Study type

Study type

Non-interventional study

Scope of the study

Safety study (incl. comparative)
Non-interventional study

Non-interventional study design

Cohort
Other

Non-interventional study design, other

Intensive monitoring schemes
Study drug and medical condition

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

INCLISIRAN

Anatomical Therapeutic Chemical (ATC) code

(C10AX16) inclisiran
inclisiran
Population studied

Age groups

Adolescents (12 to < 18 years)
Adults (18 to < 46 years)
Adults (46 to < 65 years)

Special population of interest

Pregnant women

Estimated number of subjects

500
Study design details

Main study objective

To estimate the proportion of major congenital malformations among pregnancies exposed to inclisiran during pregnancy prospectively reported to Novartis amongst (i) live births and (ii) live births plus still births plus termination of pregnancy for fetal anomaly (TOPFA).

Outcomes

Major congenital malformations (MCM) among pregnancies exposed to inclisiran during pregnancy, • To estimate the proportion of MCM among pregnancies exposed to inclisiran prior to the last menstrual period (LMP) • To compare the frequency of MCM among pregnancies exposed to inclisiran with the background frequencies • To estimate the proportion of other secondary outcomes in pregnancies exposed to inclisiran prior to LMP and during pregnancy

Data analysis plan

Data analysis will focus on the prospectively reported pregnancy cases. Retrospective pregnancy cases will also be analyzed and presented separately from the prospective cases. Data analysis will include the estimation of proportion (and 95% confidence interval) of malformations (major, minor, and overall), and of specific pregnancy outcomes such as live births, stillbirths, spontaneous abortions and elective terminations. Proportions will be estimated by timing of drug exposure in pregnancy (during pregnancy and prior to LMP, as well as by trimester if sample size allows). Descriptive analysis will be performed for all prospective pregnancy cases including case disposition (outcome known, pending, and lost to follow-up) and maternal characteristics (i.e. age, ethnicity, region) by providing the number and percentage of pregnancies in each category. Further stratified analyses may be undertaken if sample size allows.