A Worldwide Pregnancy Safety Study to Assess Maternal, Fetal, and Infant Outcomes Following Exposure to Efgartigimod During Pregnancy and/or Breastfeeding.

A Worldwide Pregnancy Safety Study To Assess Maternal, Fetal, And Infant Outcomes Following Exposure To Efgartigimod During Pregnancy And/Or Breastfeeding

This is a multi-country, prospective safety study of pregnant women exposed to efgartigimod any time within 25 days prior to conception or any time during pregnancy. Women exposed to efgartigimod only during breastfeeding will also be eligible to enroll. Background rates of major congenital malformations (MCMs) will be obtained from populations within the same countries/regions as the countries/regions in which the VYVGART IV or SC-exposed pregnancies were reported.

Study Overview

• Status: Not yet recruiting
• Conditions: Myasthenia Gravis
• Intervention / Treatment: Biological: Efgartigimod

• Study Type: Observational
• Enrollment (Estimated): 279

Contacts and Locations

• Study Contact: Name: Sabine Coppieters, MD; Phone Number: 857-350-4834; Email: clinicaltrials@argenx.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Pregnant women or women no longer pregnant at the time of study enrollment but exposed to efgartigimod any time within 25 days prior to conception or any time during the pregnancy.

Description

Inclusion Criteria:

• Women with exposure to efgartigimod any time within 25 days prior to conception or any time during pregnancy, or women with exposure to efgartigimod during breastfeeding. The timeframe of 25 days prior to conception is calculated based on five times the efgartigimod half-life, which is 3 to 5 days.
• Written informed consent or eConsent (depending on country regulations) (for adolescents under the age of majority, written informed assent or eConsent by the pregnant minor (where applicable) and written informed consent or eConsent by the parent/legal guardian).

Exclusion Criteria:

• None

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Retrospective Pregnancy; woman is no longer pregnant at time of study enrollment but was exposed to efgartigimod any time within 25 days prior to conception or any time during pregnancy	Biological: Efgartigimod; Efgartigimod IV or SC; Other Names: Vyvgart
Prospective Pregnancy; woman is pregnant or breastfeeding at time of study enrollment.	Biological: Efgartigimod; Efgartigimod IV or SC; Other Names: Vyvgart

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pregnancy outcomes	Pregnancy outcomes: Spontaneous abortion; Elective or therapeutic abortion; Fetal death/stillbirth ; Molar or ectopic pregnancy; Live birth (Preterm delivery; Fullterm delivery)	up to 10 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Congenital malformations	Congenital malformations (CDC 2020b) identified in the developing fetus, neonate, or infant: Major congenital malformations (MCMs); Minor congenital malformations	up to 10 years
The number of observed other events of interest in the developing neonate and infant (such as hospitalizations for serious illness)	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	up to 10 years
Maternal complications of pregnancy	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	up to 10 years
Maternal infections		up to 10 years
The number of observed fetal growth deficiency		up to 10 years

Collaborators and Investigators

• Sponsor: argenx

Study record dates

Study Major Dates

• Study Start (Estimated): February 29, 2024
• Primary Completion (Estimated): December 1, 2033
• Study Completion (Estimated): December 1, 2033

Study Registration Dates

• First Submitted: February 22, 2024
• First Submitted That Met QC Criteria: March 1, 2024
• First Posted (Actual): March 8, 2024

Study Record Updates

• Last Update Posted (Actual): March 8, 2024
• Last Update Submitted That Met QC Criteria: March 1, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Immune System Diseases; Neoplasms; Autoimmune Diseases of the Nervous System; Autoimmune Diseases; Neoplasms by Site; Neuromuscular Diseases; Neurodegenerative Diseases; Nervous System Neoplasms; Paraneoplastic Syndromes, Nervous System; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Myasthenia Gravis
• Other Study ID Numbers: ARGX-113-PAC-2206

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No