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

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  PALYNZIQ
• Study drug International non-proprietary name (INN) or common name:
  PEGVALIASE
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AB19) pegvaliase

  pegvaliase
• Medical condition to be studied:
  Phenylketonuria

Population studied

• Age groups:
  • Paediatric Population (< 18 years)

    • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
• Special population of interest:
  Pregnant women
• Estimated number of subjects:
  70

Study design details

• Main study objective:
  The purpose of this observational study is to further increase knowledge about the outcomes of pregnant women with PKU and their offspring exposed to pegvaliase during pregnancy and breastfeeding.
• Outcomes:
  Estimate the frequency of pregnancy outcomes (eg,spontaneous abortion,stillbirth,live birth,and termination) among subjects with PKU treated with pegvaliase during pregnancy and fetal/infant outcomes (all major congenital malformations and specifically microcephaly and congenital heart defects, FGR, small for gestational age,low birth weight,preterm birth,failure to thrive,and developmental delays, Pregnancy/infant outcomes among pegvaliase treated/untreated. Differences in pregnancy/infant outcomes by maternal Phe level. SAEs other than congenital malformations (CMs) in infants through their first year of life. Outcomes of subjects treated with pegvaliase during breastfeeding (low milk supply) and their infants (failure to thrive and SAEs) through their first year of life.
• Data analysis plan:
  The primary analysis is the prevalence of pregnancy outcomes, including major congenital malformations. A 95% exact confidence interval of the prevalence will also be calculated. Results from each pregnancy outcome will also be descriptively compared with published data on outcomes in non-pegvaliase exposed PKU pregnancies and, when appropriate, general population reference literature. Analysis of secondary endpoints will include prevalence of each pregnancy outcome by varying blood Phe concentration levels. In addition, the number and incidence of infant SAEs and infants who meet the failure to thrive criteria will be provided. The number and incidence of low milk supply among breastfeeding women will be examined as well. Additional analysis will include the number and proportion of major congenital malformations, infant hospitalization, and infant death. Tabulations of patient (and infant) characteristics at enrolment (and at birth) and during the study will be summarized.