An Observational Pregnancy Safety Study in Women with Neuromyelitis Optica Spectrum Disorder (NMOSD) Exposed to UPLIZNA® (inebilizumab-cdon) during Pregnancy (VIB0551.P4.S4 /20230064)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Observational Pregnancy Safety Study

Study drug and medical condition

• Name of medicine:
  UPLIZNA
• Study drug International non-proprietary name (INN) or common name:
  INEBILIZUMAB
• Medical condition to be studied:
  Neuromyelitis optica spectrum disorder

Population studied

• Short description of the study population:
  Study is recruiting participants ages 15 years and older.
• 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)
• Special population of interest:
  Pregnant women
• Estimated number of subjects:
  60

Study design details

• Main study objective:
  To assess pregnancy and birth outcomes in female patients with neuromyelitis optica spectrum disorder (NMOSD), exposed to UPLIZNA during pregnancy as defined by receipt of any dose during pregnancy or within 6 months preceding conception.
• Outcomes:
  • Major congenital malformation (MCM): Any major structural or chromosomal defect, or a combination of 2 or more conditional defects in live-born infants, stillbirths, or fetal losses of any gestational age (including outcomes prior to 20 weeks’ gestation or weighing < 500 g) ; • Preterm birth: An infant born at gestational age < 37 weeks ; • Low birth weight: An infant whose birth weight < 2500 g. ; • Minor congenital malformation: congenital anomalies that do not require major medical or surgical treatment, do not seriously affect health and development, and do not have significant cosmetic impact, in live-born infants, stillbirths, or fetal losses of any gestational age. ; • Developmental milestones or neurologic abnormalities in offspring of exposed mothers. ; • Abnormalities of immune system development in offspring of exposed mothers.
• Data analysis plan:
  Pregnancy outcomes will be summarized by the trimester of exposure and by preconception exposure. ; The number of infants with congenital malformations will be summarized descriptively. ; In addition, the risk of infants with congenital malformations, defined as the percentage of infants with congenital malformations among total number of infants, will be reported. ; If data permit, analyses will also be presented by the subgroups of maternal age, race/ethnicity, prior history of elective or therapeutic pregnancy termination status, prospective cases vs. retrospective cases, and other important risk factors.