Clinical Trials on Cerebrotendinous Xanthomatosis in United Kingdom

Total 9 results

  • Travere Therapeutics, Inc.
    Unknown
    Cerebrotendinous Xanthomatosis (CTX) Prevalence Study
    Cerebrotendinous Xanthomatosis (CTX)
    United States
  • US Department of Veterans Affairs
    Withdrawn
    Cholestanol in Humans
    Cerebrotendinous Xanthomatosis
    United States
  • Mirum Pharmaceuticals, Inc.
    Completed
    Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)
    CTX
    United States, Brazil
  • National Center for Research Resources (NCRR)
    Oregon Health and Science University
    Unknown
    Phase II Study of Cholesterol- and Cholestanol-Free Diet, Lovastatin, and Chenodeoxycholic Acid for Cerebrotendinous Xanthomatosis
    Cerebrotendinous Xanthomatosis
    United States
  • Albert Einstein College of Medicine
    Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators
    Enrolling by invitation
    ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program (ScreenPlus)
    Mucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditions
    United States
  • Children's Hospital of Philadelphia
    Illumina, Inc.
    Active, not recruiting
    LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
    Mucopolysaccharidoses | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2 Gangliosidosis | Zellweger... and other conditions
    United States
  • Children's Hospital of Philadelphia
    Eli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaborators
    Recruiting
    The Myelin Disorders Biorepository Project (MDBP)
    Mucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... and other conditions
    United States
  • The Hospital for Sick Children
    Active, not recruiting
    International Rare Histiocytic Disorders Registry (IRHDR) (IRHDR)
    Rare Histiocytic Disorders (RHDs) | Juvenile Xanthogranuloma (JXG) | Reticulohistiocytoma (Epithelioid Histiocytoma) | Xanthoma Disseminatum (XD) | Multicentric Reticulohistiocytosis (MRH) | Systemic Juvenile Xanthogranuloma | Erdheim-Chester Disease (ECD) | Multi-system Rosai-Dorfman Disease (RDD)
    Canada, United States, Poland, Argentina, Austria, Czechia, Germany, Italy, Russian Federation, Spain
  • RTI International
    Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators
    Enrolling by invitation
    Early Check: Expanded Screening in Newborns
    Primary 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 conditions
    United States

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