Clinical Trials on Argininosuccinic Aciduria
Total 53 results
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McGill University Health Centre/Research Institute...UnknownMethylmalonic Acidemia | Malonic AciduriaCanada
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Brendan LeeEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedAmino Acid Metabolism, Inborn Errors | Urea Cycle Disorders | Argininosuccinic AciduriaUnited States
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Baylor College of MedicineCompletedUrea Cycle Disorders | Argininosuccinic Aciduria | Argininosuccinate Lyase DeficiencyUnited States
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Baylor College of MedicineRare Diseases Clinical Research Network; Neogenis LaboratoriesCompletedUrea Cycle Disorders, Inborn | Urea Cycle Disorder | Argininosuccinic Aciduria | Argininosuccinate Lyase DeficiencyUnited States
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Emma Marie Caroline SlackUniversity Children's HospitalCompletedInborn Errors of Metabolism | Propionic Aciduria | Methylmalonic Aciduria | Urea Cycle DisorderSwitzerland
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Shanghai Vitalgen BioPharma Co., Ltd.Not yet recruitingGlutaric Acidemia Type I | Glutaric Aciduria Type IChina
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Wellstat TherapeuticsCompletedHereditary Orotic AciduriaUnited States
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National Taiwan University HospitalUnknownPropionic Acidemia (PA) , Methylmalonic Acidemia (MMA)Taiwan
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Recordati Rare DiseasesRecruitingPropionic Acidemia | Methylmalonic AcidemiaFrance, Spain, Italy, United Kingdom, Norway, Germany, Sweden
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Beijing Tongren HospitalCompleted
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Women and Infants Hospital of Rhode IslandFoundation for Society of Maternal-Fetal MedicineRecruiting
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Sheffield Teaching Hospitals NHS Foundation TrustCompleted
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West Kazakhstan Medical UniversityRecruitingOrnithine Transcarbamylase Deficiency | Biotinidase Deficiency | Citrullinemia | Glutaric Acidemia Type II | Argininosuccinic Aciduria | Maple Syrup Urine Disease | Primary Carnitine Deficiency | Homocystinuria | Carnitine Palmitoyltransferase II Deficiency | Arginase Deficiency | Very Long-chain Acyl-CoA... and other conditionsKazakhstan
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National Human Genome Research Institute (NHGRI)TerminatedCitrullinemia | Argininosuccinic Aciduria | Hyperargininemia | Ornithine Carbamoyltransferase Deficiency | Carbamoyl-Phosphate Synthase I Deficiency | N-Acetylglutamate Synthase DeficiencyUnited States
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CoA Therapeutics, Inc., a BridgeBio companyTerminatedHealthy Volunteers | Propionic Acidemia | Methylmalonic Acidemia | Organic AcidemiaUnited States
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University of SaskatchewanRecruiting
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Baylor College of MedicineChildren's Hospital of Philadelphia; Children's Hospital Colorado; Seattle Children... and other collaboratorsRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS DeficiencyUnited States
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Baylor College of MedicineChildren's National Research InstituteRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Carbamyl Phosphate Synthetase Deficiency | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS Deficiency | NAGS DeficiencyUnited States
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ModernaTX, Inc.CompletedPropionic Acidemia | Methylmalonic AcidemiaUnited States, United Kingdom, Spain, France
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HemoShear TherapeuticsPrometrika, LLC; AllStripes Research Inc.; Genome MedicalTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Canada, United Kingdom
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Target PharmaSolutions, Inc.Recordati Rare DiseasesEnrolling by invitationPropionic Acidemia | Methylmalonic Acidemia | HyperammonemiaUnited States
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Recordati Rare DiseasesTarget PharmaSolutions, Inc.RecruitingPropionic Acidemia | Methylmalonic Acidemia | HyperammonemiaUnited States
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HemoShear TherapeuticsTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Saudi Arabia, Australia
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King Abdullah International Medical Research CenterCompletedPropionic Acidemia | Methylmalonic AcidemiaSaudi Arabia
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Mendel TuchmanUniversity of Colorado, Denver; Children's Hospital of Philadelphia; University... and other collaboratorsTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States
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National Human Genome Research Institute (NHGRI)RecruitingPropionic Acidemia | Metabolic Disease | Organic AcidemiaUnited States
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Hacettepe UniversityCompletedOrganic Acidemia | Cognitive OrientationTurkey
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National Human Genome Research Institute (NHGRI)RecruitingInborn Errors of Metabolism | Methylmalonic Acidemia | Organic AcidemiaUnited States
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ModernaTX, Inc.RecruitingMethylmalonic AcidemiaUnited States, Spain, Canada, Netherlands, France, Australia, United Kingdom
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Bambino Gesù Hospital and Research InstituteCatholic University of the Sacred HeartCompletedGenetic Disease | Retinopathy | Methylmalonic Aciduria and Homocystinuria,Cblc TypeItaly
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ModernaTX, Inc.WithdrawnMetabolism, Inborn Errors | Methylmalonic Acidemia (MMA)
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Selecta Biosciences, Inc.National Human Genome Research Institute (NHGRI)SuspendedMethylmalonic Acidemia (MMA)United States
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LogicBio Therapeutics, IncAlexion Pharmaceuticals, Inc.TerminatedMethylmalonic AcidemiaUnited States
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Mendel TuchmanChildren's Hospital of Philadelphia; University of California, Los Angeles; Icahn... and other collaboratorsCompletedMethylmalonic Acidemia | Carbamoyl-Phosphate Synthase I Deficiency Disease | Propionic Acidemia, Type I and/or Type II | Ornithine Carbamoyltransferase DeficiencyUnited States
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Canada, United Kingdom
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ModernaTX, Inc.RecruitingMethylmalonic AcidemiaUnited States, Canada, Netherlands, United Kingdom
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Nicola LongoNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National...Completed
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Children's Hospital of PhiladelphiaEunice Kennedy Shriver National Institute of Child Health and Human Development...WithdrawnUrea Cycle Disorders, Inborn | Inborn Errors of Metabolism | Propionic Acidemia | Methylmalonic Acidemia | Carbamyl Phosphate Synthetase Deficiency
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University College DublinUnknownLactate Blood Increase | Exercise-Induced Lactic Acidemia
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German Diabetes CenterUniversity Children's Hospital, DüsseldorfCompletedMetabolic Syndrome | Insulin Resistance | Energy MetabolismGermany
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Spain, Netherlands, France, United Kingdom, Australia, Canada, Italy
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Canada, United Kingdom
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University of PittsburghCompleted
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LogicBio Therapeutics, IncAlexion Pharmaceuticals, Inc.Active, not recruitingMethylmalonic AcidemiaUnited States
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PTC TherapeuticsGenzyme, a Sanofi CompanyTerminatedAmino Acid Metabolism, Inborn ErrorsFrance, United Kingdom, Italy, Belgium, Germany, Switzerland
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Children's National Research InstituteNational Center for Advancing Translational Sciences (NCATS)RecruitingHypoxic-Ischemic Encephalopathy | Urea Cycle Disorder | Organic Acidemia | Fatty Acid Oxidation Disorder | Maple Syrup Urine Disease | Glutaric Acidemia IUnited States
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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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Centre Hospitalier Régional de la CitadelleSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Universitaire... 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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Raydiant Oximetry, Inc.TerminatedFetal Hypoxia | Fetal Distress | Fetal Acidemia | Fetal or Neonatal Effect of Complication of LaborUnited States
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Uta Lichter-KoneckiMedical College of Wisconsin; Children's National Research InstituteCompletedUrea Cycle Disorders | Organic AcidemiasUnited States