- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Genetic Diseases, Inborn in Ireland
Total 8115 results
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Freeline TherapeuticsCompletedHemophilia B | Blood Coagulation Disorders, Inherited | Blood Coagulation Disorder | Haematologic DiseaseUnited States, United Kingdom, Australia, Netherlands, Austria, France, Ireland, Italy, Turkey, Canada, Germany, South Africa
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Vyne Therapeutics Inc.CompletedPruritus | Atopic DermatitisUnited States
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Vanda PharmaceuticalsCompletedAtopic DermatitisUnited States
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Bioverativ Therapeutics Inc.Swedish Orphan BiovitrumCompletedHemophilia AUnited States, Poland, United Kingdom, Netherlands, South Africa, Hong Kong, Australia, Ireland
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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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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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AOBiome LLCbioRASI, LLCCompletedAtopic DermatitisUnited States
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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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Swedish Orphan BiovitrumSyneos HealthActive, not recruitingHemophilia A, SevereGermany, Spain, France, Czechia, Netherlands, Ireland, Austria, Croatia, Greece, Italy, Norway, Slovenia, Sweden, United Kingdom
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Annexon, Inc.CompletedHuntington DiseaseUnited States
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GlycoMimetics IncorporatedCompletedAnemia, Sickle CellUnited States, Canada
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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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Genzyme, a Sanofi CompanyRecruitingMucopolysaccharidosis I (MPS I)Croatia, United States, Argentina, Australia, Belgium, Brazil, Canada, Denmark, France, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Lebanon, Malaysia, Netherlands, Pakistan, Philippines, Poland, Saudi Arabia, Singapore, Slovaki... and more
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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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Seaside Therapeutics, Inc.CompletedFragile X SyndromeUnited States
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Seaside Therapeutics, Inc.TerminatedFragile X SyndromeUnited States
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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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Vertex Pharmaceuticals IncorporatedCystic Fibrosis FoundationCompletedCystic FibrosisUnited States, Canada, Australia, Ireland, France, United Kingdom, Germany, Czech Republic
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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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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Canada, Australia, Ireland, Germany, United Kingdom
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ProQR TherapeuticsEuropean CommissionCompletedCystic FibrosisUnited States, Spain, United Kingdom, Germany, Italy, Belgium, Canada, Czechia, Denmark, France
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National Human Genome Research Institute (NHGRI)CompletedMelanoma | Breast Neoplasm | Ovarian Neoplasm | Hereditary Neoplastic SyndromeUnited States, Finland, Iceland, Sweden
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Women and Infants Hospital of Rhode IslandNatera, Inc.CompletedTrisomy 21 | Trisomy 18 | Trisomy 13 | Monosomy XUnited States
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Novartis PharmaceuticalsTerminatedCystic FibrosisUnited States, Belgium, Germany, France, United Kingdom, Ireland, Romania
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Bambino Gesù Hospital and Research InstituteRadboud University Medical Center; University of Exeter; Consorzio per Valutazioni...RecruitingCystic Fibrosis | AspergillosisIreland, Spain, Netherlands, France, United Kingdom, Czechia, Germany, Greece, Italy, Portugal, Switzerland
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Vertex Pharmaceuticals IncorporatedCystic Fibrosis FoundationCompletedCystic FibrosisUnited States, France, United Kingdom, Germany, Canada, Czech Republic, Ireland, Australia
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Seaside Therapeutics, Inc.Completed
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Neurocrine BiosciencesCompletedTourette SyndromeUnited States
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Novartis PharmaceuticalsTerminatedFragile X SyndromeUnited States, Belgium, Australia, Israel, Switzerland, Germany, Denmark, Italy, Netherlands, Spain, Sweden, United Kingdom, France
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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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Novartis PharmaceuticalsTerminatedFragile X SyndromeGermany, United States, Switzerland, Australia, Canada, Denmark, France, Italy, United Kingdom, Spain
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Neurocrine BiosciencesCompletedTourette SyndromeUnited States
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Kowa Research Institute, Inc.CompletedFuchs' Endothelial Corneal DystrophyUnited States, Australia, Germany, Spain, Denmark
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BioMarin PharmaceuticalICON plcCompletedMucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVAUnited States, United Kingdom, Australia, Taiwan, Belgium, Malaysia, Austria, Canada, Portugal, France, Ireland, Czechia, Denmark, Italy, Netherlands, Poland, Puerto Rico
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Omeros CorporationTerminatedHuntington's DiseaseUnited States
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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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Kyowa Kirin Pharmaceutical Development LtdActive, not recruitingX-Linked HypophosphatemiaSpain, Italy, United Kingdom, France, Belgium, Denmark, Germany, Hungary, Portugal, Norway, Switzerland, Israel, Netherlands, Czechia, Bulgaria, Sweden, Slovenia, Ireland, Latvia, Slovakia
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Bioverativ Therapeutics Inc.CompletedSevere Hemophilia BUnited States, Italy, Belgium, Australia, Brazil, Hong Kong, India, Ireland, Japan, South Africa, United Kingdom, Netherlands, Sweden, Canada, Poland, China, France, Germany
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Vertex Pharmaceuticals IncorporatedTerminatedAlpha 1-Antitrypsin DeficiencyUnited States, Germany, Canada, Ireland
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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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American Thrombosis and Hemostasis NetworkTakedaActive, not recruiting
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Novartis PharmaceuticalsCompletedFragile X SyndromeUnited States, Switzerland, Germany, Australia, Canada, Denmark, France, Italy, Spain, United Kingdom
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OctapharmaCompleted
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Spruce BiosciencesActive, not recruitingCongenital Adrenal HyperplasiaUnited States, Australia, Brazil, Canada, Denmark, Estonia, Germany, Ireland, Italy, Korea, Republic of, Latvia, Lithuania, Netherlands, Poland, Romania, Spain, Sweden, Switzerland, Turkey, United Kingdom
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Novartis PharmaceuticalsCompletedHereditary Periodic FeversBelgium, Italy, Japan, Russian Federation, Turkey, Spain, Germany, United Kingdom, France, United States, Ireland, Israel, Canada, Hungary, Netherlands, Switzerland
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Parion SciencesVertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited Kingdom, United States, France, Ireland
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Ironwood Pharmaceuticals, Inc.Medpace, Inc.TerminatedGout | Chronic Kidney Disease (CKD)United States, Hungary, Poland, Czechia
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Novartis PharmaceuticalsCompletedFragile X SyndromeUnited States, Belgium, Australia, Israel, Switzerland, Germany, France, Canada, Denmark, Indonesia, Italy, Netherlands, Spain, Sweden, Turkey, 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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Brown, Theodore R., M.D., MPHCompletedStroke | Multiple Sclerosis | Parkinson's Disease | Brain Injury | Amyotrophic Lateral Sclerosis | Alzheimer's Disease | Spinal Cord Injury | Huntington's DiseaseUnited States