RECRUITINGOBSERVATIONAL
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
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.
Who May Be Eligible (Plain English)
Who May Qualify:
Each patient must meet all of the following criteria to be enrolled in this study:
1. Confirmed biochemical diagnosis of Type I, Type II or Type III Gaucher disease
2. Written Ethics Committee (EC) approved willing to sign a consent form obtained from the patient, or patient's parent or legal guardian and patient assent if appropriate
3. Male or Female patients, no age limitation
4. Willing and able to comply with study schedule and procedures
5. Deceased patients for whom the EC determines that patient data can be collected without a new consent from the patient
Who Should NOT Join This Trial:
Patients meeting any of the following criteria will be excluded from the study:
1. Unrelated co-morbid condition limiting life expectancy to less than 6 months
2. Patient or if applicable, parent or legal guardian is unable to comprehend, sign and date the EC approved willing to sign a consent form form and patient assent as appropriate
3. If determined unsuitable for the study by the investigator
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
Inclusion Criteria:
Each patient must meet all of the following criteria to be enrolled in this study:
1. Confirmed biochemical diagnosis of Type I, Type II or Type III Gaucher disease
2. Written Ethics Committee (EC) approved informed consent obtained from the patient, or patient's parent or legal guardian and patient assent if appropriate
3. Male or Female patients, no age limitation
4. Willing and able to comply with study schedule and procedures
5. Deceased patients for whom the EC determines that patient data can be collected without a new consent from the patient
Exclusion Criteria:
Patients meeting any of the following criteria will be excluded from the study:
1. Unrelated co-morbid condition limiting life expectancy to less than 6 months
2. 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
3. If determined unsuitable for the study by the investigator
Treatments Being Tested
OTHER
Stratified response to Enzyme Therapy
Observational study involves review of retrospective and prospective data of participants' medical history, pathology, imaging and health questionnaires.
Locations (8)
Birmingham Childrens Hospital
Birmingham, United Kingdom
New Queen Elizabeth Hospital
Birmingham, United Kingdom
Cambridge University Hospital
Cambridge, United Kingdom
Great Ormond Street Hospital
London, United Kingdom
National Hospital for Neurology and Neurosurgery
London, United Kingdom
Royal Free Hospital
London, United Kingdom
Royal Manchester Childrens Hospital
Manchester, United Kingdom
Salford Royal NHS Foundation Trust
Salford, United Kingdom