NCT05579548 A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
| NCT ID | NCT05579548 |
| Status | Recruiting |
| Phase | — |
| Sponsor | BioMarin Pharmaceutical |
| Condition | Phenylketonuria, Maternal |
| Study Type | OBSERVATIONAL |
| Enrollment | 50 participants |
| Start Date | 2022-11-22 |
| Primary Completion | 2032-10-04 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 50 participants in total. It began in 2022-11-22 with a primary completion date of 2032-10-04.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 Criteria
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
Contact & Investigator
Medical Director, MD
STUDY DIRECTOR
BioMarin Pharmaceutical
Frequently Asked Questions
Who can join the NCT05579548 clinical trial?
This trial is open to female participants only, studying Phenylketonuria, Maternal. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT05579548 currently recruiting?
Yes, NCT05579548 is actively recruiting participants. Contact the research team at medinfo@bmrn.com for enrollment information.
Where is the NCT05579548 trial being conducted?
This trial is being conducted at Wilmington, United States, Calgary, Canada, Hamburg, Germany, Milan, Italy.
Who is sponsoring the NCT05579548 clinical trial?
NCT05579548 is sponsored by BioMarin Pharmaceutical. The principal investigator is Medical Director, MD at BioMarin Pharmaceutical. The trial plans to enroll 50 participants.