A Worldwide Pregnancy Safety Study to Assess Maternal, Foetal, and Infant Outcomes Following Exposure to Efgartigimod alfa during Pregnancy and/or Breastfeeding

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Other
• If ‘other’, further details on the scope of the study:
  Pregnancy exposure registry
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Pregnancy exposure registry

Study drug and medical condition

• Name of medicine:
  VYVGART
• Study drug International non-proprietary name (INN) or common name:
  EFGARTIGIMOD ALFA
• Medical condition to be studied:
  Myasthenia gravis

Population studied

• Short description of the study population:
  Two mutually exclusive subgroups of the pregnancy safety study population are defined as follows, depending on whether the woman is still pregnant at the time of enrolment.; - Retrospective Pregnancy: woman is no longer pregnant at the time of study enrolment but exposed to efgartigimod any time within 25 days prior to conception or any time during the pregnancy.; - Prospective Pregnancy: woman is pregnant or breastfeeding at the time of study enrolment.
• Age groups:
  Adult and elderly population (≥18 years)

  Adults (18 to < 65 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Elderly (≥ 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Special population of interest:
  Nursing women

  Pregnant women
• Estimated number of subjects:
  279

Study design details

• Study design:
  This is a multi-country safety study of pregnant women exposed to efgartigimod during pregnancy or any time within 25 days prior to conception. Infants exposed in utero or through breastfeeding will be followed through 12 months of age.
• Main study objective:
  This global pregnancy safety study will assess maternal, foetal, and infant outcomes following exposure to efgartigimod during pregnancy.
• Setting:
  Two (2) mutually exclusive subgroups of the pregnancy safety study population are defined as follows, depending on whether the woman is still pregnant at the time of enrolment.; - Retrospective Pregnancy: woman is no longer pregnant at the time of study enrolment but exposed to efgartigimod any time within 25 days prior to conception or any time during the pregnancy.; - Prospective Pregnancy: woman is pregnant or breastfeeding at the time of study enrolment.; Eligible women will be recruited from the US, Japan, and all additional countries that have received authorization for efgartigimod marketing at the time of protocol approval. Recruitment will be expanded over the course of the pregnancy safety study, as efgartigimod continues to be approved in additional countries.
• Comparators:
  An external historical control will be used as a comparator.
• Outcomes:
  • Pregnancy outcomes: Spontaneous abortion; Elective or therapeutic abortion; Fetal death/stillbirth ; Molar or ectopic pregnancy; Live birth (Preterm delivery; Fullterm delivery); • Congenital malformations identified in the developing fetus, neonate, or infant: Major congenital malformations (MCMs); Minor congenital malformations ; • Other events of interest identified in the developing neonate and infant (Hospitalizations for serious illness; Potential adverse reactions to medications; Growth and development milestones as described by the Centers for Disease Control and Prevention or other accepted standard assessments; Infant developmental deficiency; Postnatal growth deficiency or failure to thrive (FTT); Neonatal and infant mortality; Infections; Transient neonatal myasthenia; Vaccination and vaccine reactions; • Maternal complications of pregnancy, including but not limited to: Premature rupture of membranes (PROM); Preterm PROM (PPROM); Pre-eclampsia; Gestational hypertension; Eclampsia; Proteinuria; Gestational diabetes; Intrauterine growth restriction (IUGR); Polyhdramnios; • Maternal infections; • Measures of fetal growth deficiency (e.g., small for gestational age)