Avalglucosidase Alfa Pregnancy Study

Recruiting

• Conditions: Pompe Disease; Pregnancy
• Interventions: Biological: avalglucosidase alfa-NGPT (GZ402666) IV; Biological: avalglucosidase alfa-NGPT (GZ402666)

• Registration Number: NCT05734521
• Lead Sponsor: Sanofi

Brief Summary

This is a worldwide, descriptive safety study collecting data on women and their offspring exposed to avalglucosidase alfa during pregnancy and/or lactation, to assess the risks of avalglucsodiase alfa on pregnancy and maternal complications and adverse effects in the developing fetus, neonate, and infant.

* Outcomes in exposed infants, including growth and development, will be assessed through at least the first year of life.

* Data will be collected for approximately 10 years.

Detailed Description

Study Design Time Perspective: Retrospective and Prospective

Study Timeline

• Study Start: 2022-10-26
• Study First Submitted: 2023-02-03
• Study First Submitted QC: 2023-02-15
• Study First Posted: 2023-02-21
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-12
• Last Update Posted: 2024-07-15
• Primary Completion: 2032-10
• Study Completion: 2032-10

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Female
• Target Recruitment: 100

Inclusion Criteria

• Women and their offspring exposed to avalglucosidase alfa during pregnancy and/or lactation for whom an Individual Case Safety Report (ICSR) has been submitted to the Sanofi Global Pharmacovigilance (PV) department, and/or,
• Women exposed to avalglucosidase alfa during pregnancy and/or lactation who have provided informed consent to enroll in the Pompe Pregnancy Sub-registry.

Exclusion Criteria

There are no exclusion criteria in this study.

The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Pregnant women exposed to avalglucosidase alfa	avalglucosidase alfa-NGPT (GZ402666) IV	Pregnant women with a confirmed diagnosis of Pompe disease and avalglucosidase alfa exposure during the pregnancy and/or lactation
Infants born to mother/father exposed to avalglucosidase alfa	avalglucosidase alfa-NGPT (GZ402666)	Infants born to mother/father with a confirmed diagnosis of Pompe disease and exposed to avalglucosidase alfa

Primary Outcome Measures

Name	Time	Method
Pregnancy outcome	through study completion, an average of 10 years	Prevalence of live births, spontaneous abortions (\<20 weeks of gestation), elective terminations, ectopic pregnancies, early fetal deaths (20 to 27 weeks of gestation), late fetal deaths (≥28 weeks of gestation), stillbirths and maternal deaths
Infant outcome	through study completion, an average of 10 years	Number of occurrences of major congenital anomalies, neonatal deaths, development delays (growth, motor, neurologic, behavioral) through first year of life.; Major congenital anomalies defined using the US Centers for Disease Control and Prevention (CDC) and growth and development assessments using CDC's Developmental Milestones 2021.
Maternal complications	through study completion, an average of 10 years	Prevalence of pregnancy/labor/delivery/postpartum maternal complications

Trial Locations

Locations (1)

Investigational site worldwide; 🇺🇸 Bridgewater, New Jersey, United States