Study type

Study topic

Human medicinal product

Study type

Non-interventional study

Scope of the study

Safety study (incl. comparative)
Non-interventional study

Non-interventional study design

Cohort
Study drug and medical condition

Name of medicine

REPATHA

Study drug International non-proprietary name (INN) or common name

EVOLOCUMAB

Anatomical Therapeutic Chemical (ATC) code

(C10AX13) evolocumab
evolocumab

Medical condition to be studied

Hypercholesterolaemia
Pregnancy
Population studied

Age groups

  • Paediatric Population (< 18 years)
    • Neonate
      • Preterm newborn infants (0 – 27 days)
      • Term newborn infants (0 – 27 days)
    • Infants and toddlers (28 days – 23 months)
    • Children (2 to < 12 years)
  • Adult and elderly population (≥18 years)
    • Adults (18 to < 65 years)
      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)

Special population of interest

Pregnant women

Estimated number of subjects

375
Study design details

Main study objective

Primary scope is to evaluate the safety of Repatha in pregnant women and their pregnancy outcomes.

Setting

A prospective observational study of pregnant women exposed to evolocumab to evaluate fetal, infant & childhood outcomes of these women & their live born offspring through the first 5 years of life to estimate incidence rates for potential safety signals of adverse pregnancy outcomes, embryo-fetal growth and development, & adverse infant and childhood outcomes related to humoral immune suppression.

Outcomes

To estimate the overall combined rate of major structural defects in infants of women with ASCVD and/or familial hypercholesterolemia (FH) exposed to evolocumab during pregnancy. Any major structural defects will be classified by a study investigator according to the Centers for Disease Control (CDC) Coding Manual: Abortion (spontaneous and elective), stillbirth, premature delivery, minor structural defects, size at gestational age, postnatal growth deficiency, postnatal serious infections, infant vaccination reactions, IgG-tetanus antibody response, adverse neurodevelopmental outcomes, breastfeeding outcomes

Data analysis plan

All statistical analyses will be performed at the 0.05 significance level using 2-sided tests or 2-sided 95%confidence intervals (CIs) unless otherwise specified. Demographics and baseline characteristics will be summarized.All continuous variables will be summarized using the following statistics: Number of non-missing data, Mean, Standard Deviation, Median, Minimum, Maximum, 1st quartile, 3rd quartile. All categorical variables will be summarized using counts and percentages. Missing data or unknown responses will not be counted in the percentages. e.g. risk estimation, measures of risk, internal/external validity.