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Gaucherite - A Study to Stratify Gaucher Disease

Recruitingn/aAges any - anyAllCambridge University Hospitals NHS Foundation Truโ€ฆ
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In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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 legal guardian and patient assent if appropriate 3. Male or Female patients, no age limitation 4. Willing and able to comply with study schedule and procedures 5. Deceased patients for whom the EC determines that patient data can be collected without a new consent from the patient Exclusion Criteria: Patients meeting any of the following criteria will be excluded from the study: 1. Unrelated co-morbid condition limiting life expectancy to less than 6 months 2. Patient or if applicable, parent or legal guardian is unable to comprehend, sign and date the EC approved informed consent form and patient assent as appropriate 3. If determined unsuitable for the study by the investigator
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What it tests

  • Stratified response to Enzyme Therapy
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Where

8 sites
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
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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