A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

Phenylketonuria (PKU) Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06305234
• Other study ID #: 165-503
• Status: Recruiting
• Start date: January 20, 2024
• Est. completion date: November 18, 2033

Study information

• Verified date: February 2024
• Source: BioMarin Pharmaceutical
• Contact: 165-503 Program Director
• Phone: 1-800-983-4587
• Email: medinfo[@]bmrn.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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 (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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: November 18, 2033
• Est. primary completion date: November 18, 2033
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• 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).

Related Conditions & MeSH terms

• Phenylketonuria (PKU)
• Phenylketonurias

Intervention

Drug:
• Pegvaliase
Must be enrolled in the 165-501 PALace study. Subjects currently receiving or who plan to receive pegvaliase treatment within 30 days after the date of enrollment in the 165-501 study, including subjects who previously received pegvaliase as part of the clinical development program and have completed study participation

Locations

Country	Name	City	State
United States	Tulane University School of Medicine	New Orleans	Louisiana

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate immunologic and inflammatory responses associated with occurrences of	Acute systemic hypersensitivity reaction; Anaphylaxis; Angioedema; Serum sickness; Severe hypersensitivity reaction; Severe or Persistent arthralgia; Severe injection site reaction	A maximum of 10 years treatment duration.
Secondary	Evaluation of immunologic and/or inflammatory responses over time	To evaluate immunologic and inflammatory responses (immunologic testing, inflammatory markers) associated with occurrences of end-organ function (eg, kidney, liver) related, immune-mediated adverse drug reactions (ADRs)*	A maximum of 10 years treatment duration.
Secondary	Evaluation of immunologic responses over time	To evaluate the potential association between immunologic responses and blood Phe levels; * A list of end-organ function related, immune-mediated ADRs is included as an appendix to the statistical analysis plan (SAP).	A maximum of 10 years treatment duration.