- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Genetic Diseases, X-Linked in United States
Total 7967 results
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Fusion Pharmaceuticals Inc.TerminatedBreast Cancer | Gastric Cancer | Colorectal Cancer | Ovarian Cancer | Lung Cancer | Advanced Solid Tumor | Head and Neck Squamous Cell Carcinoma | Liver Cancer | Bladder Carcinoma | Susceptible FGFR3 Genetic Alterations | FGFR3 | FGFR3 Overexpression | FGFR3 Receptor | FGFR3 Protein OverexpressionUnited States, Australia
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ShireCompletedHereditary Angioedema (HAE)United States, United Kingdom, Germany, Canada, Italy, Jordan, Puerto Rico
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Fondazione Angelo Bianchi BonomiSintesi Research SrlActive, not recruitingType 3 Von Willebrand's DiseaseFinland, France, Germany, Hungary, Iran, Islamic Republic of, Italy, Netherlands, Spain, Sweden, United Kingdom
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Natera, Inc.CompletedTrisomy 21 | Trisomy 18 | Trisomy 13 | Vanishing TwinUnited States
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ShireCompletedHereditary Angioedema (HAE)Spain, Israel, United States, Hungary, Germany, Canada, Romania
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ShireCompletedHereditary Angioedema (HAE)United States, Canada, Australia, Austria, Colombia, Germany, Hungary, Israel, Italy, Spain
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CSL BehringCompletedHereditary Angioedema Types I and IIUnited States, Spain, Australia, Canada, Czechia, Hungary, Israel, Italy, Romania, United Kingdom
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PTC TherapeuticsCompletedMitochondrial Diseases | Drug Resistant Epilepsy | Leigh Disease | Leigh Syndrome | Mitochondrial Encephalopathy (MELAS) | Pontocerebellar Hypoplasia Type 6 (PCH6) | Alpers Disease | Alpers SyndromeUnited States, Spain, United Kingdom, Canada, France, Italy, Poland, Sweden, Japan
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Scott R. Plotkin, MD, PhDTakeda; National Comprehensive Cancer Network; The Children's Tumor FoundationRecruitingEpendymoma | Meningioma | Neurofibromatosis Type 2 | Vestibular Schwannoma | Non-vestibular SchwannomaUnited States
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H. Lundbeck A/SRecruitingCongenital Adrenal HyperplasiaUnited Kingdom
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Sheffield Teaching Hospitals NHS Foundation TrustSociety for Endocrinology; The Clinical Endocrinology TrustCompletedCongenital Adrenal HyperplasiaUnited Kingdom
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Gilead SciencesCompletedCystic FibrosisUnited States, France, Spain, Germany, Austria, Belgium, Italy, Netherlands, Poland
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Royal Marsden NHS Foundation TrustMerck KGaA, Darmstadt, Germany; University of Oxford; University of SurreyTerminatedMicrosatellite Instability | Stage III Colon Cancer | POLE Exonuclease Mutant Colon CancerUnited Kingdom
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Respirion Pharmaceuticals Pty LtdRecruitingPseudomonas Aeruginosa | Cystic Fibrosis Lung | Respiratory Infections, Recurrent, ChronicUnited States, Australia
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Bonnie RamseyNational Heart, Lung, and Blood Institute (NHLBI)CompletedCystic FibrosisUnited States
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Otsuka Pharmaceutical Development & Commercialization...CompletedAutosomal Dominant Polycystic Kidney Disease (ADPKD)Germany, Italy, Belgium, United Kingdom
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Acceleron Pharma Inc. (a wholly owned subsidiary...TerminatedCharcot-Marie-Tooth Disease | Facioscapulohumeral Muscular DystrophyUnited States, Spain, Canada
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Genzyme, a Sanofi CompanyCompletedThyroid CancerItaly, Spain, Romania, Netherlands, Canada, United States, Switzerland, Australia
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Novartis PharmaceuticalsCompletedAcute Gouty ArthritisHungary, Germany, United States, Lithuania, Canada
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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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ShireTerminatedMetabolic Diseases | Brain Diseases | Central Nervous System Diseases | Nervous System Diseases | Demyelinating Diseases | Genetic Diseases, Inborn | Metabolism, Inborn Errors | Lysosomal Storage Diseases | Lipid Metabolism Disorders | Sphingolipidoses | Hereditary Central Nervous System Demyelinating Diseases and other conditionsUnited States, Denmark, France, Argentina, Belgium, Brazil, Canada, Germany, Japan, Turkey
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National Institute of Allergy and Infectious Diseases...National Center for Advancing Translational Sciences (NCATS); Office of Rare... and other collaboratorsEnrolling by invitationOmenn Syndrome | Reticular Dysgenesis | Severe Combined Immunodeficiency (SCID) | XSCID | Leaky SCID | ADA SCIDUnited States, Canada
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AUnited States, Macedonia, The Former Yugoslav Republic of, Serbia, Malaysia, Brazil, Turkey, Lithuania, Russian Federation, Italy, Japan, Poland, Puerto Rico, Taiwan
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AstraZenecaMerck Sharp & Dohme LLCActive, not recruitingNeurofibromatosis 1 | Plexiform Neurofibroma (PN)United States, Canada, France, Italy, Germany, China, United Kingdom, Spain, Japan, Russian Federation, Australia, Poland, Brazil
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Academic and Community Cancer Research UnitedNational Cancer Institute (NCI)RecruitingBile Duct Adenocarcinoma | Fanconi Anemia Complementation Group Gene Mutation | Metastatic Bile Duct Carcinoma | PTEN Gene DeletionUnited States
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Newcastle-upon-Tyne Hospitals NHS TrustRecruitingBethlem Myopathy | Ullrich Congenital Muscular Dystrophy 1, Digenic, Col6A1/Col6A2 | Ullrich Congenital Muscular Dystrophy 1, Autosomal Recessive | Ullrich Congenital Muscular Dystrophy 1, Autosomal Dominant | Bethlem Myopathy 1, Autosomal Recessive | UCMD | BTHLM1United Kingdom
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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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Novartis PharmaceuticalsCompletedPulmonary Infections | Pseudomonas Aeruginosa in Cystic FibrosisUnited States, Italy, Germany, France, Australia, Canada, Spain, Hungary, Mexico, Argentina
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LG ChemRecruitingHyperuricemia | Gout | Gout Flare | TophiUnited States
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Margaret RagniNational Heart, Lung, and Blood Institute (NHLBI); Duke University; University...TerminatedVon Willebrand DiseasesUnited States
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KalVista Pharmaceuticals, Ltd.RecruitingHereditary AngioedemaUnited States, Bulgaria, France, Greece, Israel, Netherlands, Spain, United Kingdom, Australia, Germany, South Africa, Slovakia, Austria, New Zealand, Romania, Canada, Japan
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Otsuka Pharmaceutical Development & Commercialization...CompletedTic Disorder | Tourette's DisorderUnited States, Germany, Spain, Mexico, Romania, Canada, Hungary, Italy, Sweden
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Rocket Pharmaceuticals Inc.Active, not recruitingFanconi Anemia Complementation Group ASpain, United Kingdom
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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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ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)United States, Canada, Belgium, Israel, Netherlands, United Kingdom, Germany, Spain, Japan, Brazil, Argentina, France, Greece, Italy
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Noema Pharma AGRecruitingTuberous Sclerosis ComplexUnited States, Turkey, Poland, Spain, United Kingdom, India, Italy, Israel
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ShireTakeda Development Center Americas, Inc.CompletedAngioedemaUnited States, France, Netherlands, Spain, Hungary, Italy, Canada, Poland, Japan, Germany
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ShireCompletedHereditary AngioedemaUnited States
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AUnited States, Netherlands, Spain, Switzerland, Taiwan, Turkey, United Kingdom, Croatia, Germany, Brazil, France, Japan, Australia, Hungary, Israel, Korea, Republic of, Lithuania, Portugal, Ukraine, Greece, Italy, Denmark, Norway, Canada and more
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AFrance, United States, Denmark, Netherlands, Sweden, Spain, Germany, Switzerland, Taiwan, United Kingdom, Croatia, Malaysia, Brazil, Australia, Hungary, Israel, Turkey, Italy, Japan, Norway, Bulgaria
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ShireTakeda Development Center Americas, Inc.CompletedHereditary Angioedema (HAE)Canada, United States, Puerto Rico
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Children's Health IrelandBelfast Health and Social Care Trust; University of Edinburgh; University Hospital... and other collaboratorsRecruitingQuality of Life | Obstructive Sleep Apnea | Down Syndrome | Behavior | Adherence, TreatmentUnited Kingdom, Ireland
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Symbollon PharmaceuticalsUnknownPain | Fibrocystic Disease of Breast | Fibrocystic Changes of Breast | Fibrocystic MastopathyUnited States
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TakedaRecruitingLennox Gastaut Syndrome (LGS) | Dravet Syndrome (DS)United States, China, Spain, France, Belgium, Australia, Brazil, Canada, Germany, Greece, Hungary, Italy, Japan, Latvia, Netherlands, Poland, Serbia, Mexico, Russian Federation, Ukraine
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Sequenom, Inc.CompletedTurner Syndrome | Down Syndrome (Trisomy 21) | Patau Syndrome (Trisomy 13) | Edwards Syndrome (Trisomy 18)United States
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Biohaven Pharmaceuticals, Inc.Active, not recruitingSpinocerebellar Ataxias | Spinocerebellar Ataxia Genotype Type 1 | Spinocerebellar Ataxia Genotype Type 2 | Spinocerebellar Ataxia Genotype Type 3 | Spinocerebellar Ataxia Genotype Type 6 | Spinocerebellar Ataxia Genotype Type 7 | Spinocerebellar Ataxia Genotype Type 8 | Spinocerebellar Ataxia Genotype...United States
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TakedaCompleted
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VA Office of Research and DevelopmentCompletedChronic Kidney Diseases | GoutUnited States
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Mirum Pharmaceuticals, Inc.CompletedAlagille SyndromeFrance, Belgium, Poland, Australia, Spain, United Kingdom
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ZevraDenmarkAvailableNiemann-Pick Disease, Type CUnited States