Clinical Trials on Pyruvate Dehydrogenase E1 Alpha Deficiency in United States
Total 153 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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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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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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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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Oregon Health and Science UniversityCompletedNormal Volunteers | Trifunctional Protein Deficiency | Very Long-chain Acyl-CoA Dehydrogenase Deficiency | Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency | Medium-chain Acyl-CoA Dehydrogenase Deficiency | Carnitine Palmitoyltransferase II Deficiency, MyopathicUnited States
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Oregon Health and Science UniversityCompletedTrifunctional Protein Deficiency | Very Long Chain Acyl Coa Dehydrogenase Deficiency | Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency | Carnitine Palmitoyltransferase Deficiency 2United States
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Baxalta now part of ShireArriva Pharmaceuticals, Inc.CompletedAlpha1-antitrypsin DeficiencyUnited States
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Horizon Pharma USA, Inc.CompletedAldehyde Dehydrogenase-2 (ALDH2) DeficiencyUnited States
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Oregon Health and Science UniversityUniversity of PittsburghCompletedVery Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | Carnitine Palmitoyltransferase 2 (CPT2) Deficiency | Mitochondrial Trifunctional Protein (TFP) Deficiency | Long-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) DeficiencyUnited States
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Baxalta now part of ShireTerminated
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Baxalta now part of ShireCompleted
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Baxalta now part of ShireArriva Pharmaceuticals, Inc.CompletedAlpha1-antitrypsin DeficiencyUnited States
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TakedaRecruitingAlpha1-Antitrypsin DeficiencyUnited States, Poland
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Ultragenyx Pharmaceutical IncCompletedCarnitine Palmitoyltransferase (CPT I or CPT II) Deficiency | Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency | Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency | Trifunctional Protein (TFP) Deficiency | Carnitine-acylcarnitine Translocase (CACT) DeficiencyUnited States, United Kingdom
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Grifols Therapeutics LLCRecruiting
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Boston Children's HospitalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsActive, not recruitingSuccinic Semialdehyde Dehydrogenase DeficiencyUnited States, United Kingdom, Germany, Spain
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TakedaEnrolling by invitationAlpha1-Antitrypsin DeficiencyUnited Kingdom, Germany, United States, Austria, Portugal
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University of PittsburghHorizon Pharma Ireland, Ltd., Dublin IrelandCompletedMedium-chain Acyl-CoA Dehydrogenase (MCAD) DeficiencyUnited States
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Baxalta now part of ShireCompletedAlpha1-antitrypsin DeficiencyUnited States, Canada
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Vertex Pharmaceuticals IncorporatedCompletedAlpha1-Antitrypsin DeficiencyUnited States, Germany, Canada, Ireland, Sweden, United Kingdom
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Z Factor LimitedHammersmith Medicines Research; Centessa Pharmaceuticals plcTerminatedAlpha1 Anti-Trypsin DeficiencyUnited Kingdom
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TakedaTakeda Development Center Americas, Inc.RecruitingAlpha1-Antitrypsin DeficiencyUnited States, Belgium, Switzerland, Canada, Australia, Germany, United Kingdom, Austria, France, Spain, Portugal, Italy, Poland, Brazil
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Ultragenyx Pharmaceutical IncCompletedLong-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency | Trifunctional Protein (TFP) Deficiency | Carnitine Palmitoyltransferase (CPT II) Deficiency | Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) DeficiencyUnited States, United Kingdom
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University of Mississippi, OxfordCompletedMalaria | Glucose 6 Phosphate Dehydrogenase DeficiencyUnited States
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University of FloridaAlpha-1 FoundationEnrolling by invitation
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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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Baxalta now part of ShireBaxalta Innovations GmbH, now part of ShireTerminatedChronic Obstructive Pulmonary Disease | Alpha1-antitrypsin DeficiencyUnited States, Canada, Australia
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Michael Campos, MDCSL BehringCompletedAlpha 1 Antitrypsin DeficiencyUnited States
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Grifols Therapeutics LLCCompletedAlpha₁-Antitrypsin DeficiencyUnited States
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Washington University School of MedicineNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); N... and other collaboratorsTerminatedLiver Cirrhosis | Alpha-1-antitrypsin DeficiencyUnited 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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Origin BiosciencesCompletedMolybdenum Cofactor Deficiency (MoCD) | Rare Autosomal Recessive Disorder | Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)United States
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Grifols Therapeutics LLCCompletedEmphysema | Alpha 1-antitrypsin Deficiency (AATD)United States
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University of PittsburghNational Heart, Lung, and Blood Institute (NHLBI)CompletedAlpha 1 Antitrypsin Deficiency | AATDUnited States
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Alnylam PharmaceuticalsTerminatedZZ Type Alpha-1 Antitrypsin Deficiency Liver DiseaseUnited Kingdom
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National Institute of Neurological Disorders and...CompletedSuccinic Semialdehyde | Dehydrogenase Deficiency | DiseasesUnited States
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University of FloridaCompletedAlpha-1 Antitrypsin Deficiency | Liver Fibrosis | AATD | AAT DeficiencyUnited States
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National Jewish HealthMedical University of South CarolinaUnknownAlpha 1-Antitrypsin DeficiencyUnited States
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Biocerna LLCCompletedAlpha 1-Antitrypsin DeficiencyUnited States
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National Center for Research Resources (NCRR)University of PittsburghTerminatedAlpha 1-Antitrypsin DeficiencyUnited States
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Alpha-1 FoundationRecruiting
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Medical University of South CarolinaAlpha-1 FoundationCompleted
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University of FloridaRecruitingAlpha-1 Antitrypsin DeficiencyUnited States
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Krystal Biotech, Inc.RecruitingAlpha 1-Antitrypsin DeficiencyUnited States
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Kamada, Ltd.CompletedAlpha-1 Antitrypsin DeficiencyUnited States
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Coram Clinical TrialsCompletedAlpha-1 Anti-trypsin DeficiencyUnited States
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Adverum Biotechnologies, Inc.Completed
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Eunice Kennedy Shriver National Institute of Child...Completed