Clinical trial NCT05579548: A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
Official title: A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Phenylketonuria, Maternal
Keywords: Phase 4
Observational
Maternal
Pregnancy Outcomes
Exposure
Breastfeeding Outcomes
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
Collaborators: name: Syneos Health
First posted: 2022-10-14
Enrollment: 50
Sex: FEMALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Pregnant women with PKU who have been treated with Palynziq (pegvaliase) within 2 weeks of their last menstrual period

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.

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.

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

Primary outcomes:
measure: Pregnancy outcome and infant development for women who have been treated with Palynziq (pegvaliase) within 2 weeks of their last menstrual period
timeFrame: 10 years
Secondary outcomes:
measure: Pregnancy Outcomes
timeFrame: 10 years
measure: Pregnancy Outcomes
timeFrame: 10 years
measure: Serious Adverse Events
timeFrame: 10 years
measure: Maternal Pegvaliase use during breastfeeding
timeFrame: 10 years
Other outcomes:
Locations:
city: Wilmington
country: United States
facility: Syneos Health
state: North Carolina
city: Hamburg
country: Germany
facility: Universitaetsklinikum Hamburg Eppendorf
city: Milano
country: Italy
facility: Ospedale San Paolo
Study officials:
affiliation: BioMarin Pharmaceutical
name: Medical Director, MD
role: STUDY_DIRECTOR
References: