Status: Ongoing First registered on: 19/07/2021
Last updated on: 19/07/2021
1. Study identification
EU PAS Register NumberEUPAS41426
Official titleA Multicentre, Observational Study to Evaluate the Real-World Outcomes of Palynziq® (Pegvaliase) in Subjects with Phenylketonuria in Germany
Study title acronymOPAL
Study typeObservational study
Brief description of the studyA multicentre observational study evaluating data from routine clinical practice in subjects receiving Pegvaliase for the treatment of PKU. Subjects with PKU who are receiving or have been recommended to receive Pegvaliase to treat their PKU are eligible for participation. The study will have an enrolment period of approximately 12 months and a planned subject data collection period of 24 months. Up to 120 subjects will be enrolled across all sites with a minimum of 70 of these being newly treated (incident) subjects. During the study, relevant medical information will be obtained via review of the subject's medical records. Subjects who consent to participate in the study will be requested to provide relevant PKU disease history, including PKU treatment history, MNT (including medical foods, intact protein foods [natural protein sources], and special low protein foods), concomitant medication use, and any historical patient-centred outcome data collected previously as part of routine clinical practice. Information on all blood Phe levels available will be collected as well as Pegvaliase dosing and titration information and prescribed dietary protein and MNT.
Was this study requested by a regulator?No
Is the study required by a Risk Management Plan (RMP)? Not applicable
Regulatory procedure number (RMP Category 1 and 2 studies only)
Other study registration identification numbers and URLs as applicable
2. Research centres and Investigator details
Coordinating study entity
Centre nameBioMarin
Centre locationSan Rafael, CA, USA
Details of (Primary) lead investigator
Title Dr
Last name Director
First name Program
Is this study being carried out with the collaboration of a research network?
No
Other centres where this study is being conducted
Multiple centres

In total how many centres are involved in this Study?10
Universitätsklinikum Hamburg-Eppendorf (UKE), Hamburg, Germany
Universitätsklinikum Münster (UKM), Munster, Germany
Kreiskliniken Reutlingen, Reutlingen, Germany
Universität Ulm, Ulm, Germany
Universität Leipzig, Leipzig, Germany
Universität Gießen, Giessen, Germany
Klinikum der Universität München, Munich, Germany
Hospital Carl-Thiem-Klinikum Cottbus, Cottbus, Germany
Universitätsklinikum Düsseldorf, Dusseldorf, Germany
Countries in which this study is being conducted
National study

Germany
3. Study timelines: initial administrative steps, progress reports and final report
PlannedActual
Date when funding contract was signed09/04/2021
Start date of data collection20/05/2021
Start date of data analysis
Date of interim report, if expected
Date of final study report31/12/2024
4. Sources of funding
Please provide estimates of the percentage of funding by source for this study
Names(s)Approximate % funding
Pharmaceutical companiesBioMarin100
Charities
Government body
Research councils
EU funding scheme
5. Contact details for enquiries
Scientific Enquiries
Title Dr
Last name Director
First name Program
Address line 1770, Lindaro Street
Address line 2 
Address line 3 
CitySan Rafael 
PostcodeCA 94901 
CountryUnited States
Phone number (incl. country code)0016515230310 
Alternative phone number 
Fax number (incl. country code) 
Email address medinfo@bmrn.com
Public Enquiries
Title Dr 
Last name Director 
First name Program 
Address line 1770, Lindaro Street 
Address line 2 
Address line 3 
CitySan Rafael 
PostcodeCA 94901 
CountryUnited States 
Phone number (incl. country code)0016515230310 
Alternative phone number 
Fax number (incl. country code) 
Email address medinfo@bmrn.com 
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