Avalglucosidase Alfa Pregnancy Study

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

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.

Study Overview

• Status: Recruiting
• Conditions: Pregnancy; Pompe Disease
• Intervention / Treatment: Biological: avalglucosidase alfa-NGPT (GZ402666) IV; Biological: avalglucosidase alfa-NGPT (GZ402666)

Detailed Description

Study Design Time Perspective: Retrospective and Prospective

• Study Type: Observational
• Enrollment (Estimated): 100

Contacts and Locations

• Study Contact: Name: Trial Transparency email recommended (Toll free for US & Canada); Phone Number: option 6 800-633-1610; Email: Contact-US@sanofi.com

Study Locations

• United States
  • New Jersey
    • Bridgewater, New Jersey, United States, 08807
      • Recruiting
      • Investigational site worldwide

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Non-Probability Sample

Study Population

Women and their offspring exposed to avalglucosidase alfa during pregnancy and/or lactation

Description

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 Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Pregnant women exposed to avalglucosidase alfa; 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; intravenous infusion; Other Names: NEXVIAZYME
Infants born to mother/father exposed to avalglucosidase alfa; Infants born to mother/father with a confirmed diagnosis of Pompe disease and exposed to avalglucosidase alfa	Biological: avalglucosidase alfa-NGPT (GZ402666); exposed via pregnancy and lactation; Other Names: NEXVIAZYME

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Sanofi
• Investigators: Study Director: Clinical Sciences & Operations, Sanofi

Study record dates

Study Major Dates

• Study Start (Actual): October 26, 2022
• Primary Completion (Estimated): October 31, 2032
• Study Completion (Estimated): October 31, 2032

Study Registration Dates

• First Submitted: February 3, 2023
• First Submitted That Met QC Criteria: February 15, 2023
• First Posted (Actual): February 21, 2023

Study Record Updates

• Last Update Posted (Estimated): January 16, 2026
• Last Update Submitted That Met QC Criteria: January 14, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Lysosomal Storage Diseases, Nervous System; Glycogen Storage Disease; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Glycogen Storage Disease Type II; GAA protein, human
• Other Study ID Numbers: OBS17641

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

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