Search clinical trials for: Mevalonate Kinase Deficiency (MKD)
Total 18240 results
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University of LiverpoolVitaflo International, Ltd; Walton Centre NHS Foundation TrustCompletedGlioblastoma | Glioblastoma Multiforme | Glioblastoma, AdultUnited Kingdom
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Birmingham and Solihull Mental Health NHS Foundation...UnknownEpilepsyUnited Kingdom
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Michigan Technological UniversityWithdrawnHyper IgD Syndrome | Mevalonate Kinase DeficiencyUnited States, Netherlands
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Novartis PharmaceuticalsCompleted
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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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Assistance Publique - Hôpitaux de ParisInstitut ImagineNot yet recruitingAutoinflammatory Disease | FMF | TRAPS | MKD | Cryopyrin Associated Periodic Syndrome | HaploinsufficiencyFrance
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Medical University of ViennaCompletedOcular Physiology | Regional Blood FlowAustria
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Royal Devon and Exeter NHS Foundation TrustWellcome Trust; Haukeland University Hospital; Hôpital Necker-Enfants MaladesCompletedDiabetes Mellitus
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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