Recruiting

Palynziq

Sponsor:

BioMarin Pharmaceutical

Code:

NCT05579548

Conditions

Phenylketonuria, Maternal

Eligibility Criteria

Sex: Female

Age: 0+

Healthy Volunteers: Not accepted

Interventions

Pegvaliase

Study Details

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.

Conditions

Phenylketonuria, Maternal

Study ID

NCT05579548

Start date

Nov 22, 2022

Status verified date

Mar, 2026

Completion date

Oct 4, 2032

Anticipated

Primary completion date

Oct 4, 2032

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Female

Age: 0+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

50 participants

Anticipated

Interventions and Outcome Measures

Arms

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.

Interventions

Pegvaliase

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

Primary outcome measure

  • Pregnancy outcome and infant development for women who have been treated with Palynziq (pegvaliase) within 2 weeks of their last menstrual period [ Time Frame: 10 years ]

Central Contacts and Locations

Central contacts

165-504 Program Director

1-800-983-4587medinfo@bmrn.com

Locations

Syneos Health

Recruiting

Wilmington, North Carolina, United States, 28403

Contacts

Coordinating Center

833-672-2203PalynziqPregnancyStudy@syneoshealth.com

Metabolics and Genetics in Calgary (MAGIC)

Recruiting

Calgary, Canada, T2E 7Z4

Contacts

Aneal Khan, Dr

587-885-3158aneal.khan@discoverydna.ca

More Information

Sponsor

BioMarin Pharmaceutical

Last update posted

Mar 24, 2026

Last verified

Mar, 2026

Keywords

  • Phase 4
  • Observational
  • Maternal
  • Pregnancy Outcomes
  • Exposure
  • Breastfeeding Outcomes

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by BioMarin Pharmaceutical on 2026-03-24.