Gaucher Disease Clinical Trials

A listing of Gaucher Disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

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Found 14 clinical trials
A Long-term Follow-up Study of Gaucher Disease

The study aims are to: a) identify the long-term natural history of Gaucher disease, b) evaluate long-term treatment efficacy of enzyme replacement therapy (ERT) and substrate reduction therapy

substrate reduction therapy
replacement therapy
  • 26 views
  • 27 Oct, 2022
  • 1 location
A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen (OPPUS)

Primary Objective: To assess prevalence of Gaucher disease (GD) diagnosed in pediatric patients presenting with unexplained splenomegaly (SMG) after exclusion of first intention-diagnoses

  • 0 views
  • 04 Oct, 2022
  • 40 locations
A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen (OPPUS)

Primary Objective: To assess prevalence of Gaucher disease (GD) diagnosed in pediatric patients presenting with unexplained splenomegaly (SMG) after exclusion of first intention-diagnoses

  • 0 views
  • 04 Oct, 2022
  • 40 locations
Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders

treatment. Patients of any age with Tay-Sachs disease, Sandhoff disease, GM1 gangliosidosis, or type 2 Gaucher disease may be eligible for this study. Participants will be admitted to the NIH

MRI
deficiency
  • 76 views
  • 25 Oct, 2022
  • 1 location