The aim of the PEGASUS Study is to see whether an investigational drug, called pegvaliase, is a safe and effective treatment for adolescents with PKU (12 to 17 years of age).
Group 1 – adolescents will be receiving pegvaliase during the study (while maintaining a stable protein diet).
Group 2 – adolescents will be managing their PKU with diet-only (maintaining a stable protein diet) for the first 72 weeks of the study. Following 72 weeks, adolescents will start treatment with pegvaliase.
All adolescents randomized to the diet-only treatment arm will receive pegvaliase treatment after 72 weeks.
Your adolescent is twice as likely to be assigned to Group 1 than to Group 2.
The study will enroll around 54 adolescents across the US and Germany.
The study duration is approximately 3 years.