Actively Recruiting
Gaucherite - A Study to Stratify Gaucher Disease
Led by Cambridge University Hospitals NHS Foundation Trust · Updated on 2024-08-06
250
Participants Needed
8
Research Sites
782 weeks
Total Duration
On this page
Sponsors
C
Cambridge University Hospitals NHS Foundation Trust
Lead Sponsor
M
Medical Research Council
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Gaucherite - A Study to Stratify Gaucher Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed biochemical diagnosis of Type I, Type II or Type III Gaucher disease
- Written Ethics Committee 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 Ethics Committee determines that patient data can be collected without a new consent from the patient
You will not qualify if you...
- Presence of 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 Ethics Committee approved informed consent form and patient assent as appropriate
- Patients deemed unsuitable for the study by the investigator
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 8 locations
1
Birmingham Childrens Hospital
Birmingham, United Kingdom
Terminated
2
New Queen Elizabeth Hospital
Birmingham, United Kingdom
Actively Recruiting
3
Cambridge University Hospital
Cambridge, United Kingdom
Actively Recruiting
4
Great Ormond Street Hospital
London, United Kingdom
Actively Recruiting
5
National Hospital for Neurology and Neurosurgery
London, United Kingdom
Actively Recruiting
6
Royal Free Hospital
London, United Kingdom
Actively Recruiting
7
Royal Manchester Childrens Hospital
Manchester, United Kingdom
Actively Recruiting
8
Salford Royal NHS Foundation Trust
Salford, United Kingdom
Actively Recruiting
Research Team
E
Elizabeth M MORRIS, RN
CONTACT
C
Chong Y TAN, MB PhD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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