Clinical Trials in the USA / 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 USA tracker
Condition(s) studied
Phenylketonuria, Maternal
Investigational drug(s) / intervention(s)
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
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)
| Facility | City | Region | Status |
|---|---|---|---|
| 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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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 the USA