Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
- Registration Number
- NCT03694353
- Lead Sponsor
- BioMarin Pharmaceutical
- Brief 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A subject who reduces to a dose of 40 mg/day or lower for 32 consecutive weeks will be discontinued from study drug and withdrawn from the study as they will have the option to transition to commercial drug.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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 37
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Pegvaliase Pegvaliase Beginning on Day 1, subjects will receive the same dose and regimen of pegvaliase they were receiving in 165-302 or PAL-003 (pegvaliase dosing should continue without interruption from the previous study). Subsequent revisions to dosing regimens are allowed following consultation with the medical monitor.
- Primary Outcome Measures
Name Time Method Number of Participants With Treatment-emergent Adverse Events (TEAEs) as Assessed by CTCAE v5.0 Up to Day 741 (approximately Week 106) A treatment-emergent AE was defined as any adverse event (AE) newly appearing or worsened in severity following initiation of study drug until 4 weeks after last dose of pegvaliase
Change in Blood Phe Concentration The Outcome Measure Data Table below uses the 'analysis visit' as defined by the mapping rule in the SAP. The last Phe measurement was mapped to 'analysis visit' week 121. The actual date of the last Phe measurement was day 836 (approximately Week 119). Change in blood phenylalanine (Phe) concentration from Parent Study baseline (naïve/pretreatment).
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (17)
University of Missouri Health Care
🇺🇸Columbia, Missouri, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
University of Florida
🇺🇸Gainesville, Florida, United States
Indiana CTSI Clinical Research Center
🇺🇸Indianapolis, Indiana, United States
Morristown Medical Center
🇺🇸Morristown, New Jersey, United States
St. Christopher's Hospital for Children
🇺🇸Philadelphia, Pennsylvania, United States
University of Nebraska Medical Center
🇺🇸Omaha, Nebraska, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Medical College of Milwaukee
🇺🇸Milwaukee, Wisconsin, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
University of South Florida
🇺🇸Tampa, Florida, United States
University of Texas Health Science Center at Houston
🇺🇸Houston, Texas, United States
Wayne State University, Center for Molecular Medicine and Genetics, Pediatrics and Pathology
🇺🇸Detroit, Michigan, United States
University of Kentucky Medical Center
🇺🇸Lexington, Kentucky, United States
University of Oklahoma Health Science Center
🇺🇸Oklahoma City, Oklahoma, United States