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Clinical Trials on Alpha-1 Antitrypsin Deficiency
Total 360 results
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Children's Hospital Los AngelesCompletedType 1 Diabetes | Vitamin D Deficiency/InsufficiencyUnited States
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IpsenTerminatedrhGH and rhIGF-1 Combination Therapy in Children With Short Stature Associated With IGF-1 DeficiencyInsulin-like Growth Factor-1 DeficiencyUnited States
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National Institute of Arthritis and Musculoskeletal...National Institutes of Health Clinical Center (CC)Completed
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William Beaumont HospitalsCompletedType 1 Diabetes MellitusUnited 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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Al-Azhar UniversityCompletedVitamin D Deficiency | Type 1 Diabetes MellitusPalestinian Territory, occupied
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Cure CMDRecruitingEmery-Dreifuss Muscular Dystrophy | Congenital Myasthenic Syndrome | Limb-Girdle Muscular Dystrophy | Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency | Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy) and other conditionsUnited States
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Jerry Vockley, MD, PhDUltragenyx Pharmaceutical IncNo longer availableGlucose Transporter 1 Deficiency SyndromeUnited States
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Indiana Hemophilia &Thrombosis Center, Inc.Active, not recruitingPlasminogen Activator Inhibitor-1 Deficiency | Cardiac FibrosisUnited States
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National Institute of Allergy and Infectious Diseases...Recruiting
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University of British ColumbiaUltragenyx Pharmaceutical IncUnknownGlucose Transporter Type 1 Deficiency SyndromeCanada
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National Institute of Allergy and Infectious Diseases...Active, not recruitingPGM3 Deficiency | Eosinophilic and/or Atopic Dermatitis | OSMR Deficiency | Primary Localized Cutaneous Amyloidosis | Hereditary Alpha-tryptasemiaUnited States
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Ultragenyx Pharmaceutical IncAvailableGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
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IpsenCompletedGrowth Disorders | Insulin-Like Growth Factor-1 DeficiencyUnited States
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Ultragenyx Pharmaceutical IncCompletedGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)United States, Spain, Australia, France, Israel, Italy, United Kingdom
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Juan PascualActive, not recruitingGlucose Transporter Type 1 Deficiency Syndrome | GLUT1DS1United States
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Janssen Sciences Ireland UCCompletedImmunodeficiency Virus Type 1, HumanUnited States, France, Poland, United Kingdom, Belgium, Germany, Spain, Canada, Russian Federation, Puerto Rico
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University of the PunjabThe National HIV/AIDS ProgrammeCompletedHIV-1-infection | Hypovitaminosis DPakistan
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Great Ormond Street Hospital for Children NHS Foundation...National Institute for Health Research, United Kingdom; The Freya FoundationRecruitingPyruvate Dehydrogenase Complex Deficiency | Pyruvate Dehydrogenase E1 Alpha Deficiency | Pyruvate Dehydrogenase E1-Beta Deficiency | Pyruvate Dehydrogenase E2 Deficiency | Pyruvate Dehydrogenase Phosphatase DeficiencyUnited Kingdom
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ANRS, Emerging Infectious DiseasesEuropean and Developing Countries Clinical Trials Partnership (EDCTP); European...RecruitingTuberculosis | HIV-1-infection | Immuno-DeficiencyUganda, Cambodia, Cameroon, Guinea, Vietnam, Zambia
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Adrian LacyUltragenyx Pharmaceutical IncCompletedGlucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)United States
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Greenwood Genetic CenterCompletedGalactosialidosis | Sialidosis | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Beta Mannosidosis | Mucolipidosis II | Mucolipidosis III | Schindler DiseaseUnited States
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Ultragenyx Pharmaceutical IncTerminatedGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)United States, Spain, France, Germany, Italy, United Kingdom
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IpsenCompletedGrowth Disorders | Insulin-Like Growth Factor-1 DeficiencyUnited States
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Rigshospitalet, DenmarkUnknownCarbohydrate Metabolism, Inborn Errors | Metabolism, Inborn Errors | Lipid Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease Type V | VLCAD Deficiency | Glycogen Storage Disease Type III | Phosphoglycerate Kinase Deficiency | Neutral Lipid Storage Disease | Carnitine... and other conditionsDenmark
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Instituto Nacional de Ciencias Medicas y Nutricion...SuspendedHIV-1-infection | Immune Deficiency | CD4+ T Lymphocytopenia | Immuno-senescenceMexico
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Vitaflo International, LtdWithdrawnEpilepsy | Glucose Transporter Type 1 Deficiency Syndrome | Ketogenic Dieting
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University of EdinburghNHS Lothian; DexCom, Inc.; Tandem Diabetes Care, Inc.; The Leona M. and Harry B...Active, not recruitingHypoglycemia | Type 1 Diabetes | Type 1 Diabetes Mellitus With Hypoglycemia | Hypoglycemia Unawareness | Glucagon Deficiency | Insulin HypoglycemiaUnited Kingdom
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Baylor College of MedicineChildren's National Research InstituteRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Carbamyl Phosphate Synthetase Deficiency | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS Deficiency | NAGS DeficiencyUnited States
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IpsenTerminatedPrimary Insulin-like Growth Factor-1 DeficiencyUnited States
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Merck Sharp & Dohme LLCWithdrawnHIV-1 | Immunodeficiency Virus Type 1, Human | Human Immunodeficiency Virus Type 1 | Human Immunodeficiency Virus 1
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Nina BhardwajNational Institute of Allergy and Infectious Diseases (NIAID); National Institutes... and other collaboratorsCompletedHIV-1 Infected Adults With Chronic HIV-1 InfectionUnited States
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National Cancer Institute (NCI)Recruiting
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UniQure Biopharma B.V.CompletedLipoprotein Lipase Deficiency | Familial Hyperlipoproteinemia Type 1 | Familial HyperchylomicronemiaGermany
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Assistance Publique - Hôpitaux de ParisRecruitingHTLV-1 Infection | Lymphoproliferation Induced by HTLV-1 | HTLV-1 Adult T-cell Lymphoma / LeukemiaFrance
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Vitaflo International, LtdCompletedIntractable Epilepsy | Glucose Transporter Type 1 Deficiency Syndrome | Ketogenic DietingUnited Kingdom
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Vitaflo International, LtdCompletedGlucose Transporter Type 1 Deficiency Syndrome | Ketogenic Dieting | Epilepsy IntractableUnited Kingdom
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Baylor College of MedicineChildren's Hospital of Philadelphia; Children's Hospital Colorado; Seattle Children... and other collaboratorsRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS DeficiencyUnited States
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CENTOGENE GmbH RostockTerminatedDeafness | Immunodeficiency | Mental Retardation | Gingival Hypertrophy | Skeletal Abnormalities | Facial DysmorphismGermany, Switzerland
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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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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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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 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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Peking Union Medical College HospitalGilead SciencesActive, not recruiting
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IpsenTerminated
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ANRS, Emerging Infectious DiseasesMerck Sharp & Dohme LLC; Pfizer; Gilead Sciences; Janssen-Cilag Ltd.Completed
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Pharvaris Netherlands B.V.RecruitingHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditionsUnited States, Bulgaria, Czechia, Hungary, Spain, France, Germany, Poland, Canada, Israel
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Pharvaris Netherlands B.V.RecruitingHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditionsUnited States, Puerto Rico
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Tibotec Pharmaceuticals, IrelandCompletedHIV Infections | HIV-1 | Human Immunodeficiency Virus Type 1United States, Australia, France, United Kingdom, Taiwan, Spain, Argentina, Portugal, Romania, South Africa, Canada, Denmark, Sweden, Netherlands, Brazil, Puerto Rico, Austria, Russian Federation, Thailand, Mexico
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Eva BonfillRecruiting