Study type

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

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

247
Study design details

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 SmPC

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.