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

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/50385
• EU PAS number:
  EUPAS35156
• Study ID:
  50385
• Official title and acronym:
  A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (pegvaliase) During Pregnancy and Breastfeeding (PALomino)
• DARWIN EU® study:
  No
• Study countries:
  Canada

  Germany

  Italy

  United States
• Study description:
  This is an observational, prospective surveillance study of subjects with Phenylketonuria (PKU) exposed to pegvaliase during pregnancy. No study medication is provided as part of participation and all direction for medication usage is at the discretion of the prescribing physician in accordance with standard practice and the local label. The assignment of a subject to pegvaliase is not decided in advance or influenced by the study protocol, and the decision to prescribe pegvaliase is independent of the decision to include the subject in the study. Retrospective data collection of pegvaliase exposure and disease data will be collected for at least 3 months prior to Last Menstrual Period (LMP). Pegvaliase exposure will also be recorded during pregnancy and breastfeeding including exposure during each trimester of pregnancy. For these measures, first trimester exposure will be defined as any dose between 2 weeks prior to the first day of LMP and 13 weeks gestation, second trimester as 14 weeks through 27 weeks gestation, and third trimester as 28 weeks gestation onwards. Additionally, ongoing pegvaliase exposure will be recorded among subjects with live-birth outcomes who initiate breastfeeding, as long as breastfeeding continues up to (and not past) infant age of 12 months.
• Study status:
  Ongoing

Research institution and networks

Institutions

BioMarin Pharmaceuticals

Study timelines

Date when funding contract was signed

Planned: 01/01/2020

Actual: 08/06/2020

Study start date

Planned: 01/12/2022

Actual: 22/11/2022

Data analysis start date

Planned: 31/08/2025

Date of interim report, if expected

Planned: 15/12/2025

Actual: 21/12/2021

Date of final study report

Planned: 31/05/2033

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

BioMarin Pharmaceutical Inc.

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)