Avalglucosidase Alfa Pregnancy Study

Study Overview
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Brief Summary
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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
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Study Design Time Perspective: Retrospective and Prospective

Official Title
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A Descriptive Safety Study Based on Data Collected From Women and Their Offspring Exposed to Nexviazyme/Nexviadyme (Avalglucosidase Alfa-ngpt/Avalglucosidase Alfa) During Pregnancy and/or Lactation in the Postmarketing Setting

Conditions
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Pompe Disease, Pregnancy

Intervention / Treatment
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* Biological: avalglucosidase alfa-NGPT (GZ402666) IV
* Biological: avalglucosidase alfa-NGPT (GZ402666)


Participation Criteria
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Eligibility Criteria
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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.
Sexes Eligible for Study
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Female

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Pregnant women exposed to avalglucosidase alfa<br>Pregnant women with a confirmed diagnosis of Pompe disease and avalglucosidase alfa exposure during the pregnancy and/or lactation | Biological: avalglucosidase alfa-NGPT (GZ402666) IV<br>* intravenous infusion<br>* Other names: NEXVIAZYME;|
| Infants born to mother/father exposed to avalglucosidase alfa<br>Infants born to mother/father with a confirmed diagnosis of Pompe disease and exposed to avalglucosidase alfa | Biological: avalglucosidase alfa-NGPT (GZ402666)<br>* exposed via pregnancy and lactation<br>* Other names: NEXVIAZYME;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Maternal complications | Prevalence of pregnancy/labor/delivery/postpartum maternal complications | through study completion, an average of 10 years | 
| Pregnancy outcome | 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 | through study completion, an average of 10 years | 
| Infant outcome | 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. | through study completion, an average of 10 years |