Actively Recruiting
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq (Pegvaliase) During Pregnancy and Breastfeeding
Led by BioMarin Pharmaceutical · Updated on 2026-03-24
50
Participants Needed
4
Research Sites
N/A
Total Duration
On this page
Sponsors
B
BioMarin Pharmaceutical
Lead Sponsor
S
Syneos Health
Collaborating Sponsor
AI-Summary
What this Trial Is About
This research aims to observe the effects of Palynziq (pegvaliase) treatment in pregnant women with phenylketonuria (PKU) and on their babies exposed to pegvaliase during pregnancy and breastfeeding. The study is a Phase 4 observational trial that evaluates maternal, fetal, and infant outcomes related to pegvaliase exposure. It includes women diagnosed with PKU who were treated with pegvaliase from two weeks before their last menstrual period (LMP) or at any time during pregnancy. Participants are pregnant women prescribed pegvaliase by their healthcare provider who enroll through a centralized call center. The study collects data retrospectively from at least three months before the LMP, through pregnancy, and during the infant's first year of life. The timing and duration of pegvaliase exposure during pregnancy and breastfeeding, including each trimester, are recorded. Individual participation lasts up to about 21 months. During the study, information will be gathered from the participant's healthcare providers and the infant's doctors. Researchers will monitor pregnancy outcomes and infant development over ten years, focusing on pegvaliase exposure effects. The study also tracks serious adverse events and pegvaliase use during breastfeeding. This long-term monitoring aims to provide detailed data on maternal and infant health following pegvaliase exposure.
CONDITIONS
Brief Title
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Subject (or legally authorized representative) consent obtained before enrollment
- Confirmed ongoing pregnancy, with classification as prospective or retrospective based on prenatal testing
- Diagnosed with phenylketonuria (PKU) per local standards
- Treated with pegvaliase from 2 weeks prior to last menstrual period (LMP) or during pregnancy
- Agrees to allow investigator to contact healthcare providers for medical information
You will not qualify if you...
- Currently participating in a BioMarin-sponsored interventional study involving any investigational product, device, or procedure
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to approximately 21 months
Participants who have been treated with pegvaliase from 2 weeks prior to their last menstrual period or during pregnancy are observed during pregnancy and throughout the infant's first year of life.
Trial Site Locations
Total: 4 locations
1
Syneos Health
Wilmington, North Carolina, United States, 28403
Actively Recruiting
2
Metabolics and Genetics in Calgary (MAGIC)
Calgary, Canada, T2E 7Z4
Actively Recruiting
3
Universitaetsklinikum Hamburg Eppendorf
Hamburg, Germany, 20246
Actively Recruiting
4
Ospedale San Paolo
Milan, Italy, 20142
Actively Recruiting
Research Team
1
165-504 Program Director
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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