RECRUITING

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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) (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.

Official Title

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

Quick Facts

Study Start:2022-06-06
Study Completion:2033-11-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05813678

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.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Documented diagnosis of PKU per local standard of care
  2. * 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.
  3. * 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.
  1. * 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.
  2. * Currently participating in an interventional study of any investigational product, device, or procedure
  3. * Previously enrolled in this study (eg, subjects who have been withdrawn from the study and wish to participate again at a later date)
  4. * German subjects \<16 years if age

Contacts and Locations

Study Contact

165-501 Program Director
CONTACT
1-800-983-4587
medinfo@bmrn.com
Medical Director, MD
CONTACT
1-800-983-4587
medinfo@bmrn.com

Principal Investigator

Medical Director, MD
STUDY_DIRECTOR
BioMarin Pharmaceutical

Study Locations (Sites)

University of Colorado
Aurora, Colorado, 80045
United States
Northwestern University
Chicago, Illinois, 60611
United States
Indiana University School of Medicine
Indianapolis, Indiana, 46202
United States
Tulane University Medical Center
New Orleans, Louisiana, 70118
United States
Oregon Health and Science University
Portland, Oregon, 97239
United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224
United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
United States

Collaborators and Investigators

Sponsor: BioMarin Pharmaceutical

  • Medical Director, MD, STUDY_DIRECTOR, 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 Registration Dates

Study Start Date2022-06-06
Study Completion Date2033-11-01

Study Record Updates

Study Start Date2022-06-06
Study Completion Date2033-11-01

Terms related to this study

Keywords Provided by Researchers

  • Observational
  • Safety Study
  • Pegvaliase
  • Palynziq
  • PKU
  • Phenylketonuria
  • Phase 4

Additional Relevant MeSH Terms

  • Phenylketonuria (PKU)