Status:
RECRUITING
Gaucherite - A Study to Stratify Gaucher Disease
Lead Sponsor:
Cambridge University Hospitals NHS Foundation Trust
Collaborating Sponsors:
Medical Research Council
National Institute for Health Research, United Kingdom
Conditions:
Gaucher Disease, Type I
Gaucher Disease, Type III
Eligibility:
All Genders
Brief 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 a...
Detailed Description
Gaucher disease is part of a rare group of genetic metabolic diseases which are caused by an inherited deficiency of the enzyme glucocerebrosidase. The most common form, Type 1 affects 1 in 40,000 to ...
Eligibility Criteria
Inclusion
- Each patient must meet all of the following criteria to be enrolled in this study:
- Confirmed biochemical diagnosis of Type I, Type II or Type III Gaucher disease
- Written Ethics Committee (EC) approved informed consent obtained from the patient, or patient's parent or legal guardian and patient assent if appropriate
- Male or Female patients, no age limitation
- Willing and able to comply with study schedule and procedures
- Deceased patients for whom the EC determines that patient data can be collected without a new consent from the patient
Exclusion
- Patients meeting any of the following criteria will be excluded from the study:
- Unrelated co-morbid condition limiting life expectancy to less than 6 months
- 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
- If determined unsuitable for the study by the investigator
Key Trial Info
Start Date :
January 1 2014
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 31 2028
Estimated Enrollment :
250 Patients enrolled
Trial Details
Trial ID
NCT03240653
Start Date
January 1 2014
End Date
December 31 2028
Last Update
August 6 2024
Active Locations (8)
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1
Birmingham Childrens Hospital
Birmingham, United Kingdom
2
New Queen Elizabeth Hospital
Birmingham, United Kingdom
3
Cambridge University Hospital
Cambridge, United Kingdom
4
Great Ormond Street Hospital
London, United Kingdom