Clinical Trials on Metabolismo de esteroides, errores innatos
Total 2782 results
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Ludwig-Maximilians - University of MunichUnknownIschemic Stroke | Cerebral Amyloid Angiopathy | Intracerebral HemorrhageGermany
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Khondrion BVNot yet recruitingMitochondrial Diseases | Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation | Maternally Inherited Diabetes and Deafness (MIDD) | Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
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University Hospital Inselspital, BerneRecruitingCerebral Amyloid Angiopathy | Cadasil | Small Vessel Cerebrovascular Disease | CAA - Cerebral Amyloid AngiopathySwitzerland
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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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New York Stem Cell Foundation Research InstituteRecruitingPTSD | Multiple Sclerosis | Cervical Cancer | Diabetes Mellitus, Type 2 | Diabetes Mellitus | Diabetes | Macular Degeneration | Diabetes Mellitus, Type 1 | Parkinson Disease | Amyotrophic Lateral Sclerosis | Dementia | Ovarian Cancer | Alzheimer Disease | ALS | Vulvar Cancer | Post Traumatic Stress Disorder | Lewy Body Disease and other conditionsUnited States
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Masonic Cancer Center, University of MinnesotaTerminatedSickle Cell Disease | Thalassemia | Cerebral Adrenoleukodystrophy | Inherited Metabolic Disorders | High Risk Hematologic DisordersUnited States
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University Hospital FreiburgCompletedMyelodysplastic Syndromes | Fanconi Anemia | Dyskeratosis Congenita | Pearson Marrow-pancreas Syndrome | Shwachman-diamond SyndromeSpain, Germany, Switzerland, Austria, Netherlands, Italy, Czech Republic, Belgium, Denmark, Ireland
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute... and other collaboratorsRecruitingCongenital Amegakaryocytic Thrombocytopenia | Paroxysmal Nocturnal Hemoglobinuria | Diamond-Blackfan Anemia | Shwachman-Diamond Syndrome | Bone Marrow Failure Syndrome | Hereditary Sideroblastic Anemia | Hematologic Neoplasm With Germline GATA2 Mutation | Hematologic Neoplasm With Germline SAMD9... and other conditionsUnited 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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Hugo W. Moser Research Institute at Kennedy Krieger...RecruitingNeurodegenerative Diseases | Ataxia | Cerebellar Ataxia | Leukodystrophy | Adrenomyeloneuropathy | LBSL | Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation | Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)United States
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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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Children's Oncology GroupNational Cancer Institute (NCI)CompletedChronic Myelomonocytic Leukemia | Fanconi Anemia | Childhood Acute Erythroleukemia (M6) | Childhood Acute Megakaryocytic Leukemia (M7) | Childhood Acute Monoblastic Leukemia (M5a) | Childhood Acute Monocytic Leukemia (M5b) | Childhood Acute Myeloblastic Leukemia With Maturation (M2) | Childhood Acute... and other conditionsUnited States
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Hospital Universitari Vall d'Hebron Research InstituteErasme University Hospital; Cyprus Institute of Neurology and Genetics; EuroBloodNet...RecruitingIron Metabolism Disorders | Sickle Cell Disease | Thalassemia | Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder | Anemia Due to Membrane Defect | CDA | Sideroblastic Anemia | Constitutional Aplastic Anemia | Hereditary AnemiaSpain
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Jakub AntczakCompletedAdrenomyeloneuropathy | Hereditary Spastic ParaplegiaPoland
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University of FloridaLive Like Bella Pediatric Cancer ResearchCompletedCommon Variable Immunodeficiency | Severe Combined Immunodeficiency | Sickle Cell Disease | Hurler Syndrome | Chronic Granulomatous Disease | Thalassemia | Wiskott-Aldrich Syndrome | Adrenoleukodystrophy | X-linked Lymphoproliferative Disease | Diamond Blackfan Anemia | Bone Marrow Failure Syndrome | Hemophagocytic... and other conditionsUnited States
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Children's Hospital of PhiladelphiaIllumina, Inc.Active, not recruitingMucopolysaccharidoses | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2 Gangliosidosis | Zellweger... and other conditionsUnited States
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Children's Hospital of PhiladelphiaEli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaboratorsRecruitingMucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... 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
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University of Milano BicoccaComitato Maria Letizia Verga,ItalyRecruitingLymphoma, Non-Hodgkin | Leukemia, Myeloid, Acute | Leukemia, B-cell | Adrenoleukodystrophy | Lymphoma, Hodgkin | Thalassemia Major | Leukemia, T Cell | DrepanocytosisItaly
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University of CincinnatiChildren's Hospital Medical Center, CincinnatiTerminatedAdrenoleukodystrophy | Zellweger Syndrome | Infantile Refsum's Disease | Bifunctional Enzyme Deficiency
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Khondrion BVJulius Clinical; ProPharma Group; CertaraCompletedMitochondrial Diseases | Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation | Maternally Inherited Diabetes and Deafness (MIDD) | Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke Like Episodes (MELAS) | Chronic Progressive External Ophthalmoplegia (CPEO)Netherlands, Denmark, United Kingdom, Germany
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Mirum Pharmaceuticals, Inc.Children's Hospital Medical Center, CincinnatiCompletedCholestasis | Peroxisomal Disorders | Adrenoleukodystrophy | Zellweger Syndrome | Infantile Refsum's DiseaseUnited States
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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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Diane GeorgeActive, not recruitingSickle Cell Disease | Hemoglobinopathies | Severe Congenital Neutropenia | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Macrophage Activation Syndrome | Bone Marrow Failure Syndrome | Amegakaryocytic Thrombocytopenia | Schwachman Diamond Syndrome | Primary Immunodeficiency Syndromes | Acquired Immunodeficiency... and other conditionsUnited States
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McGill University Health Centre/Research Institute...RecruitingPeroxisome Biogenesis Disorder | Zellweger Spectrum Disorder | RCDP - Rhizomelic Chondrodysplasia Punctata | D-Bifunctional Protein Deficiency | Alpha-Methylacyl-CoA Racemase Deficiency | Peroxisomal Acyl-CoA Oxidase Deficiency | Peroxisomal Acyl-CoA Oxidase 2 Deficiency | ATP Binding Cassette Subfamily... and other conditionsCanada
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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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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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Roswell Park Cancer InstituteActive, not recruitingAcute Myeloid Leukemia | Polycythemia Vera | Myelofibrosis | Chronic Myelomonocytic Leukemia | Waldenstrom Macroglobulinemia | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Secondary Acute Myeloid Leukemia | Sickle Cell Disease | Myelodysplastic Syndrome | Plasma Cell Myeloma | Chronic Granulomatous... and other conditionsUnited States
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Sanguine BiosciencesCompletedDepression | Melanoma | Epilepsy | Lymphoma | Multiple Sclerosis | Kidney Cancer | Cervical Cancer | Diabetes Mellitus | Leukemia | Breast Cancer | Chronic Kidney Diseases | Chronic Obstructive Pulmonary Disease | Parkinson's Disease | Systemic Lupus Erythematosus | Multiple Myeloma | Hepatitis B | Insomnia | Colorectal Cancer | HIV/AID... and other conditionsUnited States
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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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City of Hope Medical CenterTerminatedPrimary Myelofibrosis | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Iron Overload | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell... and other conditionsUnited States
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Wake Forest University Health SciencesNational Cancer Institute (NCI)CompletedPrimary Myelofibrosis | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent Adult Diffuse Large Cell Lymphoma and other conditionsUnited States