Status: Recruiting
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 severity and response to therapy of Gaucher disease especially in the areas of bone, cancer and brain conditions.
Participants needed: 250
Trial details
Biological sex: AllType: ObservationalSponsor: Cambridge University Hospitals NHS Foundation TrustUpdated: Aug 6, 2024Locations: 8
Eligibility criteria
Confirmed biochemical diagnosis of Type I, Type II or Type III Gaucher disease [+4]
Unrelated co-morbid condition limiting life expectancy to less than 6 months [+2]