Study type

Study topic

Disease /health condition
Human medicinal product

Study type

Non-interventional study

Scope of the study

Assessment of risk minimisation measure implementation or effectiveness

Data collection methods

Combined primary data collection and secondary use of data
Non-interventional study

Non-interventional study design

Cohort
Study drug and medical condition

Name of medicine

SPINRAZA

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

NUSINERSEN

Medical condition to be studied

Spinal muscular atrophy
Population studied

Short description of the study population

Pregnant participants with SMA who are exposed to nusinersen from the UK-Adult SMA REACH, ISMAR-US and SMArtCARE registries will be enrolled to obtain information on effects of nusinersen on pregnancy complications and outcomes.

Age groups

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

Special population of interest

Pregnant women

Estimated number of subjects

20
Study design details

Study design

An observational cohort prospective study

Main study objective

To evaluate pregnancy complications and outcomes in subjects with SMA, birth outcomes and adverse effects in infants born to subjects exposed to nusinersen up to 14 months before first day of last menstrual period before conception, 14.5 months before conception, and/or at any time during pregnancy.

Outcomes

Number of Pregnancy Terminations, Spontaneous Abortions, Fetal Deaths, Live Births, Neonatal, Perinatal, Infant Deaths, Major Congenital Malformations(MCMs), Infants Small for Gestational Age Birth, Ectopic, Molar Pregnancies, Maternal Deaths and Infants With Abnormal Postnatal Growth and Development, Neurobehavioral Impairment

Data analysis plan

All analyses will be conducted on an overall basis, as well as stratified by earliest trimester of exposure. For MCMs, analyses will be conducted only for participants who have exposure in the first trimester.
The prevalence and 95% CIs of spontaneous abortions, MCMs, SGA births, and abnormal postnatal growth and development will be calculated.
Other negative pregnancy outcomes will be similarly examined as the sample size permits.
Infants with minor malformations, chromosomal abnormalities, genetic syndromes, positional defects, and prematurity-related defects will be excluded from the primary analyses related to MCM prevalence, these outcomes will be reported in the interim and final reports.