Clinical trial NCT05813678: A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
Official title: A Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (Pegvaliase) in Subjects With Phenylketonuria
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Phenylketonuria (PKU)
Keywords: Observational
Safety Study
Pegvaliase
Palynziq
PKU
Phenylketonuria
Phase 4
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2023-04-14
Enrollment: 450
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All subjects with PKU followed at participating centers, currently receiving or planning to receive pegvaliase treatment.

Brief 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: 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.
timeFrame: A maximum of 10 years treatment duration.
Secondary outcomes:
measure: 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.
timeFrame: A maximum of 10 years treatment follow up duration.
Other outcomes:
Locations:
city: Aurora
country: United States
facility: University of Colorado
state: Colorado
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University School of Medicine
state: Indiana
city: New Orleans
country: United States
facility: Tulane University Medical Center
state: Louisiana
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: Cottbus
country: Germany
facility: Hospital Carl-Thiem-Klinikum Cottbus
city: Hamburg
country: Germany
facility: Universitaetsklinikum Hamburg Eppendorf
city: Münster
country: Germany
facility: Universitätsklinikum Münster (UKM)-Pädiatrische Universitätsklinik Münster
city: Ulm
country: Germany
facility: Universität Ulm
city: Bologna
country: Italy
facility: Policlinico Sant'orsola Malpighi
city: Firenze
country: Italy
facility: University Hospital Careggi
city: Milan
country: Italy
facility: Ospedale San Paolo
city: Naples
country: Italy
facility: Azienda Ospedaliera Universitaria "Federico II" Dipartimento di Pediatria
Study officials:
affiliation: BioMarin Pharmaceutical
name: Medical Director, MD
role: STUDY_DIRECTOR
References: