Trial Design of PCSK9-inhibitor, Evolocumab, in Pediatric Patients with Heterozygous Familial Hypercholesterolemia
June 18, 2018
In this video, Prof Paul Maruff discusses a recently published paper which details the design of a study investigating the PCSK9-inhibitor, evolocumab, in pediatric patients with heterozygous familial hypercholesterolemia. The FDA has been investigating reports of cognitive impairment associated with the use of lipid-lowering medications for several years, and while they did not link statins and cognitive impairment in clinical and observational studies, a safety advisory was eventually required on the label for statin medications. In this global pediatric study, the Cogstate battery was selected for the assessment of cognitive function, as the tests covering the domains of interest are appropriate for—and highly acceptable to—pediatric patients across a broad age range, established age-matched normative datasets of healthy participants are available for comparison, the tests are highly repeatable over short time periods, and the availability of translations and robustness to culture and language allow for multi-national research.