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NCT05813678 · ClinicalTrials.gov registry record
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
A clinical trial of Phenylketonuria (PKU), sponsored by BioMarin Pharmaceutical.
- Recruiting
- Registry status
- 450
- Enrollment target
- 16
- Study locations
NCT05813678: Recruiting study of Phenylketonuria (PKU), sponsored by BioMarin Pharmaceutical.
NCT05813678 is a study of Phenylketonuria (PKU) that is actively recruiting participants, run by BioMarin Pharmaceutical. The registered enrollment target is 450 participants, above the 107-participant average among 6 other Phenylketonuria (PKU) trials with a reported enrollment target (321% higher). The trial reports 16 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05813678, a study of Phenylketonuria (PKU), is actively recruiting participants, sponsored by BioMarin Pharmaceutical.
- RECRUITING
- Registry status
- 450 participants
- Enrollment target
- 16
- Study locations
Study Summary
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.
Primary Outcome
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 arthralgia Severe injection site reaction Hypophenylalaninemia
Conditions Studied
Interventions
- DRUG Pegvaliase
Study Locations (16)
Other
- Hospital Carl-Thiem-Klinikum Cottbus - Cottbus
- Universitaetsklinikum Hamburg Eppendorf - Hamburg
- Universitätsklinikum Münster (UKM)-Pädiatrische Universitätsklinik Münster - Münster
- Universität Ulm - Ulm
- Policlinico Sant'orsola Malpighi - Bologna
- University Hospital Careggi - Florence
- Ospedale San Paolo - Milan
- Azienda Ospedaliera Universitaria "Federico II" Dipartimento di Pediatria - Naples
Colorado
- University of Colorado - Aurora
Illinois
- Northwestern University - Chicago
Indiana
- Indiana University School of Medicine - Indianapolis
Louisiana
- Tulane University Medical Center - New Orleans
Oregon
- Oregon Health and Science University - Portland
Pennsylvania
- University of Pittsburgh Medical Center - Pittsburgh
Texas
- University of Texas Southwestern Medical Center - Dallas
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 450 participants |
| Start Date | 2022-06-06 |
| Est. Completion | 2033-11-01 |
What NCT05813678 shows while recruiting
NCT05813678 is an observational study that tracks outcomes without assigning an intervention. The registered 450 participants enrollment target is mid-sized for trials with a published cap, above the 107-participant average among 6 other Phenylketonuria (PKU) trials with a reported enrollment target (321% higher).
The record links to 1 condition, with Phenylketonuria (PKU) appearing as the primary indexed condition, and to 1 intervention - of which Pegvaliase is the first listed.
NCT05813678 lists 16 locations in 9 states (Other, Colorado, Illinois).
Frequently Asked Questions
What is clinical trial NCT05813678 about?
NCT05813678 is a clinical study titled "A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)". 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 subj...
What is the current status of trial NCT05813678?
This trial is currently recruiting. The enrollment target is 450 participants. The study started on 2022-06-06. Estimated completion is 2033-11-01.
What conditions does trial NCT05813678 study?
This clinical trial studies the following conditions: Phenylketonuria (PKU).
What interventions are being tested in trial NCT05813678?
The interventions under investigation include: Pegvaliase (DRUG).
Who is sponsoring clinical trial NCT05813678?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05813678 being conducted?
This trial has 16 study locations across Colorado, Illinois, Indiana, Louisiana, Oregon. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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