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

Study identification

• EU PAS number:
  EUPAS34992
• Study ID:
  49099
• Official title and acronym:
  A Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (pegvaliase) in Subjects with Phenylketonuria (PALace)
• DARWIN EU® study:
  No
• Study countries:
  Germany

  Italy

  United States
• 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 additional risk minimization measures (aRMMs) 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.
• Study status:
  Ongoing

Research institutions and networks

Institutions

BioMarin Pharmaceuticals

Study timelines

Date when funding contract was signed

Planned: 01/01/2019

Actual: 18/11/2020

Study start date

Planned: 28/02/2022

Actual: 23/06/2022

Data analysis start date

Planned: 09/08/2023

Actual: 24/08/2023

Date of interim report, if expected

Planned: 21/12/2025

Actual: 20/12/2021

Date of final study report

Planned: 31/05/2033

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

BioMarin Pharmaceutical Inc.

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)