Avalglucosidase Alfa Pregnancy Study

Sanofi

Status: Enrolling

Conditions: Pompe Disease; Pregnancy

Treatments: Biological: avalglucosidase alfa-NGPT (GZ402666); Biological: avalglucosidase alfa-NGPT (GZ402666) IV

Study type: Observational

Funder types: Industry

Identifiers

NCT05734521

OBS17641

Details and patient eligibility

About

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.

Full description

Study Design Time Perspective: Retrospective and Prospective

Enrollment: 100 estimated patients

Sex: Female

Volunteers: No Healthy Volunteers

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.

Trial design

100 participants in 2 patient groups

Pregnant women exposed to avalglucosidase alfa

Description:

Pregnant women with a confirmed diagnosis of Pompe disease and avalglucosidase alfa exposure during the pregnancy and/or lactation

Treatment:

Biological: avalglucosidase alfa-NGPT (GZ402666) IV

Infants born to mother/father exposed to avalglucosidase alfa

Description:

Infants born to mother/father with a confirmed diagnosis of Pompe disease and exposed to avalglucosidase alfa

Treatment:

Biological: avalglucosidase alfa-NGPT (GZ402666)