Clinical Trials on Creatine Deficiency, X-linked
Total 1554 results
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Ultragenyx Pharmaceutical IncTerminatedCreatine Deficiency, X-linkedUnited States, Canada
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IRCCS Fondazione Stella MarisRecruitingCreatine Deficiency, X-linked | Creatine Deficiency Syndrome Due to Gamt DeficiencyItaly
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Hospices Civils de LyonRecruitingCreatine Transporter DefectFrance
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Great Ormond Street Hospital for Children NHS Foundation...UnknownX-linked Severe Combined ImmunodeficiencyUnited Kingdom
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National Institute of Allergy and Infectious Diseases...RecruitingX-linked Severe Combined Immunodeficiency (XSCID)United States
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St. Jude Children's Research HospitalNational Heart, Lung, and Blood Institute (NHLBI); Assisi Foundation; California...SuspendedSevere Combined Immunodeficiency Disease, X-linkedUnited States
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David WilliamsActive, not recruitingGene Therapy | Severe Combined Immunodeficiency, X LinkedUnited States, United Kingdom
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Assistance Publique - Hôpitaux de ParisCompletedX-linked Severe Combined ImmunodeficiencyFrance
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University of MiamiJackson Health SystemNo longer available
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National Institute of Allergy and Infectious Diseases...UnknownX-Linked Combined Immunodeficiency DiseasesUnited States
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National Institute of Allergy and Infectious Diseases...CompletedSevere Combined ImmunodeficiencyUnited States
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National Institute of Allergy and Infectious Diseases...TerminatedGrowth Failure | X-linked Severe Combined Immunodeficiency (XSCID) | Growth Hormone ResistenceUnited States
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Shenzhen Geno-Immune Medical InstituteUnknownSCID, X LinkedChina
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Children's Hospital of Chongqing Medical UniversityRecruiting
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Great Ormond Street Hospital for Children NHS Foundation...RecruitingSevere Combined Immunodeficiency, X-LinkedUnited Kingdom
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The Hospital for Sick ChildrenCompletedX-linked Lymphoproliferative Syndrome Type 2 (XLP-2)Canada
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VegaVect, Inc.National Eye Institute (NEI)Active, not recruiting
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Eunice Kennedy Shriver National Institute of Child...University of WashingtonUnknownWiskott-Aldrich Syndrome | Leukocyte Adhesion Deficiency Syndrome | X-Linked Agammaglobulinemia | X-Linked Hyper IgM 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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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute...CompletedSevere Combined Immunodeficiency | Adenosine Deaminase Deficiency | Immune System Disorder | Autosomal Recessive Disorder | Purine-Nucleoside Phosphorylase Deficiency | Severe Combined Immunodeficiency With Absence of T and B Cells | X-Linked Severe Combined ImmunodeficiencyUnited States
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National Eye Institute (NEI)CompletedRetinoschisis | X-LinkedUnited States
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West China HospitalRecruiting
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IRCCS San RaffaeleFondazione TelethonRecruitingChronic Granulomatous Disease X-linked (X-CGD)Italy
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Bio Products LaboratoryCompletedCommon Variable Immunodeficiency | Primary Immune Deficiency Disorders | Hyper-IgM Syndrome | X-linked AgammaglobulinaemiaUnited States, United Kingdom, Hungary
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Newcastle-upon-Tyne Hospitals NHS TrustRecruitingX-linked Myotubular Myopathy | Centronuclear Myopathy | Myotubular Myopathy | Myotubular Myopathy 1 | Myotubular (Centronuclear) Myopathy | Centronuclear Myopathy, X-LinkedUnited Kingdom
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University of California, Los AngelesNational Heart, Lung, and Blood Institute (NHLBI); Boston Children's Hospital; California Institute for Regenerative Medicine (CIRM) and other collaboratorsRecruitingGranulomatous Disease, Chronic, X-linkedUnited States
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Retina Foundation of the SouthwestDSM Nutritional Products, Inc.; Foundation Fighting BlindnessCompletedRetinitis Pigmentosa | X-linked Genetic DiseasesUnited States
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Bio Products LaboratoryCompletedCommon Variable Immunodeficiency | Primary Immune Deficiency Disorders | X-linked Agammaglobulinaemia | Hyperimmunoglobulin M SyndromeUnited States
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Hubert Serve, Prof., MDUnknown
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Astellas Gene TherapiesCompletedX-linked Myotubular MyopathyUnited States, France, United Kingdom, Canada, Germany
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University of FloridaAudentes TherapeuticsCompletedX-linked Myotubular MyopathyUnited States
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University of South FloridaMerck Sharp & Dohme LLCTerminatedX-linked Agammaglobulinemia | Specific Antibody Deficiency | Common Variable Immune DeficiencyUnited States
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James DowlingCanadian Institutes of Health Research (CIHR); Cures Within Reach; The Joshua... and other collaboratorsRecruitingX Linked Myotubular MyopathyUnited Kingdom, Canada, United States
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Yale UniversityNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)CompletedHypophosphatemic Rickets, X Linked DominantUnited States
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Murdoch Childrens Research InstituteUniversity of Sydney; Sydney Children's Hospitals Network; Women's and Children... and other collaboratorsActive, not recruitingAutosomal Recessive Disorder | X-Linked Genetic DiseasesAustralia
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Astellas Gene TherapiesCompletedMales With X-linked Myotubular Myopathy (XLMTM)United States, United Kingdom
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Fairview University Medical CenterTerminatedImmunologic Deficiency Syndromes | Common Variable Immunodeficiency | Severe Combined Immunodeficiency | Chronic Granulomatous Disease | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | Graft Versus Host Disease | X-linked Agammaglobulinemia | Chediak-Higashi Syndrome | Virus-Associated... and other conditionsUnited States
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GenethonActive, not recruitingX-Linked Chronic Granulomatous DiseaseFrance
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Kyowa Kirin Co., Ltd.Active, not recruiting
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National Eye Institute (NEI)CompletedRetinoschisis | X-LinkedUnited States
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Edimer PharmaceuticalsCompletedX-linked Hypohidrotic Ectodermal DysplasiaUnited States
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GenethonActive, not recruitingX-Linked Chronic Granulomatous DiseaseUnited Kingdom
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Great Ormond Street Hospital for Children NHS Foundation...National Institute for Health Research, United Kingdom; The Freya FoundationRecruitingPyruvate Dehydrogenase Complex Deficiency | Pyruvate Dehydrogenase E1 Alpha Deficiency | Pyruvate Dehydrogenase E1-Beta Deficiency | Pyruvate Dehydrogenase E2 Deficiency | Pyruvate Dehydrogenase Phosphatase DeficiencyUnited Kingdom
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University of California, DavisUniversity of Alberta; St. Justine's HospitalRecruitingNeurobehavioral Manifestations | Genetic Diseases, X-Linked | Intellectual Disability | Fragile X Syndrome | Sex Chromosome Disorders | Fragile X Mental Retardation Syndrome | Trinucleotide Repeat Expansion | Fra(X) Syndrome | FXS | Mental Retardation, X LinkedUnited States, Canada
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Hospices Civils de LyonUnknownX-linked Mental RetardationFrance
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Mitsubishi Tanabe Pharma America Inc.RecruitingErythropoietic Protoporphyria (EPP) | X-Linked Protoporphyria (XLP)United States, Spain, France, United Kingdom, Japan, Italy, Australia, Bulgaria, Czechia, Netherlands, Poland
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Edimer PharmaceuticalsCompletedX Linked Hypohidrotic Ectodermal DysplasiaUnited States
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Cure CMDUniversity of Michigan; Congenital Muscle Disease International RegistrCompleted
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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS); Rare Diseases...RecruitingPyruvate Dehydrogenase Complex Deficiency DiseaseUnited States
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Rare Thyroid Therapeutics International ABVitaccess LtdCompletedAllan-Herndon-Dudley Syndrome | Monocarboxylate Transporter 8 (MCT8) DeficiencyUnited Kingdom