Clinical Trial

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

Study acronym: PEGASUS
Active, Not Recruiting Phase 3
View on ClinicalTrials.gov →
Summary
This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.
Protocol Amendment History 18 amendments
This ClinicalTrials.gov record has been amended 18 times since 2022-02-25; most recent amendment 2026-06-23.
Status change: Recruiting → Active, Not Recruiting 2023-09-19
Status change: Not Yet Recruiting → Recruiting 2022-06-06
Trial Details
NCT Number NCT05270837
Lead Sponsor BioMarin Pharmaceutical
Conditions Phenylketonuria (PKU)
Enrollment 55 participants
Start Date 2022-06-17
Primary Completion 2025-01-14 (estimated)
Study Completion 2027-10 (estimated)
Updated on ClinicalTrials.gov 2026-07-21