- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Congenital Abnormalities in Australia
Total 1196 results
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PTC TherapeuticsTerminatedNervous System Diseases | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Musculoskeletal Diseases | Muscular Diseases | Neuromuscular Diseases | Muscular Dystrophies | Muscular Disorders, Atrophic | Muscular Dystrophy, DuchenneUnited States, Spain, Korea, Republic of, Belgium, France, Canada, Australia, United Kingdom, Israel, Italy, Germany, Brazil, Bulgaria, Chile, Czechia, Poland, Sweden, Switzerland, Turkey
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Novo Nordisk A/SCompletedGrowth Hormone Disorder | Adult Growth Hormone DeficiencyUnited States, Poland, India, Japan, Russian Federation, United Kingdom, Germany, Sweden, Israel, Malaysia, Turkey, South Africa, Australia, Latvia, Lithuania, Romania, Brazil, Norway, Ukraine
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Children's Oncology GroupNational Cancer Institute (NCI)CompletedUnspecified Childhood Solid Tumor, Protocol Specific | Ewing Sarcoma of Bone | Ewing Sarcoma | Childhood Acute Lymphoblastic Leukemia | Wilms Tumor and Other Childhood Kidney Tumors | Childhood Rhabdomyosarcoma | Childhood Soft Tissue Sarcoma | Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor...United States, Australia, Canada
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Pompe Disease (Late-Onset) | Glycogenesis Type II | Acid Maltase Deficiency (AMD)United States, France, Canada, Netherlands, Australia
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Hoffmann-La RocheCompletedHuntingtons DiseaseUnited States, United Kingdom, Australia, Canada, Japan, Poland, Netherlands, Germany, Russian Federation, France, Spain, Argentina, New Zealand, Austria, Denmark, Italy, Switzerland, Chile
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BioCryst PharmaceuticalsCompletedHereditary Angioedema | Prophylaxis | HAEAustria, Denmark, France, Germany, Hungary, Israel, Italy, North Macedonia, Poland, Switzerland, United Kingdom, United States, Australia, Hong Kong, Korea, Republic of, New Zealand, Serbia, Slovakia, South Africa, Spain
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Sarepta Therapeutics, Inc.Active, not recruitingDuchenne Muscular DystrophyUnited States, Korea, Republic of, Italy, United Kingdom, Canada, Poland, Spain, France, Germany, Belgium, Australia, Czechia, Serbia, Greece, Argentina, Bulgaria, Denmark, Hungary, India, Ireland, Israel, Mexico, Russian Federation, ...
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Novartis Gene TherapiesActive, not recruitingSMA | Spinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type I | Spinal Muscular Atrophy Type IIIUnited States, Belgium, France, Japan, United Kingdom, Italy, Taiwan, Australia, Canada
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KalVista Pharmaceuticals, Ltd.TerminatedAngioedema, Hereditary, Types I and IIUnited States, Czechia, Germany, Hungary, Italy, North Macedonia, United Kingdom, Australia, Bulgaria, Canada, France, New Zealand, Puerto Rico
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Kastle Therapeutics, LLCCompletedHypercholesterolemia | Heterozygous FamilialUnited States, Italy, Poland, Ukraine, United Kingdom, Germany, Hong Kong, Taiwan, Spain, Argentina, Belgium, Israel, Malaysia, South Africa, Turkey, Netherlands, Korea, Republic of, Canada, New Zealand, Russian Federation, Brazil, Austral... and more
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GlaxoSmithKlineTerminatedMuscular DystrophiesFrance, Bulgaria, Denmark, Germany, Belgium, Canada, Spain, Turkey, United Kingdom, Korea, Republic of, Brazil, Netherlands, Italy, Japan, Czech Republic, Chile, Israel, Hungary, Russian Federation, Australia, Poland, Norway, Argentina and more
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Boehringer IngelheimCompletedAcute Respiratory Distress SyndromeSpain, Germany, Italy, Belgium, Mexico, Netherlands, India, France, Malaysia, Austria, Brazil, Russian Federation, Turkey
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BayerRegeneron PharmaceuticalsCompletedAflibercept for Retinopathy of Prematurity - Intravitreal Injection Versus Laser Therapy (FIREFLEYE)Retinopathy of Prematurity (ROP)Spain, Singapore, Hong Kong, Korea, Republic of, Malaysia, Japan, Taiwan, Sweden, Portugal, Belgium, Argentina, Bulgaria, Italy, Austria, Brazil, Czechia, Greece, Hungary, Israel, Netherlands, Poland, Romania, Russian Federation, Slov... and more
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Genzyme, a Sanofi CompanyCompletedSphingomyelin LipidosisUnited States, Argentina, Australia, Belgium, Brazil, Bulgaria, Chile, France, Germany, Italy, Japan, Netherlands, Portugal, Spain, Tunisia, Turkey, United Kingdom
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KalVista Pharmaceuticals, Ltd.CompletedHereditary AngioedemaUnited States, Austria, Czechia, Germany, Hungary, Italy, Netherlands, North Macedonia, Poland, United Kingdom
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Santhera PharmaceuticalsTerminatedDuchenne Muscular Dystrophy (DMD)United States, Spain, Netherlands, Sweden, Germany, France, Belgium, United Kingdom, Italy, Ireland, Switzerland, Austria, Bulgaria, Hungary, Israel
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Children's Oncology GroupNational Cancer Institute (NCI)CompletedKidney CancerUnited States, Canada, Puerto Rico, Australia, Netherlands, New Zealand, Switzerland
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Alnylam PharmaceuticalsCompletedAcute Hepatic Porphyria | Acute Intermittent Porphyria | Porphyria, Acute Intermittent | Acute Porphyria | Hereditary Coproporphyria (HCP) | Variegate Porphyria (VP) | ALA Dehydratase Deficient Porphyria (ADP)United States, Spain, United Kingdom, Korea, Republic of, Australia, Bulgaria, Canada, Denmark, Finland, France, Germany, Italy, Japan, Mexico, Netherlands, Poland, Sweden, Taiwan
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ExelixisCompletedRenal Cell CarcinomaUnited States, Belgium, Spain, Taiwan, Canada, Germany, Korea, Republic of, France, Austria, Chile, Australia, Hungary, Russian Federation, Italy, Turkey, Netherlands, Sweden, United Kingdom, Czechia, Ireland, Slovakia, Argentina, D... and more
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Baxalta now part of ShireTakeda Development Center Americas, Inc.Active, not recruitingHemophilia ASpain, United States, Denmark, Italy, Taiwan, Malaysia, Belgium, Thailand, Turkey, Austria, Bulgaria, Canada, Finland, France, Germany, Hong Kong, Hungary, Korea, Republic of, Netherlands, Norway, Singapore, Ukraine, United Kingdom
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Swedish Orphan BiovitrumCompletedHereditary Tyrosinemia, Type IBelgium, Denmark, France, Germany, Austria, Czechia, Finland, Hungary, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, United Kingdom
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Novartis PharmaceuticalsTerminatedAtopic DermatitisUnited States, United Kingdom, Germany, Hungary, Russian Federation, Taiwan, Belgium, Netherlands, Japan, Austria, Finland, Slovakia, Czechia, Canada, Iceland, Poland
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BayerCompletedHemophilia A, Hemophilia BItaly, China, New Zealand, Russian Federation, Taiwan, Singapore, Bulgaria, United States, Denmark, Germany, Hungary, South Africa, United Kingdom, Romania, Colombia, Mexico, Turkey, France, India, Japan, Australia, Netherlands, Poland and more
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Spain, United Kingdom, New Zealand, Israel, Australia, Ireland, Germany, Sweden, Czechia, Portugal, Hungary
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Australia, Canada, Germany, Switzerland, Israel, Netherlands, Belgium, Italy, United Kingdom, France
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BayerCompletedHemophilia A | Blood Coagulation DisordersSpain, Taiwan, Austria, United States, Germany, Thailand, United Kingdom, Italy, Turkey, Croatia, Serbia, Poland, Israel, Sweden, Indonesia, Argentina, Norway, South Africa, India, Hong Kong, Denmark, Pakistan
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TakedaCompletedHereditary AngioedemaBelgium, Bulgaria, Estonia, Spain, Hungary, Israel, Germany, Czechia, Portugal, Greece, Lithuania, Poland, Latvia, Croatia, Slovakia, Austria, Canada, Ireland, North Macedonia, Romania, Serbia, Slovenia
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Genzyme, a Sanofi CompanyTerminatedFabry DiseaseAustria, Brazil, France, United States, Argentina, Belgium, Canada, Finland, Hungary, Norway, Poland, Spain, Taiwan, United Kingdom
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Eli Lilly and CompanyIncyte CorporationCompletedAtopic DermatitisBelgium, Netherlands, Japan, Spain, Switzerland, United Kingdom, Germany, France, Austria, Russian Federation, Poland, Italy, Brazil, Finland
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Hoffmann-La RocheCompletedHuntington DiseaseUnited States, Canada, Netherlands, Germany, Spain, United Kingdom, Austria, Italy
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Teva Branded Pharmaceutical Products R&D, Inc.Nuvelution TS Pharma, Inc.TerminatedTourette SyndromeUnited States, Argentina, Australia, Canada, Colombia, Denmark, Hungary, Italy, Korea, Republic of, Mexico, Poland, Russian Federation, Serbia, Spain, Ukraine
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AmgenRecruitingAtopic DermatitisUnited States, Japan, Korea, Republic of, Australia, Taiwan, Spain, Switzerland, Germany, Austria, Denmark, Netherlands, Hungary, Czechia, Poland, Brazil, France, Greece, Portugal, Italy, Finland, Argentina, Slovenia, Estonia, United... and more
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AstraZenecaRecruitingNeurofibromatosis Type 1Portugal, Spain, United Kingdom, Israel, France, Italy, Switzerland, Germany, Austria
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McMaster UniversityCanadian Institutes of Health Research (CIHR)CompletedRespiratory Insufficiency of PrematurityUnited States, Singapore, United Kingdom, Canada, Ireland, Qatar, Austria, Belgium, Netherlands, Sweden
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AmgenActive, not recruitingAtopic DermatitisUnited States, Spain, United Kingdom, Korea, Republic of, Australia, Denmark, Germany, Belgium, Canada, Japan, Sweden, Poland, Romania, Estonia, Brazil, Czechia, Portugal, Finland, Mexico, Turkey, South Africa
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TakedaCompletedLennox Gastaut Syndrome (LGS)United States, China, Canada, France, Hungary, Australia, Poland, Spain, Japan, Belgium, Greece, Serbia, Germany, Italy, Latvia, Netherlands, Russian Federation, Ukraine
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Medical University of ViennaMedical University of Graz; Charite University, Berlin, Germany; Hannover Medical... and other collaboratorsCompletedSarcoma | Hematologic Diseases | Leukemia | Brain Tumor | Neurofibromatoses | NeuroblastomaAustria, Germany, Switzerland, Italy
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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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AlexionRecruitingWolman Disease | Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 | Acid Lipase Deficiency | LIPA Deficiency | LAL-DeficiencyFrance, Belgium, United States, Spain, Germany, Greece, Israel, Italy, Slovenia, United Kingdom, Brazil, Canada, Denmark, Australia, Croatia, Czechia, Ireland, Mexico, Netherlands, Poland, Portugal, Saudi Arabia
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Corbus Pharmaceuticals Inc.Cystic Fibrosis FoundationCompletedCystic FibrosisUnited States, United Kingdom, Sweden, Canada, France, Austria, Germany, Netherlands, Italy, Belgium, Portugal, Bulgaria, Czechia, Greece, Hungary, Poland, Romania, Russian Federation, Serbia, Slovakia, Spain
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Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseUnited States, Netherlands, Australia, Hungary, United Kingdom, Canada, Czechia, Finland, France, Italy, Norway, Slovenia, Spain
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Ardea Biosciences, Inc.CompletedGoutUnited States, South Africa, Spain, Belgium, Australia, Germany, Canada, New Zealand, Poland, Ukraine, Switzerland
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AstraZenecaRecruitingAdv Solid Malig - H&N SCC, ATM Pro / Def NSCLC, Gastric, Breast and Ovarian CancerSpain, United States, Belgium, United Kingdom, France, Hungary, Canada, Korea, Republic of, Australia
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Genzyme, a Sanofi CompanyRecruitingGlycogen Storage Disease Type II | Pompe DiseaseHungary, Croatia, Serbia, United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, China, Colombia, Denmark, France, Germany, Greece, Hong Kong, India, Indonesia, Israel, Italy, Japan, Jordan, Korea, Republic of, Kuwait and more
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Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid PolyneuropathiesUnited States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
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BioMarin PharmaceuticalCompletedHyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) DeficiencySweden, France, Italy, Spain, Germany, Netherlands, Austria, Slovakia, Portugal
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Novartis PharmaceuticalsCompletedRetinopathy of PrematurityUnited States, Austria, Belgium, Croatia, Czechia, Denmark, Egypt, Estonia, France, Germany, Greece, Hungary, India, Italy, Japan, Lithuania, Malaysia, Mexico, Poland, Romania, Russian Federation, Saudi Arabia, Slovakia, Taiwan, Turkey, United...
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Gilead SciencesCovance; ClinPhone, Inc.; Chiltern International Inc.CompletedCystic FibrosisBelgium, France, Germany, Italy, Netherlands, Spain, United States, Ireland, United Kingdom, Portugal, Austria, Denmark, Switzerland
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Wyeth is now a wholly owned subsidiary of PfizerCompletedHemophilia BBelgium, Spain, Italy, Austria, United Kingdom, Netherlands, Sweden, Germany, Portugal, France
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Gilead SciencesCompletedCystic FibrosisUnited States, France, Spain, Germany, Austria, Belgium, Italy, Netherlands, Poland