A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
ConditionPhenylketonuria (PKU)
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorBioMarin Pharmaceutical
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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Previously enrolled in this study (eg, subjects who have been withdrawn from the study and wish to participate again at a later date)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
Trial population
All subjects with PKU followed at participating centers, currently receiving or planning to receive pegvaliase treatment.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Pegvaliase
DrugThis is an uncontrolled, single-cohort, Phase 4 observational study not falling under the definition of an Applicable Clinical Trial (ACT) (i.e., non-interventional).
Treatment groups
No treatment groups listed
Trial outcomes
Primary outcomes
1
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 arthralgia Severe injection site reaction Hypophenylalaninemia
Time frame
A maximum of 10 years treatment duration.Secondary outcomes
1
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
Time frame
A maximum of 10 years treatment follow up duration.Sponsors and contacts
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Source ClinicalTrials.gov