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Clinical Trials on Alpha 1-Antitrypsin Deficiency in United States
Total 220 results
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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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William Beaumont HospitalsCompletedType 1 Diabetes MellitusUnited 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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IpsenCompletedGrowth Disorders | Insulin-Like Growth Factor-1 DeficiencyUnited States
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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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Juan PascualActive, not recruitingGlucose Transporter Type 1 Deficiency Syndrome | GLUT1DS1United States
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Adrian LacyUltragenyx Pharmaceutical IncCompletedGlucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)United States
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IpsenCompletedGrowth Disorders | Insulin-Like Growth Factor-1 DeficiencyUnited 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 IncCompletedGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)United States, Spain, Australia, France, Israel, Italy, United Kingdom
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IpsenTerminatedPrimary Insulin-like Growth Factor-1 DeficiencyUnited States
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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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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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Ultragenyx Pharmaceutical IncTerminatedGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)United States, Spain, France, Germany, Italy, United Kingdom
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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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National Cancer Institute (NCI)Recruiting
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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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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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Bristol-Myers SquibbCompletedHuman Immunodeficiency Virus Type 1 (HIV-1)United States
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National Institute of Allergy and Infectious Diseases...RecruitingChronic Granulomatous Disease (CGD) | X-Linked Severe Combined Immune Deficiency (XSCID) | Leukocyte Adhesion Deficiency 1 (LAD) | Graft Versus Host Disease (cGvHD)United States
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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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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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Bristol-Myers SquibbCompletedHIV Infections | Human Immunodeficiency Virus Type 1 (HIV-1)United States, Netherlands
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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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National Institute of Allergy and Infectious Diseases...RecruitingMulti-Drug Resistant Hiv-1 Infection | HIV-1 InfectionUnited States
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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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Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis | Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Krabbe Disease | Adrenoleukodystrophy (ALD) | Hunter Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Metachromatic Leukodystrophy (MLD) | Maroteaux-Lamy SyndromeUnited States
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Masonic Cancer Center, University of MinnesotaWithdrawnHIV-1-infection | ART | Interleukin | Cd4+ Lymphocyte Deficiency | Lymphoid Tissue; InfectionUnited States
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National Human Genome Research Institute (NHGRI)RecruitingMetabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditionsUnited States
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Ruth M. Rothstein CORE CenterVircoUnknownHIV Infections | HIV-1United States
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Vitaflo International, LtdCompletedGlucose Transporter Type 1 Deficiency Syndrome | Ketogenic Dieting | Epilepsy IntractableUnited Kingdom
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Eunice Kennedy Shriver National Institute of Child...University of WashingtonUnknownWiskott-Aldrich Syndrome | Leukocyte Adhesion Deficiency Syndrome | X-Linked Agammaglobulinemia | X-Linked Hyper IgM SyndromeUnited States
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Vitaflo International, LtdCompletedIntractable Epilepsy | Glucose Transporter Type 1 Deficiency Syndrome | Ketogenic DietingUnited 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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National Institute of Allergy and Infectious Diseases...CompletedHIV Infections | HIV-1 | AIDS VaccinesUnited States
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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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Inovio PharmaceuticalsCompletedStudy of PENNVAX™-B (Gag, Pol, Env) + Electroporation in HIV-1 Infected Adult Participants (HIV-001)HIV Infections | HIV-1 InfectionUnited States
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ST Pharm Co., Ltd.RecruitingHIV-1-infectionUnited 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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Masonic Cancer Center, University of MinnesotaTerminatedSphingolipidoses | Niemann-Pick Disease, Type C | Krabbe Disease | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Hurler's Syndrome | Maroteaux-Lamy Syndrome | Wolman's Disease | Niemann-Pick Disease Type BUnited States
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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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Ultragenyx Pharmaceutical IncTerminatedGlucose Transporter Type 1 Deficiency SyndromeUnited States, Spain, Denmark, United Kingdom, Australia
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Icahn School of Medicine at Mount SinaiNational Heart, Lung, and Blood Institute (NHLBI)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.Active, not 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, Poland, Germany, Austria, Bulgaria, Canada, Ireland, Italy, United Kingdom
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Genzyme, a Sanofi CompanyTerminatedFabry Disease | Alpha Galactosidase A DeficiencyUnited States, Austria, United Kingdom
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Gilead SciencesTerminatedHuman Immunodeficiency Virus Type 1 (HIV-1) InfectionUnited States
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University of North Carolina, Chapel HillNational Heart, Lung, and Blood Institute (NHLBI)Completed