WAYLIVRA® Post-Authorisation Safety Study (PASS) and Product Registry (WAY4001)

Study type

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

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Cohort Driven, Non-interventional observational study

Study drug and medical condition

• Name of medicine:
  WAYLIVRA
• Anatomical Therapeutic Chemical (ATC) code:
  (C10AX18) volanesorsen

  volanesorsen
• Additional medical condition(s):
  Familial Chylomicronaemia Syndrome (FCS)

Population studied

• Age groups:
  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)
• Estimated number of subjects:
  200

Study design details

• Study design:
  The first phase of the study is the PASS phase and will be concluded after a study term of 5 years. Following the PASS phase, the study will continue as the WAYLIVRA Product Registry.
• Main study objective:
  To evaluate the safety of WAYLIVRA on severe thrombocytopenia and bleeding in FCS patients according to the dose recommendation and dose algorithm in the Summary of Product Characteristics.
• Setting:
  This study will be conducted in Europe at centers that manage patients with FCS, as well as by physicians specializing in lipid disorders (e.g., endocrinologists, cardiologists).; Patients from each participating site will be invited to participate in the study in order to minimize selection bias. For eligible patients who do not enroll, reason for non-enrolment will be collected.
• Outcomes:
  Rate of Adverse Event Reporting of Severe Thrombocytopenia and Severe Bleeding; Rate and severity of adverse events with a focus on immunological events, hepatoxicity, renal toxicity and severe injection site reactions; Adherence rate to platelet monitoring and association of serious bleeding events; Dose and dose reduction rates; Pregnancy outcomes; Summarization of triglyceride reduction, pancreatitis prevention, and reduction in abdominal pain frequency and severity.
• Data analysis plan:
  A PASS final analysis will be performed after the conclusion of the PASS phase which will be after 5 years in which at least data on 247 person-years of exposure have been collected in patients with genetically confirmed FCS and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate, at which point a study report will be written. Complete analytical specifications for the study report, including tables and listings, will be included in the SAP, which will be prepared separately.