NCT05579548

A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 50 (estimated)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: Female
• Age: —
• Healthy volunteers: —

Summary

This is a Phase 4 observational study designed to assess the impact of Palynziq ® (pegvaliase) treatment in pregnant women with PKU and on their offspring who were exposed to pegvaliase at any time during pregnancy and breastfeeding.

Detailed description

Women prescribed pegvaliase by their healthcare provider (HCP) who become pregnant during treatment may decide to participate in the study and enroll via a centralized call center model. Upon consent, data will be collected from the patient's HCP(s) retrospectively for at least 3 months prior to Last Menstrual Period (LMP). Data will be collected during pregnancy and throughout the infant's first year of life. Pegvaliase exposure will be recorded during pregnancy and breastfeeding including exposure during each trimester of pregnancy. Duration of individual subject participation will be up to approximately 21 months.

Conditions

• Phenylketonuria, Maternal

Interventions

Type	Name	Description
DRUG	Pegvaliase	Pregnant women exposed to pegvaliase within 2 weeks prior to LMP.

Timeline

• Start date: 2022-11-22
• Primary completion: 2032-10-04
• Completion: 2032-10-04
• First posted: 2022-10-14
• Last updated: 2026-03-24

Locations

4 sites across 4 countries: United States, Canada, Germany, Italy

Regulatory

• FDA-regulated drug study