Search clinical trials for: mevalonate kinase deficiency
Total 18234 results
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Oregon Health and Science UniversityTerminatedImmune System Diseases | Lipid Metabolism, Inborn Errors | Mevalonic Aciduria | Mevalonate Kinase Deficiency | Periodic Fever Syndromes, HereditaryUnited States
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Michigan Technological UniversityWithdrawnHyper IgD Syndrome | Mevalonate Kinase DeficiencyUnited States, Netherlands
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Novartis PharmaceuticalsCompleted
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Rocket Pharmaceuticals Inc.Not yet recruitingPyruvate Kinase DeficiencySpain, United States
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Sun Yat-Sen Memorial Hospital of Sun Yat-Sen UniversityRecruiting
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7 Hills Pharma, LLCNot yet recruitingMelanoma | Kidney Cancer | Renal Cell Carcinoma | Hepatocellular Carcinoma | Colorectal Cancer | NSCLC | Non Small Cell Lung Cancer | Skin Cancer | Metastasis | Advanced Solid Tumor | Advanced Cancer | Renal Cell Cancer | Hepatocellular Cancer | Mismatch Repair Deficiency | Pleural Mesothelioma | MSI-High | Anaplastic Lymphoma... and other conditionsUnited States
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Hospital Universitari Vall d'Hebron Research InstituteErasme University Hospital; Cyprus Institute of Neurology and Genetics; EuroBloodNet...RecruitingIron Metabolism Disorders | Sickle Cell Disease | Thalassemia | Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder | Anemia Due to Membrane Defect | CDA | Sideroblastic Anemia | Constitutional Aplastic Anemia | Hereditary AnemiaSpain
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Institut für Klinische Krebsforschung IKF GmbH...Recruiting
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Pamela MunsterNational Cancer Institute (NCI)RecruitingBRCA1 Mutation | BRCA2 Mutation | PALB2 Gene Mutation | ATM Gene Mutation | BRCA Mutation | Checkpoint Kinase 2 Gene MutationUnited States
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Johns Hopkins UniversityNational Institute of Allergy and Infectious Diseases (NIAID); National Institute...RecruitingHIV Infections | AgingUnited 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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National Cancer Institute (NCI)RecruitingInherited Bone Marrow Failure Syndrome | Familial Platelet Disorder With Predisposition to Myeloid MalignanciesUnited States
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PfizerArvinas Estrogen Receptor, Inc.RecruitingBreast CancerUnited States, China, Spain, Hungary, Japan, Australia, Brazil, Italy, Slovakia, Switzerland
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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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Brown UniversityTaiho Oncology, Inc.RecruitingSoft Tissue Sarcoma | Advanced Breast Cancer | SCLC | Bladder Cancer | Endometrial Cancer | Treatment-Refractory Solid Tumors | Retinoblastoma DeficiencyUnited States
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Eva BonfillRecruiting
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Ikena OncologyRecruitingSolid Tumor | Solid Tumors, Adult | Malignant Pleural Mesothelioma (MPM) | Epithelioid Hemangioendothelioma (EHE) | NF2 Deficient Mesothelioma | Other NF2 Deficient Solid Tumors and Solid Tumors With YAP1/TAZ Fusion Genes | NF2 Deficiency | YAP1 or TAZ Gene FusionsUnited States, United Kingdom
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Agios Pharmaceuticals, Inc.RecruitingPediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic AnemiaUnited States, Spain, Germany, Netherlands, Canada, France, Israel, Switzerland, Italy
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Yonsei UniversityNot yet recruitingNon Small Cell Lung CancerKorea, Republic of
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Massachusetts General HospitalAgios Pharmaceuticals, Inc.RecruitingAcute Myeloid Leukemia | Myelodysplastic Syndromes | Myeloproliferative Neoplasm | Myelodysplastic/Myeloproliferative Neoplasm | Clonal Cytopenia of Undetermined Significance | Pyruvate Kinase Deficiency | Pyruvate Kinase Deficiency Anemia | Hereditary Hemolytic Anemia | Clonal Myeloid Neoplasm | Other... and other conditionsUnited States
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Agios Pharmaceuticals, Inc.RecruitingPyruvate Kinase DeficiencyUnited States, Germany, Spain, Italy, United Kingdom, Czechia, France, Canada, Korea, Republic of, Netherlands, Turkey, Portugal, Denmark, Ireland, Switzerland, Thailand
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Children's Hospital of Fudan UniversityRecruitingActivated PI3K-delta Syndrome | Immunodeficiency PrimaryChina
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Novartis PharmaceuticalsRecruitingAdvanced Solid Tumors Which Are cMET-dependentGermany, Korea, Republic of, Belgium, Italy, Singapore, United States, Denmark, Spain, France, Canada
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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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Agios Pharmaceuticals, Inc.Active, not recruitingPyruvate Kinase DeficiencyUnited States, Canada, Netherlands, United Kingdom, France, Italy
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Agios Pharmaceuticals, Inc.Active, not recruitingPediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic AnemiaUnited States, Spain, Denmark, Turkey, Netherlands, Czechia, United Kingdom, Germany, Canada, Switzerland
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Agios Pharmaceuticals, Inc.Enrolling by invitationAnemia, Hemolytic | Pyruvate Kinase DeficiencyJapan
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Agios Pharmaceuticals, Inc.Active, not recruitingPyruvate Kinase DeficiencyUnited States, Spain, Japan, Canada, United Kingdom, Korea, Republic of, Ireland, Thailand, Italy, Turkey, Denmark, Brazil, France, Germany, Netherlands, Switzerland
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National Institute of Allergy and Infectious Diseases...Enrolling by invitationSafety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory ComplicationsChronic Granulomatous Disease | Inflammatory Gastrointestinal Disease | Inflammatory Skin Disease | Inflammatory Lung DiseaseUnited States
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West China HospitalActive, not recruitingCarcinoma, Renal Cell | Immunotherapy | Fumarate Hydratase DeficiencyChina
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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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Rocket Pharmaceuticals Inc.Active, not recruiting
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Loulou FoundationActive, not recruitingCyclin-Dependent Kinase-Like 5 Deficiency DisorderUnited States, Spain, Italy, France, Canada, Germany, United Arab Emirates
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Columbia UniversityUniversitat Autonoma de Barcelona; Instituto de Salud Carlos III; University... and other collaboratorsActive, not recruitingThymidine Kinase 2 Deficiency | Mitochondrial DNA Depletion Syndrome 2 Myopathic TypeUnited States
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UCB BIOSCIENCES, Inc.Zogenix, Inc.Active, not recruitingThymidine Kinase 2 DeficiencyUnited States, Israel, Spain
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Sierra Oncology LLC - a GSK companyCompletedPrimary Myelofibrosis | Post-polycythemia Vera Myelofibrosis | Post-essential Thrombocythemia MyelofibrosisKorea, Republic of, Canada, Hungary, Israel, Spain, United States, Australia, Taiwan, Singapore, Germany, Italy, France, Belgium, New Zealand, Austria, United Kingdom, Denmark, Bulgaria, Poland, Czechia, Romania, Sweden
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Agios Pharmaceuticals, Inc.CompletedPyruvate Kinase DeficiencyUnited States, Italy, Netherlands
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Agios Pharmaceuticals, Inc.TerminatedAnemia | Anemia, Hemolytic | Pyruvate Kinase Deficiency | Pyruvate Kinase Deficiency AnemiaUnited States, United Kingdom, Italy, Spain, Netherlands
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Zogenix MDS, Inc.Zogenix, Inc.WithdrawnThymidine Kinase 2 Deficiency
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Zogenix MDS, Inc.Zogenix, Inc.CompletedThymidine Kinase 2 DeficiencySpain, United States, Russian Federation, Turkey
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University of WashingtonNational Cancer Institute (NCI); BayerCompletedAcute Myeloid Leukemia | Myelodysplastic Syndrome | Myeloproliferative Neoplasm | de Novo Myelodysplastic Syndrome | Acute Biphenotypic LeukemiaUnited States
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MEI Pharma, Inc.TerminatedChronic Lymphocytic Leukemia (CLL) | Small Lymphocytic Lymphoma (SLL) | Mantle Cell Lymphoma (MCL) | Diffuse Large B-cell Lymphoma (DLBCL) | Follicular Lymphoma (FL) | Marginal Zone B Cell Lymphoma | High Grade Non-Hodgkin's LymphomaUnited States, Switzerland
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CENTOGENE GmbH RostockCompletedPyruvate Kinase DeficiencyUnited States
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TakedaOvid Therapeutics Inc.Completed15q Duplication Syndrome | CDKL5 Deficiency DiseaseUnited States
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Agios Pharmaceuticals, Inc.CompletedAnemia, Hemolytic | Pyruvate Kinase DeficiencyUnited States, Spain, Switzerland, Turkey, Germany, France, United Kingdom, Japan, Netherlands, Brazil, Canada, Czechia, Denmark, Italy, Korea, Republic of, Thailand
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University of Maryland, BaltimoreGlaxoSmithKline; Novartis PharmaceuticalsTerminatedBreast Neoplasms | Neoplasm Metastasis | Endocrine Breast DiseasesUnited States
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Agios Pharmaceuticals, Inc.CompletedAnemia, Hemolytic | Pyruvate Kinase DeficiencyUnited States, United Kingdom, Netherlands, Canada, Denmark, France, Italy, Ireland, Thailand
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Novartis PharmaceuticalsTerminatedNeoplasms, BreastSpain, United States, Argentina, Austria, Italy, Russian Federation, Thailand, Brazil, Peru, Philippines, Hong Kong, Mexico
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Amsterdam UMC, location VUmcTerminated
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Novartis PharmaceuticalsCompletedBreast NeoplasmsUnited States, France, Spain, United Kingdom, Croatia, Turkey, Netherlands, Argentina, Australia, Canada, Czechia, Germany, Italy, Poland, Russian Federation, Brazil, Bulgaria, Chile, Denmark, Hungary, Ireland, Peru, South Africa, Col... and more