VPRIV® Non-Interventional Study in Patients Previously Treated with Other Enzyme Replacement Therapies (ERTs)/Substrate Reduction Therapies (SRTs) (SHP669-405)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Other
• If ‘other’, further details on the scope of the study:
  Treatment patterns
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Phase 4, observational, retrospective/prospective, non-controlled, non-comparative, multicenter study

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (A16AB10) velaglucerase alfa

  velaglucerase alfa
• Medical condition to be studied:
  Gaucher's disease

Population studied

• Short description of the study population:
  Patients with GD1 transitioning from other ERTs/SRTs to VPRIV in a routine clinical practice setting in Canada.; ; Inclusion Criteria; 1. Patients with GD1 and currently being treated with another ERT/SRT for at least 6 months before baseline enrolment.; 2. Patient or legally authorized representative has provided written informed consent.; ; Exclusion Criteria; Patients are excluded from the study if any of the following criteria are met.; 1. Patient is at high risk of non-compliance in the investigator's opinion.; 2. Patient is in the opinion of the investigator, unsuitable in any other way to participate in this study.; 3. Patient is pregnant
• Age groups:
  • Preterm newborn infants (0 – 27 days)
  • Term newborn infants (0 – 27 days)
  • Infants and toddlers (28 days – 23 months)
  • Children (2 to < 12 years)
  • Adolescents (12 to < 18 years)
  • Adults (18 to < 46 years)
  • Adults (46 to < 65 years)
  • Adults (65 to < 75 years)
  • Adults (75 to < 85 years)
  • Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Gaucher disease patients
• Estimated number of subjects:
  24

Study design details

• Main study objective:
  The main objective is to describe the safety in patients with GD1 transitioning from other ERTs/SRTs to VPRIV across all age groups.
• Outcomes:
  1. Number of Participants with Adverse Events (AEs) Following the Transition From Other ERTs/SRTs to VPRIV, 1. Describe VPRIV treatment dosing and administration patterns for all patients 2. Change From Baseline in Use of Glucosylspingosine (Lyso-Gb1) Biomarker 3. Change From Baseline in Gaucher Disease Questionnaire Score at Month 12
• Data analysis plan:
  The statistical evaluation of all collected data will be done on a descriptive basis. No statistical hypotheses will be tested.