Avalglucosidase Alfa Pregnancy Study

NCT05734521 | Recruiting FDA Drug

• 1 other identifier: OBS17641
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 1

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.

Conditions

Pompe Disease; Pregnancy

Outcome Measures

Primary Outcomes (3)

• 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

Study Arms (2)

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

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)

Interventions

avalglucosidase alfa-NGPT (GZ402666) IV BIOLOGICAL

intravenous infusion

Also known as: NEXVIAZYME

Pregnant women exposed to avalglucosidase alfa

avalglucosidase alfa-NGPT (GZ402666) BIOLOGICAL

exposed via pregnancy and lactation

Also known as: NEXVIAZYME

Infants born to mother/father exposed to avalglucosidase alfa

Eligibility Criteria

• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Sanofi: lead

Study Sites (1)

Investigational site worldwide

Bridgewater, New Jersey, 08807, United States

RECRUITING

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Interventions

GAA protein, human

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Central Study Contacts

Trial Transparency email recommended (Toll free for US & Canada)

CONTACT

800-633-1610; Contact-US@sanofi.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 3, 2023
• First Posted: February 21, 2023
• Study Start: October 26, 2022
• Primary Completion (Estimated): October 31, 2032
• Study Completion (Estimated): October 31, 2032
• Last Updated: January 16, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share

Locations

US (1)