Gaucherite - A Study to Stratify Gaucher Disease
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.
Study identifier: NCT03240653. Status: RECRUITING.
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 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
Participating locations
- Birmingham Childrens Hospital, Birmingham
- New Queen Elizabeth Hospital, Birmingham
- Cambridge University Hospital, Cambridge
- Great Ormond Street Hospital, London
- National Hospital for Neurology and Neurosurgery, London
- Royal Free Hospital, London
- Royal Manchester Childrens Hospital, Manchester
- Salford Royal NHS Foundation Trust, Salford
Study records are sourced from ClinicalTrials.gov. Confirm current eligibility, recruiting status, and locations in the official record.