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RECRUITING
NCT03240653

Gaucherite - A Study to Stratify Gaucher Disease

Sponsor: Cambridge University Hospitals NHS Foundation Trust

View on ClinicalTrials.gov

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

OTHER

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