Clinical Trials on Inborn Errors of Metabolism in United States
Total 3225 results
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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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University of Alabama at BirminghamLakeshore Foundation; YMCA of Greater BirminghamActive, not recruitingStroke | Multiple Sclerosis | Cerebral Palsy | Parkinson Disease | Traumatic Brain Injury | Spinal Cord Injuries | Spina BifidaUnited States
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BioCryst PharmaceuticalsCompletedHereditary AngioedemaGermany, United Kingdom
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University of South FloridaUniversity of Pennsylvania; University of Toronto; University of Western Ontario...CompletedVasculitis | Microscopic Polyangiitis | Systemic Vasculitis | Takayasu's Arteritis | Cryoglobulinemic Vasculitis | Behcet's Disease | CNS Vasculitis | Temporal Arteritis | Polyarteritis Nodosa (PAN) | Urticarial Vasculitis | Henoch-Schoenlein Purpura | Wegener Granulomatosis | Eosinophilic GranulomatosisUnited States
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University of PlymouthCompletedDiabetic Peripheral Neuropathy | Limited Joint Mobility Syndrome | Ankle Joint Range of Motion | Forefoot Peak Plantar Pressure | Foot and Ankle Mobilisations | Home Exercise Programme / StretchesUnited Kingdom
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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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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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University of BirminghamActive, not recruitingWolfram SyndromeFrance, United Kingdom, Poland, Spain
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The Methodist Hospital Research InstituteActive, not recruitingNon-Small Cell Lung Cancer | DNA Repair-Deficiency Disorders | Head and Neck Squamous Cell Carcinoma | Urothelial Carcinoma Bladder | Classical Hodgkin Lymphoma | Malignant MelanomaUnited States
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Intellia TherapeuticsEnrolling by invitationHereditary AngioedemaFrance, New Zealand, United Kingdom, Netherlands
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Pharming Technologies B.V.CompletedHereditary AngioedemaIsrael, Italy, United States, Czechia, Germany, Hungary, Poland, Romania, Slovakia, North Macedonia
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Molecular NeuroImagingInstitute for Neurodegenerative DisordersCompletedAlzheimer's Disease (AD) | Tauopathies | Parkinson's Disease (PD) | Progressive Supranuclear Palsy (PSP) | Chronic Traumatic Encephalopathy (CTE) | Frontal Temporal Dementia (FTD) | Pick's DiseaseUnited States
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ExonhitCompletedMild Cognitive Impairment | Primary Progressive Aphasia | Vascular Dementia | Alzheimer's Disease | Mixed Dementia | Fronto-temporal Dementia | Parkinson' Disease DementiaUnited States
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Clementia Pharmaceuticals Inc.TerminatedExostoses, Multiple HereditaryUnited States, Spain, Canada, Japan, Italy, Portugal, United Kingdom, Turkey, Australia, Belgium, France, Netherlands
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Liminal BioSciences Ltd.CompletedDiabetes | Inflammation and FibrosisUnited Kingdom
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University of South FloridaBoston University; Yale UniversityCompletedGiant Cell Arteritis | Microscopic Polyangiitis | Churg-Strauss Syndrome | Polyarteritis Nodosa | Takayasu's Arteritis | Behcet's Disease | CNS Vasculitis | Henoch-Schoenlein Purpura | Wegener GranulomatosisUnited States
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University of South FloridaBoston University; Duke University; Data Management and Coordinating Center (DMCC)CompletedGiant Cell Arteritis | Vasculitis, Central Nervous System | Wegener's Granulomatosis | Microscopic Polyangiitis | Churg-Strauss Syndrome | Polyarteritis Nodosa | Takayasu's Arteritis | Kawasaki Disease | Behcet's Disease | Henoch-schoenlein Purpura | Drug-induced Necrotizing VasculitisUnited States
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Christopher DvorakNo longer availableAcute Myeloid Leukemia | Leukocyte Disorders | Acute Lymphoblastic Leukemia | Chronic Myeloid Leukemia | Myelodysplastic Syndrome | Cytopenias | Immune Deficiency | Lymphomas | Bone Marrow Failure | Osteopetrosis | Hemoglobinopathy | Anemia Due to Intrinsic Red Cell AbnormalityUnited States
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National Institute of Allergy and Infectious Diseases...RecruitingObstructive Sleep Apnea | Sleep Disordered Breathing | Tonsillitis | Periodic Fever, Aphthous Stomatitis, Pharyngitis, And Cervical Adenitis (Pfapa) | Tonsil DisorderUnited States
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AstraZenecaCompletedMcCune-Albright SyndromeFrance, Italy, United Kingdom, Germany
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BioCryst PharmaceuticalsCompletedHereditary Angioedema | HAEFrance, United Kingdom, Germany, United States, Belgium, Hungary, Italy, Canada
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Alector Inc.Active, not recruitingFrontotemporal DementiaSpain, United States, Portugal, United Kingdom, France, Germany, Netherlands, Belgium, Australia, Italy, Turkey, Switzerland, Argentina, Canada, Greece, Sweden
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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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University of MinnesotaWithdrawnMild Cognitive Impairment (MCI) | Amyotrophic Lateral Sclerosis (ALS) | Dementia With Lewy Bodies (DLB) | Alzheimer Disease (AD) | Frontotemporal Lobar Degeneration (FTLD) | Parkinsons Disease With Dementia (PDD) | Transient Epileptic Amnesia (TEA) | Temporal Lobe Epilepsy (TLE) | Spinocerebellar Ataxias... and other conditionsUnited States
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Wave Life Sciences Ltd.TerminatedALS | FTDNetherlands, United Kingdom
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Johns Hopkins UniversityNational Cancer Institute (NCI); National Institutes of Health (NIH); ChiRhoClin...RecruitingLynch Syndrome | Pancreas Cancer | Gene Mutation | Peutz-Jeghers Syndrome (PJS) | Germline Mutation CarrierUnited States
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University of South FloridaBoston University; University of Pennsylvania; Data Management and Coordinating...CompletedGiant Cell Arteritis | Vasculitis, Central Nervous System | Microscopic Polyangiitis | Churg-Strauss Syndrome | Polyarteritis Nodosa | Takayasu's Arteritis | Behcet's Disease | Henoch-Schoenlein Purpura | Wegener GranulomatosisUnited States
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Indiana UniversityRecruitingAortic Valve Disease | Marfan Syndrome | Bicuspid Aortic Valve | Thoracic Aortic Aneurysm | Thoracic Aortic Dissection | Turner Syndrome | Vascular Ehlers-Danlos Syndrome | Familial Thoracic Aortic Aneurysm and Aortic Dissection | PHACE Syndrome | Aortopathies | Thoracic Aortic Disease | Thoracic Aortic Rupture and other conditionsUnited States
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Intellia TherapeuticsActive, not recruitingHereditary AngioedemaFrance, New Zealand, Germany, United Kingdom, Netherlands, Australia
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CSL BehringCompletedHereditary AngioedemaBulgaria, United States, Poland, Spain, Hungary, United Kingdom, Russian Federation, Macedonia, The Former Yugoslav Republic of, Argentina, Australia, Canada, Czech Republic, Israel, Romania, Sweden
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ShireTakeda Development Center Americas, Inc.No longer availableAngioedema | Hereditary Angioedema (HAE)United States, Canada, Germany, Hungary, Spain
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University of Colorado, DenverNational Institute of Nursing Research (NINR); University of California, San...CompletedParkinson Disease | Alzheimer Disease | Lewy Body Disease | Parkinsonism | Supranuclear Palsy, Progressive | Parkinsonism Vascular | Multiple System Atrophy | Corticobasal Degeneration | Frontotemporal Dementia | Primary Progressive Aphasia | Vascular DementiaUnited States
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Wake Forest University Health SciencesNational Cancer Institute (NCI)CompletedPain | Lymphoma | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Multiple Myeloma and Plasma Cell Neoplasm | Precancerous Condition | Psychosocial Effects of Cancer and Its Treatment | Lymphoproliferative Disorder | Myelodysplastic/Myeloproliferative NeoplasmsUnited States
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Novartis PharmaceuticalsRecruitingPrimary Prevention of Atherosclerotic Cardiovascular DiseaseUnited States, China, Italy, Spain, France, Netherlands, Poland, Switzerland, Taiwan, Greece, Thailand, Brazil, India, Hungary, Latvia, Mexico, Israel, Malaysia, Turkey, Argentina, Singapore, Australia, Belgium, Bulgaria, Denmark, Croatia, South... and more
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POINT BiopharmaActive, not recruitingSoft Tissue Sarcoma | Colorectal Cancer | Esophageal Cancer | Cholangiocarcinoma | Head and Neck Squamous Cell Carcinoma | Pancreatic Ductal Adenocarcinoma | Melanoma (Skin)United States, Canada
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Ionis Pharmaceuticals, Inc.CompletedHereditary AngioedemaUnited States, France, Netherlands, Spain, United Kingdom, Belgium, Bulgaria, Canada, Denmark, Germany, Israel, Italy, Poland, Turkey
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Boston Children's HospitalNational Eye Institute (NEI); Howard Hughes Medical InstituteRecruitingOcular Motility Disorders | Facial Palsy | Synkinesis | Sixth Nerve Palsy | Mobius Syndrome | Congenital Fibrosis of Extraocular Muscles | Duane Retraction Syndrome | Duane Radial Ray Syndrome | Brown Syndrome | Marcus Gunn Syndrome | Strabismus Congenital | Horizontal Gaze Palsy | Horizontal Gaze Palsy With Progressive... and other conditionsUnited States
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University of California, San FranciscoUniversity of Colorado, Denver; National Institute on Aging (NIA); HealthPartners... and other collaboratorsActive, not recruitingDementia | Frontotemporal Dementia | Dementia, Vascular | Dementia With Lewy Bodies | Dementia AlzheimersUnited States
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Molnlycke Health Care ABRecruitingDiabetic Foot Ulcer | Wound; Foot | Venous Leg Ulcer | Wounds | Wound of Skin | Wound LegUnited States
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Anglia Ruskin UniversityUniversity of Melbourne; Alzheimer's Society; Norwegian Academy of Music; Cambridgeshire... and other collaboratorsCompletedDepression | Quality of Life | Dementia | Dementia, Vascular | Dementia, Mixed | Dementia With Lewy Bodies | Dementia Alzheimers | Behavioural and Psychiatric Symptoms of DementiaUnited Kingdom, Australia, Germany, Norway, Poland
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ShireCompletedHereditary Angioedema (HAE)United States, Germany, Spain, Sweden
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Ionis Pharmaceuticals, Inc.RecruitingHereditary AngioedemaUnited States, Puerto Rico, Belgium, Bulgaria, Canada, France, Germany, Israel, Italy, Netherlands, Poland, Spain, Turkey, United Kingdom
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CSL BehringCompletedHereditary AngioedemaUnited States, Germany, Japan, Hungary, Canada, Netherlands, Israel
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DePuy InternationalTerminatedOsteoarthritis | Avascular Necrosis | Post-traumatic Arthritis | Congenital Hip Dysplasia | Slipped Capital Femoral Epiphysis | Traumatic Femoral Fractures | Nonunion of Femoral Fractures | Collagen DisorderUnited Kingdom
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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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KalVista Pharmaceuticals, Ltd.CompletedHereditary AngioedemaUnited States, Austria, Czechia, Germany, Hungary, Italy, Netherlands, North Macedonia, Poland, United Kingdom
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ShireCompletedHereditary AngioedemaUnited States
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Pharming Technologies B.V.CompletedHereditary AngioedemaUnited States, Bulgaria, Canada, Hungary, Israel, Italy, Macedonia, The Former Yugoslav Republic of, Poland, Romania, Serbia, South Africa
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Baylor College of MedicineUniversity of California, San Diego; University of Alabama at Birmingham; Boston... and other collaboratorsCompletedPrader-Willi Syndrome | Rett Syndrome | Angelman Syndrome | Sleep ProblemsUnited States
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DePuy InternationalTerminatedOsteoarthritis | Rheumatoid Arthritis | Avascular Necrosis | Post-traumatic Arthritis | Congenital Hip Dysplasia | Slipped Capital Femoral Epiphysis | Collagen Disorders | Traumatic Femoral Fractures | Nonunion of Femoral FracturesUnited Kingdom