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Niemann-Pick Disease, Type C

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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

    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

    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

    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

      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

      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

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

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

      Sphingomyelin Lipidosis Trial (olipudase alfa (GZ402665))

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

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

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

      Acid Sphingomyelinase Deficiency Disease and Gaucher Disease in

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

        Adult and Pediatric Acid Sphingomyelinase Deficiency on Early

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

        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

        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

        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 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

        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

          Complement Activation in the Lysosomal Storage Disorders

          Withdrawn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

          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, Morristown (Hydroxypropyl-beta-cyclodextrin)

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

          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

          Niemann-Pick Disease, Type C Trial in Worldwide (arimoclomol, Placebo)

          Active, not recruiting
          • Niemann-Pick Disease, Type C
          • Oakland, California
          • +13 more
          Apr 14, 2021

          Pediatric Patients With Unexplained Enlarged Spleen

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

            Niemann-Pick Disease, Type C Trial (VTS-270)

            Withdrawn
            • Niemann-Pick Disease, Type C
            • (no location specified)
            Jul 20, 2020