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

BioMarin Pharmaceutical

Status: Enrolling

Conditions: Phenylketonuria, Maternal

Treatments: Drug: Pegvaliase

Study type: Observational

Funder types: Other; Industry

Identifiers

NCT05579548

165-504

Details and patient eligibility

About

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.

Full 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.

Enrollment: 50 estimated patients

Sex: Female

Volunteers: No Healthy Volunteers

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

Trial design

50 participants in 1 patient group

Pregnant Women with PKU

Description:

This study will enroll pregnant women diagnosed with PKU who have been treated with pegvaliase from 2 weeks prior to LMP or at any time during pregnancy.

Treatment:

Drug: Pegvaliase