Clinical Trials on Pediatric Pyruvate Kinase Deficiency
Total 116 results
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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.Enrolling by invitationAnemia, Hemolytic | Pyruvate Kinase DeficiencyJapan
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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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Great Ormond Street Hospital for Children NHS Foundation...National Institute for Health Research, United Kingdom; The Freya FoundationRecruitingPyruvate Dehydrogenase Complex Deficiency | Pyruvate Dehydrogenase E1 Alpha Deficiency | Pyruvate Dehydrogenase E1-Beta Deficiency | Pyruvate Dehydrogenase E2 Deficiency | Pyruvate Dehydrogenase Phosphatase DeficiencyUnited Kingdom
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Rocket Pharmaceuticals Inc.Not yet recruitingPyruvate Kinase DeficiencySpain, United States
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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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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.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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Boston Children's HospitalAgios Pharmaceuticals, Inc.CompletedPyruvate Kinase Deficiency | Congenital Non-Spherocytic Hemolytic AnemiaUnited States, Germany, Canada, Czechia, Italy, Netherlands
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Vanderbilt UniversityCompletedPhosphoglycerate Kinase (PGK) DeficiencyUnited States
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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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Loulou FoundationActive, not recruitingCyclin-Dependent Kinase-Like 5 Deficiency DisorderUnited States, Spain, Italy, France, Canada, Germany, United Arab Emirates
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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS); Rare Diseases...RecruitingPyruvate Dehydrogenase Complex Deficiency DiseaseUnited States
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Jerry Vockley, MD, PhDUltragenyx Pharmaceutical IncNo longer availableBarth Syndrome | Mitochondrial Trifunctional Protein Deficiency | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2) | Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency | Glycogen Storage Disorders | Pyruvate Carboxylase Deficiency... and other conditionsUnited States
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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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Zogenix MDS, Inc.Zogenix, Inc.WithdrawnThymidine Kinase 2 Deficiency
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OPKO Health, Inc.CompletedSafety and Efficacy Phase 2 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient ChildrenPediatric Growth Hormone DeficiencyGreece, Hungary, Slovakia
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Jirair Krikor BedoyanUltragenyx Pharmaceutical IncRecruitingPyruvate Dehydrogenase Complex DeficiencyUnited States
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University of FloridaEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsActive, not recruitingPyruvate Dehydrogenase Complex DeficiencyUnited States
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Fondazione TelethonCompletedPyruvate Dehydrogenase Complex DeficiencyItaly
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Changchun GeneScience Pharmaceutical Co., Ltd.Not yet recruitingPediatric Growth Hormone Deficiency (PGHD)China
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Changchun GeneScience Pharmaceutical Co., Ltd.Not yet recruitingPediatric Growth Hormone Deficiency (PGHD)China
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Changchun GeneScience Pharmaceutical Co., Ltd.Not yet recruitingPediatric Growth Hormone Deficiency (PGHD)China
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Changchun GeneScience Pharmaceutical Co., Ltd.Not yet recruitingPediatric Growth Hormone Deficiency (PGHD)China
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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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PfizerNot yet recruiting
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Columbia UniversityCompletedVitamin D Deficiency | Obesity, Morbid | Obesity, Childhood | Obesity, PediatricUnited States
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Oslo University HospitalActive, not recruiting
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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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Namik Kemal UniversityNot yet recruitingCan the Geriatric Questionnaires Predict Adverse Effects in Geriatric Breast Cancer Patients Treated With Cyclin-dependent Kinase 4 and 6 InhibitorsTurkey
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Amsterdam Molecular TherapeuticsInternational Antiviral Therapy Evaluation CenterUnknownFamilial Lipoprotein Lipase DeficiencyCanada
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Jessica RiguttoUniversity of Oxford; Jomo Kenyatta University of Agriculture and Technology... and other collaboratorsCompletedIron-deficiency | Iron Deficiency Anemia | Pediatric ALL | VaccinationKenya, Switzerland
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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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Xinhua Hospital, Shanghai Jiao Tong University...CompletedVitamin D Deficiency | Obesity, ChildhoodChina
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University of British ColumbiaMcMaster UniversityCompleted
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University of Cape TownUniversity of Stellenbosch; University of KwaZuluRecruitingIron Deficiency Anemia | Anaemia | Iron | Prospective Studies | Paediatric Surgery | Interventional StudiesSouth Africa
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West China HospitalNot yet recruitingSepsis | Vitamin A Deficiency | Pediatric
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Adiyaman UniversityCompletedVitamin D Deficiency | Optic Nerve Diseases | Pediatric Disorder | Retinal Disease | Choroidal DiseaseTurkey
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Children's Hospital of Eastern OntarioMcGill University; Ottawa Hospital Research Institute; The Ottawa Hospital; Children...CompletedVitamin D Deficiency | Heart Defects, Congenital | Thoracic Surgery | Pediatric DisordersCanada
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PfizerWithdrawnPediatric Growth Hormone Deficiency
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Alexion PharmaceuticalsTerminatedLysosomal Acid Lipase DeficiencyUnited Kingdom, Finland, United States, Italy