Pompe Disease, Pregnancy
Conditions
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
Interventions
BIOLOGICALavalglucosidase alfa-NGPT (GZ402666) IV
intravenous infusion
BIOLOGICALavalglucosidase alfa-NGPT (GZ402666)
exposed via pregnancy and lactation
Sponsors
Sanofi
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
FEMALE
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Maternal complications | through study completion, an average of 10 years | Prevalence of pregnancy/labor/delivery/postpartum maternal complications |
| 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. |
Countries
United States
Contacts
CONTACTTrial Transparency email recommended (Toll free for US & Canada)
STUDY_DIRECTORClinical Sciences & Operations
Sanofi
Outcome results
None listed