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

NCT ID: NCT05579548

Last Updated: 2025-11-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

50 participants

Study Classification

OBSERVATIONAL

Study Start Date

2022-11-22

Study Completion Date

2032-10-04

Brief Summary

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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

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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.

Conditions

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Phenylketonuria, Maternal

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Pregnant Women with PKU

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.

Pegvaliase

Intervention Type DRUG

Pregnant women exposed to pegvaliase within 2 weeks prior to LMP.

Interventions

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Pegvaliase

Pregnant women exposed to pegvaliase within 2 weeks prior to LMP.

Intervention Type DRUG

Eligibility Criteria

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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
Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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Syneos Health

OTHER

Sponsor Role collaborator

BioMarin Pharmaceutical

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director, MD

Role: STUDY_DIRECTOR

BioMarin Pharmaceutical

Locations

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Syneos Health

Wilmington, North Carolina, United States

Site Status RECRUITING

Metabolics and Genetics in Calgary (MAGIC)

Calgary, , Canada

Site Status RECRUITING

Universitaetsklinikum Hamburg Eppendorf

Hamburg, , Germany

Site Status RECRUITING

Ospedale San Paolo

Milan, , Italy

Site Status RECRUITING

Countries

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United States Canada Germany Italy

Central Contacts

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165-504 Program Director

Role: CONTACT

1-800-983-4587

Facility Contacts

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Coordinating Center

Role: primary

833-672-2203

Aneal Khan, Dr

Role: primary

587-885-3158

Professor Muntau

Role: primary

Dr. Rovelli

Role: primary

Related Links

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Other Identifiers

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165-504

Identifier Type: -

Identifier Source: org_study_id

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