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

BioMarin Pharmaceutical16 sites in 2 countries55 target enrollmentJune 17, 2022

ConditionsPhenylketonuria (PKU)

InterventionsPegvaliase Diet Only

DrugsPegvaliase

Overview

Phase: Phase 3
Intervention: Pegvaliase
Conditions: Phenylketonuria (PKU)
Sponsor: BioMarin Pharmaceutical
Enrollment: 55
Locations: 16
Primary Endpoint: Change in blood Phe concentration
Status: Active, not recruiting
Last Updated: 7 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

June 17, 2022

End Date

October 1, 2027

Last Updated

7 months ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

BioMarin Pharmaceutical

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arms & Interventions

Pegvaliase

Intervention: Pegvaliase

Diet Only

Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72, initiating pegvaliase treatment beginning Week 73 and, from Weeks 73 through 215.

Intervention: Diet Only