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Gaucherite - A Study to Stratify Gaucher Disease

NCT03240653 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 250

Last updated 2024-08-06

No results posted yet for this study

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.

Conditions

  • Gaucher Disease, Type I
  • Gaucher Disease, Type III

Interventions

OTHER

Stratified response to Enzyme Therapy

Observational study involves review of retrospective and prospective data of participants' medical history, pathology, imaging and health questionnaires.

Sponsors & Collaborators

  • Medical Research Council

    collaborator OTHER_GOV

  • National Institute for Health Research, United Kingdom

    collaborator OTHER_GOV

  • Cambridge University Hospitals NHS Foundation Trust

    lead OTHER

Principal Investigators

  • Timothy M Cox, MD · University of Cambridge

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-01-01
Primary Completion
2028-09-30
Completion
2028-12-31

Countries

  • United Kingdom

Study Locations

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Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03240653 on ClinicalTrials.gov