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Clinical Trials on Alpha and Beta Thalassemia
Total 722 results
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LMU KlinikumSeventh Framework Programme; NBIA AllianceRecruitingNeurodegeneration With Brain Iron Accumulation (NBIA) | Pantothenate Kinase-associated Neurodegeneration (PKAN) | Aceruloplasminemia | Beta-Propeller Protein-Associated Neurodegeneration (BPAN) | Mitochondrial Membrane Protein Associated Neurodegeneration (MPAN) | Fatty Acid Hydroxylase-associated... and other conditionsCanada, Czechia, Germany, Italy, Netherlands, Poland, Serbia, Spain
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Aeglea BiotherapeuticsTerminatedHomocystinuria Due to Cystathionine Beta-Synthase DeficiencyUnited States, United Kingdom, Australia
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Baqiyatallah Medical Sciences UniversityGuilan University of Medical Sciences; Baqiyatallah Research Center for Gastroenterology... and other collaboratorsCompletedHepatitis C | ThalassemiaIran, Islamic Republic of
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Ala'a ShararaGilead SciencesCompletedHepatitis C | ThalassemiaLebanon
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HealthCore-NERINational Heart, Lung, and Blood Institute (NHLBI)CompletedHepatitis C | ThalassemiaUnited States, United Kingdom, Canada
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UCSF Benioff Children's Hospital OaklandFood and Drug Administration (FDA)CompletedSickle Cell Disease | Pulmonary Hypertension | ThalassemiaUnited States
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University of Southern CaliforniaKaiser PermanenteCompletedObesity | Physical Activity | Insulin Resistance | Genetics | Air Pollution | Gestational Diabetes | Nutrition | Diabetes Type 2 | Adipokines | Beta Cell Function
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Talaris Therapeutics Inc.Duke UniversityTerminatedNiemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...United 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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Stanford UniversityUniversity of Alabama at Birmingham; University of MinnesotaCompletedSickle Cell Disease | Thalassemia | Diamond-Blackfan AnemiaUnited States
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Genzyme, a Sanofi CompanyTerminatedFabry Disease | Alpha Galactosidase A DeficiencyUnited States, Austria, United Kingdom
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IRCCS Policlinico S. DonatoTerminated
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University of ValenciaRecruitingCerebrospinal Fluid Levels of Amyloid Beta 1-42, H-tau and Phospho-tau Proteins | Cognitive Impairment and Its Evolution After Hydrocephalus Treatment | Motor Impairment and Its Evolution After Hydrocephalus Treatment | Ventricular Size and Its Evolution After Implantation of a Ventriculoperitoneal...Spain
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National Heart, Lung, and Blood Institute (NHLBI)CompletedDiabetes Mellitus | Heart Disease | Thalassemia | HemochromatosisUnited States
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Duke UniversityCompletedHemoglobinopathies | Thalassemia | Lysosomal Storage Disease | Immunodeficiencies | Inborn Errors of Metabolism | Sickle Cell | Non Malignant Disorders | Congenital Marrow FailuresUnited States
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University Hospital of North NorwayNorwegian Extra Foundation for Health and RehabilitationCompletedStroke | Quality of Life | Fatigue | Cognitive Impairment | Post-stroke Depression | Neuropsychology | Post-Stroke Fatigue | Meta-cognitionNorway
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UCSF Benioff Children's Hospital OaklandNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Medical... and other collaboratorsCompletedSickle Cell Disease | Thalassemia | Diamond-Blackfan AnemiaUnited Kingdom, United States, Germany
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InvicroAC Immune SAActive, not recruitingParkinson's Disease | Dementia With Lewy Bodies | Multisystem Atrophy | Alpha-SynucleinopathyUnited States
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Chelsea TherapeuticsCompletedMultiple System Atrophy | Neurogenic Orthostatic Hypotension | Dopamine Beta Hydroxylase Deficiency | Non-Diabetic Autonomic NeuropathyUnited States, Australia, Canada, New Zealand
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HaEmek Medical Center, IsraelCompletedAnorexia | Sickle Cell Anemia | Thalassemia | Chronic Renal Failure | Suicide AttemptIsrael
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Passage Bio, Inc.Active, not recruitingGM1 Gangliosidosis | GM1 Gangliosidosis, Type I | GM1 Gangliosidosis, Type 2 | Beta-Galactosidase-1 (GLB1) DeficiencyUnited States, Canada, United Kingdom, Turkey, Brazil
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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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National Cerebral and Cardiovascular CenterMinistry of Health, Labor and Welfare (Japan)TerminatedMyocardial Infarction | Impaired Glucose ToleranceJapan
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Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis I | Mucopolysaccharidosis VI | Mannosidosis | Mucolipidosis Type II (I-cell Disease)United States
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Novartis PharmaceuticalsRecruitingAcute Myeloid Leukemia | Primary Myelofibrosis | Polycythemia Vera | Thalassemia | Graft Versus Host DiseaseKorea, Republic of, Italy, Belgium, Japan, Germany, Australia, Bulgaria, Chile, China, Denmark, Greece, Hungary, Israel, Lebanon, Poland, Portugal, Russian Federation, Slovakia, South Africa, Spain, Sweden, Thailand, India, Mexico, Turkey, Fran...
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Genzyme, a Sanofi CompanyRecruitingGaucher Disease | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency DiseaseHungary, Croatia, United States, Australia, Belgium, Brazil, Bulgaria, Canada, Denmark, Germany, Greece, Hong Kong, India, Indonesia, Italy, Jordan, Korea, Republic of, Kuwait, Lebanon, Malaysia, Pakistan, Philippines, Poland, Saudi Arabia and more
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Novartis PharmaceuticalsCompletedMyelofibrosis | Sickle Cell Disease | Thalassemia | Diamond Blackfan AnemiaUnited States, Netherlands, Taiwan, Thailand, Italy, Germany, Spain, Canada, Belgium, Greece, Turkey, United Kingdom
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UCSF Benioff Children's Hospital OaklandNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Medical... and other collaboratorsCompletedSickle Cell Disease | Thalassemia | Diamond-Blackfan AnemiaUnited States, United Kingdom, Germany
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Chelsea TherapeuticsTerminatedParkinson's Disease | Pure Autonomic Failure | Dopamine Beta Hydroxylase Deficiency | Symptomatic Neurogenic Orthostatic Hypotension | Multiple Systems AtrophyUnited 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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Genzyme, a Sanofi CompanyCompletedGaucher Disease, Type 1 | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency Disease | Gaucher Disease, Non-Neuronopathic FormUnited States, United Kingdom, Spain, Italy, Brazil, Canada, Poland
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Genzyme, a Sanofi CompanyCompletedCerebroside Lipidosis Syndrome | Glucosylceramide Beta-Glucosidase Deficiency Disease | Gaucher Disease Type I | Clucocerebrosidase Deficiency Disease | Gaucher Disease, Non-Neuronopathic FormUnited States
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AC Immune SAWorldwide Clinical TrialsRecruitingAlzheimer's Disease | Prodromal Alzheimer's Disease | Amyloid Plaque | Beta-Amyloid | Alzheimer's Disease in Down SyndromeUnited States, Spain, United Kingdom
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Genzyme, a Sanofi CompanyCompletedGaucher Disease, Type 1 | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency Disease | Gaucher Disease, Non-Neuronopathic FormUnited States, Israel, Mexico, Russian Federation, Argentina, Italy
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Ohio State University Comprehensive Cancer CenterImmunoGen, Inc.; Clovis Oncology, Inc.Active, not recruitingBRCA1 Gene Mutation | BRCA2 Gene Mutation | Folate Receptor Alpha Positive | Recurrent Fallopian Tube Carcinoma | Recurrent Ovarian Carcinoma | Recurrent Primary Peritoneal Carcinoma | Recurrent Uterine Corpus Carcinoma | Recurrent Uterine Serous Carcinoma | Recurrent Uterine Carcinosarcoma | Platinum...United States
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University of California, San FranciscoCompletedSickle Cell Disease | Hemoglobinopathies | Thalassemia | Hematologic Malignancies | Immunodeficiencies | Fanconi's Anemia | Nonmalignant Diseases | Genetic Inborn Errors of MetabolismUnited States
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University of California, San FranciscoThrasher Research FundCompletedSickle Cell Disease | Hemoglobinopathies | Fanconi Anemia | Thalassemia | Hematologic Malignancies | Immunodeficiencies | Nonmalignant Diseases | Genetic Inborn Errors of MetabolismUnited States
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University of California, San FranciscoCompletedSickle Cell Disease | Hemoglobinopathies | Thalassemia | Hematologic Malignancies | Immunodeficiencies | Fanconi's Anemia | Nonmalignant Diseases | Genetic Inborn Errors of MetabolismUnited States
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Masonic Cancer Center, University of MinnesotaNational Marrow Donor ProgramCompletedSickle Cell Disease | Thalassemia | Severe Congenital Neutropenia | Diamond-Blackfan Anemia | Shwachman-Diamond SyndromeUnited States
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University of PittsburghRecruitingLysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditionsUnited States
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BeiGeneNot yet recruitingMetastatic Solid Tumor | Advanced Hepatocellular Carcinoma | Metastatic Hepatocellular Carcinoma | Alpha-fetoprotein (AFP)-Producing Gastric Cancer | Extragonadal Yolk Sac Tumors | Glypican-3 (GPC3)-Positive Squamous Non-small Cell Lung Cancer
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National Medical Research Center for Rehabilitation...Pirogov Russian National Research Medical University; DNKOM LLC; The Orenburg...RecruitingCardiovascular Diseases | Nervous System Diseases | Urologic Diseases | Diabetes | Neuromuscular Diseases | Hypercholesterolemia | Gout | Hyperbilirubinemia | Hyper IgE Syndrome | Hyper-LDL-cholesterolemia | Hyper Beta Lipoproteinemia | Hyper Eosinophilic SyndromeRussian Federation
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University of California, San FranciscoCompletedSickle Cell Disease | Hemoglobinopathies | Fanconi Anemia | Thalassemia | Hematologic Malignancies | Immune Deficiency | Nonmalignant Diseases | Genetic Inborn Errors of MetabolismUnited States
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West Kazakhstan Medical UniversityRecruitingOrnithine Transcarbamylase Deficiency | Biotinidase Deficiency | Citrullinemia | Glutaric Acidemia Type II | Argininosuccinic Aciduria | Maple Syrup Urine Disease | Primary Carnitine Deficiency | Homocystinuria | Carnitine Palmitoyltransferase II Deficiency | Arginase Deficiency | Very Long-chain Acyl-CoA... and other conditionsKazakhstan
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Cardiologs TechnologiesCompletedAtrial Fibrillation | Bradycardia | Tachycardia | Premature Ventricular Contraction | Premature Supraventricular BeatUnited States
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City of Hope Medical CenterNational Cancer Institute (NCI); Mustang Bio, Inc.Active, not recruitingAcute Myeloid Leukemia | Recurrent Adult Acute Myeloid Leukemia | Secondary Acute Myeloid Leukemia | Untreated Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia in Remission | Refractory Acute Myeloid Leukemia | Blastic Plasmacytoid Dendritic Cell Neoplasm | Minimal Residual Disease | Acute... and other conditionsUnited States
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Hospital Universitari Vall d'Hebron Research InstituteERN-EuroBloodNet (European Reference Network on Rare Hematological DiseasesRecruitingLeukemia | Anemia | Iron Metabolism Disorders | Anemia, Sickle Cell | Thalassemia | Bleeding Disorder | Myeloma | Lymphoid Neoplasm | Blood Cancer | Bone Marrow Failure | Red Cell Membrane and Enzyme Abnormalities | Myeloma, MalignantSpain
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American Thrombosis and Hemostasis NetworkNovo Nordisk A/S; Sanofi; Pfizer; Genentech, Inc.; CSL Behring; Hemophilia of Georgia... and other collaboratorsRecruitingHemophilia | Thrombosis | Thrombophilia | Sickle Cell Disease | Thalassemia | Bleeding Disorder | Von Willebrand Diseases | Factor IX Deficiency | Connective Tissue Disorder | Factor VIII Deficiency | Hematologic Disorder | Rare Bleeding Disorder | Platelet DisorderUnited 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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ImmunoGen, Inc.Not yet recruitingRecurrent Ovarian Cancer | Folate Receptor-Alpha PositiveUnited States, Spain, Australia, Belgium, Canada, France, Ireland, Italy