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Niemann-Pick Diseases

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Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

Recruiting
  • Niemann-Pick Disease, Type C
    • Bethesda, Maryland
      National Institutes of Health Clinical Center
    Feb 2, 2023

    Asmd, Visceral Type Trial in Amsterdam (MR Spectroscopy (MRS), MR Elastography (MRE))

    Recruiting
    • Asmd, Visceral Type
    • MR Spectroscopy (MRS)
    • MR Elastography (MRE)
    • Amsterdam, Noord-Holland, Netherlands
      Amsterdam UMC
    Jun 6, 2023

    PREDIGA 2: Spanish Acronym of "Educational and Diagnostic

    Not yet recruiting
    • Gaucher Disease
    • +4 more
    • Patient with splenomegaly or splenectomy
    • Barcelona, Spain
      Hospital Vall d'Hebron
    Dec 13, 2022

    GM2 Gangliosidosis, Niemann-Pick Disease, Type C Trial in Curitiba, Porto Alegre, Rio De Janeiro (AZ-3102 (Dose 1), Placebo,

    Not yet recruiting
    • GM2 Gangliosidosis
    • Niemann-Pick Disease, Type C
    • AZ-3102 (Dose 1)
    • +2 more
    • Curitiba, Brazil
    • +2 more
    Feb 24, 2023

    Functional Near-Infrared Spectroscopy Combined With Diffuse

    Recruiting
    • Juvenile Neuronal Ceroid Lipofuscinosis
    • +3 more
      • Bethesda, Maryland
        National Institutes of Health Clinical Center
      Jan 30, 2023

      Sphingomyelin Lipidosis Trial (olipudase alfa (GZ402665))

      Approved for marketing
      • Sphingomyelin Lipidosis
      • olipudase alfa (GZ402665)
      • (no location specified)
      Sep 15, 2022

      Niemann-Pick Disease, Type C1 Trial in Worldwide (Hydroxypropyl-beta-cyclodextrin, Placebo)

      Recruiting
      • Niemann-Pick Disease, Type C1
      • Oakland, California
      • +13 more
      Jan 9, 2023

      Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II Trial in Durham (DUOC-01)

      Recruiting
      • Adrenoleukodystrophy
      • +11 more
      • DUOC-01
      • Durham, North Carolina
        Duke University Medical Center
      Oct 24, 2022

      Adult and Pediatric Acid Sphingomyelinase Deficiency on Early

      Recruiting
      • Acid Sphingomyelinase Deficiency (ASMD)
      • Olipudase alfa
      • France, France
        Investigational site number France
      Jun 13, 2022

      Acid Sphingomyelinase Deficiency Disease and Gaucher Disease in

      Recruiting
      • Gaucher Disease
      • Acid SphingoMyelinase Deficiency
        • Ponferrada, León, Spain
        • +11 more
        Aug 8, 2023

        Niemann-Pick Type C Disease Trial in United States (Adrabetadex)

        Completed
        • Niemann-Pick Type C Disease
        • Little Rock, Arkansas
        • +16 more
        Oct 7, 2022

        Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy Trial in Denver, Valhalla, Salt Lake City (Human

        Completed
        • Mucopolysaccharidosis I
        • +15 more
        • Human Placental Derived Stem Cell
        • Denver, Colorado
        • +2 more
        Oct 24, 2022

        Niemann-Pick Disease, Type C Trial in United States (Arimoclomol)

        Available
        • Niemann-Pick Disease, Type C
        • Birmingham, Alabama
        • +14 more
        Aug 18, 2022

        Niemann-Pick Disease, Type C1 Trial run by the (VTS-270)

        Completed
        • Niemann-Pick Disease, Type C1
        • Bethesda, Maryland
          National Institutes of Health Clinical Center, 9000 Rockville Pi
        Jul 19, 2021

        The Natural History of Metachromatic Leukodystrophy

        Recruiting
        • MLD
        • +26 more
        • Palliative Care
        • Hematopoetic Stem Cell Transplantation
        • Pittsburgh, Pennsylvania
          The Program for the Study of Neurodevelopment in Rare Disorders,
        Nov 2, 2021

        Complement Activation in the Lysosomal Storage Disorders

        Withdrawn
        • Fabry Disease
        • +3 more
        • Complement measurements
        • Melbourne, Victoria, Australia
          The Royal Melbourne Hospital
        Jun 2, 2021

        Biomarker for Niemann Pick Type C Disease (BioNPC)

        Active, not recruiting
        • Niemann-Pick Disease
        • Niemann-Pick Disease, Type C
          • Rostock, Germany
          • +3 more
          May 12, 2021

          Sphingomyelin Lipidosis Trial in Worldwide (Olipudase alfa)

          Completed
          Has Results
          • Sphingomyelin Lipidosis
          • Olipudase alfa
          • New York, New York
          • +5 more
          Mar 15, 2022

          Niemann-Pick Disease, Type C1 Trial in Oakland (Hydroxypropyl-ß-cyclodextrin)

          Active, not recruiting
          • Niemann-Pick Disease, Type C1
          • Oakland, California
            UCSF Benioff Children's Hospital Oakland
          Jun 16, 2021

          ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn

          Enrolling by invitation
          • Acid Sphingomyelinase Deficiency
          • +13 more
          • Confirmatory Testing
          • Bronx, New York
          • +9 more
          May 9, 2022

          Health Insurance Literacy and Challenges in Accessing Health

          Completed
          • Niemann-Pick Diseases
            • New York, New York
              Icahn School of Medicine at Mount Sinai
            Oct 5, 2020

            Pediatric Patients With Unexplained Enlarged Spleen

            Recruiting
            • Gaucher Disease, Splenomegaly
            • Acid SphingoMyelinase Deficiency
              • Amiens, France
              • +39 more
              Aug 19, 2022

              Niemann-Pick Disease, Type C1 Trial in Oakland, Morristown (Hydroxypropyl-beta-cyclodextrin)

              Completed
              • Niemann-Pick Disease, Type C1
              • Oakland, California
              • +1 more
              Feb 17, 2021

              Niemann-Pick Disease, Type C Trial in Worldwide (Parts A/B: Adrabetadex, Parts A/B: Sham Control)

              Active, not recruiting
              • Niemann-Pick Disease, Type C
              • Parts A/B: Adrabetadex
              • Parts A/B: Sham Control
              • Birmingham, Alabama
              • +28 more
              Jul 23, 2021

              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