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

NCT05270837 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 55

Last updated 2025-09-12

No results posted yet for this study

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.

Conditions

  • Phenylketonuria (PKU)

Interventions

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

Sponsors & Collaborators

Principal Investigators

  • Study Director · BioMarin Pharmaceutical

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
12 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-06-17
Primary Completion
2025-01-14
Completion
2027-10-31
FDA Drug
Yes

Countries

  • United States
  • Germany

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05270837 on ClinicalTrials.gov