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- US Clinical Trials Registry
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Search clinical trials for: methylmalonic aciduria with homocystinuria
Total 158174 results
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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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Fondazione I.R.C.C.S. Istituto Neurologico Carlo...RecruitingNeurologic Disorder | Rare Diseases | Metabolic Disease | Inherited Disease | Undiagnosed Disease | Neuro-Degenerative DiseaseItaly
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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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Bambino Gesù Hospital and Research InstituteCatholic University of the Sacred HeartCompletedGenetic Disease | Retinopathy | Methylmalonic Aciduria and Homocystinuria,Cblc TypeItaly
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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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ModernaTX, Inc.RecruitingMethylmalonic AcidemiaUnited States, Spain, Canada, Netherlands, France, Australia, United Kingdom
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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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Travere Therapeutics, Inc.Recruiting
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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
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ModernaTX, Inc.RecruitingMethylmalonic AcidemiaUnited States, Canada, Netherlands, United Kingdom
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Recordati Rare DiseasesRecruitingPropionic Acidemia | Methylmalonic AcidemiaFrance, Spain, Italy, United Kingdom, Norway, Germany, Sweden
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Canada, United Kingdom
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LogicBio Therapeutics, IncAlexion Pharmaceuticals, Inc.TerminatedMethylmalonic AcidemiaUnited States
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Travere Therapeutics, Inc.Active, not recruiting
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HemoShear TherapeuticsTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Saudi Arabia, Australia
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HemoShear TherapeuticsPrometrika, LLC; AllStripes Research Inc.; Genome MedicalTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Canada, United Kingdom
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CoA Therapeutics, Inc., a BridgeBio companyTerminatedHealthy Volunteers | Propionic Acidemia | Methylmalonic Acidemia | Organic AcidemiaUnited States
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Aeglea BiotherapeuticsTerminatedHomocystinuria Due to Cystathionine Beta-Synthase DeficiencyUnited States, United Kingdom, Australia
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Selecta Biosciences, Inc.National Human Genome Research Institute (NHGRI)SuspendedMethylmalonic Acidemia (MMA)United 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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Indiana UniversityCompletedKidney CalculiUnited States
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ModernaTX, Inc.WithdrawnMetabolism, Inborn Errors | Methylmalonic Acidemia (MMA)
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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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Universität des SaarlandesInstitute of Alternative and Sustainable Nutrition (IFANE)CompletedCobalamin DeficiencyGermany
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Assistance Publique - Hôpitaux de ParisCompleted
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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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Wellstat TherapeuticsCompletedHereditary Orotic AciduriaUnited States
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Uta Lichter-KoneckiMedical College of Wisconsin; Children's National Research InstituteCompletedUrea Cycle Disorders | Organic AcidemiasUnited States
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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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McGill University Health Centre/Research Institute...UnknownMethylmalonic Acidemia | Malonic AciduriaCanada
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McGill University Health Centre/Research Institute...March of DimesCompleted
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Omer AyhanRecruiting
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Grupo LusófonaCentro Hospitalar Universitario do Algarve; Universidade Lusófona de Humanidades... and other collaboratorsNot yet recruitingBreast Cancer | Chemotherapy EffectPortugal
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Central South UniversityNot yet recruiting
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Quanta TherapeuticsNot yet recruitingAdvanced Solid Tumor
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Arab American University (Palestine)Not yet recruitingAutism Spectrum Disorder
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Merck Sharp & Dohme LLCNot yet recruiting
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Merck Sharp & Dohme LLCNot yet recruitingColorectal Cancer | Biliary Tract Cancer | Pancreatic Ductal Carcinoma
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Massachusetts General HospitalUnited States Department of DefenseNot yet recruiting
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Novartis PharmaceuticalsNot yet recruitingLeukemia, Myelogenous, Chronic, BCR-ABL Positive
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I.R.C.C.S. Fondazione Santa LuciaNot yet recruiting
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The First Affiliated Hospital with Nanjing Medical...Not yet recruiting
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Tufts UniversityNot yet recruitingOral Lichen Planus | Oral Lichenoid ReactionUnited States
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Sichuan Kelun Pharmaceutical Research Institute...Not yet recruitingAdvanced Solid Tumors
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Galderma R&DRecruiting
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University of Kansas Medical CenterNot yet recruiting
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Malmö UniversityNot yet recruitingDiabetes Mellitus | Skin and Connective Tissue Diseases
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BayerNot yet recruitingChronic Kidney Disease | Arterial Hypertension | Non-proliferative Diabetic RetinopathyGermany
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Boston Medical CenterCharles H. Hood FoundationNot yet recruiting