- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Genetic Diseases, X-Linked in Australia
Total 967 results
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QED Therapeutics, Inc.RecruitingAchondroplasiaUnited States, Spain, Singapore, Canada, United Kingdom, France, Australia, Norway, Argentina, Germany, Italy
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Canada, Australia, Ireland, Germany, United Kingdom
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Ataxia Study GroupRecruitingSpinocerebellar AtaxiaAustria, Belgium, France, Germany, Hungary, Italy, Netherlands, Poland, Spain, United Kingdom
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Vertex Pharmaceuticals IncorporatedModerna, IncRecruitingCystic FibrosisUnited States, United Kingdom, Australia, Canada
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Wyeth is now a wholly owned subsidiary of PfizerCompleted
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Vertex Pharmaceuticals IncorporatedCystic Fibrosis FoundationCompletedCystic FibrosisUnited States, Canada, Australia, Ireland, France, United Kingdom, Germany, Czech Republic
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Amicus TherapeuticsCompletedPompe Disease (Late-onset)United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
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BioMarin PharmaceuticalActive, not recruitingHereditary Angioedema | HAESpain, United States, Australia
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Australia, Canada, United Kingdom, Germany, Belgium, Austria, Ireland, Italy, France
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ActelionCompletedGaucher Disease Type 1United States, Taiwan, Canada, Spain, Australia, France, Brazil, Hungary, Czechia, Germany, Italy, Netherlands, United Kingdom
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The University of Texas Health Science Center,...Baxter Healthcare Corporation; Rho, Inc.CompletedHemophilia AUnited States, Austria, Italy, Netherlands, Sweden
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Genzyme, a Sanofi CompanyActive, not recruitingHemophiliaChina, Hungary, Turkey, Canada, Korea, Republic of, United States, Australia, Denmark, France, Germany, India, Ireland, Israel, Italy, Japan, Malaysia, South Africa, Taiwan, Ukraine, United Kingdom
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Ardea Biosciences, Inc.CompletedCombination Treatment Study in Subjects With Tophaceous Gout With Lesinurad and Febuxostat (CRYSTAL)Tophaceous GoutPoland, United States, Canada, Australia, New Zealand, Switzerland
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TakedaEnrolling by invitationAlpha1-Antitrypsin DeficiencyUnited Kingdom, Germany, United States, Austria, Portugal
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Vertex Pharmaceuticals IncorporatedCystic Fibrosis FoundationCompletedCystic FibrosisUnited States, France, United Kingdom, Germany, Canada, Czech Republic, Ireland, Australia
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Humanigen, Inc.CompletedCystic FibrosisUnited States, Australia, Israel, New Zealand
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Genzyme, a Sanofi CompanyCompletedThyroid CancerItaly, Spain, Romania, Netherlands, Canada, United States, Switzerland, Australia
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Massachusetts General HospitalNational Center for Complementary and Integrative Health (NCCIH); University...TerminatedHuntington's DiseaseUnited States, Canada, New Zealand, Australia
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HP Therapeutics FoundationHuntington Study GroupUnknownHuntington DiseaseUnited States, Canada, Australia
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Amicus TherapeuticsTerminatedLate-onset Pompe DiseaseUnited States, Canada, Australia, Belgium
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Arrowhead PharmaceuticalsActive, not recruitingFamilial ChylomicronemiaKorea, Republic of, Canada, Australia, Singapore, Belgium, Spain, Japan, United States, Austria, Germany, Ireland, Oman, Poland, Israel, New Zealand, Argentina, France, Croatia, Mexico, Serbia, Turkey
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Seelos Therapeutics, Inc.TerminatedSpinocerebellar Ataxia Type 3United States, Spain, Korea, Republic of, Australia, United Kingdom, Brazil, Germany, Portugal
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Alexion Pharmaceuticals, Inc.RecruitingHypophosphatasiaIsrael, Korea, Republic of, Taiwan, Sweden, Germany, Italy, Turkey, China, Poland, United States, France, Australia, Thailand, United Kingdom, Mexico, Vietnam, Argentina, Belgium, Brazil, Canada, Spain
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QED Therapeutics, Inc.Enrolling by invitationAchondroplasiaAustralia, United States, Argentina, Canada, France, Germany, Italy, Norway, Singapore, Spain, United Kingdom
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Otsuka Pharmaceutical Europe LtdUnknownAutosomal Dominant Polycystic Kidney DiseaseSpain, United Kingdom, Germany, France, Belgium, Switzerland, Austria
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Alexion Pharmaceuticals, Inc.TerminatedWilson DiseaseSpain, Korea, Republic of, France, Germany, Japan, Australia, Poland, United Kingdom
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ErydelRecruitingAtaxia TelangiectasiaUnited States, Australia, Belgium, Germany, India, Italy, Poland, Spain, Tunisia
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GRIN Therapeutics, Inc.RecruitingTuberous Sclerosis Complex | Focal Cortical DysplasiaSpain, United Kingdom, Australia, Belgium, Canada, Italy, Poland
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Baxalta now part of ShireBaxter Innovations GmbHTerminatedHemophilia APoland, United States, Bulgaria, Serbia, Canada, Russian Federation, Germany, Netherlands, Lithuania, Czechia, Austria, Spain
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CSL BehringCompletedHemophilia A | Severe Hemophilia AUnited States, Australia, Austria, Canada, Czechia, France, Georgia, Germany, Hungary, Ireland, Italy, Japan, Lebanon, Malaysia, Netherlands, Philippines, Poland, Portugal, Romania, South Africa, Spain, Switzerland, Thailand, Ukraine, United...
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Jaeb Center for Health ResearchFoundation Fighting BlindnessRecruitingRetinitis Pigmentosa | Inherited Retinal DegenerationUnited States, United Kingdom, France, Finland, Israel, Netherlands, Switzerland, Australia, Canada, Brazil, Mexico
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Wuerzburg University HospitalCompletedCystic FibrosisUnited States, Austria, Canada, France, Germany, Netherlands, Switzerland, United Kingdom
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Ardea Biosciences, Inc.CompletedGoutUnited States, Canada, Australia, New Zealand, Switzerland, Poland
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ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)Denmark, Germany, Japan, Australia, France, Brazil, Czechia
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Hoffmann-La RocheCompletedHemophilia ACanada, Australia, Belgium, India, Germany, Israel, Italy, Spain, Colombia, United Kingdom, Netherlands, Saudi Arabia, Hungary, Guatemala, Russian Federation, Poland, Switzerland, Finland, Panama, Brazil, Mexico, Portugal, Romania, Sw...
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BioMarin PharmaceuticalCompletedPhenylketonuriaItaly, Turkey, United Kingdom, Germany, Netherlands, Slovakia, Austria, Belgium, Czechia
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New York State Institute for Basic ResearchEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsUnknownAlzheimer Disease | Down SyndromeUnited States, Canada, Australia, United Kingdom
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University of Alabama at BirminghamNational Cancer Institute (NCI); Children's Hospital of Philadelphia; Washington... and other collaboratorsCompletedNeurofibromatosis Type 1United States, Australia
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Vertex Pharmaceuticals IncorporatedEnrolling by invitationCystic FibrosisUnited States, Australia, Germany, Sweden, Switzerland, France, Netherlands, United Kingdom
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Vertex Pharmaceuticals IncorporatedActive, not recruitingCystic FibrosisUnited States, Australia, Germany, Sweden, Switzerland, France, Netherlands, United Kingdom
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Alexion PharmaceuticalsCompletedWilson DiseaseUnited States, Germany, Austria, Poland, United Kingdom
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University of IowaJohns Hopkins University; University of Colorado, Denver; King's College Hospital... and other collaboratorsRecruitingCharcot-Marie-Tooth Disease, Type Ia (Disorder) | HMSNUnited States, Italy, United Kingdom, Australia
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase DeficiencyFrance, Poland, United Kingdom, Spain, Mexico, Turkey, Japan, Australia, Russian Federation, United States, Germany, Italy, Czechia, Argentina, Croatia
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IntraBio IncActive, not recruitingNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Germany, Slovakia, Netherlands, Czechia, Switzerland
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Sergiusz JozwiakMedical University of Vienna; Charite University, Berlin, Germany; Institut National... and other collaboratorsUnknownEpilepsy | Tuberous Sclerosis Complex | TSCPoland, Austria, Belgium, Czech Republic, France, Germany, Italy, Netherlands
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AlexionCompletedWilson DiseaseUnited States, Germany, Austria, Poland, United Kingdom
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Baxalta now part of ShireAmerican Thrombosis and Hemostasis Network; UDC Rare Bleeding and Clotting...CompletedProtein C DeficiencyUnited States, United Kingdom, Germany, Italy, Netherlands, Austria
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European Friedreich's Ataxia Consortium for Translational...RecruitingFriedreich's AtaxiaAustria, Germany, Italy, Spain, Belgium, Czechia, France, Greece, Ireland, United Kingdom
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AAustria, Spain, United States, Germany, Serbia, Taiwan, Bulgaria
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ShireTakeda Development Center Americas, Inc.RecruitingHereditary Angioedema (HAE)France, Austria, Australia, Spain, Czechia, Italy, United Kingdom, Germany, Israel, Ireland, Brazil, Denmark, Greece, Sweden