Clinical Trials on Creatine Deficiency, X-linked
Total 1558 results
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Cyprium Therapeutics, Inc.Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedMenkes Disease | Occipital Horn Syndrome | Unexplained Copper DeficiencyUnited States
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Baylor College of MedicineCompleted
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Baylor College of MedicineChildren's Hospital of Philadelphia; Children's Hospital Colorado; Seattle Children... and other collaboratorsRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS DeficiencyUnited 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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National Human Genome Research Institute (NHGRI)Active, not recruitingWiskott- Aldrich Syndrome | ADA Deficient SCIDUnited States
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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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International Rett Syndrome FoundationBaylor College of Medicine; Children's Hospital of Philadelphia; University of... and other collaboratorsRecruitingNervous System Diseases | Neurologic Manifestations | Neurobehavioral Manifestations | Genetic Diseases, X-Linked | Intellectual Disability | Neurodevelopmental Disorders | Neurologic Disorder | Rett Syndrome | Genetic Disease | Rett Syndrome, AtypicalUnited States
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Universitair Ziekenhuis BrusselMarguerite - Marie Delacroix FoundationNot yet recruitingClassical Lissencephalies and Subcortical Band HeterotopiasBelgium
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Medialis Ltd.Metabolic Support UKCompletedX-linked Hypophosphatemia (XLH)United Kingdom
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Bambino Gesù Hospital and Research InstituteAutour Des Williams; AceaUnknownFragile X Syndrome | Williams SyndromeItaly
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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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Fred Hutchinson Cancer CenterCompletedMetabolism, Inborn Errors | Granulomatous Disease, ChronicUnited States
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Shanghai Children's Medical CenterUnknown
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National Center for Research Resources (NCRR)University of North CarolinaCompletedHemophilia B | Factor IX Deficiency
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Minoryx Therapeutics, S.L.RecruitingX-linked AdrenoleukodystrophyFrance
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National Human Genome Research Institute (NHGRI)TerminatedCitrullinemia | Argininosuccinic Aciduria | Hyperargininemia | Ornithine Carbamoyltransferase Deficiency | Carbamoyl-Phosphate Synthase I Deficiency | N-Acetylglutamate Synthase DeficiencyUnited States
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Andrea GropmanChildren's National Research InstituteCompletedOrnithine Transcarbamylase Deficiency
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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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China Academy of Chinese Medical SciencesUnknownEfficiency of Genuine Regional and Non-genuine Regional Rhizoma Atractylodis in Treating FD
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Andrea GropmanNational Center for Research Resources (NCRR); Rare Diseases Clinical Research... and other collaboratorsCompletedBrain Diseases, Metabolic, Inborn | Ornithine Transcarbamylase Deficiency | Urea Cycle DisorderUnited States
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Ionis Pharmaceuticals, Inc.RecruitingMethyl CpG Binding Protein 2 (MECP2) Duplication SyndromeUnited States
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National Institute of Allergy and Infectious Diseases...CompletedMycosesUnited States
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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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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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University Hospital, BrestUnknownDysautonomia | Masculinity | MECP2-Related Severe Neonatal EncephalopathyFrance
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National Institute of Allergy and Infectious Diseases...TerminatedChronic Granulomatous DiseaseUnited States
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Shenzhen Geno-Immune Medical InstituteUnknown
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Federal Research Institute of Pediatric Hematology...UnknownChronic Granulomatous DiseaseRussian Federation
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National Institute of Allergy and Infectious Diseases...Terminated
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University Hospital, GrenobleTerminatedChronic Granulomatous DiseaseFrance
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National Institute of Allergy and Infectious Diseases...CompletedChronic Granulomatous DiseaseUnited States
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Rocket Pharmaceuticals Inc.Recruiting
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University of California, San DiegoRocket Pharmaceuticals Inc.Recruiting
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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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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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InnoVec Biotherapeutics Inc.Peking Union Medical College HospitalRecruitingEye Diseases | Retinal Degeneration | Retinal Disease | RetinoschisisChina
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BiogenCompletedChoroideremiaUnited States, Finland, France, Denmark, Netherlands, Germany, United Kingdom, Canada
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Cooperative International Neuromuscular Research...Carolinas Medical Center lead study siteCompletedBecker Muscular Dystrophy | Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency) | Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency) | Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)United States
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Mendel TuchmanChildren's Hospital of Philadelphia; University of California, Los Angeles; Icahn... and other collaboratorsCompletedMethylmalonic Acidemia | Carbamoyl-Phosphate Synthase I Deficiency Disease | Propionic Acidemia, Type I and/or Type II | Ornithine Carbamoyltransferase DeficiencyUnited States
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University of Colorado, DenverCompletedChronic Granulomatous DiseaseUnited States