Gaucherite stratified medicine project

Condition

Gaucher Disease

Full Study Name

Predictive measures to stratify clinical outcomes in children and adults with Gaucher disease and responses to specific therapies

Sponsor

Cambridge University Hospitals NHS Foundation Trust

Lead Site

Cambridge

Chief Investigator

Timothy Cox

Local Principal Investigator

Simon Jones

Study Status

recruiting

Main Aims

To develop a clinical stratification of Gaucher disease

Inclusion Criteria
  • Confirmed biochemical diagnosis of Type 1 or Type 3 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 Aged ≥5 years
  • Willing to comply with study schedule and procedures
Exclusion Criteria
  • Unrelated comorbid
  • condition limiting life expectancy to less than 6 months.
  • Confirmed Type 2 Neurological Gaucher Disease.
  • 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.
Open Sites

Birmingham, Great Ormond Street, Salford, UCLH, Manchester, Royal Free

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