NCT03694353

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

An Open-label Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase (> 40 mg/Day Dose) in Adults With Phenylketonuria

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 37 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 3 open-label extension study enrolling adult patients with PKU who were previously treated with pegvaliase in Studies PAL-003 (NCT00924703) or 165-302 (NCT02468570). The study is designed to evaluate the long-term safety and efficacy of pegvaliase administered at doses \> 40 mg/day to 60 mg/day.

Detailed description

Pegvaliase dosing will continue without interruption from Parent Study 165-302 (NCT02468570) or Parent Study PAL-003 (NCT00924703). Beginning on Day 1, subjects will receive the same dose and regimen of pegvaliase they were receiving in either 165-302 or PAL-003. A subject who reduces to a dose of 40 mg/day or lower for 32 consecutive weeks will be discontinued from study drug and withdrawn from the study as they will have the option to transition to commercial drug. Dose reductions may be performed if warranted due to AEs or hypophenylalaninemia. Dose increases to up to 60 mg/day may be performed per investigator discretion in consultation with the sponsor's medical monitor. Dosing will continue for approximately 121 weeks.

Conditions

• Phenylketonuria (PKU)

Interventions

Type	Name	Description
DRUG	Pegvaliase	40-60mg/day

Timeline

• Start date: 2018-09-13
• Primary completion: 2021-01-13
• Completion: 2021-01-13
• First posted: 2018-10-03
• Last updated: 2022-02-17
• Results posted: 2022-02-17

Locations

17 sites across 1 country: United States

Regulatory

• FDA-regulated drug study