A Multi-Center, Prospective, Longitudinal, Study Evaluating Immunologic, Inflammatory, and Laboratory Parameters Associated With Long-Term Palynziq® (Pegvaliase) Treatment in Subjects With Phenylketonuria (PKU) in the United States
Investigating Long-Term Effects of Investigational Treatment in Phenylketonuria (PKU)
Melissa Lah, MD
Primary Investigator
Brief description of study
This is a 10-year multi-center, prospective, longitudinal, single arm study evaluating immunologic, inflammatory and laboratory parameters associated with long-term Palynziq treatment in subjects with phenylketonuria (PKU) in the United States (US). Subjects in the US for whom a clinical decision has been made that they will receive pegvaliase to treat their PKU within 30 days following the date of enrollment in Study 165-501 (incident-users) or who have previously started treatment with pegvaliase at the date of enrollment in Study 165-501 (prevalent-users) are eligible for participation in Study 165-503.
Detailed description of study
Subjects participating in the 165-501 study who consent to participate in this study (165-503) will be requested to provide pre-dose blood samples for Phe, immunologic and inflammatory marker testing approximately every 3 months for the first 3 years of participation, then every 6 months for the remainder of the study. Subjects will be requested to provide additional pre-dose blood samples at the time of Study 165-501 -protocol defined safety events. Additional pre-dose samples will be drawn as per standard of care as detailed in protocol 165-501. The blood Phe, immunologic (PAL IgG, PEG IgG, PEG IgM, anti-pegvaliase IgE), and inflammatory (C3/C4, hsCRP) markers, will be sent to a central laboratory for processing. Data collected in the 165-501 study will be combined with the data collected in this study to decrease burden on sites for data entry and to avoid duplication of ADR reporting (see Criteria for Evaluation). The combined data will be reported in the Clinical Study Report for this study. Subjects can withdraw from Study 165-503 and remain on Study 165-501, but they cannot withdraw from Study 165-501 and remain on Study 165-503 as safety events and other data-points are collected in Study 165-501.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Phenylketonuria, PKU, Riley
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Age: 100 years or below
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Gender: All
- Subjects enrolled at US sites participating in the 165-501 study.
Exclusion Criteria:
- Legal incapacity or limited legal capacity without legal guardian representation.
- Subject is unable or unwilling to provide informed consent for the additional interventional burden of the study (blood sampling).
This study investigates the effects of long-term treatment on people with phenylketonuria (PKU), a condition where the body cannot break down an amino acid called phenylalanine, which can lead to harmful levels in the body. The purpose of this study is to evaluate immune and inflammatory responses in participants receiving an investigational medication over a long period.
Participants will provide blood samples for testing of phenylalanine levels and immune markers approximately every 3 months for the first 3 years, then every 6 months thereafter. These samples will help researchers understand the body's response to the treatment. The study will combine data from another ongoing study to reduce the burden on study sites and participants.
- Who can participate: Participants must be enrolled in a related study at US sites and able to provide consent. Those with legal incapacity without a guardian or unwilling to provide consent for blood sampling are excluded.
- Study details: Participants will provide blood samples at specified intervals to measure phenylalanine and immune markers. These samples will be used to evaluate the body's response to the investigational treatment.
- Study timelines: The study will last 10 years.
