lysosomal storage diseases

Showing 1 - 16 of 16

STUDY
CONDITIONS
INTERVENTIONS
LOCATIONS

Pompe Disease, Muscle Loss, Obesity Trial (Multi-ingredient supplement (PDT-MIS), Placebo (PLA))

Not yet recruiting
  • Pompe Disease
  • +7 more
  • Multi-ingredient supplement (PDT-MIS)
  • Placebo (PLA)
  • (no location specified)
Nov 8, 2023

Lysosomal Storage Diseases, Metachromatic Leukodystrophy Trial in Milan (OTL-200)

Recruiting
  • Lysosomal Storage Diseases
  • Metachromatic Leukodystrophy
  • OTL-200
  • Milan, Italy
    Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR
Nov 16, 2022

Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2 Trial in Worldwide (SPK-3006)

Active, not recruiting
  • Pompe Disease
  • +6 more
  • SPK-3006
  • Phoenix, Arizona
  • +28 more
Jul 22, 2022

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

Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2 Trial in Worldwide (Neutralizing Antibody to SPK-3006

Completed
  • Pompe Disease
  • +5 more
  • Neutralizing Antibody to SPK-3006 capsid
  • Phoenix, Arizona
  • +17 more
Jul 14, 2021

Fabry Disease, Fabry Disease, Cardiac Variant, Lysosomal Storage Diseases Trial in Wuerzburg (blood sampling

Enrolling by invitation
  • Fabry Disease
  • +4 more
  • blood sampling (alpha-Galactosidase & LysoGb3)
  • Wuerzburg, Bavaria, Germany
    University Hospital Wuerzburg
Jul 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Withdrawn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

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

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

    Pompe Disease, Glycogen Storage Disease Type II, Lysosomal Storage Diseases Trial in Gainesville (Diet and Exercise)

    Completed
    • Pompe Disease
    • +2 more
    • Diet and Exercise
    • Gainesville, Florida
      University of Florida Clinical Research Center
    May 13, 2021

    Children With Metachromatic Leukodystrophy

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

      Fabry Disease, Lysosomal Storage Diseases Trial in Worldwide (FLT190)

      Unknown status
      • Fabry Disease
      • Lysosomal Storage Diseases
      • FLT190
      • Berlin, Germany
      • +4 more
      Feb 2, 2021

      Fabry Disease, Lysosomal Storage Diseases Trial in London (FLT190)

      Recruiting
      • Fabry Disease
      • Lysosomal Storage Diseases
      • FLT190
      • London, United Kingdom
        Royal Free London
      Sep 18, 2020

      Lysosomal Storage Disorders Diseases in Minority Groups

      Unknown status
      • Lysosomal Storage Diseases
      • Enzyme assay and molecular sequencing
      • Fairfax, Virginia
        LDRTC
      Jan 18, 2019

      Cellular Pharmacodynamics of Small Molecules in Lysosomal

      Unknown status
      • Lysosomal Storage Diseases
        • Fairfax, Virginia
          LDRTC
        Jan 18, 2019

        Lysosomal Storage Diseases, Inborn Errors of Metabolism Trial in Durham (ALD-601)

        Withdrawn
        • Lysosomal Storage Diseases
        • Inborn Errors of Metabolism
        • ALD-601
        • Durham, North Carolina
        • +1 more
        Jun 27, 2017

        Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression Trial in Torrance, Helsinki (laronidase)

        Terminated
        • Mucopolysaccharidosis I
        • +2 more
        • Torrance, California
        • +1 more
        Feb 20, 2013