E78.3
Conditions
Interventions
Group 1: Patients with genetically confirmed FCS (Familial Chylomicronaemia Syndrome) who were treated or who are currently treated with Waylivra (according to SmPC) are asked to complete a FCS questi
Sponsors
Akcea Therapeutics, Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Adult patients (= 18-years-old) prescribed WAYLIVRA™ in accordance with the approved Summary of Product Characteristics. 2. Treated in Germany. 3. Have provided written informed consent.
Exclusion criteria
Exclusion criteria: • Patients for whom WAYLIVRA is not advised including: • Patients with chronic or unexplained thrombocytopenia. Treatment should not be initiated in patients with thrombocytopenia (platelet count < 140 × 109/L). • Patients with hypersensitivity to the active substance or to any of the following excipients: o Sodium hydroxide (for pH adjustment). o Hydrochloric acid (for pH adjustment).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary 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. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Safety Objectives • To determine real-world incidence rates of mild, moderate and severe thrombocytopenia, and associated bleeding events, overall and by event grading • To describe prescribers’ adherence to recommendations for platelet monitoring and dose reduction to minimise the risk of thrombocytopenia as outlined in the SmPC • To determine real-world incidence rates and severity of: o immunogenicity/immunological events o hepatotoxicity o renal toxicity o severe injection site reactions o mild, moderate and severe thrombocytopenia with or without an associated serious bleeding episode in patients weighing less than 70kg, overall and by event grading. • To describe the safety profile in the following patient sub-groups for patients treated with WAYLIVRA: o patients with hepatic impairment o patients with renal impairment o elderly patients (= 65 years of age) • To describe the long-term safety profile of WAYLIVRA • To describe outcomes for WAYLIVRA-exposed pregnancies; specifically, gestational outcomes (e.g., live birth, spontaneous abortion, etc.) and major congenital malformations observed at pregnancy conclusion Secondary Efficacy Objectives • To evaluate the long-term efficacy of WAYLIVRA with respect to triglyceride reduction, pancreatitis prevention, and reduction in abdominal pain frequency and severity • To evaluate the effect of WAYLIVRA on health-related quality of life assessed by FCS-related symptoms and impacts on daily lives. | — |
Countries
Austria, France, Germany, Greece, Italy, Netherlands, Spain, Sweden, United Kingdom
Contacts
Public ContactRichard Jones
Akcea Therapeutics Ireland Ltd
Outcome results
None listed