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Clinical Trials in Italy / NCT05579548
Recruiting Observational

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

NCT05579548 · tracked via the Priya Life Science Italy tracker
Sponsor
BioMarin Pharmaceutical
Phase
Observational
Started
2022-11-22
Last updated
2026-03-24

Condition(s) studied

Phenylketonuria, Maternal

Investigational drug(s) / intervention(s)

Pegvaliase →

Pegvaliase: Pregnant women exposed to pegvaliase within 2 weeks prior to LMP.

Study 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.

Eligibility

Sex
FEMALE
Min age
—
Max age
—
Healthy volunteers
No
Inclusion Criteria: * Subject (or a legally authorized representative) consent obtained prior to enrollment. Consent will be obtained in compliance with any country-specific regulations or requirements * Confirmation of ongoing pregnancy. Subjects with unknown outcomes will be classified as prospective pregnancies; subjects who have undergone prenatal testing (eg, targeted ultrasound, amniocentesis) regardless of findings will be classified as retrospective pregnancies * Diagnosed with PKU per local standard of care * Documentation that the subject was treated with pegvaliase at any point starting from 2 weeks prior to the date of LMP * Agrees to permit the Investigator (ie, CRP, CCA, PI) to contact the subject's HCPs (eg, PCP, PKU-treating physician, OB, nurse, midwife) and the infant's HCP (eg, pediatrician, neonatologist) for medical information Exclusion Criteria: • Currently participating in a BioMarin sponsored interventional study of any investigational product, device, or procedure

Primary outcome measure(s)

  • Pregnancy outcome and infant development for women who have been treated with Palynziq (pegvaliase) within 2 weeks of their last menstrual period — 10 years
    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.

Trial sites (4)

FacilityCityRegionStatus
Syneos Health Wilmington North Carolina Recruiting
Metabolics and Genetics in Calgary (MAGIC) Calgary Canada Recruiting
Universitaetsklinikum Hamburg Eppendorf Hamburg Germany Recruiting
Ospedale San Paolo Milan Italy Recruiting

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Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia Recruiting
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia Active, not recruiting
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment Active, not recruiting
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05579548 on ClinicalTrials.gov ↗ ← All trials in Italy