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Search clinical trials for: severe deficiency
Total 18143 results
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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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David WilliamsActive, not recruitingGene Therapy | Severe Combined Immunodeficiency, X LinkedUnited States, United Kingdom
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AEterna ZentarisNovo Nordisk A/SActive, not recruitingGrowth Hormone DeficiencyUnited States, Italy, Turkey, Georgia, Romania, Poland, Armenia, Germany, Serbia, Slovakia, Slovenia
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St. Jude Children's Research HospitalNational Heart, Lung, and Blood Institute (NHLBI); Assisi Foundation; California...SuspendedSevere Combined Immunodeficiency Disease, X-linkedUnited States
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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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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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Bellicum PharmaceuticalsTerminatedHemoglobinopathies | Hemophagocytic Lymphohistiocytosis | Metabolic Disorders | Primary Immune Deficiency Disorders | Inherited Bone Marrow Failure SyndromeUnited States
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LogicBio Therapeutics, IncAlexion Pharmaceuticals, Inc.TerminatedMethylmalonic AcidemiaUnited States
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Fondazione TelethonEnrolling by invitationImmunologic Deficiency SyndromesItaly
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Fondazione TelethonActive, not recruitingSevere Combined Immunodeficiency Due to ADA DeficiencyItaly
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Medical University of GrazActive, not recruitingPrimary Immunodeficiency | Immune Thrombocytopenia | Autoimmune Hemolytic Anemia | Evans SyndromeAustria
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Hydrocephalus and Neuroscience InstituteCompletedFolic Acid Deficiency | Neural Tube Defects | Spina Bifida | Anencephaly-Spina BifidaUnited States
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Paul SzabolcsEnrolling by invitationChronic Granulomatous Disease (CGD) | Wiskott-Aldrich Syndrome | Severe Chronic Neutropenia | Severe Combined Immunodeficiency (SCID) | Immunodeficiency With Predominant T-cell Defect, Unspecified | Hyper IgE Syndromes | Hyper IgM Deficiencies | Mendelian Susceptibility to Mycobacterial Disease | Common...United States
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University GhentInstitut de Recherche en Sciences de la Sante, Burkina Faso; Centre Muraz; University...CompletedSevere Acute Malnutrition | Kwashiorkor | Marasmus | Nutritional EdemaBurkina Faso
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PfizerIn Expanded Access, treating physicians are the SponsorAvailableSevere Uncontrolled Atopic Dermatitis
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Fondazione TelethonCompletedSevere Combined Immunodeficiency Due to ADA DeficiencyItaly
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David WilliamsUniversity of California, Los Angeles; Boston Children's Hospital; Children's...CompletedSevere Combined ImmunodeficiencyUnited States
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University GhentInstitut de Recherche en Sciences de la Sante, Burkina Faso; Centre Muraz; University...CompletedSevere Acute Malnutrition | Kwashiorkor | Marasmus | Nutritional EdemaBurkina Faso
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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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Assistance Publique - Hôpitaux de ParisCompletedSeptic Shock | Meningococcal Infections | Severe SepsisFrance
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Bioverativ, a Sanofi companyCompletedFactor VIII DeficiencyUnited States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, France, Germany, Greece, Hungary, Italy, Japan, Korea, Republic of, Mexico, Netherlands, Spain, Taiwan, United Kingdom
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University of California, Los AngelesGreat Ormond Street Hospital for Children NHS Foundation TrustEnrolling by invitationAdenosine Deaminase Deficiency | Severe Combined Immunodeficiency (SCID)United States, United Kingdom
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Chiesi Farmaceutici S.p.A.CompletedSevere Combined Immunodeficiency | Adenosine Deaminase DeficiencyUnited States
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Grifols Therapeutics LLCTerminatedCOVID-19United States, Brazil, Chile, Colombia, Mexico
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Children's Hospital of Fudan UniversityCompletedEpstein-Barr Virus Infections | ImmunodeficiencyChina
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Sanofi Pasteur, a Sanofi CompanyCompletedHuman Immunodeficiency Virus | Dengue Fever | Dengue Hemorrhagic FeverBrazil
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Prof. Dr. Jörg LeuppiCompletedCovid19 | Vitamin D Deficiency | Corona Virus Infection | ARDS | SARS-CoV Infection | CoronavirusSwitzerland
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Great Ormond Street Hospital for Children NHS Foundation...Orchard TherapeuticsCompletedSevere Combined Immunodeficiency Due to ADA DeficiencyUnited Kingdom
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Joanne Kurtzberg, MDDuke University; Miltenyi Biotec, Inc.AvailableHematologic Malignancies | Immune Deficiencies | Inborn Errors of Metabolism DisordersUnited States
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University of California, Los AngelesOrchard Therapeutics; California Institute for Regenerative Medicine (CIRM)CompletedSevere Combined Immunodeficiency Due to ADA DeficiencyUnited States
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University of California, Los AngelesWithdrawnSevere Combined Immunodeficiency Due to ADA DeficiencyUnited States
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University Hospital of SplitActive, not recruitingCovid-19 | Vitamin D Deficiency | Respiratory Distress SyndromeCroatia
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Hoffmann-La RocheCompletedHaemophilia A | Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor | Severe Hereditary Factor VIII Deficiency Disease Without InhibitorItaly
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Centre Leon BerardWithdrawnMyelodysplastic Syndromes | Severe Combined Immunodeficiency | Acute Leukemia | Haematological Malignancy | Hemoglobinopathy in Children | Bone Marrow AplasiaFrance
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International Centre for Diarrhoeal Disease Research...CompletedChildren | Iron Deficiency Anemia | Helicobacter Pylori Infection | Not Severely Malnourished | No Systemic InfectionBangladesh
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National Institute of CancerologíaInstituto Nacional de Enfermedades RespiratoriasCompletedKaposi Sarcoma | Human Immunodeficiency Virus | Immune Reconstitution SyndromeMexico
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Children's Hospital of Fudan UniversityCompletedSevere Combined Immunodeficiency | Neonatal ScreeningChina
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Bio Products LaboratoryCompletedFactor 10 DeficiencyUnited States
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National Institute of Allergy and Infectious Diseases...CompletedHIV Infections | CytopeniasUnited States
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National Institute of Allergy and Infectious Diseases...Applied ImmunesciencesCompleted
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Merck Sharp & Dohme LLCCompletedHuman Immunodeficiency Virus (HIV) InfectionUnited States, Germany
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Washington University School of MedicineNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); N... and other collaboratorsTerminatedLiver Cirrhosis | Alpha-1-antitrypsin DeficiencyUnited States
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Oslo University HospitalActive, not recruitingIron-deficiency | Severe Aortic StenosisNorway
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Assistance Publique - Hôpitaux de ParisCompletedX-linked Severe Combined ImmunodeficiencyFrance
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Great Ormond Street Hospital for Children NHS Foundation...Orchard TherapeuticsCompletedAdenosine Deaminase Deficiency | Severe Combined Immunodeficiencies (SCID)United Kingdom
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University of Wisconsin, MadisonCompletedSeptic Shock | Ascorbic Acid DeficiencyUnited States
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University Hospital, CaenEnrolling by invitationIdiopathic Infantile Hypercalcemia - Severe FormFrance
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Association Pour l'Aide a la Recherche en Immunologie...CompletedSevere Combined ImmunodeficiencyFrance
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National Heart, Lung, and Blood Institute (NHLBI)CompletedCystic Fibrosis | Psuedomas InfectionUnited States