Clinical Trials on Methylmalonic Aciduria cblA Type
Total 30 results
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McGill University Health Centre/Research Institute...UnknownMethylmalonic Acidemia | Malonic AciduriaCanada
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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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National Taiwan University HospitalUnknownPropionic Acidemia (PA) , Methylmalonic Acidemia (MMA)Taiwan
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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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Recordati Rare DiseasesRecruitingPropionic Acidemia | Methylmalonic AcidemiaFrance, Spain, Italy, United Kingdom, Norway, Germany, Sweden
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CoA Therapeutics, Inc., a BridgeBio companyTerminatedHealthy Volunteers | Propionic Acidemia | Methylmalonic Acidemia | Organic AcidemiaUnited 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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HemoShear TherapeuticsPrometrika, LLC; AllStripes Research Inc.; Genome MedicalTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Canada, United Kingdom
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ModernaTX, Inc.RecruitingMethylmalonic AcidemiaUnited States, Canada, Netherlands, United Kingdom
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ModernaTX, Inc.RecruitingMethylmalonic AcidemiaUnited States, Spain, Canada, Netherlands, France, Australia, United Kingdom
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Target PharmaSolutions, Inc.Recordati Rare DiseasesEnrolling by invitationPropionic Acidemia | Methylmalonic Acidemia | HyperammonemiaUnited States
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ModernaTX, Inc.CompletedPropionic Acidemia | Methylmalonic AcidemiaUnited States, United Kingdom, Spain, France
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LogicBio Therapeutics, IncAlexion Pharmaceuticals, Inc.TerminatedMethylmalonic AcidemiaUnited 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)RecruitingInborn Errors of Metabolism | Methylmalonic Acidemia | Organic 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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Shanghai Vitalgen BioPharma Co., Ltd.RecruitingGlutaric Acidemia Type I | Glutaric Aciduria Type IChina
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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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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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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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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States
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Centre Hospitalier Universitaire de LiegeSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... 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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Therapeutic Advances in Childhood Leukemia ConsortiumRecruitingNeurofibromatosis 1 | JMML | Leukemia, Juvenile Myelomonocytic | JCML | CBL SyndromeUnited States
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Masonic Cancer Center, University of MinnesotaRecruitingAcute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Acute Lymphoblastic Leukemia | AML | Acute Leukemia | Neurofibromatosis 1 | Hematologic Malignancy | Myelodysplasia | Minimal Residual Disease | Chromosome Abnormality | Monosomy 7 | Remission | Somatic Mutation | Cytogenetic Abnormality | TP53 | Fetal Hemoglobin | Intrachromosomal Amplification of Chromosome... and other conditionsUnited States
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National Human Genome Research Institute (NHGRI)RecruitingMetabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditionsUnited States