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.
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 Type
OBSERVATIONAL
Enrollment
50
Pregnant women exposed to pegvaliase within 2 weeks prior to LMP.
Syneos Health
Wilmington, North Carolina, United States
RECRUITINGMetabolics and Genetics in Calgary (MAGIC)
Calgary, Canada
RECRUITINGUniversitaetsklinikum Hamburg Eppendorf
Hamburg, Germany
RECRUITINGPregnancy outcome and infant development for women who have been treated with Palynziq (pegvaliase) within 2 weeks of their last menstrual period
To estimate the frequency of pregnancy outcomes (eg, spontaneous abortion, stillbirth, live birth, and termination) among subjects with PKU treated with pegvaliase during pregnancy and fetal/infant outcomes (all major congenital malformations \[MCMs\] and specifically microcephaly and congenital heart defects\], FGR, small for gestational age \[SGA\], low birth weight, preterm birth, failure to thrive, and developmental delays) among their offspring exposed to pegvaliase during pregnancy.
Time frame: 10 years
Pregnancy Outcomes
To compare the frequency of pregnancy outcomes (eg, spontaneous abortion, stillbirth, live birth, and termination) and fetal/infant outcomes (all MCMs and specifically microcephaly and congenital heart defects, FGR, SGA, low birth weight, preterm birth, failure to thrive, and developmental delays) among subjects with PKU treated with pegvaliase during pregnancy and their offspring to information on those same outcomes in non-pegvaliase exposed, pregnant women with PKU as described in reference literature
Time frame: 10 years
Pregnancy Outcomes
To examine differences in the frequency of pregnancy outcomes (eg, spontaneous abortion, stillbirth, live birth, and termination) and fetal/infant outcomes (all MCMs and specifically microcephaly and congenital heart defects, FGR, SGA, low birth weight, preterm birth, failure to thrive, and developmental delays) among subjects with PKU treated with pegvaliase during pregnancy and their offspring by maternal blood Phe levels
Time frame: 10 years
Serious Adverse Events
To estimate the frequency of serious adverse events (SAEs) other than CMs in infants exposed to pegvaliase during pregnancy through their first year of life
Time frame: 10 years
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Ospedale San Paolo
Milan, Italy
RECRUITINGMaternal Pegvaliase use during breastfeeding
To estimate the frequency of selected outcomes in subjects with PKU treated with pegvaliase during breastfeeding (low milk supply) and their infants (failure to thrive and SAEs) through their first year of life
Time frame: 10 years