Gaucherite - A Study to Stratify Gaucher Disease
Predictive Measures to Stratify Clinical Outcomes in Children and Adults With Gaucher Disease and Responses to Specific Therapies
Cambridge University Hospitals NHS Foundation Trust
250 participants
Jan 1, 2014
OBSERVATIONAL
Conditions
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 and response to therapy of Gaucher disease especially in the areas of bone, cancer and brain conditions.
Eligibility
Inclusion Criteria6
- 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 Criteria4
- 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
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Interventions
Observational study involves review of retrospective and prospective data of participants' medical history, pathology, imaging and health questionnaires.
Locations(8)
View Full Details on ClinicalTrials.gov
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NCT03240653