NCT05270837

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

A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) With Phenylketonuria Featuring an Open-Label Randomized Two-Arm (Active vs Diet-Only Control) Design

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 55 (estimated)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 12 Years – 17 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This Phase 3 multicenter study is designed to evaluate the safety and efficacy of pegvaliase administered daily to adolescents (ages 12 to 17 years old (US), inclusive, and 12 to 15 years old (EU), inclusive 12-17) with phenylketonuria (PKU). Participants will be randomized in a 2:1 ratio to the active (pegvaliase) and control (diet-only) treatment arms, respectively, with 36 participants receiving pegvaliase and 18 participants managing their PKU with diet alone.

Conditions

• Phenylketonuria (PKU)

Interventions

Type	Name	Description
DRUG	Pegvaliase	Pegvaliase 2.5mg/10mg/20mg/40mg/60mg self-administered from 1 time up to 7 times a week
OTHER	Diet Only	Diet Control

Timeline

• Start date: 2022-06-17
• Primary completion: 2025-01-14
• Completion: 2027-10-01
• First posted: 2022-03-08
• Last updated: 2025-09-12

Locations

16 sites across 2 countries: United States, Germany

Regulatory

• FDA-regulated drug study