Gaucherite - A Study to Stratify Gaucher Disease
Key facts
- Where
- Birmingham, Cambridge, London, Manchester, and Salford, United KingdomSee the 8 sites
Where in United Kingdom
- New Queen Elizabeth HospitalBirmingham, United KingdomTaking people
- Birmingham Childrens HospitalBirmingham, United KingdomNo longer taking part
- Cambridge university hospitalCambridge, United KingdomTaking people
- Great Ormond Street HospitalLondon, United KingdomTaking people
- National Hospital For Neurology and NeurosurgeryLondon, United KingdomTaking people
- Royal Free HospitalLondon, United KingdomTaking people
- Royal Manchester Childrens HospitalManchester, United KingdomTaking people
- Salford Royal NHS Foundation TrustSalford, United KingdomTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Cambridge University Hospitals NHS Foundation Trust
- Trial code
- NCT03240653
- Record updated
- 6 August 2024
- Original description
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.Read full original descriptionShow less
Predictive Measures to Stratify Clinical Outcomes in Children and Adults With Gaucher Disease and Responses to Specific Therapies
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.
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: Unrelated co-morbid condition limiting life expectancy to less than 6 months; Patients meeting any of the following criteria will be excluded from the study:; 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.