Study identification

PURI

https://redirect.ema.europa.eu/resource/50667

EU PAS number

EUPAS41426

Study ID

50667

Official title and acronym

A Multicentre, Observational Study to Evaluate the Real-World Outcomes of Palynziq® (Pegvaliase) in Subjects with Phenylketonuria (OPAL)

DARWIN EU® study

No

Study countries

Germany
Italy
United States

Study description

A 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 32 months and a planned subject data collection period of 24 months. Up to 150 subjects will be enrolled across all sites. 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 patient-centred outcome data collected 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.

Study status

Ongoing
Research institution and networks

Institutions

University of Ulm
Germany
First published:
01/02/2024
Institution
Educational Institution
Universitätsklinikum Münster (UKM) Munster, Germany
Kreiskliniken Reutlingen Reutlingen, Germany
Universität Leipzig Leipzig, Germany
Universität Gießen Giessen, Germany
Hospital Carl-Thiem-Klinikum Cottbus Cottbus, Germany
Universitätsklinikum Düsseldorf Dusseldorf, Germany
Klinikum Bremen Mitte Bremen,Germany
Universitäasklinikum Zentrum fur Kinder und Jugenmedizin Mainz, Germany
Policlinico S.Orsola Malpighi, AOU di Bologna Bologna, Italy
Ospedale San Paolo Milano, Italy
University of Kentucky Lexington, KY, United States
St. Christopher's Hospital for Children Philadelphia, PA, United States
Oregon Health & Science University Portland, OR, United States
Indiana University School of Medicine Indianapolis, IN, United States
Children's Hospital of Pittsburgh Pittsburgh, PA, United States
University of Texas Southwestern Medical Center Dallas, TX, United States
University of Texas Houston Medical School Houston, TX, United States
Emory University Decatur, Georgia, United States
Children's Hospital Colorado Aurora, Colorado, United States
Tulane University Medical Center New Orleans, LA, United States
Ann and Robert H. Lurie Children's Hospital of Chicago Chicago, IL, United States
University of Florida (Gainesville) Gainesville, FL, United States

Contact details

165-508 Program Director

Primary lead investigator
Study timelines

Date when funding contract was signed

Actual:

Study start date

Actual:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

BioMarin
Regulatory

Was the study required by a regulatory body?

No

Is the study required by a Risk Management Plan (RMP)?

Not applicable