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NCT05579548Recruiting

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

BioMarin Pharmaceutical

Start Date

11/22/2022

Completion Date

10/4/2032

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.

Detailed Description

Women prescribed pegvaliase by their healthcare provider (HCP) who become pregnant during treatment may decide to participate in the study and enroll via a centralized call center model. Upon consent, data will be collected from the patient's HCP(s) retrospectively for at least 3 months prior to Last Menstrual Period (LMP). Data will be collected during pregnancy and throughout the infant's first year of life. Pegvaliase exposure will be recorded during pregnancy and breastfeeding including exposure during each trimester of pregnancy. Duration of individual subject participation will be up to approximately 21 months.

Eligibility Criteria

Age Range: No minimum to No maximum

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

Interventions

DRUG

Pegvaliase

Interested in This Trial?

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Conditions

Phenylketonuria, Maternal

Locations

Syneos Health

Wilmington, North Carolina 28403

United States

Metabolics and Genetics in Calgary (MAGIC)

Calgary, T2E 7Z4

Canada

Universitaetsklinikum Hamburg Eppendorf

Hamburg, 20246

Germany

Ospedale San Paolo

Milan, 20142

Italy