NCT05813678 A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
| NCT ID | NCT05813678 |
| Status | Recruiting |
| Phase | — |
| Sponsor | BioMarin Pharmaceutical |
| Condition | Phenylketonuria (PKU) |
| Study Type | OBSERVATIONAL |
| Enrollment | 450 participants |
| Start Date | 2022-06-06 |
| Primary Completion | 2033-11-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 450 participants in total. It began in 2022-06-06 with a primary completion date of 2033-11-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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.
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
Contact & Investigator
Medical Director, MD
STUDY DIRECTOR
BioMarin Pharmaceutical
Frequently Asked Questions
Who can join the NCT05813678 clinical trial?
This trial is open to participants of all sexes, studying Phenylketonuria (PKU). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT05813678 currently recruiting?
Yes, NCT05813678 is actively recruiting participants. Contact the research team at medinfo@bmrn.com for enrollment information.
Where is the NCT05813678 trial being conducted?
This trial is being conducted at Aurora, United States, Atlanta, United States, Chicago, United States, Indianapolis, United States and 11 additional locations.
Who is sponsoring the NCT05813678 clinical trial?
NCT05813678 is sponsored by BioMarin Pharmaceutical. The principal investigator is Medical Director, MD at BioMarin Pharmaceutical. The trial plans to enroll 450 participants.