | Leiomyosarcoma | Stem Cell Transplant Complications | Solid Organ Transplant Complications | Epstein-Barr Virus (EBV)-Associated Diseases | EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD) | Allogeneic Hematopoietic Cell Transplant | EBV+ Sarcomas | EBV+ Lymphoproliferative... and other conditionsUnited States, Austria, Belgium, France, Italy, Spain, United Kingdom
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Reata, a wholly owned subsidiary of BiogenCompletedFocal Segmental Glomerulosclerosis | IgA Nephropathy | Autosomal Dominant Polycystic Kidney | CKD Associated With Type 1 DiabetesUnited States
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Reata, a wholly owned subsidiary of BiogenTerminatedChronic Kidney Diseases | Autosomal Dominant Polycystic Kidney | Alport SyndromeUnited States, France, Australia, Japan, Spain, Puerto Rico
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Children's Oncology GroupNational Cancer Institute (NCI)CompletedMedulloblastoma | Nevoid Basal Cell Carcinoma Syndrome | Desmoplastic/Nodular Medulloblastoma | Medulloblastoma With Extensive NodularityUnited States, Canada, Puerto Rico, Australia
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American Thrombosis and Hemostasis NetworkSanofi; Pfizer; Genentech, Inc.; CSL Behring; Hemophilia of Georgia, Inc.; Hemab TherapeuticsRecruitingHemophilia | Thrombosis | Thrombophilia | Sickle Cell Disease | Thalassemia | Bleeding Disorder | Von Willebrand Diseases | Factor IX Deficiency | Connective Tissue Disorder | Factor VIII Deficiency | Hematologic Disorder | Rare Bleeding Disorder | Platelet DisorderUnited States
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Merck Sharp & Dohme LLCRecruitingVon Hippel-Lindau Disease | Pancreatic Neuroendocrine Tumor | Pheochromocytoma/Paraganglioma | Advanced Gastrointestinal Stromal Tumor | HIF-2α Mutated CancersCanada, United States, Denmark, France, Germany, Hungary, Italy, Russian Federation, Spain, Sweden, Turkey, Israel, United Kingdom, Netherlands, Australia, China, Singapore, Japan
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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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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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OMNI Medical Services, LLCOMNI Medical Services IncRecruitingDepression | COVID-19 | Epilepsy | Multiple Sclerosis | Glaucoma | Cancer | Covid19 | Chronic Pain | Parkinson Disease | Inflammatory Bowel Diseases | Hepatitis C | Insomnia | HIV/AIDS | Amyotrophic Lateral Sclerosis | Fibromyalgia | Anxiety | Bipolar Disorder | Seizures | Crohn Disease | Traumatic Brain Injury | Ulcerative Colitis | Sickle... and other conditionsUnited States
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Children's Oncology GroupNational Cancer Institute (NCI)Active, not recruitingStage I Kidney Wilms Tumor | Stage II Kidney Wilms Tumor | Stage III Kidney Wilms Tumor | Stage IV Kidney Wilms Tumor | Adult Kidney Wilms Tumor | Beckwith-Wiedemann Syndrome | Childhood Kidney Wilms Tumor | Diffuse Hyperplastic Perilobar Nephroblastomatosis | Rhabdoid Tumor of the Kidney | Stage V Kidney...United States, Canada, Australia, New Zealand, Puerto Rico, Israel
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LLS PedAL Initiative, LLCNational Cancer Institute (NCI); Children's Oncology GroupRecruitingAcute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Acute Lymphoblastic Leukemia | Myelodysplastic Syndrome | Myeloid Leukemia Associated With Down Syndrome | Mixed Phenotype Acute Leukemia | Acute Myeloid Leukemia Post Cytotoxic Therapy | Myelodysplastic Syndrome Post Cytotoxic TherapyUnited States, Canada, Australia, Puerto Rico, New Zealand