Study type

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

MAYZENT

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

SIPONIMOD

Anatomical Therapeutic Chemical (ATC) code

(L04AA42) siponimod
siponimod

Medical condition to be studied

Multiple sclerosis
Population studied

Age groups

Preterm newborn infants (0 – 27 days)
Term newborn infants (0 – 27 days)
Infants and toddlers (28 days – 23 months)
Adolescents (12 to < 18 years)
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)

Special population of interest

Pregnant women

Estimated number of subjects

867
Study design details

Main study objective

The primary objective is to estimate and compare the prevalence of major structural defects in siponimod exposed pregnant women versus 1) disease-matched pregnant women not exposed to siponimod, and 2) healthy pregnant women.

Outcomes

The primary outcome of the study is major structural defects.

Secondary outcomes:
- Spontaneous abortion/miscarriage.
- Stillbirth.
- Elective termination.
- Preterm delivery.
- Preeclampsia / eclampsia.
- Pattern of 3 or more minor structural defects.
- Small for gestational age.
- Postnatal growth small for age at approximately one year of age.
- Developmental performance at approximately one year of age.
- Serious or opportunistic infections in the first year of life.

Data analysis plan

The primary population for analysis will be those enrolled in the prospective cohort study comparing siponimod-exposed pregnancies with MS to the disease-matched cohort and the non-diseased cohort. Statistical analyses of those enrolled in the exposure series who do not meet the cohort study criteria will be descriptive only. All relevant exposure, outcome, and covariate data within each study group will be summarized using descriptive statistics annually. Means and standard deviations will be presented for continuous variables and frequencies and percentages will be presented for categorical variables. At the completion of the study, a final analysis with adjusted comparisons between cohort groups will be performed.