Risdiplam Single-Arm Pregnancy Safety Study

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:
  EVRYSDI
• Medical condition to be studied:
  Spinal muscular atrophy

Population studied

• Age groups:
  • Paediatric Population (< 18 years)

    • Adolescents (12 to < 18 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:
  46

Study design details

• Main study objective:
  To collect and describe selected pregnancy outcomes and complications in women with SMA exposed to risdiplam during the defined exposure window, and to collect and describe selected fetal/neonatal/infant outcomes at birth and through up to first year of life of infants born to women exposed to risdiplam during the defined pregnancy exposure window and during breastfeeding up to 1 year after birth.
• Outcomes:
  1) Spontaneous Abortions 2) Fetal Death or Stillbirth 3) Live Birth 4) Elective or Therapeutic Pregnancy Terminations 5) Preterm Birth 6) Congenital Malformations 7) Size for Gestational Age 8) Low Birth Weight 9) Failure to Thrive 10) Ectopic Pregnancies 11) Molar Pregnancies 12) Hospitalization of Infants 13) Neonatal Death 14) Perinatal Death 15) Infant Death 16) Maternal Death
• Data analysis plan:
  Data analyses will be reported in aggregate and analyzed separately for prospectively and retrospectively enrolled pregnancies. For prospectively enrolled pregnancies, descriptive analysis will be stratified considering the availability of informative prenatal testing prior to enrolment. When an informative prenatal test result is known, analyses will be reported separately for positive and negative test results, if sample size permits. Stratified analysis by prenatal test result will also be conducted for retrospectively enrolled pregnancies. Cases where the infant is diagnosed with SMA will also be reported separately. Descriptive analyses will be performed to understand the qualitative and quantitative nature of the data collected and the characteristics of the sample studied.