Clinical Trials on Pyruvate Metabolism, Inborn Errors
Total 4308 results
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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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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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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.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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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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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS); Rare Diseases...RecruitingPyruvate Dehydrogenase Complex Deficiency DiseaseUnited States
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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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Boston Children's HospitalAgios Pharmaceuticals, Inc.CompletedPyruvate Kinase Deficiency | Congenital Non-Spherocytic Hemolytic AnemiaUnited States, Germany, Canada, Czechia, Italy, Netherlands
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PTC TherapeuticsAxio Research. LLCCompleted
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Oslo University HospitalActive, not recruiting
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PTC TherapeuticsCompletedLeigh SyndromeUnited States
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Aadi Bioscience, Inc.Withdrawn
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Taysha Gene Therapies, Inc.Withdrawn
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National Human Genome Research Institute (NHGRI)RecruitingMitochondrial Disorders | Leigh Disease | Oxidative Phosphorylation Deficiencies | Electron Transport Chain Disorders, MitochondrialUnited States
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Khondrion BVJulius Clinical; ProPharma Group; Europees Fonds voor Regionale Ontwikkeling... and other collaboratorsRecruitingMitochondrial Diseases | MELAS | Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation | Subacute Necrotizing EncephalomyelopathyNetherlands
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Khondrion BVDrug Research Unit Ghent, BelgiumCompletedMitochondrial Disease | MELAS | Leigh Syndrome | LHON | Mitochondrial DNA tRNALeu(UUR) m.3243A<G MutationBelgium
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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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Columbia UniversityEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationMitochondrial Disease | Mitochondrial Disorders | Melas | Kearns Sayer | NARP | MNGIE | LHON | Mitochondrial Depletion Syndrome | Leigh's DiseaseUnited States
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Horizon Pharma USA, Inc.CompletedInherited Mitochondrial Disease, Including Leigh SyndromeUnited States
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The University of Texas Health Science Center,...RecruitingLeigh Disease | Leigh Syndrome | Leigh's Necrotizing Encephalopathy | Subacute Necrotizing Encephalomyopathy | Subacute Necrotizing EncephalomyelopathyUnited 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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University of MiamiHRSA/Maternal and Child Health BureauCompletedPhenylketonuria | Inborn Errors of Metabolism | GalactosemiaUnited States
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Sciotec Diagnostic Technologies GmbHNot yet recruitingFructose IntoleranceAustria
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Augusta UniversityUniversity of Iowa; Teikyo UniversityRecruitingFructose IntoleranceUnited States
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Augusta UniversityUnknownFructose IntoleranceUnited States
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Mayo ClinicNational Institute of Neurological Disorders and Stroke (NINDS)Enrolling by invitationCongenital Disorders of GlycosylationUnited States
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University of LausanneCompletedGlucose Metabolism Disorders | Fructose Metabolism, Inborn Errors | Hereditary Fructose Intolerance
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Maastricht University Medical CenterPfizerCompleted
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Augusta UniversityTerminatedDietary Fructose IntoleranceUnited States
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University of LausanneUniversity of LiegeCompletedHereditary Fructose IntoleranceBelgium
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University of LausanneRecruiting
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Assistance Publique - Hôpitaux de ParisCompletedInborn Errors of MetabolismFrance
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PTC TherapeuticsCompletedMitochondrial Diseases | Drug Resistant Epilepsy | Leigh Disease | Leigh Syndrome | Mitochondrial Encephalopathy (MELAS) | Pontocerebellar Hypoplasia Type 6 (PCH6) | Alpers Disease | Alpers SyndromeUnited States, Spain, United Kingdom, Canada, France, Italy, Poland, Sweden, Japan
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CENTOGENE GmbH RostockRecruitingInborn Errors of MetabolismPakistan
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University of LausanneCompletedInborn Errors of MetabolismSwitzerland
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CENTOGENE GmbH RostockCompletedInborn Errors of Metabolism | BiomarkerEgypt, Sri Lanka, Albania, Romania, Georgia, India, Lithuania, Pakistan
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Universitaire Ziekenhuizen KU LeuvenUnknownLipid Metabolism, Inborn ErrorsBelgium
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University Children's Hospital, ZurichRecruitingInborn Errors of Metabolism | TransitionSwitzerland, Austria
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National Human Genome Research Institute (NHGRI)RecruitingInborn Errors of Metabolism | Methylmalonic Acidemia | Organic AcidemiaUnited States