Ignite Creation Date:
2024-05-06 @ 8:14 PM
Last Modification Date:
2024-10-26 @ 3:23 PM
Study NCT ID:
NCT06305234
Status:
RECRUITING
Last Update Posted:
2024-03-12
First Post:
2024-01-30
Brief Title:
A Long Term Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU PALisade
Sponsor:
BioMarin Pharmaceutical
Organization:
BioMarin Pharmaceutical
Study Overview
Official Title:
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
Status:
RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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:
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 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 C3C4 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None