A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

Phenylketonuria (PKU) Clinical Trial

— PALace  

Official title:

A Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (Pegvaliase) in Subjects With Phenylketonuria

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05813678
• Other study ID #: 165-501
• Secondary ID: EUPAS34992
• Status: Recruiting
• Start date: June 6, 2022
• Est. completion date: November 1, 2033

Study information

• Verified date: May 2024
• Source: BioMarin Pharmaceutical
• Contact: 165-501 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, global, observational study to further characterize the safety profile of pegvaliase, including hypersensitivity reactions, long-term safety and tolerability, and the effectiveness of the additional risk minimization measures (aRMMs) (European Union (EU) only) in subjects receiving pegvaliase for the treatment of PKU. Subjects 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 (incident-users) or have previously started treatment with pegvaliase at the date of enrollment (prevalent-users) are eligible for participation in this study.

Description:

This is a 10-year multi-center, global, observational study to further characterize the safety profile of pegvaliase, including hypersensitivity reactions, long-term safety and tolerability, and the effectiveness of the aRMMs (EU only) in subjects receiving pegvaliase for the treatment of PKU. Subjects 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 (incident-users) or have previously started treatment with pegvaliase at the date of enrollment (prevalent-users) are eligible for participation in this study. Once the subject has been enrolled, the investigator (ie, subject's primary physician) will be asked to provide information about the subject's medical history, treatment(s) received to manage their blood Phe levels, treatment with pegvaliase, and concomitant medication use. The investigator will also be asked to provide evaluation of the following safety concerns: Acute systemic hypersensitivity reaction, Anaphylaxis, Angioedema, Serum sickness, Severe hypersensitivity reaction, Severe or Persistent arthralgia, Severe injection site reaction, Hypophenylalaninemia and other protocol-defined safety events, following the initiation of pegvaliase therapy. There are no protocol-mandated visits or procedures associated with this study. Enrolled subjects should be followed per routine clinical practice at the institution based upon their diagnosis, with clinical outcomes assessed at regular intervals (typically every month during the induction and titration phases of pegvaliase treatment and every 6 months once the maintenance phase is reached). No experimental treatment or assessments are planned as part of this observational study. An Independent Adjudication Committee (IAC) will periodically review protocol-defined safety events, including Acute systemic hypersensitivity reaction and Serum sickness.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 450
• Est. completion date: November 1, 2033
• Est. primary completion date: November 1, 2033
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Documented diagnosis of PKU per local standard of care

• Currently receiving or planned to receive pegvaliase treatment within 30 days after the date of enrollment, including subjects who previously received pegvaliase as part of the clinical development program and have completed study participation.

• Subject (or legally authorized 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.

Exclusion Criteria:

• Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or comply with any aspect of the study.

• Currently participating in an interventional study of any investigational product, device, or procedure

• Previously enrolled in this study (eg, subjects who have been withdrawn from the study and wish to participate again at a later date)

• German subjects <16 years if age

Related Conditions & MeSH terms

• Phenylketonuria (PKU)
• Phenylketonurias

Intervention

Drug:
• Pegvaliase
This is an uncontrolled, single-cohort, Phase 4 observational study not falling under the definition of an Applicable Clinical Trial (ACT) (i.e., non-interventional).

Locations

Country	Name	City	State
Germany	Hospital Carl-Thiem-Klinikum Cottbus	Cottbus
Germany	Universitaetsklinikum Hamburg Eppendorf	Hamburg
Germany	Universitätsklinikum Münster (UKM)-Pädiatrische Universitätsklinik Münster	Münster
Germany	Universität Ulm	Ulm
Italy	Policlinico Sant'orsola Malpighi	Bologna
Italy	University Hospital Careggi	Firenze
Italy	Ospedale San Paolo	Milan
Italy	Azienda Ospedaliera Universitaria "Federico II" Dipartimento di Pediatria	Naples
United States	University of Colorado	Aurora	Colorado
United States	Northwestern University	Chicago	Illinois
United States	University of Texas Southwestern Medical Center	Dallas	Texas
United States	Indiana University School of Medicine	Indianapolis	Indiana
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	Tulane University Medical Center	New Orleans	Louisiana
United States	University of Pittsburgh Medical Center	Pittsburgh	Pennsylvania
United States	Oregon Health and Science University	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

United States,  Germany,  Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To quantify and characterize the risk of protocol-defined safety events in incident-users receiving pegvaliase for the treatment of PKU in a real¬ world setting.	Analyses on incident-users are considered primary. The primary analysis is the incidence rate of: Acute systemic hypersensitivity reaction; Anaphylaxis; Angioedema; Serum sickness; Severe hypersensitivity reaction; Severe or Persistent (= 6 months) or arthralgiaSevere injection site reactionHypophenylalaninemia.	A maximum of 10 years treatment duration.
Secondary	To quantify and characterize the risk of protocol-defined safety events in subjects receiving pegvaliase for the treatment of PKU in a real-world setting.	Secondary analyses under safety events will include: The incidence rate of: End-organ damage associated with immune-complex formation, or PEG accumulation.; SAEs• Severe ADRs; ADRs leading to treatment interruption or discontinuation and/or study discontinuation	A maximum of 10 years treatment follow up duration.