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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 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.

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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.

Conditions Studied

Phenylketonuria (PKU)

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

FieldValue
Enrollment Target 450 participants
Start Date 2022-06-06
Est. Completion 2033-11-01

Sponsor

BioMarin Pharmaceutical

80 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT05813678

The ClinicalTrials.gov registry entry for NCT05813678 describes a study currently listed as recruiting, categorized as an unspecified phase. The registered enrollment target is 450 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 107-participant average among 6 other Phenylketonuria (PKU) trials with a reported enrollment target (321% higher). The listed sponsor is BioMarin Pharmaceutical, which has 80 total studies on file at ClinicalTrials.gov.

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 reports 16 study locations spanning 9 distinct geographic areas - top geographies include 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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Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.