Search clinical trials for: Inherited Metabolic Disorder
Total 165253 results
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Washington University School of MedicineAlzheimer's Association; Eisai Inc.Not yet recruitingDementia | Alzheimer's Disease | Alzheimer's Disease, FamilialUnited States, Australia, United Kingdom
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Royal Marsden NHS Foundation TrustRecruitingLynch Syndrome | Familial Adenomatous Polyposis | Hereditary Diffuse Gastric Cancer | Peutz-Jeghers Syndrome | Cowden Syndrome | Li Fraumeni Syndrome | MutYH-associated Polyposis | SMAD4 Gene MutationUnited Kingdom
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Assistance Publique - Hôpitaux de ParisNot yet recruitingHemophilia A | Hemophilia B | Cystic Fibrosis | Sickle Cell Disease | Muscular Dystrophy, Duchenne | Fragile X Syndrome | Huntington Disease | Myotonic Dystrophy | Autosomal Recessive Polycystic Kidney Disease | Neurofibromatosis-Noonan Syndrome | Muscular Dystrophy, Becker | Invasive PreNatal Diagnosis in a... and other conditionsFrance
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UK Kidney AssociationRecruitingVasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditionsUnited Kingdom
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Boston Children's HospitalChildren's Hospital Medical Center, CincinnatiRecruitingShwachman-Diamond Syndrome | Shwachman-Diamond Syndrome-LikeUnited States
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Institut National de la Santé Et de la Recherche...RecruitingNeurofibromatosis Type 1 | Ichthyosis | Ectodermal Dysplasia | Albinism | Pemphigus | Mucous Membrane Pemphigoid | Inherited Epidermolysis Bullosa | Palmoplantar Keratoderma | Incontinentia PigmentiFrance
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Hereditary Neuropathy FoundationRecruitingCharcot-Marie-Tooth Disease | Charcot-Marie-Tooth Disease, Type IA | Charcot-Marie-Tooth Disease Type 2A | Charcot-Marie-Tooth | Charcot-Marie-Tooth Disease, Type IB | Charcot-Marie-Tooth Disease Type 2 | Charcot-Marie-Tooth Disease, Type 2C | Charcot-Marie-Tooth Disease Type 2A2B | Charcot-Marie-Tooth... 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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National Cancer Institute (NCI)RecruitingFanconi Anemia | Inherited Bone Marrow Failure SyndromeUnited States
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Tsung-Ying LiRecruitingHemophilia | Vitamin D Deficiency | Body Composition | Sarcopenia | Osteoporosis | Hemophilic ArthropathyTaiwan
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University Hospital, GrenobleRecruitingSchizophrenia | Inherited Metabolic Disorder of Nervous SystemFrance
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Mayo ClinicHopital du Sacre-Coeur de MontrealRecruitingAutosomal Dominant Polycystic Kidney Disease | Nephrogenic Diabetes Insipidus | Acquired Nephrogenic Diabetes Insipidus | Congenital Nephrogenic Diabetes InsipidusUnited States
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National Human Genome Research Institute (NHGRI)RecruitingDiabetes | Undiagnosed Diseases | Inherited Metabolic Disorders | Batten's Disease | Tay SachsUnited States
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Aspa TherapeuticsRecruitingCanavan DiseaseUnited States
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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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Assistance Publique - Hôpitaux de ParisRecruiting
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Memorial Sloan Kettering Cancer CenterRecruitingHemoglobinopathies | AML | MDS | Bone Marrow Failure | ALL | Inherited Metabolic Disorders | HLH | MPD Withou Myelofibrosis | NHL or HLUnited States
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Paul SzabolcsRecruitingCongenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary AnemiasUnited States
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PD Dr. Andreas StraußBayerRecruiting
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OHSU Knight Cancer InstituteNational Cancer Institute (NCI); Oregon Health and Science UniversityRecruitingMalignant Solid Neoplasm | Lynch Syndrome | Hematopoietic and Lymphoid System Neoplasm | Breast Ductal Carcinoma In Situ | Hereditary Neoplastic Syndrome | BRCA1/2-Associated Hereditary Breast and Ovarian Cancer SyndromeUnited States
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Johns Hopkins All Children's HospitalRecruitingMetabolic Disease | Primary Immune Deficiency DisordersUnited States
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National Institute of Neurological Disorders and...RecruitingAmyotrophic Lateral Sclerosis Type 4 | Inherited Neurological Disorders of RNA ProcessingUnited States
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Centre Hospitalier Universitaire de Saint EtienneMinistry of Health, FranceRecruiting
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Aspa TherapeuticsRecruiting
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University of NebraskaNational Cancer Institute (NCI); VA Nebraska Western Iowa Health Care System; Virginia Mason Hospital/Medical CenterRecruitingDiabetes Mellitus, Type 2 | PreDiabetes | Genetic Predisposition to Disease | Chronic Pancreatitis | Inherited Disease | Pancreas CystUnited States
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Assistance Publique Hopitaux De MarseilleRecruitingInherited Metabolic Diseases Requiring Restrictive and Specific DietFrance
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Foundation Fighting BlindnessRecruitingRetinitis Pigmentosa | Choroideremia | Usher Syndrome | Batten Disease | Leber Congenital Amaurosis | Goldmann-Favre Syndrome | Kearns-Sayre Syndrome | Retinal Disease | Bardet-Biedl Syndrome | Stargardt Disease | Cone Dystrophy | Retinoschisis | Achromatopsia | Gyrate Atrophy | Eye Diseases Hereditary | Bassen-Kornzweig... and other conditionsUnited States
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Joanne Kurtzberg, MDThe Marcus FoundationRecruitingMucopolysaccharidosis II | Brain Diseases, Metabolic, Inborn | Adrenoleukodystrophy | Batten Disease | Leukodystrophy, Metachromatic | Leukodystrophy, Globoid Cell | Alpha-Mannosidosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | Neimann Pick Disease | Sanfilippo MucopolysaccharidosesUnited States
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Masonic Cancer Center, University of MinnesotaRecruitingSickle Cell Disease | Severe Congenital Neutropenia | Paroxysmal Nocturnal Hemoglobinuria | Glanzmann Thrombasthenia | Shwachman-Diamond Syndrome | Diamond Blackfan Anemia | Transfusion Dependent Alpha- or Beta- Thalassemia | Non-Malignant Hematologic DisordersUnited States
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Masonic Cancer Center, University of MinnesotaRecruitingHurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditionsUnited States
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Paul SzabolcsRecruitingCongenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias | Systemic Juvenile Idiopathic Arthritis (sJIA)United States
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The Familial Hypercholesterolemia FoundationRecruitingHypercholesterolemiaUnited States
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National Institute of Neurological Disorders and...RecruitingMotor Neuron Disease | Neuromuscular Disease | Inherited Neuromuscular Conditions | Inherited NeuropathiesUnited States
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Washington University School of MedicineRecruitingSevere Combined Immunodeficiency | Chronic Granulomatous Disease | DiGeorge Syndrome | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | X-linked Lymphoproliferative Syndrome | X-linked Agammaglobulinemia | Chediak-Higashi Syndrome | IPEX | Autoimmune Lymphoproliferative Syndrome | Common Variable... and other conditionsUnited States
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Sanford HealthNational Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaboratorsRecruitingMitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditionsUnited States, Australia
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Columbia UniversityNational Institute of Neurological Disorders and Stroke (NINDS)RecruitingMitochondrial Diseases | Mitochondrial Disorders | Mitochondrial Genetic Disorders | Disorder of Mitochondrial Respiratory Chain Complexes | Deletion and Duplication of Mitochondrial DNAUnited States, Canada
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Masonic Cancer Center, University of MinnesotaRecruitingHemoglobinopathies | Immunodeficiencies | Hematologic DisordersUnited States
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Masonic Cancer Center, University of MinnesotaRecruitingCommon Variable Immunodeficiency | Chronic Granulomatous Disease | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | X-linked Lymphoproliferative Disease | Langerhan's Cell Histiocytosis | Chediak-Higashi Syndrome | Griscelli Syndrome | Bare Lymphocyte Syndrome | SCID | Omenn's Syndrome | Reticular... and other conditionsUnited States
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Washington University School of MedicineNational Institute on Aging (NIA)RecruitingAlzheimer's DiseaseUnited States, Argentina, Australia, Brazil, Canada, Colombia, Germany, Japan, Korea, Republic of, Mexico, United Kingdom, Spain
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Andrea GropmanEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsRecruitingBrain Diseases, Metabolic, Inborn | Amino Acid Metabolism, Inborn Errors | Urea Cycle DisordersUnited States, Canada, Germany, Switzerland
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National Institute of Allergy and Infectious Diseases...RecruitingChronic Granulomatous Disease (CGD) | X-Linked Severe Combined Immune Deficiency (XSCID) | Leukocyte Adhesion Deficiency 1 (LAD) | Graft Versus Host Disease (cGvHD)United States
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National Human Genome Research Institute (NHGRI)RecruitingPulmonary Fibrosis | Healthy Volunteers | Hermansky-Pudlak Syndrome (HPS)United States
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National Heart, Lung, and Blood Institute (NHLBI)Recruiting
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National Cancer Institute (NCI)RecruitingFanconi Anemia | Dyskeratosis Congenita | Diamond Blackfan Anemia | Shwachman Diamond Syndrome | Inherited Bone Marrow Failure Syndrome, Aplastic AnemiaUnited States
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National Institute of Neurological Disorders and...RecruitingMotor Neuron Disease | Muscular Dystrophy | Peripheral Nervous System Disease | Muscular DiseaseUnited States, Mali
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National Human Genome Research Institute (NHGRI)RecruitingHermansky-Pudlak Syndrome (HPS)United States
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Cyclo Therapeutics, Inc.Active, not recruitingNiemann-Pick Disease, Type C1United States, Taiwan, United Kingdom, Israel, Spain, Brazil, Italy, Australia, Turkey, Saudi Arabia, Argentina, Poland, Germany
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Hoffmann-La RocheActive, not recruitingHemophilia A | Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor | Moderate Hereditary Factor VIII Deficiency Disease Without InhibitorUnited States, Netherlands, Belgium, France, Germany, South Africa, Spain, Canada, United Kingdom, Poland
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National Human Genome Research Institute (NHGRI)CompletedUndiagnosed Disease | Healthy Volunteer (Adult With Typically Developing Child) | Inherited Neurodegenerative Disorders | Inherited Metabolic Disorders (Inborn Errors of Metabolism) | Alzheimer's Disease and Related DementiasUnited States
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AmgenCompletedFamilial HypercholesterolemiaCanada, Italy, Norway, Belgium, United States, Turkey, Australia, Austria, South Africa, Poland, Switzerland, Spain, Portugal, Slovenia, Brazil, Russian Federation, Hungary, United Kingdom, Colombia, Czechia, Netherlands, Greece, Ma...