What is this study trying to find out about phenylketonuria (PKU) in pregnancy?

This study is observing the effects of pegvaliase treatment on pregnant women with PKU and their babies exposed to pegvaliase during pregnancy and breastfeeding. The research aims to understand maternal, fetal, and infant outcomes over time. It is sponsored by BioMarin Pharmaceutical and helps provide important information for families affected by PKU.

Who may be eligible for this phenylketonuria (PKU) clinical trial?

Women who are pregnant, diagnosed with PKU, and have been treated with pegvaliase within two weeks before their last menstrual period or during pregnancy may be eligible. The study is for females only and requires consent to share medical information with the study team. Please review eligibility criteria for more details.

Is there any cost to participate in this phenylketonuria (PKU) clinical trial?

Study-related care, including the study drug or procedure and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered. Participants should be prepared for any travel costs related to study visits.

Actively Recruiting

FEMALE

ID05579548

Global Study on Effects of Palynziq Pegvaliase Exposure During Pregnancy and Breastfeeding in Women with PKU

Led by BioMarin Pharmaceutical · Updated on 2026-03-24

Participants Needed

Research Sites

N/A

Total Duration

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 infants 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 participants healthcare providers and the infants 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

FEMALE

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

Research Team

165-504 Program Director

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