- 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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Ardea Biosciences, Inc.CompletedGoutUnited States, Germany, New Zealand, South Africa, Spain, Canada, Switzerland, Australia, Belgium, Poland, Ukraine
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Savara Inc.CelerionCompletedBronchiectasis | Cystic Fibrosis | Acute Respiratory Distress Syndrome | Pulmonary Alveolar ProteinosisUnited Kingdom
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University of North Carolina, Chapel HillThe Hospital for Sick Children; Washington University School of Medicine; McGill... and other collaboratorsRecruitingPrimary Ciliary Dyskinesia | Kartagener Syndrome | Primary Immune DeficiencyUnited States, Canada
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Sidney Kimmel Comprehensive Cancer Center at Johns...Massachusetts General Hospital; Washington University School of Medicine; Gla... and other collaboratorsCompletedNeurofibromatosis 2 | Vestibular Schwannoma | Acoustic Neuroma | NF2 | Auditory TumorUnited States
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Santhera PharmaceuticalsCompletedFriedreich's AtaxiaNetherlands, Austria, United Kingdom, Belgium, France, Germany
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Daiichi Sankyo, Inc.TerminatedAtherosclerosis | Heterozygous Familial HypercholesterolemiaUnited States, United Kingdom, Spain, South Africa, Israel, Canada, Netherlands, Norway, Sweden
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ShireTakeda Development Center Americas, Inc.No longer availableAngioedema | Hereditary Angioedema (HAE)United States, Canada, Germany, Hungary, Spain
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Arbor Research Collaborative for HealthNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)SuspendedLiver Diseases | Alpha 1-Antitrypsin Deficiency | Alagille SyndromeUnited States, Canada
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Otsuka Pharmaceutical Europe LtdUnknownAutosomal Dominant Polycystic Kidney DiseaseSpain, United Kingdom, Germany, France, Belgium, Switzerland, Austria
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University of UlmUniversity of Pisa; IRCCS Azienda Ospedaliero-Universitaria di Bologna; Medical... and other collaboratorsRecruitingLipodystrophy Acquired | Lipodystrophy CongenitalAustria, France, Germany, Hungary, Italy, Portugal, Spain, Netherlands, Russian Federation, Slovenia, Switzerland, Turkey, United Kingdom
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BiomX, Inc.Active, not recruitingCystic Fibrosis | Chronic Pseudomonas Aeruginosa InfectionUnited States, Israel, Spain, Czechia, Netherlands
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Novartis PharmaceuticalsCompletedCancerFrance, Australia, United States, Canada, United Kingdom
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Clementia Pharmaceuticals Inc.TerminatedExostoses, Multiple HereditaryUnited States, Spain, Canada, Japan, Italy, Portugal, United Kingdom, Turkey, Australia, Belgium, France, Netherlands
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Vertex Pharmaceuticals IncorporatedActive, not recruitingCystic FibrosisUnited States, Spain, United Kingdom, Belgium, Netherlands, Denmark, Israel, New Zealand, France, Australia, Ireland, Germany, Sweden, Canada, Czechia, Switzerland, Portugal, Italy, Austria, Hungary, Norway, Poland, Greece
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Australia, Canada, France, United Kingdom, Germany, Spain, Switzerland, Belgium, Israel, Netherlands, Denmark, Italy, Austria, Ireland, Sweden
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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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Pfizer's Upjohn has merged with Mylan to form Viatris...CompletedArthritis, GoutyCanada, United States, Spain, Russian Federation, Colombia, Costa Rica, Peru, Taiwan, Korea, Republic of, Mexico, Philippines, Thailand
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Savient PharmaceuticalsCompletedGoutUnited States, Canada, Mexico
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Otsuka Pharmaceutical Development & Commercialization...TerminatedTourette's Disorder (TD)United States, Canada, Hungary
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AeRang KimActive, not recruitingOsteosarcoma | Ewing Sarcoma | Neuroblastoma | Rhabdomyosarcoma | Wilms Tumor | Desmoid Tumor | Germ Cell Tumor | Relapsed Pediatric Solid Tumors | Refractory Pediatric Solid Tumors | Hepatic TumorUnited States
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Manchester University NHS Foundation TrustManchester Metropolitan UniversityCompletedMuscle Weakness | Neurofibromatosis Type 1United Kingdom
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Genzyme, a Sanofi CompanyCompletedGaucher Disease, Type 1 | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency Disease | Gaucher Disease, Non-Neuronopathic FormUnited States, United Kingdom, Spain, Italy, Brazil, Canada, Poland
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MD Stem CellsRecruitingStroke | Nervous System Diseases | Diabetic Neuropathies | Neurodegenerative Diseases | Cognitive Impairment | Amyotrophic Lateral Sclerosis | Dementia | Neurological Disorders | Alzheimer Disease | ALS | Traumatic Brain Injury | Cerebral Infarction | Lewy Body Disease | Cerebral Hemorrhage | Frontotemporal Dementia | Progressive... and other conditionsUnited States, United Arab Emirates
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NHS TaysideUniversity of DundeeCompletedAtaxia-TelangiectasiaUnited 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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University of NottinghamNottingham University Hospitals NHS Trust; Royal Papworth HospitalActive, not recruitingAtaxia TelangiectasiaUnited Kingdom
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Agios Pharmaceuticals, Inc.RecruitingPyruvate Kinase DeficiencyUnited States, Germany, Spain, Italy, United Kingdom, Czechia, France, Canada, Korea, Republic of, Netherlands, Turkey, Portugal, Denmark, Ireland, Switzerland, Thailand
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Novo Nordisk A/SRecruitingHaemophilia A With Inhibitors | Haemophilia ATaiwan, Canada, Spain, Netherlands, Korea, Republic of, India, United States, United Kingdom, China, Portugal, Lithuania, Turkey, South Africa, Ireland, Italy, France, Japan, Belgium, Malaysia, Poland, Austria, Bulgaria, Denmark, Germany and more
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Istituto Auxologico ItalianoFondazione TelethonCompletedOsteoporosis | Cystic FibrosisItaly, Holy See (Vatican City State)
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Gilead SciencesCompletedCystic Fibrosis | Pseudomonas Aeruginosa | Lung InfectionUnited States, Australia, Canada
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Huntington Study GroupNational Institute of Neurological Disorders and Stroke (NINDS); National Human...UnknownHuntington DiseaseUnited States, Canada
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Agios Pharmaceuticals, Inc.RecruitingPediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic AnemiaUnited States, Spain, Germany, Netherlands, Canada, France, Israel, Switzerland, Italy
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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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Johns Hopkins UniversityNational Cancer Institute (NCI); National Institutes of Health (NIH); ChiRhoClin...RecruitingLynch Syndrome | Pancreas Cancer | Gene Mutation | Peutz-Jeghers Syndrome (PJS) | Germline Mutation CarrierUnited States
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Northwell HealthCompletedPure Red Cell Aplasia | Diamond Blackfan Anemia | Blackfan Diamond Syndrome | DBA | Congenital Hypoplastic AnemiaUnited States
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Craig L Slingluff, JrTheraclionActive, not recruitingMelanoma | Cervical Cancer | Breast Cancer | Hepatocellular Carcinoma | Gastric Cancer | Esophageal Cancer | Ovarian Cancer | Non Small Cell Lung Cancer | Small-cell Lung Cancer | Urothelial Carcinoma | Merkel Cell Carcinoma | Microsatellite Instability High | Squamous Cell CancerUnited States
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Grifols Therapeutics LLCActive, not recruitingPulmonary Emphysema in Alpha-1 PI DeficiencyUnited States, Australia, Denmark, New Zealand, Estonia, Poland, Russian Federation, Sweden, Brazil, Canada, France, Finland
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Amicus TherapeuticsActive, not recruitingPompe Disease (Late-onset)United States, Germany, Taiwan, Belgium, Australia, Slovenia, Spain, Netherlands, Hungary, Japan, Austria, United Kingdom, France, Argentina, Canada, Sweden, Denmark, New Zealand, Greece, Korea, Republic of, Bosnia and Herzegovina, Ita... and more
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Astellas Pharma Global Development, Inc.No longer availableAcute Myeloid Leukemia (AML) | FMS-like Tyrosine Kinase-3 (FLT3) MutationsAustralia, Italy, United Kingdom
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Grifols Therapeutics LLCTerminatedCOVID-19United States, Brazil, Chile, Colombia, Mexico
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National Institute of Allergy and Infectious Diseases...National Center for Advancing Translational Sciences (NCATS); Primary Immune...Enrolling by invitationADA-SCID | Omenn Syndrome | SCID | Reticular Dysgenesis | XSCID | Leaky SCIDUnited States, Canada
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University of PlymouthRecruitingAtaxia Telangiectasia | Ataxia Telangiectasia in ChildrenUnited Kingdom
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Sidney Kimmel Cancer Center at Thomas Jefferson...CompletedPrimary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent... and other conditionsUnited States
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MD Stem CellsEnrolling by invitationAlzheimer Disease | Traumatic Brain Injury | Autism Spectrum Disorder | Lewy Body Disease | Vascular Dementia | Autism | Dementia, Mixed | Alzheimer Dementia | Dementia, Multi-Infarct | Cadasil | Parkinson-Dementia Syndrome | Chronic Traumatic Encephalopathy | Autistic Behavior | Lewy Body Dementia With Behavioral... and other conditionsUnited States, United Arab Emirates
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Newcastle UniversityLudwig-Maximilians - University of Munich; LGMD2i Research Fund; CureLGMD2iRecruitingLimb Girdle Muscular Dystrophy | Congenital Muscular Dystrophy | LGMDR9 | LGMD2I | Walker-Warburg Syndrome | Muscle-Eye-Brain Disease | FKRP Gene MutationUnited Kingdom
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University of Alabama at BirminghamCompletedNeurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PNUnited States
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Genzyme, a Sanofi CompanyTerminatedPompe DiseaseUnited States
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Ardea Biosciences, Inc.CompletedGoutUnited States, South Africa, Spain, Belgium, Australia, Germany, Canada, New Zealand, Poland, Ukraine, Switzerland
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TakedaCompleted
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MacroGenicsCompletedOsteosarcoma | Ewing Sarcoma | Neuroblastoma | Rhabdomyosarcoma | Wilms Tumor | Desmoplastic Small Round Cell TumorUnited States