Clinical Trials on Steroid Metabolism, Inborn Errors
Total 4351 results
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Centre Leon BerardRecruitingMyelodysplastic Syndromes | Severe Combined Immunodeficiency | Acute Leukemia | Haematological Malignancy | Hemoglobinopathy in Children | Bone Marrow AplasiaFrance
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Matrix Biomed, Inc.The University of Texas Health Science Center, HoustonNot yet recruitingAtaxia Telangiectasia | Ataxia Telangiectasia Louis-Bar | Ataxia Telangiectasia in ChildrenUnited States
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National Heart, Lung, and Blood Institute (NHLBI)RecruitingParkinson's Disease | Atherosclerosis | Cardiomyopathy | Li-Fraumeni Syndrome | Cardiovascular CapacityUnited States
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Quince Therapeutics S.p.A.CompletedGenetic Syndrome | Nervous System DiseaseIndia, United States, Australia, Germany, Italy, Norway, Poland, Spain, Tunisia, United Kingdom, Israel, Belgium
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University of ChicagoPfizer; Biogen; APDM Wearable TechnologiesActive, not recruitingSpinocerebellar Ataxia Type 3 | Friedreich Ataxia | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6United States
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Argo SanteGenSight BiologicsCompleted
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University of PittsburghCompletedPulmonary Hypertension | Friedreich Ataxia | Iron-sulfur Cluster DeficiencyUnited States
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Beth Israel Deaconess Medical CenterNational Institute of Neurological Disorders and Stroke (NINDS)CompletedAmyotrophic Lateral Sclerosis | Muscular Dystrophy | Radiculopathy | Trauma Injury | Orthopedic Disorder | Myopathy | Polyneuropathy and MononeuropathiesUnited States
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Stanford UniversityCompletedEpidermolysis Bullosa Simplex | Epidermolysis Bullosa Simplex Kobner | Weber-Cockayne SyndromeUnited States
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University of BirminghamActive, not recruitingWolfram SyndromeFrance, United Kingdom, Poland, Spain
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Centre d'Etude des Cellules SouchesGenethonRecruitingWolfram SyndromeFrance, Spain
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Amylyx Pharmaceuticals Inc.Active, not recruiting
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Ospedale San RaffaeleRecruiting
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Washington University School of MedicineEnrolling by invitation
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Reata, a wholly owned subsidiary of BiogenCompletedFocal Segmental Glomerulosclerosis | IgA Nephropathy | Autosomal Dominant Polycystic Kidney | CKD Associated With Type 1 DiabetesUnited States
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San Raffaele UniversityUnita' di Gastroenterologia - Policlinico Universitario di Bari; Unita' di... and other collaboratorsRecruitingLynch Syndrome | MLH1 Gene Mutation | MSH2 Gene Mutation | MSH6 Gene Mutation | PMS2 Gene Mutation | Lynch Syndrome II | Small Bowel Adenocarcinoma | Lynch Syndrome IItaly
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National Cancer Institute (NCI)CompletedMelanoma | Skin Cancer | Basal Cell Carcinoma | Squamous Cell Carcinoma | Xeroderma PigmentosumUnited States
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RWJ Barnabas Health at Jersey City Medical CenterUnknownLynch Syndrome | Endometrial Cancer | Somatic Mutation | Cancer Gene MutationUnited States
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute...CompletedSevere Combined Immunodeficiency | Adenosine Deaminase Deficiency | Immune System Disorder | Autosomal Recessive Disorder | Purine-Nucleoside Phosphorylase Deficiency | Severe Combined Immunodeficiency With Absence of T and B Cells | X-Linked Severe Combined ImmunodeficiencyUnited States
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Assistance Publique - Hôpitaux de ParisCompleted
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National Heart, Lung, and Blood Institute (NHLBI)CompletedNeoplasms | Myelodysplastic Syndromes | Hematologic Diseases | Leukemia | Severe Combined Immunodeficiency | Fanconi Anemia | Anemia, Aplastic | Hematopoietic Stem Cell Transplantation
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University of PlymouthRecruitingAtaxia Telangiectasia | Ataxia Telangiectasia in ChildrenUnited Kingdom
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Ohio State University Comprehensive Cancer CenterActive, not recruitingLynch Syndrome | Relatives | Endometrial Carcinoma | Endometrial AdenocarcinomaUnited States
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Assistance Publique - Hôpitaux de ParisTerminatedNeuromuscular Diseases | Amyotrophic Lateral Sclerosis | Duchenne Muscular DystrophyFrance
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Vanderbilt University Medical CenterRecruitingPostural Orthostatic Tachycardia Syndrome | Acute Hepatic PorphyriaUnited States
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National Neuroscience InstituteNanyang Technological University; Institute for Infocomm Research; Institute... and other collaboratorsActive, not recruitingMotor Neuron Disease | Amyotrophic Lateral Sclerosis | Muscular Dystrophies | Spinal Cord Injuries | Tetraplegia | Tetraplegia/Tetraparesis | Locked-in SyndromeSingapore
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National Institute of Neurological Disorders and...CompletedMotor Neuron Disease | Spinal and Bulbar Muscular Atrophy (SBMA)United States
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Sheba Medical CenterCompletedFatigue | Ataxia | Dystonia | Parkinsonism | ChoreaIsrael
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TakedaNot yet recruitingHereditary Angioedema (HAE)China
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ACADIA Pharmaceuticals Inc.RecruitingHyperphagia in Prader-Willi SyndromeUnited States, Canada, United Kingdom
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CSL BehringRecruitingHereditary Angioedema (HAE)United States, Germany, Israel, Australia, Canada
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TakedaRecruitingHereditary Angioedema (HAE)United Kingdom
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TakedaRecruitingHereditary Angioedema (HAE)Japan
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Nanfang Hospital of Southern Medical UniversityNot yet recruitingOsteogenesis Imperfecta | Tooth Socket | Total Absence of Permanent Teeth
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TakedaRecruitingHereditary Angioedema (HAE)Poland
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ShireTakeda Development Center Americas, Inc.RecruitingHereditary Angioedema (HAE)France, Austria, Australia, Spain, Czechia, Italy, United Kingdom, Germany, Israel, Ireland, Brazil, Denmark, Greece, Sweden
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ACADIA Pharmaceuticals Inc.Enrolling by invitationHyperphagia in Prader-Willi SyndromeUnited States
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University of Colorado, DenverPKD FoundationCompletedPolycystic Kidney, Autosomal DominantUnited States
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ShireTakeda Development Center Americas, Inc.CompletedHereditary Angioedema (HAE)Canada, United States, Puerto Rico
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TakedaCompletedHereditary Angioedema (HAE)France, Germany, Austria, Greece
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ShireCompletedHereditary Angioedema (HAE)United States, Jordan, Canada
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ShireDyax Corp.CompletedHereditary Angioedema (HAE)United States, Canada, Germany, Italy, Jordan, Puerto Rico, United Kingdom
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ShireWithdrawnHereditary Angioedema (HAE)
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BioCryst PharmaceuticalsCompletedHereditary Angioedema (HAE)Austria, Denmark, France, Germany, Hungary, Israel, Italy, North Macedonia, Poland, Romania, Switzerland, United Kingdom
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ShireCompletedHereditary Angioedema (HAE)Germany, Spain, United Kingdom, France, Italy, Belgium
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BioCryst PharmaceuticalsApproved for marketingHereditary Angioedema | Prophylaxis | HAE
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University Hospital, GrenobleTerminatedHealthy Volunteers | Hereditary AngioedemaFrance
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Michael RafiiJanssen Research & Development, LLCCompleted
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Rambam Health Care CampusSchneider Children's HospitalUnknownFamilial Mediterranean FeverIsrael