Gaucherite - A Study to Stratify Gaucher Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorCambridge University Hospitals NHS Foundation Trust
About this trial
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 criteria
Qualifiers
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
Disqualifiers
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
Trial design
Treatments tested in this trial
- Stratified response to Enzyme Therapy
Treatment groups
250 Participants
are divided into 2 treatment groupsSponsors and collaborators
Cambridge University Hospitals NHS Foundation Trust
Lead sponsor
Medical Research Council
Collaborator
National Institute for Health Research, United Kingdom
Collaborator
Source ClinicalTrials.gov