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Clinical Trial 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.


Clinical Trial 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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05579548
Study type Observational
Source BioMarin Pharmaceutical
Contact 165-504 Program Director
Phone 1-800-983-4587
Email medinfo@bmrn.com
Status Recruiting
Phase
Start date November 22, 2022
Completion date October 4, 2032