Clinical Trials on Carbamoyl Phosphate Synthetase 1 Deficiency in New Zealand
Total 80 results
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Mendel TuchmanChildren's Hospital of Philadelphia; University of California, Los Angeles; Icahn... and other collaboratorsCompletedMethylmalonic Acidemia | Carbamoyl-Phosphate Synthase I Deficiency Disease | Propionic Acidemia, Type I and/or Type II | Ornithine Carbamoyltransferase DeficiencyUnited States
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Arrowhead PharmaceuticalsCompleted
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Dicerna Pharmaceuticals, Inc.WithdrawnAlpha 1-Antitrypsin DeficiencyNew Zealand
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyCompleted
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Baxalta now part of ShireCompletedAlpha 1-Antitrypsin DeficiencyNew Zealand, Australia
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Baxalta now part of ShireBaxter Healthcare, Ltd. (New Zealand), Baxter Healthcare Pty. Ltd. (Australia)CompletedAlpha 1-Antitrypsin DeficiencyAustralia, New Zealand
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Inhibrx, Inc.CompletedAlpha-1 Antitrypsin Deficiency | AATDUnited States, New Zealand, United Kingdom
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Nicholas Ah MewIcahn School of Medicine at Mount Sinai; Boston Children's HospitalRecruitingN-acetylglutamate Synthase (NAGS) DeficiencyUnited States
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Grifols Therapeutics LLCEnrolling by invitationPulmonary Emphysema in Alpha-1 Antitrypsin DeficiencyUnited States, New Zealand, Denmark, Australia, Finland, Estonia, Canada, Poland, Russian Federation, Sweden, France
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Grifols Therapeutics LLCActive, not recruitingPulmonary Emphysema in Alpha-1 PI DeficiencyUnited States, Australia, Denmark, New Zealand, Estonia, Poland, Russian Federation, Sweden, Brazil, Canada, France, Finland
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyActive, not recruitingAlpha 1-Antitrypsin DeficiencyUnited Kingdom, United States, Ireland, Belgium, New Zealand, Spain, Netherlands, Portugal, Canada, Australia, France, Austria, Germany, Sweden
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University of PittsburghNational Heart, Lung, and Blood Institute (NHLBI)CompletedAlpha 1 Antitrypsin Deficiency | AATDUnited States
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Murdoch Childrens Research InstituteRoyal Children's HospitalCompletedPrimary Immunodeficiency | Invasive Pneumococcal Disease, Protection Against | Invasive Pneumococcal Disease, Recurrent Isolated, 1 | Invasive Pneumococcal Disease, Recurrent Isolated, 2Australia, New Zealand
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Inhibrx, Inc.RecruitingEmphysema | Alpha 1-Antitrypsin DeficiencyUnited States, Australia, United Kingdom, New Zealand
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Grifols Therapeutics LLCCompletedAlpha 1-Antitrypsin DeficiencyUnited States
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TakedaEnrolling by invitationAlpha1-Antitrypsin DeficiencyUnited Kingdom, Germany, United States, Austria, Portugal
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Grifols Therapeutics LLCCompletedAlpha 1-Antitrypsin DeficiencyUnited States, United Kingdom
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Columbia UniversityAlpha-1 Foundation; Stony Wold-Herbert Fund, Inc.CompletedChronic Obstructive Pulmonary Disease | Emphysema | Alpha-1 Antitrypsin DeficiencyUnited States
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Columbia UniversityAlpha-1 FoundationRecruitingChronic Obstructive Pulmonary Disease | Acute Exacerbation Copd | Alpha-1 Anti-trypsin 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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IpsenTerminatedPrimary Insulin-like Growth Factor-1 DeficiencyUnited States
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Vertex Pharmaceuticals IncorporatedActive, not recruitingAlpha-1 Antitrypsin DeficiencyUnited States, United Kingdom, Germany, Ireland
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Gerard TurinoCompletedEmphysema | Alpha 1-Antitrypsin DeficiencyUnited States, Canada
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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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University of Alabama at BirminghamNational Institutes of Health (NIH); Mereo BioPharmaCompletedEmphysema or COPD | Alpha-1 Antitrypsin Deficiency (AATD) | Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I MutationsUnited States
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TakedaTakeda Development Center Americas, Inc.RecruitingAlpha1-Antitrypsin DeficiencyUnited States, Belgium, Switzerland, Canada, Australia, Germany, United Kingdom, Austria, France, Spain, Portugal, Italy, Poland
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Arrowhead PharmaceuticalsCompletedAlpha 1-Antitrypsin DeficiencyUnited States, Spain, Netherlands, Germany, Italy, Portugal
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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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Vertex Pharmaceuticals IncorporatedTerminatedAlpha 1-Antitrypsin DeficiencyUnited States, Germany, Canada, Ireland
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CSL BehringCompletedEmphysema | Alpha 1-proteinase Inhibitor DeficiencyAustralia, Canada, Czech Republic, Denmark, Estonia, Finland, Germany, Ireland, Poland, Romania, Sweden
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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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Baxalta now part of ShireBaxalta Innovations GmbH, now part of ShireTerminatedChronic Obstructive Pulmonary Disease | Alpha1-antitrypsin DeficiencyUnited States, Canada, Australia
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Kirby InstituteGilead Sciences; Melanoma Institute Australia; St Vincent's Hospital, Sydney; Royal... and other collaboratorsActive, not recruitingCOVID-19 | Cancer | Primary Immune Deficiency Disorder | HIV-1-infection | Immunosuppression DisordersAustralia
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Aeglea BiotherapeuticsTerminatedHomocystinuria Due to Cystathionine Beta-Synthase DeficiencyUnited States, United Kingdom, Australia
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Alpha-1 FoundationCompletedChronic Obstructive Pulmonary Disease | Alpha-1 Antitrypsin DeficiencyUnited States, Puerto Rico
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ST Pharm Co., Ltd.RecruitingHIV-1-infectionUnited States
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Gilead SciencesActive, not recruitingHIV-1-infectionUnited States, Spain, France, Taiwan, Japan, Thailand, Dominican Republic, Germany, Canada, Italy, South Africa
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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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Columbia UniversityNational Heart, Lung, and Blood Institute (NHLBI); Alpha-1 FoundationActive, not recruitingAlpha 1-Antitrypsin Deficiency | Genetic DiseaseUnited States
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John LevineNational Cancer Institute (NCI)CompletedGVHD | Graft-versus-host-diseaseUnited States
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Grifols Therapeutics LLCTerminatedType 1 Diabetes MellitusUnited 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, Israel, Mexico, Russian Federation, Argentina, Italy
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Icahn School of Medicine at Mount SinaiNational Heart, Lung, and Blood Institute (NHLBI)Completed
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Arbor Research Collaborative for HealthNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)RecruitingLiver Diseases | Alpha 1-Antitrypsin Deficiency | Alagille SyndromeUnited States, Canada
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Gilead SciencesTerminatedHuman Immunodeficiency Virus Type 1 (HIV-1) InfectionUnited States
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MacroGenicsNational Institute of Allergy and Infectious Diseases (NIAID); National Institutes... and other collaboratorsActive, not recruitingHuman Immunodeficiency Virus I Infection | Immunodeficiency Virus Type 1, Human | Human Immunodeficiency Virus Type 1United States
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CSL BehringCompletedEmphysema | Alpha1-proteinase Inhibitor DeficiencyAustralia, Russian Federation, United States, Denmark, Estonia, Finland, Germany, Ireland, Poland, Romania, Sweden, Canada, Czech Republic
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University of North Carolina, Chapel HillNational Institute on Drug Abuse (NIDA); National Institute of Mental Health...CompletedHIV Infections | HIV-1United States, Puerto Rico
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Gilead SciencesActive, not recruitingPre-Exposure Prophylaxis of HIV-1 InfectionUnited States, Austria, Denmark, United Kingdom, Netherlands, Italy, Germany, Canada, Ireland, France, Spain
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Ultragenyx Pharmaceutical IncTerminatedGlucose Transporter Type 1 Deficiency SyndromeUnited States, Spain, Denmark, United Kingdom, Australia