Clinical Trials on Inherited Retinal Dystrophy Associated With RPE65 Mutations in United States
Total 52 results
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Spark TherapeuticsActive, not recruitingConfirmed Biallelic RPE65 Mutation-associated Retinal DystrophyUnited 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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HuidaGene Therapeutics Co., Ltd.Cholgene Therapeutics, Inc.RecruitingLeber Congenital Amaurosis | Inherited Retinal Diseases Caused by RPE65 MutationsUnited States, China
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University of WashingtonRecruitingInherited Retinal Dystrophy Primarily Involving Sensory RetinaUnited States
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Columbia UniversityLudwig-Maximilians - University of Munich; Moorfields Eye Hospital NHS Foundation... and other collaboratorsRecruitingRetinitis Pigmentosa Associated With CNGB1 MutationsUnited States, France, Germany, United Kingdom
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National Eye Institute (NEI)TerminatedInherited Retinal Degeneration | Inherited Ophthalmic DiseasesUnited States
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Jaeb Center for Health ResearchFoundation Fighting BlindnessRecruitingRetinitis Pigmentosa | Inherited Retinal DegenerationUnited States, United Kingdom, France, Brazil, Finland, Israel, Netherlands, Switzerland, Australia, Canada, Mexico
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Cook Children's Health Care SystemNo longer availableDravet Syndrome | Epileptic Encephalopathies Associated With SCN1A MutationsUnited States
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University of OxfordCompletedAge-related Macular Degeneration | Retinitis Pigmentosa | Inherited Retinal Dystrophies | Optic Nerve PathologyUnited Kingdom
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Catalyst BiosciencesTerminatedPrevious Diagnosis With a Complement-mediated Disease and/or With Clinical Manifestations Reasonably Associated With Complement Factor I DeficiencyUnited States
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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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PfizerCompletedMacular Edema Associated With Diabetes MellitusUnited States, Colombia, Australia, Germany, Italy, South Africa, Greece, France, Austria, United Kingdom, Denmark, India, Portugal, Brazil, Canada, Czechia, Netherlands, Switzerland
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MedtronicNeuroCompletedChronic Refractory Pain Associated With Failed Back Surgery Syndrome, Epidural Fibrosis, Peripheral Causalgia, Complex Regional Pain SyndromeUnited States
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Cure CMDRecruitingEmery-Dreifuss Muscular Dystrophy | Congenital Myasthenic Syndrome | Limb-Girdle Muscular Dystrophy | Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency | Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy) and other conditionsUnited States
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Lundquist Institute for Biomedical Innovation at...CompletedEnd-stage Renal Disease | Chronic Kidney Failure | Disorders Associated With Peritoneal DialysisUnited States
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FibroGenAstraZenecaWithdrawnAnemia Associated With Chronic Kidney DiseaseUnited States
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FibroGenAstraZenecaCompletedAnemia Associated With End Stage Renal Disease (ESRD)United States
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FibroGenAstraZenecaCompletedAnemia Associated With End Stage Renal DiseaseUnited States
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Akebia TherapeuticsCompletedAnemia Associated With Chronic Kidney DiseaseUnited States
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BiogenRecruitingAmyotrophic Lateral Sclerosis Associated With a SOD1 Gene MutationUnited States, Canada, Spain, Australia, Germany, Italy, Japan, Korea, Republic of, United Kingdom, Belgium, Brazil, France, Poland, Sweden
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Mayo ClinicAstellas Pharma Inc; Casimir, LLCRecruitingMitochondrial Diseases | Mitochondrial Myopathies | Kearns-Sayre Syndrome | Mitochondrial DNA Mutation | Chronic Progressive External Ophthalmoplegia With MyopathyUnited States
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI); ExelixisActive, not recruitingRenal Cell Carcinoma | Metastatic Renal Cell Carcinoma | Stage IV Renal Cell Cancer AJCC v8 | Sarcomatoid Renal Cell Carcinoma | Chromophobe Renal Cell Carcinoma | Papillary Renal Cell Carcinoma | Malignant Neoplasms of Urinary Tract | Renal Cell Carcinoma Associated With Xp11.2 Translocations/TFE3... and other conditionsUnited States
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University of Alabama at BirminghamNational Institutes of Health (NIH); Mereo BioPharmaCompletedEmphysema or COPD | Alpha-1 Antitrypsin Deficiency (AATD) | Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I MutationsUnited States
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Peloton Therapeutics, Inc., a subsidiary of Merck...Active, not recruitingVHL - Von Hippel-Lindau Syndrome | VHL Gene Mutation | VHL Syndrome | VHL Gene Inactivation | VHL-Associated Renal Cell Carcinoma | VHL-Associated Clear Cell Renal Cell CarcinomaUnited States, Denmark, United Kingdom, France
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National Cancer Institute (NCI)CompletedMetastatic Transitional Cell Cancer of the Renal Pelvis and Ureter | Recurrent Bladder Cancer | Recurrent Transitional Cell Cancer of the Renal Pelvis and Ureter | Regional Transitional Cell Cancer of the Renal Pelvis and Ureter | Stage III Bladder Cancer | Stage IV Bladder Cancer | Transitional... and other conditionsUnited States
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Akebia TherapeuticsCompletedAnemia Associated With Chronic Kidney Disease (CKD)United States
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)CompletedMetastatic Transitional Cell Cancer of the Renal Pelvis and Ureter | Recurrent Bladder Cancer | Recurrent Transitional Cell Cancer of the Renal Pelvis and Ureter | Regional Transitional Cell Cancer of the Renal Pelvis and Ureter | Stage III Bladder Cancer | Stage IV Bladder Cancer | Transitional... and other conditionsUnited States
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National Cancer Institute (NCI)TerminatedMetastatic Transitional Cell Cancer of the Renal Pelvis and Ureter | Recurrent Bladder Cancer | Recurrent Transitional Cell Cancer of the Renal Pelvis and Ureter | Regional Transitional Cell Cancer of the Renal Pelvis and Ureter | Stage III Bladder Cancer | Stage IV Bladder Cancer | Transitional... and other conditionsUnited States
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University Hospital Birmingham NHS Foundation TrustCompletedWegener's Granulomatosis | Microscopic Polyangiitis | Renal Limited VasculitisUnited Kingdom
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National Cancer Institute (NCI)TerminatedMetastatic Transitional Cell Cancer of the Renal Pelvis and Ureter | Recurrent Bladder Cancer | Recurrent Transitional Cell Cancer of the Renal Pelvis and Ureter | Stage IV Bladder Cancer | Transitional Cell Carcinoma of the Bladder | Distal Urethral Cancer | Proximal Urethral Cancer | Recurrent... and other conditionsUnited States
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National Cancer Institute (NCI)CompletedMetastatic Transitional Cell Cancer of the Renal Pelvis and Ureter | Recurrent Bladder Cancer | Recurrent Transitional Cell Cancer of the Renal Pelvis and Ureter | Stage III Bladder Cancer | Stage IV Bladder Cancer | Transitional Cell Carcinoma of the Bladder | Adenocarcinoma of the Bladder | Distal... and other conditionsUnited States
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National Cancer Institute (NCI)CompletedRecurrent Bladder Cancer | Recurrent Transitional Cell Cancer of the Renal Pelvis and Ureter | Stage IV Bladder Cancer | Transitional Cell Carcinoma of the Bladder | Distal Urethral Cancer | Proximal Urethral Cancer | Recurrent Urethral Cancer | Urethral Cancer Associated With Invasive Bladder CancerUnited States, China, Korea, Republic of
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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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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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Epizyme, Inc.CompletedSynovial Sarcoma | Epithelioid Sarcoma | Renal Medullary Carcinoma | Malignant Rhabdoid Tumors (MRT) | Rhabdoid Tumors of the Kidney (RTK) | Atypical Teratoid Rhabdoid Tumors (ATRT) | Selected Tumors With Rhabdoid Features | INI1-negative Tumors | Malignant Rhabdoid Tumor of Ovary | Poorly Differentiated... and other conditionsFrance, United States, Australia, Taiwan, Belgium, United Kingdom, Canada, Germany, Italy
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute...CompletedClear Cell Renal Cell Carcinoma | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Diffuse Small Cleaved Cell Lymphoma | Recurrent Grade 1 Follicular Lymphoma | Recurrent... and other conditionsUnited States, Germany
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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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23andMe, Inc.Active, not recruitingClear Cell Renal Cell Carcinoma | Fallopian Tube Cancer | Solid Tumor | Neuroendocrine Tumors | Epithelial Ovarian Cancer | Primary Peritoneal Carcinoma | Extensive-stage Small-cell Lung Cancer | MSI-H Cancer | Cancer With A High Tumor Mutational BurdenUnited States, Canada
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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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Roswell Park Cancer InstituteActive, not recruitingAcute Myeloid Leukemia | Polycythemia Vera | Myelofibrosis | Chronic Myelomonocytic Leukemia | Waldenstrom Macroglobulinemia | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Secondary Acute Myeloid Leukemia | Sickle Cell Disease | Myelodysplastic Syndrome | Plasma Cell Myeloma | Chronic Granulomatous... and other conditionsUnited States
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Sanjiv Sam GambhirNational Cancer Institute (NCI)CompletedUnspecified Adult Solid Tumor, Protocol Specific | Recurrent Pancreatic Cancer | Stage IV Pancreatic Cancer | Male Breast Cancer | Stage IV Breast Cancer | Stage IIIA Non-small Cell Lung Cancer | Stage IIIB Non-small Cell Lung Cancer | Stage IIIA Breast Cancer | Stage IIIB Breast Cancer | Tongue Cancer | Stage... and other conditionsUnited States
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National Cancer Institute (NCI)CompletedRecurrent Pancreatic Cancer | Stage III Pancreatic Cancer | Stage IV Pancreatic Cancer | Stage IIIB Non-small Cell Lung Cancer | Stage IV Renal Cell Cancer | Stage IV Colon Cancer | Stage IV Rectal Cancer | Recurrent Colon Cancer | Recurrent Rectal Cancer | Recurrent Metastatic Squamous Neck Cancer With... and other conditionsUnited States
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National Cancer Institute (NCI)CompletedRecurrent Prostate Cancer | Unspecified Adult Solid Tumor, Protocol Specific | Stage III Renal Cell Cancer | Recurrent Melanoma | Male Breast Cancer | Stage IV Breast Cancer | Stage IV Melanoma | Stage IV Ovarian Epithelial Cancer | Stage IIIB Breast Cancer | Stage IV Renal Cell Cancer | Recurrent Renal Cell... and other conditionsUnited States
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National Cancer Institute (NCI)CompletedHIV Infection | Clear Cell Renal Cell Carcinoma | Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell... and other conditionsUnited States
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Roswell Park Cancer InstituteNational Cancer Institute (NCI)CompletedUnspecified Childhood Solid Tumor, Protocol Specific | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent Adult Diffuse Large Cell Lymphoma | Recurrent Adult Diffuse Mixed Cell Lymphoma 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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Children's Oncology GroupNational Cancer Institute (NCI)RecruitingRenal Cell Carcinoma | Wilms Tumor | Clear Cell Sarcoma of the Kidney | Papillary Renal Cell Carcinoma | Kidney Medullary Carcinoma | Diffuse Hyperplastic Perilobar Nephroblastomatosis | Rhabdoid Tumor of the Kidney | Congenital Mesoblastic Nephroma | Renal Cell Carcinoma Associated With Xp11.2 Translocations... and other conditionsUnited States, Canada, Puerto Rico, Australia, New Zealand, Israel, Switzerland
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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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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute...CompletedClear Cell Renal Cell Carcinoma | 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, Germany, Italy
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National Cancer Institute (NCI)CompletedRecurrent Prostate Cancer | Fallopian Tube Cancer | Bone Metastases | Lung Metastases | Stage III Renal Cell Cancer | Liver Metastases | Recurrent Thyroid Cancer | Stage IV Follicular Thyroid Cancer | Stage IV Papillary Thyroid Cancer | Inflammatory Breast Cancer | Metastatic Transitional Cell Cancer of the... and other conditionsUnited States