- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Genetic Diseases, X-Linked in United States
Total 7970 results
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Baylor College of MedicineActive, not recruitingFocal Dermal Hypoplasia (FDH) | Goltz SyndromeUnited States
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PTC TherapeuticsMuscular Dystrophy AssociationCompletedDuchenne Muscular DystrophyUnited States
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Bio Products LaboratoryCompletedPrimary Immunodeficiency | Wiskott-Aldrich Syndrome | Hypogammaglobulinemia | Common Variable Hypogammaglobulinemia | X-linked Hypogammaglobulinemia | Immunodeficiency With Hyper-IgMUnited States
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NS Pharma, Inc.Nippon Shinyaku Co., Ltd.RecruitingDuchenne Muscular DystrophyUnited States
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Sarepta Therapeutics, Inc.CompletedMuscular Dystrophy, DuchenneUnited States
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Sheffield Children's NHS Foundation TrustSheffield Hallam University; National Institute for Health Research, United... and other collaboratorsActive, not recruiting
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GenethonInstitute of MyologyUnknownDuchenne Muscular DystrophyUnited Kingdom, France
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NightstaRx Ltd, a Biogen CompanyCompletedX-Linked Retinitis PigmentosaUnited States, France, United Kingdom, Finland, Netherlands, Germany, Canada
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CureDuchenneRecruitingDuchenne Muscular Dystrophy | Becker Muscular DystrophyUnited States
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University of California, San FranciscoUniversity of Washington; California Institute of TechnologyRecruiting
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National Institute of Allergy and Infectious Diseases...TerminatedChronic Granulomatous Disease | Inflammatory Bowel Disease (IBD) | Crohn'S-like IBDUnited States
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Minoryx Therapeutics, S.L.Active, not recruitingAdrenoleukodystrophySpain, United States, Hungary, France, Germany, Italy, Netherlands, United Kingdom
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Catabasis PharmaceuticalsTerminatedDuchenne Muscular DystrophyUnited States, Canada, Australia, United Kingdom, Germany, Sweden
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Medexus Pharma, Inc.CompletedHemophilia BUnited States, France, United Kingdom, India, Israel, Italy, Poland
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New York Presbyterian HospitalCompletedHemophilia A | Hemophilia BUnited States
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National Institute of Allergy and Infectious Diseases...RecruitingChronic Granulomatous Disease | CGDUnited States
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Sanguine BiosciencesTerminatedChronic Granulomatous DiseaseUnited States
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StemCells, Inc.CompletedPelizaeus-Merzbacher DiseaseUnited States
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bluebird bioCompletedCerebral Adrenoleukodystrophy (CALD)United States, France, Germany, Italy, Netherlands, United Kingdom
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Weill Medical College of Cornell UniversityBayer; University Hospital, BonnCompletedHemophilia A | Hemophilia BUnited States, Israel, Australia, Italy, Sweden, Germany, Japan, Netherlands, Taiwan
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Sarepta Therapeutics, Inc.British Medical Research CouncilCompletedDuchenne Muscular DystrophyUnited Kingdom
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Santhera PharmaceuticalsNo longer availableDuchenne Muscular DystrophyUnited States
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StemCells, Inc.CompletedPelizaeus-Merzbacher Disease | PMDUnited States
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Wave Life Sciences Ltd.TerminatedDuchenne Muscular DystrophyUnited States, Belgium, United Kingdom, Czechia, Canada, France, Italy, Sweden
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National Institute of Allergy and Infectious Diseases...Recruiting
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Sarepta Therapeutics, Inc.Active, not recruitingDuchenne Muscular DystrophyUnited States, Belgium, Spain, United Kingdom, Netherlands, Germany, Canada, Italy
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Astellas Pharma IncTerminatedDuchenne Muscular Dystrophy (DMD)United States
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University College, LondonRadboud University Medical Center; Groupe Hospitalier Pitie-Salpetriere; Leiden... and other collaboratorsCompletedDuchenne Muscular DystrophyNetherlands, France, United Kingdom
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Imperial College LondonSarepta Therapeutics, Inc.; Department of Health, United KingdomCompletedDuchenne Muscular DystrophyUnited Kingdom
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Icahn School of Medicine at Mount SinaiNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)CompletedErythropoietic Protoporphyria | EPP | X-linked Protoporphyria | XLPUnited States
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Children's Healthcare of AtlantaWithdrawnScoliosis | Duchenne's Muscular DystrophyUnited States
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Baxalta now part of ShireCompletedHemophilia A | Hemophilia BUnited States
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bluebird bioCenter for International Blood and Marrow Transplant ResearchRecruitingCerebral Adrenoleukodystrophy (CALD)United States
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Avidity Biosciences, Inc.RecruitingDuchenne Muscular Dystrophy | Exon 44United States
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Jett Foundation, Inc.Pfizer; Hoffmann-La Roche; Italfarmaco; Nationwide Children's Hospital; Capricor... and other collaboratorsCompletedDuchenne Muscular Dystrophy | Rare Diseases | Disability Physical | Burden, Dependency | Impairment | Disease ManagementUnited States
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Vanderbilt University Medical CenterRecruitingDuchenne Muscular DystrophyUnited States
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Sarepta Therapeutics, Inc.Hoffmann-La RocheRecruitingDuchenne Muscular DystrophyBelgium, United States, Japan, Spain, Italy, United Kingdom
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ReveraGen BioPharma, Inc.University of Pittsburgh; Newcastle University; European Union; Cooperative International...CompletedDuchenne Muscular DystrophyUnited States, Belgium, Canada, United Kingdom, Spain, Netherlands, Sweden, Czechia, Australia, Greece, Israel
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University of RochesterNational Institute of Neurological Disorders and Stroke (NINDS); Newcastle... and other collaboratorsCompletedDuchenne Muscular DystrophyUnited States, Canada, United Kingdom, Germany, Italy
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Santhera PharmaceuticalsTerminatedDuchenne Muscular DystrophyUnited States, Belgium, United Kingdom, Austria, France, Germany, Italy, Spain, Switzerland
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Lancashire Teaching Hospitals NHS Foundation TrustDuchenne UKRecruiting
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Novo Nordisk A/SEnrolling by invitationHaemophilia BNetherlands, United Kingdom
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Virginia Commonwealth UniversityCSL BehringCompletedHemophilia A | Hemophilia B | Vitamin D DeficiencyUnited States
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PfizerActive, not recruitingHemophilia BUnited States, Canada, Australia, Turkey
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ShireCompletedHunter SyndromeSpain, United States, Canada, United Kingdom, France, Australia, Mexico
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Sarepta Therapeutics, Inc.Hoffmann-La RocheEnrolling by invitationDuchenne Muscular DystrophyUnited States, Spain
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GlaxoSmithKlineCompletedMuscular DystrophiesUnited States
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PTC TherapeuticsGenzyme, a Sanofi CompanyTerminatedDuchenne Muscular DystrophyUnited States
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Institute for Neurodegenerative DisordersCompletedParkinson Disease | Alzheimer Disease | Mild Cognitive Impairment | Fragile X Syndrome | Huntington Disease | Autistic Spectrum DisordersUnited States