Clinical Trials on X-linked Severe Combined Immunodeficiency
Total 1631 results
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Green Cross CorporationCompleted
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Lundquist Institute for Biomedical Innovation at...Active, not recruitingMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VIUnited States
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ShireCompleted
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Green Cross CorporationUnknownThe Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) PatientsHunter SyndromeKorea, Republic of
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SwanBio Therapeutics, Inc.RecruitingAMN | AMN Gene Mutation | X-ALDNetherlands, United States
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Eunice Kennedy Shriver National Institute of Child...TerminatedFragile X Syndrome | Premature Ovarian Failure | Premature Menopause | FMR1 Premutation | Primary Ovairan InsufficiencyUnited States
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Janssen Research & Development, LLCActive, not recruitingX-Linked Retinitis PigmentosaUnited States, United Kingdom
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Duke UniversityRecruiting
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National Institute of Arthritis and Musculoskeletal...CompletedIchthyosis | Ichthyosis, X-Linked | Ichthyosis, Lamellar | Darier Disease | Hailey-Hailey Disease | Hyperkeratosis, Epidermolytic | KeratodermaUnited States
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ShireCompletedHunter SyndromeUnited States, United Kingdom
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National Taiwan University HospitalUnknownRett Syndrome | Tourette Syndrome | Tic DisordersTaiwan
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University Hospital, MontpellierAgence de La BiomédecineActive, not recruitingFragile X Syndrome | Huntington Disease | Myotonic Dystrophy 1France
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Brazil
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Kyowa Kirin Co., Ltd.Active, not recruitingX-linked Hypophosphatemia (XLH)China
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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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ShireCompleted
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ShireCompletedHunter Syndrome | Mucopolysaccharidosis (MPS)United States, Argentina, Mexico, Spain, United Kingdom
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National Institute of Neurological Disorders and...CompletedMotor Neuron DiseaseUnited States
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Psyadon PharmaCompletedLesch-Nyhan DiseaseUnited States
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IpsenRecruitingModerate to Severe Upper Facial LinesGermany, France
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University of OxfordUniversity College, London; University of Southampton; Moorfields Eye Hospital... and other collaboratorsCompleted
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University of British ColumbiaVancouver General HospitalWithdrawn
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TakedaTakeda Development Center Americas, Inc.RecruitingHunter Syndrome | Mucopolysaccharidosis (MPS)United States
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National Institute of Neurological Disorders and...RecruitingMotor Neuron Disease | Spinal and Bulbar Muscular Atrophy | Kennedys DiseaseUnited States
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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-Linked HypophosphatemiaKorea, Republic of, Canada, United States, Australia, Japan, United Kingdom, Sweden
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ShireCompletedHunter SyndromeUnited Kingdom, United States, Brazil
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NightstaRx Ltd, a Biogen CompanyCompletedX-Linked Retinitis PigmentosaUnited States, France, United Kingdom, Finland, Netherlands, Germany, Canada
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AB2 Bio Ltd.CompletedNLRC4-MAS | XIAP DeficiencyUnited States, Canada, Germany
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AB2 Bio Ltd.Active, not recruitingNLRC4-MAS | XIAP DeficiencyUnited States, Canada, Germany
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Medical University of GrazCompletedBipolar Affective DisorderAustria
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Canada
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Kyowa Kirin Co., Ltd.Active, not recruitingX-linked Hypophosphatemia (XLH)China
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Lundquist Institute for Biomedical Innovation at...TerminatedMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VIUnited States
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Kiora Pharmaceuticals, Inc.RecruitingRetinitis Pigmentosa | ChoroideremiaAustralia
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Center for Eye Research AustraliaAustralian National University; Commonwealth Scientific and Industrial Research... and other collaboratorsEnrolling by invitation
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MeiraGTx UK II LtdSyne Qua Non Limited; Bionical EmasCompletedGene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)X-Linked Retinitis PigmentosaUnited Kingdom, United States
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Mobius Medical Pty Ltd.University of Melbourne; Australian National University; Centre for Eye Research... and other collaboratorsCompleted
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States
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Jirair Krikor BedoyanUltragenyx Pharmaceutical IncRecruitingPyruvate Dehydrogenase Complex DeficiencyUnited States
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Cyprium Therapeutics, Inc.Enrolling by invitation
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University of FloridaEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsActive, not recruitingPyruvate Dehydrogenase Complex DeficiencyUnited States
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Fondazione TelethonCompletedPyruvate Dehydrogenase Complex DeficiencyItaly
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Bicetre HospitalCompletedX Linked Hypophosphatemic Rickets
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Baylor College of MedicineUniversity of California, San Diego; University of Alabama at Birmingham; Boston... and other collaboratorsCompletedPrader-Willi Syndrome | Rett Syndrome | Angelman Syndrome | Sleep ProblemsUnited States
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Eunice Kennedy Shriver National Institute of Child...Completed
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Center for Eye Research AustraliaCompleted
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ShireCovance; PRA Health Sciences; PharmaNetCompletedMucopolysaccharidosis II | Hunter Syndrome | MPS IIBrazil, Poland, Taiwan
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University of California, San DiegoBioMarin PharmaceuticalWithdrawnBehavioral Manifestations of Lesch-Nyhan DiseaseUnited States