Assessing the effects of Palynziq during pregnancy and breastfeeding
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
BioMarin Pharmaceutical · NCT05579548
This study looks at how the medication Palynziq affects women with PKU and their babies during pregnancy and breastfeeding.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 50 (estimated) |
| Sex | Female |
| Sponsor | BioMarin Pharmaceutical (industry) |
| Locations | 4 sites (Wilmington, North Carolina and 3 other locations) |
| Trial ID | NCT05579548 on ClinicalTrials.gov |
What this trial studies
This observational study aims to evaluate the maternal, fetal, and infant outcomes of women with phenylketonuria (PKU) who are treated with Palynziq (pegvaliase) during pregnancy and breastfeeding. Participants will be women who have been prescribed pegvaliase and become pregnant, with data collected retrospectively from healthcare providers for at least three months prior to the last menstrual period. The study will track pegvaliase exposure throughout pregnancy and the infant's first year of life, with participation lasting up to approximately 21 months.
Who should consider this trial
Good fit: Ideal candidates for this study are pregnant women diagnosed with PKU who have been treated with pegvaliase.
Not a fit: Patients who are not currently pregnant or who have not been treated with pegvaliase will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the safety and effects of pegvaliase exposure during pregnancy and breastfeeding for both mothers and their infants.
How similar studies have performed: While this study is observational and focuses on a specific population, similar studies assessing drug exposure during pregnancy have shown varying degrees of success in understanding maternal and infant outcomes.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Wilmington, North Carolina and 3 other locations
- Syneos Health — Wilmington, North Carolina, United States (RECRUITING)
- Metabolics and Genetics in Calgary (MAGIC) — Calgary, Canada (RECRUITING)
- Universitaetsklinikum Hamburg Eppendorf — Hamburg, Germany (RECRUITING)
- Ospedale San Paolo — Milan, Italy (RECRUITING)
Study contacts
- Study coordinator: 165-504 Program Director
- Email: medinfo@bmrn.com
- Phone: 1-800-983-4587
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Phenylketonuria, Maternal, Phase 4, Observational, Maternal, Pregnancy Outcomes, Exposure, Breastfeeding Outcomes