Search clinical trials for: Inherited Metabolic Disease
Total 381604 results
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Enzyvant Therapeutics GmBHCompletedFarber Disease | Farber's Disease | Farber Lipogranulomatosis | Acid Ceramidase Deficiency | Ceramidase Deficiency | N-Laurylsphingosine Deacylase Deficiency | ASAH1 MutationUnited States, Egypt, Canada, Italy, Turkey, Argentina, Germany, India, Sweden
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Emory UniversityCompletedSickle Cell Disease | Fanconi Anemia | Hurler Syndrome | Chronic Granulomatous Disease | Severe Congenital Neutropenia | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Shwachman-Diamond Syndrome | Thalassemia Major | Leukocyte... and other conditionsUnited States
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Jesse RomanGenentech, Inc.UnknownInterstitial Lung Disease | Hermansky Pudlak SyndromeUnited States, Puerto Rico
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Roswell Park Cancer InstituteCompletedPrimary Myelofibrosis | Polycythemia Vera | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma and other conditionsUnited States
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National Human Genome Research Institute (NHGRI)CompletedCancer | Myelofibrosis | Pulmonary Fibrosis | Gaucher Disease | Hermansky-Pudlak Syndrome (HPS)United States
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Masonic Cancer Center, University of MinnesotaCompletedWolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Sanfilippo Syndrome | GM1 Gangliosidosis | Globoid Cell Leukodystrophy | Tay Sachs Disease | Sandhoffs Disease | I-Cell DiseaseUnited States
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Nutricia UK LtdCompletedPhenylketonurias | Hyperphenylalaninaemia, Type IUnited Kingdom
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Nutricia UK LtdWithdrawn
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University of MiamiMassachusetts General HospitalCompletedAmyotrophic Lateral SclerosisUnited States
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Novartis PharmaceuticalsWithdrawnInherited Metabolic Disorders IMD
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Fate TherapeuticsTerminatedMetabolic DisordersUnited States
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Novo Nordisk A/SCompletedHaemophilia A, Haemophilia B, ObesityUnited States
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University of Colorado, DenverCompletedHomocystinuriaUnited States
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Horizon Pharma USA, Inc.Terminated
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Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis | Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Krabbe Disease | Adrenoleukodystrophy (ALD) | Hunter Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Metachromatic Leukodystrophy (MLD) | Maroteaux-Lamy SyndromeUnited States
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Masonic Cancer Center, University of MinnesotaCompletedLysosomal Storage Disease | Peroxisomal DisorderUnited States
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Horizon Pharma USA, Inc.CompletedInherited Mitochondrial Disease, Including Leigh SyndromeUnited States
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William Gahl, M.D.National Human Genome Research Institute (NHGRI)CompletedPulmonary Fibrosis | Inborn Errors of Metabolism | Albinism | Oculocutaneous Albinism | Platelet Storage Pool DeficiencyUnited States
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Emory UniversityChildren's Healthcare of AtlantaTerminatedSickle Cell Disease | Fanconi Anemia | Thalassemia | Severe Congenital Neutropenia | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Leukocyte Adhesion Deficiency | Dyskeratosis-congenita | Chediak-Higashi Syndrome | Chronic-granulomatous Disease | Schwachman-Diamond...United States
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National Institute of Allergy and Infectious Diseases...WithdrawnColitis | Cytokines | Hermanski-Pudlak Syndrome | Lymphocytes | Drug EvaluationUnited States
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National Human Genome Research Institute (NHGRI)CompletedHealthy VolunteersUnited States
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Hospital Universitari Vall d'Hebron Research InstituteUniversidad de MurciaUnknownCongenital Heart Diseases | Antithrombin III Deficiency | Congenital Disorder of Glycosylation | Conotruncal DefectsSpain
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Children's Hospital Los AngelesLucile Packard Children's HospitalTerminatedMetabolic Diseases | Stem Cell Transplantation | Chronic Granulomatous Disease | Bone Marrow Transplantation | Thalassemia | Wiskott-Aldrich Syndrome | Genetic Diseases | Peripheral Blood Stem Cell Transplantation | Pediatrics | Diamond-Blackfan Anemia | Allogeneic Transplantation | Combined Immune Deficiency | X-linked Lymphoproliferative Disease
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NovartisCompletedMyelodysplastic Syndromes | Hemoglobinopathies | Diamond-Blackfan Anemia | Transfusional Iron Overload | Other Inherited or Acquired Anaemia | MPD Syndrome | Other Rare AnaemiasAustralia
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Eric HoffmanNational Institute on Minority Health and Health Disparities (NIMHD); University... and other collaboratorsCompletedObesity | Diabetes Mellitus, Type 2 | Metabolic SyndromeUnited States
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The Hospital for Sick ChildrenCanadian Hemophilia SocietyCompletedHemophilia A | Hemophilia B | Factor IX Deficiency | Factor VIII DeficiencyCanada
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Tarix PharmaceuticalsWithdrawnHematologic Malignancy | Cord Blood Transplant | Inherited Metabolic DiseaseUnited States
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Children's Hospital Los AngelesCompletedGranuloma | Anemia | Thrombocytopenia | Neutropenia | Sickle Cell Disease | Thalassemia | Niemann-Pick Disease | Wiskott-Aldrich Syndrome | Osteopetrosis | Fucosidosis | Chediak Higashi Syndrome | Hurler DiseaseUnited States
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Virginia Commonwealth UniversityCSL BehringCompletedHemophilia A | Hemophilia B | Vitamin D DeficiencyUnited States
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Helsinki University Central HospitalEli Lilly and CompanyCompleted
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Baylor College of MedicineThe Methodist Hospital Research InstituteTerminatedInherited Metabolic Storage DiseasesUnited States
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Fred Hutchinson Cancer CenterCompletedMetabolism, Inborn Errors | Granulomatous Disease, ChronicUnited States
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Children's University Hospital, IrelandUniversity College Dublin; Health Research Board, Ireland; Medical Research Charities...Completed
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AldagenTerminatedMucopolysaccharidosis | Inborn Errors of Metabolism | Lysosomal Storage Disorders | Inherited Metabolic Diseases | Peroxisomal Storage DiseasesUnited States
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National Human Genome Research Institute (NHGRI)TerminatedPulmonary Fibrosis | Metabolic Disease | Oculocutaneous Albinism | Hermansky-Pudlak Syndrome (HPS) | Platelet Storage Pool DeficiencyUnited States
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Oslo University HospitalCompletedHereditary Factor VIII Deficiency Disease With InhibitorNorway
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Mayo ClinicNational Institutes of Health (NIH)CompletedObesityUnited States
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UMC UtrechtUnknownCardiovascular Disease | HaemophiliaNetherlands, United Kingdom
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Fairview University Medical CenterTerminatedImmunologic Deficiency Syndromes | Common Variable Immunodeficiency | Severe Combined Immunodeficiency | Chronic Granulomatous Disease | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | Graft Versus Host Disease | X-linked Agammaglobulinemia | Chediak-Higashi Syndrome | Virus-Associated... and other conditionsUnited States
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Grifols Therapeutics LLCCompletedCommon Variable Immunodeficiency | Severe Combined Immunodeficiency | Wiskott-Aldrich Syndrome | Agammaglobulinemia | Immunologic Deficiency SyndromeCanada, United States
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National Institute of Arthritis and Musculoskeletal...CompletedMyositis | Dermatomyositis | Polymyositis | Glycogen Storage Disease Type II | Glycogen Storage Disease Type VIIUnited States
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National Institute of Mental Health (NIMH)Completed
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National Center for Research Resources (NCRR)James Whitcomb Riley Hospital for ChildrenCompletedThrombocytopenia | Dyskeratosis Congenita | Fanconi's Anemia | Shwachman Syndrome
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McGill UniversityRecruiting
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Penelope J. Hallett, Ph.D.National Institute of Neurological Disorders and Stroke (NINDS)Not yet recruitingParkinson DiseaseUnited States
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University of LeipzigRhythm Pharmaceuticals, Inc.RecruitingChild Obesity | Hunger | Gene AbnormalityGermany
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Hospital Universitario de BurgosRecruiting
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Oregon Health and Science UniversityNot yet recruiting
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UGECAM Rhône-AlpesRecruiting
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Odense University HospitalUniversity of Ulm; University of Birmingham; Hospital of South West JutlandNot yet recruitingOsteoporosis SecondaryDenmark