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Gaucherite - A Study to Stratify Gaucher Disease
Sponsor: Cambridge University Hospitals NHS Foundation Trust
Summary
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.
Official title: Predictive Measures to Stratify Clinical Outcomes in Children and Adults With Gaucher Disease and Responses to Specific Therapies
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
250
Start Date
2014-01-01
Completion Date
2028-12-31
Last Updated
2024-08-06
Healthy Volunteers
No
Interventions
Stratified response to Enzyme Therapy
Observational study involves review of retrospective and prospective data of participants' medical history, pathology, imaging and health questionnaires.
Locations (8)
Birmingham Childrens Hospital
Birmingham, United Kingdom
New Queen Elizabeth Hospital
Birmingham, United Kingdom
Cambridge University Hospital
Cambridge, United Kingdom
Great Ormond Street Hospital
London, United Kingdom
National Hospital for Neurology and Neurosurgery
London, United Kingdom
Royal Free Hospital
London, United Kingdom
Royal Manchester Childrens Hospital
Manchester, United Kingdom
Salford Royal NHS Foundation Trust
Salford, United Kingdom