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)