A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
Recruiting now
Conditions studied: Phenylketonuria, Maternal
In brief
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.
Key facts
- Study ID
- NCT05579548
- Run by
- BioMarin Pharmaceutical
- People needed
- 50
- Starts
- 2022-11-22
- Expected to finish
- 2032-10-04
- Last updated by the study team
- 2026-03-24
Who can join
Age: any. Sex: female. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- Currently participating in a BioMarin sponsored interventional study of any investigational product, device, or procedure
Where it is running
- Syneos Health — Wilmington, North Carolina, United States (enrolling)
- Metabolics and Genetics in Calgary (MAGIC) — Calgary, Canada (enrolling)
- Universitaetsklinikum Hamburg Eppendorf — Hamburg, Germany (enrolling)
- Ospedale San Paolo — Milan, Italy (enrolling)
Full record on ClinicalTrials.gov
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