- ICH GCP
- US Clinical Trials Registry
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Clinical Trials on Genetic Diseases, Inborn in Ireland
Total 8115 results
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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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Genzyme, a Sanofi CompanyRecruitingFabry DiseaseCroatia, United States, Argentina, Australia, Belgium, Bulgaria, Canada, Germany, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Malaysia, Philippines, Poland, Romania, Singapore, Slovakia, Taiwan, Thailand, United Kingdom, Vietn... and more
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Eli Lilly and CompanyTerminatedAtopic DermatitisSpain, Puerto Rico, United States, Canada, Japan, Italy, Argentina, Austria, Czechia, France, Germany, Hungary, Mexico
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PfizerCompletedAtopic DermatitisUnited States, Canada, Australia, Germany, Hungary
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Women and Infants Hospital of Rhode IslandSequenom, Inc.CompletedDown Syndrome | Trisomy 21United States, Canada, Israel, Hungary, Ireland, Argentina, Australia, Czech Republic, Italy, Spain
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BioMarin PharmaceuticalCompletedMucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)United States, France, Australia, Germany, Ireland, Austria, Belgium, Italy, Lithuania, Netherlands, Portugal, Sweden, United Kingdom
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CSL BehringActive, not recruitingHemophilia BUnited States, Belgium, Netherlands, Denmark, Germany, Ireland, Sweden, United Kingdom
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AbbVieEnrolling by invitationAtopic DermatitisUnited States, Australia, Austria, Belgium, Canada, China, Czechia, France, Germany, Greece, Hong Kong, Hungary, Ireland, Israel, Italy, Japan, Netherlands, New Zealand, Norway, Puerto Rico, Slovakia, Spain, Sweden, United Kingdom
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CelgeneCompletedEczema | Dermatitis, AtopicUnited States, Canada, Japan, Poland, China, Czechia
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PfizerCompletedAtopic DermatitisGermany, United States, Canada, Denmark, Hungary, Australia, Latvia, Poland, Bulgaria, Japan
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Eli Lilly and CompanyIncyte CorporationCompletedAtopic DermatitisUnited States, Puerto Rico, Canada
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A | Haemophilia BBelgium, Germany, France, United Kingdom, Italy, Hungary, Austria, Sweden, Iran, Islamic Republic of, Argentina, Ireland, Poland, Portugal, Slovakia
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Vertex Pharmaceuticals IncorporatedActive, not recruitingAlpha-1 Antitrypsin DeficiencyUnited States, United Kingdom, Germany, Ireland
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ASLAN PharmaceuticalsCompletedAtopic DermatitisUnited States, Australia, Singapore, India, Canada, New Zealand, Dominican Republic, Poland
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Eli Lilly and CompanyCompletedAtopic DermatitisUnited States
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AbbVieCompletedA Study to Evaluate Risankizumab in Adults and Adolescents With Moderate to Severe Atopic DermatitisDermatitisUnited States, Australia, Canada, Japan, Puerto Rico
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Zynerba Pharmaceuticals, Inc.RecruitingFragile X SyndromeUnited States, Australia, United Kingdom, Ireland
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LEO PharmaTerminatedAtopic DermatitisUnited States, Netherlands, United Kingdom, France, Canada, Ireland, Austria, Germany, Poland
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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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Arcutis Biotherapeutics, Inc.CompletedAtopic Dermatitis EczemaUnited States, Canada
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AbbVieCompletedAtopic DermatitisUnited States, Australia, Canada, Croatia, Czechia, Finland, France, Germany, Hungary, Ireland, Israel, Italy, Malaysia, Netherlands, New Zealand, Norway, Poland, Singapore, Spain, Taiwan, Ukraine, United Kingdom
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MallinckrodtTerminatedSickle Cell Disease | Pulmonary Hypertension | Lung Disease | Lung Transplant | Cardiac TransplantUnited States
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Regeneron PharmaceuticalsSanofiCompletedAtopic DermatitisHungary, Netherlands, Spain, United Kingdom, United States, Australia, Austria, Belgium, Bulgaria, Canada, China, Czechia, Denmark, Estonia, Finland, France, Germany, Ireland, Italy, Japan, Korea, Republic of, Lithuania, New Zealand, ... and more
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National Cancer Institute (NCI)RecruitingCancer | HIV | Chronic Granulomatous Disease | Dock 8 DeficiencyUnited States
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Galderma R&DCompletedAtopic DermatitisUnited States, France, Poland, Germany, Australia, Canada
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Eli Lilly and CompanyDermira, Inc.CompletedAtopic DermatitisUnited States, Spain, Korea, Republic of, Australia, France, Canada, Estonia, Latvia, Lithuania, Poland
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Regeneron PharmaceuticalsSanofiCompletedAtopic DermatitisUnited States, Germany, Poland, Japan, Canada, Hungary, Czechia
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AnaptysBio, Inc.CompletedAtopic DermatitisUnited States, Poland, Germany, United Kingdom, Czechia, Canada
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Canada, Australia, Ireland, United Kingdom
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Novartis PharmaceuticalsCompletedCystic FibrosisSpain, Germany, Switzerland, United Kingdom, Ireland
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited Kingdom, France, Germany, Australia, Switzerland, Ireland, Belgium, Denmark, Poland
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Sequenom, Inc.CompletedDown Syndrome | AneuploidyUnited States
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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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Vanda PharmaceuticalsTerminatedAtopic DermatitisUnited States
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AbbVieCompletedAtopic DermatitisUnited States, Australia, Canada, Croatia, Czechia, Finland, France, Germany, Hungary, Ireland, Israel, Italy, Malaysia, Netherlands, New Zealand, Norway, Poland, Singapore, Spain, Sweden, Taiwan, Ukraine, United Kingdom
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Canada, United Kingdom, Australia, Ireland
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsSlovakia, Spain, Argentina, Germany, Croatia, Italy, Canada, United Kingdom, Ireland, Sweden, France, United States, Czech Republic, Switzerland
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Canada, Australia, Ireland, United Kingdom
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Rhode Island HospitalCystic Fibrosis FoundationRecruitingCystic Fibrosis | Cystic Fibrosis-related Diabetes | Cystic Fibrosis With Intestinal ManifestationsUnited States
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Eunice Kennedy Shriver National Institute of Child...UnknownDown Syndrome | Chromosome AbnormalitiesUnited States
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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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AbbVieActive, not recruitingAtopic DermatitisUnited States, Australia, Austria, Belgium, Bulgaria, Canada, Croatia, Czechia, Denmark, France, Germany, Greece, Hungary, Ireland, Italy, Korea, Republic of, Netherlands, New Zealand, Portugal, Singapore, Spain, Taiwan, United Kingdom
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Baxalta now part of ShireCompleted
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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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Novartis PharmaceuticalsCompletedElevated Cholesterol | Homozygous Familial Hypercholesterolemia | Heterozygous Familial Hypercholesterolemia | ASCVDUnited States, Canada, Czechia, Denmark, Germany, Hungary, Netherlands, Poland, South Africa, Spain, Sweden, Ukraine, United Kingdom
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Genzyme, a Sanofi CompanyCompletedHemophiliaUnited States, Australia, China, Denmark, France, Ireland, Israel, Italy, Japan, Korea, Republic of, Malaysia, Turkey, Ukraine, United Kingdom, Mexico
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Arcutis Biotherapeutics, Inc.Active, not recruitingAtopic Dermatitis EczemaUnited States, Canada
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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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Bristol-Myers SquibbRecruitingAnemiaThailand, China, Canada, Turkey, Greece, Taiwan, Italy, Hong Kong
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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