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

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-510875-39-00

Acronym

165-306

Enrollment

Registered

2024-07-04

Start date

2022-10-25

Completion date

Unknown

Last updated

2025-12-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Phenylketonuria (PKU)

Brief summary

Safety will be evaluated by assessment of AEs, SAEs, laboratory test results (chemistry, hematology, and urinalysis), vital signs physical examination, ECG and anti-BMN165 immunogenicity test results

Detailed description

Change from baseline in total dietary protein intake (ie, medical food and/or intact food)., Plasma sample for trough PK

Interventions

DRUGPalynziq 20 mg solution for injection in pre-filled syringe

DRUGPalynziq 10 mg solution for injection in pre-filled syringe

DRUGPalynziq 2.5 mg solution for injection in pre-filled syringe

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Safety will be evaluated by assessment of AEs, SAEs, laboratory test results (chemistry, hematology, and urinalysis), vital signs physical examination, ECG and anti-BMN165 immunogenicity test results	—

Secondary

Measure	Time frame
Change from baseline in total dietary protein intake (ie, medical food and/or intact food)., Plasma sample for trough PK	—

Countries

Germany

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026