Clinical Trials on X-Linked Combined Immunodeficiency Diseases
Total 4409 results
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Bambino Gesù Hospital and Research InstituteAutour Des Williams; AceaUnknownFragile X Syndrome | Williams SyndromeItaly
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Minoryx Therapeutics, S.L.RecruitingX-linked AdrenoleukodystrophyFrance
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Leipzig University Medical CenterRecruitingX-linked AdrenoleukodystrophyGermany
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Leipzig University Medical CenterEuropean Leukodystrophy AssociationCompletedX-linked AdrenoleukodystrophyGermany
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Fondazione TelethonOspedale San RaffaeleActive, not recruitingWiskott-Aldrich SyndromeItaly, United States
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David WilliamsActive, not recruitingWiskott-Aldrich SyndromeUnited States
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GenethonInstitute of Child Health; Great Ormond Street Hospital for Children NHS Foundation...Completed
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GlaxoSmithKlineCompletedWiskott-Aldrich SyndromeUnited States
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GenethonHôpital Necker-Enfants MaladesCompleted
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Ionis Pharmaceuticals, Inc.RecruitingMethyl CpG Binding Protein 2 (MECP2) Duplication SyndromeUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingAutism Spectrum Disorder | Fragile X Syndrome | Developmental Delay | Genetic Disorder | Asperger DisorderUnited States
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Rare Thyroid Therapeutics International ABEgetis Therapeutics; AnovoRxAvailableMonocarboxylate Transporter 8 Deficiency | Allan-Herndon-Dudley SyndromeUnited States
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Rare Thyroid Therapeutics International ABPremier Research Group plc; Egetis TherapeuticsRecruitingMonocarboxylate Transporter 8 Deficiency | Allan-Herndon-Dudley SyndromeUnited States, United Kingdom, Netherlands
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Universitair Ziekenhuis BrusselMarguerite - Marie Delacroix FoundationNot yet recruitingClassical Lissencephalies and Subcortical Band HeterotopiasBelgium
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University Hospital ErlangenPervormance GmbHCompletedShort Term Effects and Risks of Physical Exercise in Subjects With Hypohidrotic Ectodermal DysplasiaX-Linked Hypohidrotic Ectodermal Dysplasia | Exercise-induced Rise of Body TemperatureGermany
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Rocket Pharmaceuticals Inc.Recruiting
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University of California, San DiegoRocket Pharmaceuticals Inc.Recruiting
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GenethonActive, not recruitingWiskott-Aldrich SyndromeUnited Kingdom, France
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Capital Research Institute of PediatricsUnknownWiskott-Aldrich Syndrome
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St. Jude Children's Research HospitalCompletedWiskott-Aldrich SyndromeUnited States
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University of Alabama at BirminghamUniversity of Colorado, Denver; Eunice Kennedy Shriver National Institute of... and other collaboratorsCompletedRett Syndrome, Preserved Speech Variant | Mecp2 Duplication Syndrome | Rett-related DisordersUnited States
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InnoVec Biotherapeutics Inc.Peking Union Medical College HospitalRecruitingEye Diseases | Retinal Degeneration | Retinal Disease | RetinoschisisChina
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BiogenCompletedChoroideremiaUnited States, Germany, France
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Duke UniversityFoundation Fighting BlindnessWithdrawnChoroideremiaUnited States
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University of AlbertaCanadian Institutes of Health Research (CIHR); Imperial College London; University... and other collaboratorsCompleted
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University of AlbertaTerminated
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National Institute of Allergy and Infectious Diseases...Rare Diseases Clinical Research Network; Primary Immune Deficiency Treatment...CompletedWiskott-Aldrich SyndromeUnited States, Canada
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The Korean Society of Pediatric Hematology OncologyCompletedWiskott-Aldrich SyndromeKorea, Republic of
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TakedaRecruitingSpinal and Bulbar Muscular AtrophyJapan
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Novartis PharmaceuticalsCompletedSpinal and Bulbar Muscular AtrophyUnited States, Germany, Italy, Denmark
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University of California, DavisEunice Kennedy Shriver National Institute of Child Health and Human Development...Enrolling by invitationIntellectual Disability | Down Syndrome | Attention Deficit Hyperactivity Disorder | Fragile X SyndromeUnited States
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Rush University Medical CenterEmory UniversityEnrolling by invitationTuberous Sclerosis | Fragile X Syndrome | Rett Syndrome | Hamartoma Syndrome, Multiple | Angelman Syndrome | Creatine Deficiency, X-linked | Telomeric 22Q13 Monosomy Syndrome | Chromosome 15Q, Partial DeletionUnited States
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4D Molecular TherapeuticsActive, not recruitingChoroideremiaUnited States
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4D Molecular TherapeuticsTerminated
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University of OxfordUniversity College, London; Moorfields Eye Hospital NHS Foundation TrustCompleted
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STZ eyetrialUniversity Hospital TuebingenCompleted
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National Institute of Allergy and Infectious Diseases...RecruitingChronic Granulomatous Disease (CGD) | X-Linked Chronic Granulomatous DiseaseUnited States
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Fondazione TelethonOspedale San RaffaeleCompleted
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Federal Research Institute of Pediatric Hematology...UnknownHematopoietic Stem Cell Transplantation | Wiskott-Aldrich Syndrome | Graft FailureRussian Federation
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University Hospital, BrestUnknownDysautonomia | Masculinity | MECP2-Related Severe Neonatal EncephalopathyFrance
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Rocket Pharmaceuticals Inc.Completed
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BiogenCompletedChoroideremiaUnited States, Finland, France, Denmark, Netherlands, Germany, United Kingdom, Canada
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AnnJi Pharmaceutical Co., Ltd.Active, not recruitingSpinal and Bulbar Muscular Atrophy | Kennedy's DiseaseUnited States
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Rocket Pharmaceuticals Inc.Active, not recruitingDanon DiseaseUnited States
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Gianni SoraruMario Negri Institute for Pharmacological ResearchRecruitingSpinal and Bulbar Muscular AtrophyItaly
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Assistance Publique - Hôpitaux de ParisCompleted
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Ramathibodi HospitalAstraZenecaCompletedSpinobulbar Muscular Atrophy | Kennedy's DiseaseThailand
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National Institute of Allergy and Infectious Diseases...CompletedGranulomatous Disease, Chronic | Infection | Job's SyndromeUnited States