A Multicentre, Observational Study to Evaluate the Real-World Outcomes of Palynziq® (Pegvaliase) in Subjects with Phenylketonuria (OPAL)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Other
• If ‘other’, further details on the scope of the study:
  Provide real-world evidence data on use of Palynziq in PKU

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Observational study on routine clinical practice

Study drug and medical condition

• Name of medicine:
  PALYNZIQ
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AB19) pegvaliase

  pegvaliase
• Medical condition to be studied:
  Phenylketonuria

Population studied

• Age groups:
  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Special population of interest:
  Pregnant women
• Estimated number of subjects:
  150

Study design details

• Main study objective:
  The efficacy and safety of Pegvaliase has been established in clinical trials as required for regulatory approval. There remains an important need to provide data to prescribers and the phenylketonuria (PKU) community on the real-world usage of Pegvaliase and associated outcomes to support clinical practice optimisation and provide greater understanding of important clinical issues in PKU.
• Outcomes:
  To evaluate outcomes achieved with Pegvaliase in a real-world setting as measured by blood phenylalanine (Phe) level over time. Describe: dosing intervals and dosing of Pegvaliase in each phase of treatment and length of titration phase, reported MNT in each treatment phase, relationship between changes in reported MNT and blood Phe, co-medications of interest used in each treatment phase, impact of blood Phe on subjects' wellbeing and health-related Quality of Life and impact of blood Phe on socioeconomic parameters.
• Data analysis plan:
  Data will be collected from patients who are receiving Pegvaliase as part of their routine clinical care undertaken by their PKU treating physician. No formal hypothesis testing or inference will be made. The study will use descriptive summary statistics and various statistical analysis methods for longitudinal, repeated measures data for estimation and summarisation of outcomes, including statistical modelling and graphical methods.