drugset
drugset / Trial / NCT05270837

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

NCT05270837 ↗

Phase 3 Active not recruiting 55 enrolled BioMarin Pharmaceutical
RandomizedParallel-groupOpen-labelTreatment

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.

Timeline

Start
2022-06-17
Primary completion
2025-01-14
Completion
2027-10

Drugs

EvaluationDrugModalityDoseRoute
Subject Pegvaliase Protein / enzyme biologic 2.5 mg Subcutaneous
Subject Pegvaliase Protein / enzyme biologic 10 mg Subcutaneous
Subject Pegvaliase Protein / enzyme biologic 20 mg Subcutaneous
Subject Pegvaliase Protein / enzyme biologic 40 mg Subcutaneous
Subject Pegvaliase Protein / enzyme biologic 60 mg Subcutaneous

Indications

phenylketonuria