Gaucherite - A Study to Stratify Gaucher Disease
Recruiting
Observational Study
Gaucher Disease, Type I
Gaucher Disease, Type III
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 250 (estimated)
- Sponsor
- Cambridge University Hospitals NHS Foundation Trust · Other
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About This Trial
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.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
Each patient must meet all of the following criteria to be enrolled in this study:
1. Confirmed biochemical diagnosis of Type I, Type II or Type III Gaucher disease
2. Written Ethics Committee (EC) approved informed consent obtained from the patient, or patient's parent or lega…
Contacts