Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
- Conditions
- Phenylketonuria (PKU)
- Interventions
- Other: Diet Only
- Registration Number
- NCT05270837
- Lead Sponsor
- BioMarin Pharmaceutical
- 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 55
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Drug: Diet Only 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. Pegvaliase Pegvaliase -
- Primary Outcome Measures
Name Time Method Change in blood Phe concentration Treatment naïve baseline following 72 weeks on study Incidence of treatment-emergent adverse events as assessed by CTCAE v5.0 Treatment naïve baseline following 72 weeks on study
- Secondary Outcome Measures
Name Time Method Change in total dietary protein intake Treatment naïve baseline following 72 weeks on study
Trial Locations
- Locations (16)
Ann and Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
University of Pittsburgh Medical Center
🇺🇸Pittsburgh, Pennsylvania, United States
Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
University of South Florida
🇺🇸Tampa, Florida, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
University of Utah Medical Center
🇺🇸Salt Lake City, Utah, United States
Children's Hospital of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Charité - Universitätsmedizin Berlin
🇩🇪Berlin, Germany
McGovern Medical School, University of Texas Health Science Center at Houston (UTHealth)
🇺🇸Houston, Texas, United States
IU Health University Hospital
🇺🇸Indianapolis, Indiana, United States
Children's Hospital of Colorado
🇺🇸Aurora, Colorado, United States
University of Kentucky College of Medicine
🇺🇸Lexington, Kentucky, United States
University of Virginia School of Medicine
🇺🇸Charlottesville, Virginia, United States
Universitat Mainz
🇩🇪Mainz, Germany
Universitätsklinikum Hamburg-Eppendorf
🇩🇪Hamburg, Germany