NCT03240653 Gaucherite - A Study to Stratify Gaucher Disease
| NCT ID | NCT03240653 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Cambridge University Hospitals NHS Foundation Trust |
| Condition | Gaucher Disease, Type I |
| Study Type | OBSERVATIONAL |
| Enrollment | 250 participants |
| Start Date | 2014-01-01 |
| Primary Completion | 2028-09-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 250 participants in total. It began in 2014-01-01 with a primary completion date of 2028-09-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 Criteria
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
Contact & Investigator
Timothy M Cox, MD
PRINCIPAL INVESTIGATOR
University of Cambridge
Frequently Asked Questions
Who can join the NCT03240653 clinical trial?
This trial is open to participants of all sexes, studying Gaucher Disease, Type I. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT03240653 currently recruiting?
Yes, NCT03240653 is actively recruiting participants. Contact the research team at liz.morris@addenbrookes.nhs.uk for enrollment information.
Where is the NCT03240653 trial being conducted?
This trial is being conducted at Birmingham, United Kingdom, Birmingham, United Kingdom, Cambridge, United Kingdom, London, United Kingdom and 4 additional locations.
Who is sponsoring the NCT03240653 clinical trial?
NCT03240653 is sponsored by Cambridge University Hospitals NHS Foundation Trust. The principal investigator is Timothy M Cox, MD at University of Cambridge. The trial plans to enroll 250 participants.