Clinical Trials on X-linked Creatine Deficiency
Total 1553 results
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University Hospital, Basel, SwitzerlandNovartisCompletedRett's SyndromeSwitzerland
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Stanford UniversityCompletedIntellectual Disability | Fragile X SyndromeUnited States
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Parc de Salut MarHospital Infantil Universitario Niño Jesús, Madrid, Spain; Hospital Universitario... and other collaboratorsCompletedDown Syndrome | Fragile X SyndromeSpain
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Jazz PharmaceuticalsGW PharmaceuticalsTerminatedRett Syndrome | RTTUnited States, Canada, Italy, Spain, United Kingdom, Australia
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Children's Hospital of Fudan UniversityRecruiting
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Taysha Gene Therapies, Inc.RecruitingRett SyndromeUnited States, United Kingdom
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Taysha Gene Therapies, Inc.RecruitingRett SyndromeUnited States, Canada
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Georgetown UniversityInternational Rett Syndrome Foundation Rettsyndrome.orgRecruiting
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Rett Syndrome Research TrustBaylor College of Medicine; Children's Hospital of Philadelphia; Boston Children... and other collaboratorsRecruiting
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Pitié-Salpêtrière HospitalRecruitingLaminopathies | EmerinopathiesFrance
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ACADIA Pharmaceuticals Inc.CompletedRett SyndromeUnited States
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Edison Pharmaceuticals IncWithdrawn
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Anavex Life Sciences Corp.Completed
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University of South FloridaEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompleted
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Neuren Pharmaceuticals Limitedrettsyndrome.orgCompleted
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Telethon Kids InstituteRigshospitalet, Denmark; University of Haifa; Ariel UniversityUnknownRett SyndromeDenmark, Israel
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Rigshospitalet, DenmarkCompleted
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Hugo W. Moser Research Institute at Kennedy Krieger...The Johns Hopkins Institute for Clinical and Translational Research (ICTR)CompletedRett SyndromeUnited States
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Edison Pharmaceuticals IncCompleted
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Boston Children's HospitalInternational Rett Syndrome FoundationCompleted
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The Cleveland ClinicTerminated
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Boston Children's HospitalAutism Speaks; International Rett Syndrome FoundationCompleted
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National Taiwan University HospitalUnknown
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Maastricht University Medical CenterCompleted
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Eunice Kennedy Shriver National Institute of Child...Baylor College of MedicineCompletedRett Syndrome
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Eunice Kennedy Shriver National Institute of Child...Baylor College of MedicineCompleted
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Novartis PharmaceuticalsRecruitingNLRC4-GOF, AIFEC (Autoinflammation With Infantile Enterocolitis), XIAP Deficiency, CDC42 MutationsSpain, France, United States, Canada, Japan, United Kingdom, Turkey, Czechia, Italy
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Ovid Therapeutics Inc.Completed
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bluebird bioTerminatedCerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)United States, United Kingdom, Argentina, Canada, Germany, Italy, Netherlands
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University Hospital, ToursCompletedHealthy Volunteers | Autism | Fragile-X Syndrome (FXS)France
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Hospices Civils de LyonNot yet recruitingX-Linked Hypophosphatemic Rickets
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University of PennsylvaniaTerminatedOrnithine Transcarbamylase Deficiency Disease
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Children's Hospital of PhiladelphiaNational Institute of Neurological Disorders and Stroke (NINDS); Vanderbilt...RecruitingRett Syndrome | Rett Syndrome, Atypical | RTTUnited States
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bluebird bioActive, not recruitingCerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)United States, Argentina, Australia, Brazil, France, Germany, Netherlands, United Kingdom, Italy
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Eunice Kennedy Shriver National Institute of Child...Children's National Research InstituteTerminatedPhase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase DeficiencyOrnithine Transcarbamylase Deficiency Disease
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Center for Rare Neurological Diseases, Norcross...Ultragenyx Pharmaceutical Inc; Rett Syndrome Research TrustUnknownRett SyndromeUnited States
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Sunshine Care FoundationMassachusetts General Hospital; Jose R. Reyes Memorial Medical CenterCompletedX-Linked Dystonia ParkinsonismPhilippines
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Kyowa Kirin Pharmaceutical Development LtdCompletedX-Linked HypophosphataemiaUnited Kingdom
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CSL BehringCompletedCommon Variable Immunodeficiency | X-linked Agammaglobulinemia | Autosomal Recessive AgammaglobulinemiaGermany, United Kingdom, France, Italy, Poland, Romania, Spain, Sweden, Switzerland
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Children's Hospital ColoradoUniversity of Colorado, Denver; University of Pennsylvania; University of Alabama... and other collaboratorsRecruiting
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National Taiwan University HospitalCompletedNeurologic Disorder | Rett Syndrome
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Jazz PharmaceuticalsTerminatedRett Syndrome | RTTUnited States, Spain, Italy, United Kingdom
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Universitaire Ziekenhuizen KU LeuvenKU LeuvenCompletedCommunication | Parent-Child Relations | Behavior, ChildBelgium
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Ultragenyx Pharmaceutical IncActive, not recruitingOrnithine Transcarbamylase (OTC) DeficiencyCanada, United States, France, Spain, United Kingdom
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Ultragenyx Pharmaceutical IncRecruitingOTC DeficiencyUnited States, Portugal, Australia, Italy, Spain, United Kingdom, Japan, Canada, Germany, France, Argentina, Netherlands, Brazil
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University College, LondonRecruitingOrnithine Transcarbamylase DeficiencyUnited Kingdom
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Arcturus Therapeutics, Inc.Active, not recruitingOrnithine Transcarbamylase DeficiencyUnited States
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The Hospital for Sick ChildrenCanadian Hemophilia SocietyCompletedHemophilia A | Hemophilia B | Factor IX Deficiency | Factor VIII DeficiencyCanada
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Virginia Commonwealth UniversityCSL BehringCompletedHemophilia A | Hemophilia B | Vitamin D DeficiencyUnited States
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Children's Hospital Medical Center, CincinnatiNot yet recruitingAutism Spectrum Disorder | Fragile X Syndrome