Study type

Study type

Non-interventional study

Scope of the study

Drug utilisation
Effectiveness study (incl. comparative)
Non-interventional study

Non-interventional study design

Case-control
Study drug and medical condition

Anatomical Therapeutic Chemical (ATC) code

(C10AX) Other lipid modifying agents
Other lipid modifying agents
(C10AX13) evolocumab
evolocumab
(C10AX14) alirocumab
alirocumab
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

80
Study design details

Main study objective

- To describe the patients who fail to achieve adequate LDL-C lowering on PCSK9inhibitor therapy compared to controls, due to the fact that they a) do not respond to the therapy (Group A), or b) experience adverse events. (Group B)- To unravel the cause of adverse effects and inefficacy of PCSK9-inhibition.

Outcomes

Identification of genetic variants in genes possibly leading to reducedefficacy of PCSK9 inhibitors (e.g. those involved in lipid metabolism), Identification of differences in DNA expression in patients not responding totherapy compared to controls.(Semi-)quantification of proteins (e.g. anti-drug antibodies, PCSK9 protein levels, other proteins involved in lipid metabolism) in patients not responding to therapy compared to controls.ยท (Semi-)quantification of metabolites (e.g. lipid composition)

Data analysis plan

Data will be obtained on the patient's medical history, family history, as well as routine laboratory results. Descriptional statistics will be used to describe the current cohort. We will perform targeted sequencing of the PCSK9 gene and other genes known to be involved in lipid-metabolism, as well as proteome and metabolome analysis by ELISA and/or protein mass spectrometry. We will store samples in a biobank for further research.