Clinical Trials on Mevalonate Kinase Deficiency
Total 9122 results
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Vanderbilt UniversityCompletedPhosphoglycerate Kinase (PGK) DeficiencyUnited States
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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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Novartis PharmaceuticalsCompleted
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UCB BIOSCIENCES, Inc.Zogenix, Inc.Active, not recruitingThymidine Kinase 2 DeficiencyUnited States, Israel, Spain
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Zogenix MDS, Inc.Zogenix, Inc.CompletedThymidine Kinase 2 DeficiencySpain, United States, Russian Federation, Turkey
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Agios Pharmaceuticals, Inc.Enrolling by invitationAnemia, Hemolytic | Pyruvate Kinase DeficiencyJapan
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Michigan Technological UniversityWithdrawnHyper IgD Syndrome | Mevalonate Kinase DeficiencyUnited States, Netherlands
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Zogenix MDS, Inc.Zogenix, Inc.WithdrawnThymidine Kinase 2 Deficiency
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Groupe Hospitalier Pitie-SalpetriereVanderbilt University Medical CenterCompleted
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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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Agios Pharmaceuticals, Inc.RecruitingPediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic AnemiaUnited States, Spain, Germany, Netherlands, Canada, France, Israel, Switzerland, 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.TerminatedAnemia | Anemia, Hemolytic | Pyruvate Kinase Deficiency | Pyruvate Kinase Deficiency AnemiaUnited States, United Kingdom, Italy, Spain, Netherlands
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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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Moscow Regional Research and Clinical Institute...TerminatedGlycogen Synthase Kinase 3Russian Federation
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AlexionRecruitingWolman Disease | Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 | Acid Lipase Deficiency | LIPA Deficiency | LAL-DeficiencyFrance, Belgium, United States, Spain, Germany, Greece, Israel, Italy, Slovenia, United Kingdom, Brazil, Canada, Denmark, Australia, Croatia, Czechia, Ireland, Mexico, Netherlands, Poland, Portugal, Saudi Arabia
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Amsterdam Molecular TherapeuticsThe Clinical Trial CompanyCompletedFamilial Lipoprotein Lipase DeficiencyCanada
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Amsterdam Molecular TherapeuticsInternational Antiviral Therapy Evaluation CenterUnknownFamilial Lipoprotein Lipase DeficiencyCanada
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Rigshospitalet, DenmarkUnknownCarbohydrate Metabolism, Inborn Errors | Metabolism, Inborn Errors | Lipid Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease Type V | VLCAD Deficiency | Glycogen Storage Disease Type III | Phosphoglycerate Kinase Deficiency | Neutral Lipid Storage Disease | Carnitine... and other conditionsDenmark
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AlexionCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited States, Canada, United Kingdom, Italy, France
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Central Hospital, Nancy, FranceCompletedMuscular Diseases | Creatine KinaseFrance
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Johnson & Johnson Pharmaceutical Research & Development...Janssen Pharmaceutica N.V., BelgiumCompletedPharmacokinetics | Advanced Malignancies | Solid Malignancies | Protein Kinase Inhibitors | Maximum Tolerated Dose | Antineoplastic Protocols | Clinical Trial, Phase 1
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Pamela MunsterNational Cancer Institute (NCI)Not yet recruitingBRCA1 Mutation | BRCA2 Mutation | PALB2 Gene Mutation | ATM Gene Mutation | BRCA Mutation | Checkpoint Kinase 2 Gene MutationUnited States
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Northwestern UniversityCompletedCancer | Communication | Medication Adherence | Cancer, Treatment-Related | Tyrosine Kinase InhibitorUnited States
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Alexion PharmaceuticalsTerminatedLysosomal Acid Lipase DeficiencyUnited Kingdom, Finland, United States, Italy
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Astellas Pharma IncCompletedSolid Tumor | B-Cell Lymphoma | Advanced Malignancies | Positive for Anaplastic Lymphoma Kinase | Positive for Proto-Oncogene Tyrosine-Protein Kinase ROSUnited 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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Agios Pharmaceuticals, Inc.Active, not recruitingPyruvate Kinase DeficiencyUnited States, Canada, Netherlands, United Kingdom, France, Italy
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Rocket Pharmaceuticals Inc.Active, not recruiting
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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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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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Hospices Civils de LyonUnknownLiver Post-transplant PatientsFrance
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Zogenix MDS, Inc.CompletedThymidine Kinase 2 (TK2)United States, Spain, Israel
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Hospices Civils de LyonUnknownPatients Waiting for a Liver Transplant.France
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National Institute of Arthritis and Musculoskeletal...TerminatedGraft Versus Host Disease | JNS Kinase | Topical AdministrationUnited States
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Agios Pharmaceuticals, Inc.CompletedPyruvate Kinase DeficiencyUnited States, Italy, Netherlands
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CENTOGENE GmbH RostockCompletedPyruvate Kinase DeficiencyUnited 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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Alexion PharmaceuticalsCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited Kingdom, France, United States, Egypt, Ireland
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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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Nanfang Hospital of Southern Medical UniversityEnrolling by invitationAcute Myeloid Leukemia | FLT3-ITD Mutation | Brutons Tyrosine KinaseChina
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD) | LAL-DeficiencyFrance, United States, United Kingdom, Czechia
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CENTOGENE GmbH RostockWithdrawnCholesterol Ester Storage Disease | Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Wolman TypeGermany, India, Sri Lanka
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Susan J. HayflickOregon Health and Science University; Oregon Clinical and Translational Research...CompletedPantothenate Kinase-associated Neurodegeneration (PKAN)United States
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase Deficiency | LAL-Deficiency | Cholesterol Ester Storage Disease (CESD)France, United Kingdom, United States, Canada, Czechia
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University of Colorado, DenverCompletedLipoprotein Lipase Deficiency
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University Medical Center GroningenRadboud University Medical Center; Maastricht University Medical Center; Erasmus... and other collaboratorsRecruitingCarcinoma, Non-Small-Cell Lung | Lung Cancer | Drug Monitoring | Anaplastic Lymphoma Kinase Gene Mutation | Anaplastic Lymphoma Kinase Gene TranslocationNetherlands, France
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Université de SherbrookeInstitut de Recherches Cliniques de MontrealRecruitingLipoprotein Lipase DeficiencyCanada
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Poznan University of Physical EducationNational Science Centre, Poland; Faculty of Physical Education and Sport at... and other collaboratorsRecruitingSupplementation | Sport | Sports Nutrition | Sports Dietetics | Protein Kinases | Aerobic Capacity | Fat Free MassCzechia, Poland
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Amsterdam Molecular TherapeuticsThe Clinical Trial CompanyUnknown