Spinraza (nusinersen) SMA Pregnancy Exposure Study Within Existing SMA Registries

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

• Medicinal product name:
  SPINRAZA
• Study drug International non-proprietary name (INN) or common name:
  NUSINERSEN
• Anatomical Therapeutic Chemical (ATC) code:
  (M09AX07) nusinersen

  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-USSMArtCARE, and Polish SMA Patient 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:
  The primary objectives of the study are to prospectively evaluate pregnancy complications and outcomes in participants with SMA, birth outcomes and adverse effects in infants born to participants with SMA, who were exposed to nusinersen 12 or 50/28 milligrams (mg) up to 14 months prior to the first day of their last menstrual period (LMP) before conception, 14.5 months before the date of conception, and/or at any time during their pregnancy. The secondary objective of the study is to evaluate pregnancy outcomes in participants with SMA exposed to nusinersen 12 or 50/28 mg as compared with participants without SMA who were not exposed to nusinersen (e.g., participants from external, general population comparators).
• Comparators:
  Women without SMA who were not exposed to nusinersen (e.g., women from external, general population ; comparators).
• 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.