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

Phenylketonuria (PKU) Clinical Trial

Official title:
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

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT05270837
Study type	Interventional
Source	BioMarin Pharmaceutical
Contact
Status	Active, not recruiting
Phase	Phase 3
Start date	June 17, 2022
Completion date	October 2027

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See also
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