RecruitingNCT03240653

Gaucherite - A Study to Stratify Gaucher Disease

Predictive Measures to Stratify Clinical Outcomes in Children and Adults With Gaucher Disease and Responses to Specific Therapies


Sponsor

Cambridge University Hospitals NHS Foundation Trust

Enrollment

250 participants

Start Date

Jan 1, 2014

Study Type

OBSERVATIONAL

Conditions

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.


Eligibility

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying Stratified response to Enzyme Therapy for people with gaucher disease, type i and gaucher disease, type iii. The study is currently recruiting participants at 8 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

OTHERStratified 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

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NCT03240653


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