A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Pegvaliase.
- Who it may be relevant to
- Registry conditions: Phenylketonuria, Maternal. Basic parameters: No limits · Female.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Canada, Germany, Italy
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
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.
Interventions
- Drug Pegvaliase
Pregnant women exposed to pegvaliase within 2 weeks prior to LMP.
Primary outcome measures
- 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]
Secondary outcome measures (4)
- Pregnancy Outcomes [Time frame: 10 years]
- Pregnancy Outcomes [Time frame: 10 years]
- Serious Adverse Events [Time frame: 10 years]
- Maternal Pegvaliase use during breastfeeding [Time frame: 10 years]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Study design
- Observational model
- Cohort
Study locations
United States · 1 center
- Syneos Health — Wilmington
Canada · 1 center
- Metabolics and Genetics in Calgary (MAGIC) — Calgary
Germany · 1 center
- Universitaetsklinikum Hamburg Eppendorf — Hamburg
Italy · 1 center
- Ospedale San Paolo — Milan
Identifiers
NCT: NCT05579548 · 165-504