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 4 clinical trials
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

PRV-GD2-101 is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose PR001 in infants diagnosed with Type 2 Gaucher disease (GD2). For each patient, the

type 1 gaucher disease
  • 0 views
  • 19 Jul, 2021
  • 5 locations
Gaucherite - A Study to Stratify Gaucher Disease

The purpose of this research is to review data already collected and to collect new data from adults and children in England with Gaucher Disease to determine clinical factors which predict

enzyme therapy
cancer
  • 87 views
  • 28 Jul, 2021
  • 8 locations
A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease

The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants under 5 years old with Gaucher disease. Symptoms will be checked with blood

velaglucerase alfa
vpriv
  • 0 views
  • 20 Jul, 2021
  • 1 location
Natural History of Glycosphingolipid and Glycoprotein Lysosomal Storage 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
  • 01 Aug, 2021
  • 1 location