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sphingolipidoses

Showing 1 - 20 of 20
STUDY
CONDITIONS
INTERVENTIONS
LOCATIONS
LAST UPDATED

Sphingolipidoses, Enzyme Deficiency Trial in San Francisco (no intervention)

Recruiting
  • Sphingolipidoses
  • Enzyme Deficiency
  • no intervention
  • San Francisco, California
    University of California San Francisco
Jul 1, 2022

Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome Trial in Minneapolis (Stem Cell Transplantation, IMD

Recruiting
  • Mucopolysaccharidosis Disorders
  • +28 more
  • Stem Cell Transplantation
  • +7 more
  • Minneapolis, Minnesota
    Masonic Cancer Center, University of Minnesota
Nov 1, 2022

Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease

Completed
  • Lysosomal Storage Diseases
  • +2 more
    • Tirana, Albania
    • +7 more
    May 27, 2021

    Children With Metachromatic Leukodystrophy

    Terminated
    • Lipid Metabolism Disorders
    • +13 more
      • Torrance, California
      • +13 more
      Mar 16, 2021

      Fabry Disease Trial in Melbourne, Perth, Taipei (4D-310)

      Active, not recruiting
      • Fabry Disease
      • 4D-310
      • Melbourne, Australia
      • +2 more
      Jan 30, 2023

      Krabbe Disease Trial in Ann Arbor (FBX-101)

      Recruiting
      • Krabbe Disease
      • FBX-101
      • Ann Arbor, Michigan
        University of Michigan Hospitals - Michigan Medicine
      Feb 13, 2023

      Fabry Disease Trial in United States (4D-310)

      Recruiting
      • Fabry Disease
      • 4D-310
      • Birmingham, Alabama
      • +5 more
      Mar 24, 2022

      Ultra-rare Inherited Metabolic and Degenerative Neurological

      Recruiting
      • Inherited Disease
      • +5 more
      • collection of data
      • Milano, Italy
        Fondazione IRCCS Istituto Neurologico Carlo Besta
      May 6, 2021

      Adrenoleukodystrophy Trial in Baltimore (glyceryl trierucate/glyceryl trioleate)

      Terminated
      Has Results
      • Adrenoleukodystrophy
      • glyceryl trierucate/glyceryl trioleate
      • Baltimore, Maryland
      • +1 more
      Oct 22, 2020

      Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome Trial in Minneapolis (Campath-1H, Cyclophosphamide, Busulfan)

      Completed
      Has Results
      • Mucopolysaccharidosis
      • +12 more
      • Minneapolis, Minnesota
        Masonic Cancer Center, University of Minnesota
      Jan 9, 2018

      Farber Disease Natural History Study

      Completed
      • Farber Disease
      • +6 more
        • Oakland, California
        • +14 more
        Jan 14, 2020

        Hurler's Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome Trial in Minneapolis (Stem Cell Transplantation, Cyclophosphamide,

        Terminated
        Has Results
        • Hurler's Syndrome
        • +10 more
        • Stem Cell Transplantation
        • +3 more
        • Minneapolis, Minnesota
          University of Minnesota, Fairview
        Dec 3, 2017

        Infantile Refsum's Disease, Zellweger Syndrome, Bifunctional Enzyme Deficiency Trial (chenodeoxycholic acid, cholic acid,

        Terminated
        • Infantile Refsum's Disease
        • +3 more
        • chenodeoxycholic acid
        • +2 more
        • (no location specified)
        Mar 24, 2015

        Adrenoleukodystrophy Trial (glyceryl trierucate, glyceryl trioleate, interferon beta)

        Completed
        • Adrenoleukodystrophy
        • glyceryl trierucate
        • +3 more
        • (no location specified)
        Mar 24, 2015

        Gaucher's Disease, Osteopenia Trial in Cincinnati, Dayton, Jerusalem (alendronate sodium, calcium carbonate, cholecalciferol)

        Completed
        • Gaucher's Disease
        • Osteopenia
        • alendronate sodium
        • +2 more
        • Cincinnati, Ohio
        • +2 more
        Mar 24, 2015

        Blood Cell Lines From Patients With Gaucher Disease

        Completed
        • Gaucher Disease
        • Blood sample
        • San Francisco, California
        • +6 more
        Aug 17, 2010

        Gaucher's Disease Trial in Pittsburgh (glucocerebrosidase)

        Unknown status
        • Gaucher's Disease
        • Pittsburgh, Pennsylvania
          University of Pittsburgh
        Jun 23, 2005

        Gaucher's Disease Trial in Pittsburgh (human glucocerebrosidase gene into autologous peripheral blood stem cells)

        Completed
        • Gaucher's Disease
        • human glucocerebrosidase gene into autologous peripheral blood stem cells
        • Pittsburgh, Pennsylvania
          University of Pittsburgh
        Jun 23, 2005

        Pulmonary Complications in Pediatric Storage Disorders

        Unknown status
        • I Cell Disease
        • +11 more
          • Minneapolis, Minnesota
            Fairview University Medical Center
          Jun 23, 2005

          Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia Trial in Los Angeles (Stem Cell Transplantation)

          Completed
          • Thrombocytopenia
          • +5 more
          • Stem Cell Transplantation
          • Los Angeles, California
            University of California Los Angeles Medical Center
          Jun 23, 2005