- ICH GCP
- US Clinical Trials Registry
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Clinical Trials on Genetic Diseases, X-Linked in Ireland
Total 396 results
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Novo Nordisk A/SCompletedChronic Kidney Disease | Turner Syndrome | Noonan Syndrome | Chronic Renal Insufficiency | Growth Hormone Disorder | Growth Hormone Deficiency in Children | Adult Growth Hormone Deficiency | Genetic Disorder | Foetal Growth Problem | Small for Gestational AgeIsrael, Saudi Arabia, Spain, Argentina, Russian Federation, Germany, Netherlands, Italy, Belgium, United Kingdom, Slovenia, Ireland, Sweden, Denmark, Hungary, Finland, Norway, France, Czechia, Lithuania, Luxembourg, Serbia, Switzerland
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Regeneron PharmaceuticalsSanofiCompletedAtopic DermatitisUnited States, Italy, United Kingdom, Spain, Japan, Canada, Netherlands, New Zealand, Hungary, Czechia, Poland, Australia, Romania, Korea, Republic of
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Olatec Therapeutics LLCRecruitingArthritis | Joint Pain | Gout | Gout Flare | Gouty Arthritis | Acute Gout Flare | Gout AttackUnited States, Spain, France
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AmgenRecruitingAtopic DermatitisUnited States, Korea, Republic of, Taiwan, Spain, Croatia, China, Greece, Belgium, Thailand, Puerto Rico, Hungary, Poland, Germany, Brazil, Italy, Japan, Canada, France, Romania, Chile
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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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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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St George's, University of LondonTerminatedEndometrial Cancer | Hereditary Non-polyposis Colon Cancer (hmsh2, hmlh1, hpms1, hpms2)United Kingdom
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GlycoMimetics IncorporatedCompletedAnemia, Sickle CellUnited States, Canada
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Boehringer IngelheimTerminatedTourette SyndromeUnited States, Germany
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AbbVieActive, not recruitingAtopic DermatitisUnited States, Argentina, Australia, Bosnia and Herzegovina, Bulgaria, Canada, China, Colombia, Croatia, Denmark, Estonia, Finland, France, Germany, Italy, Japan, Malaysia, New Zealand, Puerto Rico, Romania, Russian Federation, Sw... and more
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Vertex Pharmaceuticals IncorporatedTerminatedAlpha 1-Antitrypsin DeficiencyUnited States, Germany, Canada, Ireland
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Vertex Pharmaceuticals IncorporatedCystic Fibrosis FoundationCompletedStudy of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation (ENVISION)Cystic FibrosisUnited States, Australia, Canada, France, Germany, Ireland, United Kingdom
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Rhythm Pharmaceuticals, Inc.RecruitingObesity | Genetic ObesityUnited States, Spain, Germany, Netherlands, United Kingdom, Greece, Puerto Rico, Canada, France, Israel
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Eli Lilly and CompanyDermira, Inc.Active, not recruitingAtopic DermatitisUnited States, Spain, Canada, France, Korea, Republic of, Taiwan, Australia, Germany, Bulgaria, Estonia, Latvia, Lithuania, Mexico, Poland, Singapore, Ukraine
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BioMarin PharmaceuticalTerminatedLate-onset Pompe Patients Untreated or Treated With rhGAASpain, Slovenia, Serbia, Korea, Republic of, Greece, Australia, Singapore, Brazil, Canada, Taiwan, Ireland, Poland, Romania
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Mukoviszidose Institut gGmbHCompletedCystic FibrosisGermany, Italy, Ireland, Austria, Belgium, Hungary, Poland, Spain, Sweden
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Ascendis Pharma A/SCompletedAchondroplasiaUnited States, Australia, Austria, Germany, China, Canada, Denmark, France, Ireland, Italy, New Zealand, Portugal, Spain, Switzerland, United Kingdom
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Bristol-Myers SquibbRecruitingAnemiaThailand, China, Canada, Turkey, Greece, Taiwan, Italy, Hong Kong
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Hoffmann-La RocheChugai PharmaceuticalCompletedHemophilia AAustralia, Taiwan, Korea, Republic of, France, Germany, Italy, South Africa, Spain, United States, Japan, Ireland, Poland, United Kingdom, Costa Rica
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American Thrombosis and Hemostasis NetworkTakedaActive, not recruiting
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Insmed IncorporatedCompletedCystic FibrosisFrance, Poland, United Kingdom, Germany, Spain, Belgium, Bulgaria, Serbia, Denmark, Greece, Netherlands, Hungary, Ireland, Slovakia, Canada, Italy, Austria
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Boehringer IngelheimCompletedTourette SyndromeUnited States, Germany
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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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Bioverativ, a Sanofi companyCompletedHemophilia ATurkey, Australia, Canada, France, Germany, Hungary, Ireland, Italy, Netherlands, Spain, Sweden, Switzerland, Taiwan, United Kingdom, United States
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Parion SciencesVertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited Kingdom, United States, France, Ireland
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OctapharmaCompleted
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Israel, Ireland, United Kingdom
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Kowa Research Institute, Inc.CompletedFuchs' Endothelial Corneal DystrophyUnited States, Australia, Germany, Spain, Denmark
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Novartis PharmaceuticalsCompletedSickle Cell Disease (SCD)United Kingdom, Spain, Greece, Brazil, Turkey, France, United States, Netherlands, Lebanon, Ireland, Panama
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Vertex Pharmaceuticals IncorporatedCompletedAlpha1-Antitrypsin DeficiencyUnited States, Germany, Canada, Ireland, Sweden, United Kingdom
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BayerCompletedHaemophilia ASpain, Russian Federation, United States, Hungary, Italy, Mexico, Norway, Romania, Bulgaria, Canada, Argentina, Denmark, Israel, Poland, Ireland, Latvia, Lithuania
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Belgium, Netherlands, Australia, Germany, Ireland, United Kingdom
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Spain, Australia, United Kingdom, Germany, Ireland
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University of Southern CaliforniaNational Institute on Aging (NIA); Alzheimer's Therapeutic Research Institute; Alzheimer's Clinical Trials ConsortiumRecruitingDementia | Alzheimer Disease | Down SyndromeUnited States, Spain, United Kingdom
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Prana Biotechnology LimitedCompletedHuntington DiseaseUnited States, Australia
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Friedreich's Ataxia Research AllianceRecruitingFriedreich AtaxiaUnited States, Canada, New Zealand, Australia, France, Austria, Belgium, Brazil, Czechia, Germany, Greece, Ireland, Italy, Spain, United Kingdom, India, Netherlands
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PfizerCompletedHemophilia AUnited States, Argentina, Austria, Colombia, Croatia, Jordan, Mexico, New Zealand, Oman, Peru, Poland, Romania, Turkey
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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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PfizerCompletedHyperlipidemia | Hypercholesterolemia, FamilialAustralia, Canada, United States, France, South Africa, Sweden, Norway, Denmark, Iceland
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Novo Nordisk A/SActive, not recruitingShort Stature Children Born Small for Gestational Age (SGA)France, Russian Federation, United States, India, Spain, Estonia, Japan, Italy, Algeria, Austria, Hungary, Ireland, Israel, Latvia, Poland, Serbia, Thailand, Ukraine
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Sequenom, Inc.ObstetrixCompleted
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BioCryst PharmaceuticalsCompleted
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Regeneron PharmaceuticalsCompleted
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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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Symbollon PharmaceuticalsUnknownPain | Fibrocystic Disease of Breast | Fibrocystic Changes of Breast | Fibrocystic MastopathyUnited States
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Germany, United Kingdom, Canada, Australia, Switzerland, France, Ireland, Belgium, Denmark, Poland
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Agios Pharmaceuticals, Inc.Active, not recruitingPyruvate Kinase DeficiencyUnited States, Spain, Japan, Canada, United Kingdom, Korea, Republic of, Ireland, Thailand, Italy, Turkey, Denmark, Brazil, France, Germany, Netherlands, Switzerland
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Jiangsu Atom Bioscience and Pharmaceutical Co.,...Enrolling by invitationGoutUnited States, Georgia, Australia, Guatemala, Taiwan
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Teva Branded Pharmaceutical Products R&D, Inc.CompletedHuntington DiseaseUnited States, Canada
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Longboard PharmaceuticalsRecruitingDravet Syndrome | Lennox-Gastaut Syndrome | Developmental and Epileptic EncephalopathyUnited States, Australia