A Multicenter, Observational Study to Evaluate the Real-World Outcomes of Palynziq (Pegvaliase) in Subjects with Phenylketonuria (OPAL)
M
Melissa Lah, MD
Primary Investigator
Enrolling By Invitation
17 years or below
All
Phase
N/A
5 participants needed
1 Location
Brief description of study
What is the purpose of this study?
This is a multicenter, observational study of adults receiving pegvaliase for the treatment of PKU. Pegvaliase is a Phe-metabolising enzyme indicated to reduce blood Phe concentrations in patients with PKU who have uncontrolled blood Phe concentrations > 600 μmol/L on existing management. The study will collect data from routine clinical practice and questionnaires, and evaluate outcomes achieved with pegvaliase in a real-world setting as measured by blood phenylalanine (Phe) level over time. The study hopes to learn about real-world usage of pegvaliase and associated outcomes to support clinical practice optimization and provide greater understanding of important clinical issues in PKU.
THIS STUDY IS ENROLLING BY INVITATION ONLY - Potential subjects will be identified from the Palynziq (Pegvaliase) Clinic held at the Pediatric Care Center, and staffed by the PI and colleagues. They will be contacted in person and/or via phone.
Detailed description of study
What happens during study participation?
- The decision to treat the subject with pegvaliase will have already been made independently of the subject’s enrolment onto this study. Treatment with pegvaliase will, therefore, not be supplied by BioMarin as part of the subject’s participation on this study. Pegvaliase will be prescribed by the subject’s treating physician in accordance with routine clinical practice.
- Participants who have been prescribed pegvaliase should undergo monthly testing of their blood Phe levels; however, the specific requirements for testing may vary based on routine clinical practice for individual participants at each site.
- Information on all blood Phe levels available will be collected for this study.
- Their pegvaliase dosing and titration information and prescribed dietary protein and MNT will be recorded by the treating physician as in current clinical practice.
- Each participant will be followed for approximately 24 months after study enrollment.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Phenylketonuria, PKU, Riley
-
Age: 17 years or below
-
Gender: All
Inclusion Criteria:
1. Documented diagnosis of PKU per routine clinical practice.
2. Planned to receive pegvaliase treatment as per SmPC or USPI or currently receiving
pegvaliase and has not reached controlled blood Phe levels (defined as blood Phe
levels of > 600 μmol/L, demonstrated by 3 recent consecutive blood Phe
measurements), documented as part of their routine clinical care.
3. Subject or legally authorised representative is willing and able to provide written
informed consent after the nature of the study has been explained and prior to any
data collection.
4. Fluent in written and spoken local language.
1. Documented diagnosis of PKU per routine clinical practice.
2. Planned to receive pegvaliase treatment as per SmPC or USPI or currently receiving
pegvaliase and has not reached controlled blood Phe levels (defined as blood Phe
levels of > 600 μmol/L, demonstrated by 3 recent consecutive blood Phe
measurements), documented as part of their routine clinical care.
3. Subject or legally authorised representative is willing and able to provide written
informed consent after the nature of the study has been explained and prior to any
data collection.
4. Fluent in written and spoken local language.
Exclusion Criteria:
1. Legal incapacity or limited legal capacity without a legally authorized
representative.
2. Currently participating in an interventional study of any investigational product,
device, or procedure.
1. Legal incapacity or limited legal capacity without a legally authorized
representative.
2. Currently participating in an interventional study of any investigational product,
device, or procedure.
Updated on
08 Apr 2025.
Study ID: MMGE-BIOMARIN-165-508, 20145
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