- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05813678
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) (PALace)
January 3, 2024 updated by: BioMarin Pharmaceutical
A Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (Pegvaliase) in Subjects With Phenylketonuria
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.
Study Overview
Detailed 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 aRMMs (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.
Once the subject has been enrolled, the investigator (ie, subject's primary physician) will be asked to provide information about the subject's medical history, treatment(s) received to manage their blood Phe levels, treatment with pegvaliase, and concomitant medication use.
The investigator will also be asked to provide evaluation of the following safety concerns: Acute systemic hypersensitivity reaction, Anaphylaxis, Angioedema, Serum sickness, Severe hypersensitivity reaction, Severe or Persistent arthralgia, Severe injection site reaction, Hypophenylalaninemia and other protocol-defined safety events, following the initiation of pegvaliase therapy.
There are no protocol-mandated visits or procedures associated with this study.
Enrolled subjects should be followed per routine clinical practice at the institution based upon their diagnosis, with clinical outcomes assessed at regular intervals (typically every month during the induction and titration phases of pegvaliase treatment and every 6 months once the maintenance phase is reached).
No experimental treatment or assessments are planned as part of this observational study.
An Independent Adjudication Committee (IAC) will periodically review protocol-defined safety events, including Acute systemic hypersensitivity reaction and Serum sickness.
Study Type
Observational
Enrollment (Estimated)
450
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Medical Director, MD
- Phone Number: 1-800-983-4587
- Email: medinfo@bmrn.com
Study Contact Backup
- Name: 165-501 Program Director
- Phone Number: 1-800-983-4587
- Email: medinfo@bmrn.com
Study Locations
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Cottbus, Germany
- Recruiting
- Hospital Carl-Thiem-Klinikum Cottbus
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Hamburg, Germany
- Recruiting
- Universitaetsklinikum Hamburg Eppendorf
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Münster, Germany
- Recruiting
- Universitätsklinikum Münster (UKM)-Pädiatrische Universitätsklinik Münster
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Ulm, Germany
- Recruiting
- Universitat Ulm
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Bologna, Italy
- Recruiting
- Policlinico Sant'Orsola Malpighi
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Firenze, Italy
- Recruiting
- University Hospital Careggi
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Milan, Italy
- Recruiting
- Ospedale San Paolo
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Naples, Italy
- Recruiting
- Azienda Ospedaliera Universitaria "Federico II" Dipartimento di Pediatria
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Indiana
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Indianapolis, Indiana, United States, 46202
- Recruiting
- Indiana University School of Medicine
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Oregon
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Portland, Oregon, United States, 97239
- Recruiting
- Oregon Health and Science University
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Sampling Method
Non-Probability Sample
Study Population
All subjects with PKU followed at participating centers, currently receiving or planning to receive pegvaliase treatment.
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
Time Frame: A maximum of 10 years treatment duration.
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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 arthralgiaSevere injection site reactionHypophenylalaninemia. |
A maximum of 10 years treatment duration.
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
Time Frame: A maximum of 10 years treatment duration.
|
Secondary analyses under safety events will include: The incidence rate of:
|
A maximum of 10 years treatment duration.
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Medical Director, MD, BioMarin Pharmaceutical
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
June 6, 2022
Primary Completion (Estimated)
November 1, 2033
Study Completion (Estimated)
November 1, 2033
Study Registration Dates
First Submitted
April 3, 2023
First Submitted That Met QC Criteria
April 3, 2023
First Posted (Actual)
April 14, 2023
Study Record Updates
Last Update Posted (Actual)
January 5, 2024
Last Update Submitted That Met QC Criteria
January 3, 2024
Last Verified
January 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 165-501
- EUPAS34992 (Registry Identifier: European Network of Centres for Pharmacoepidemiology and Pharmacovigilance)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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