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NCT05270837 · ClinicalTrials.gov registry record · Phase 3

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

A Phase 3 study of Phenylketonuria (PKU), sponsored by BioMarin Pharmaceutical.

Active
Registry status
Phase 3
Development phase
55
Enrollment target
16
Study locations

NCT05270837 is a Phase 3 study of Phenylketonuria (PKU) that is active but no longer recruiting, run by BioMarin Pharmaceutical. The registered enrollment target is 55 participants, below the 173-participant average among 6 other Phenylketonuria (PKU) trials with a reported enrollment target (68% lower). The trial reports 16 study locations across 14 states.

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The verdict

NCT05270837, a Phase 3 study of Phenylketonuria (PKU), is active but no longer recruiting, sponsored by BioMarin Pharmaceutical.

ACTIVE NOT RECRUITING
Registry status
Phase 3
Development phase
55 participants
Enrollment target
16
Study locations

Study Summary

This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.

Conditions Studied

Interventions

  • DRUG Pegvaliase
  • OTHER Diet Only

Study Locations (16)

Other

  • Charité - Universitätsmedizin Berlin - Berlin
  • Universitätsklinikum Hamburg-Eppendorf - Hamburg
  • Universitat Mainz - Mainz

Arizona

  • Phoenix Children's Hospital - Phoenix

Arkansas

  • Arkansas Children's Hospital - Little Rock

Colorado

  • Children's Hospital of Colorado - Aurora

Florida

  • University of South Florida - Tampa

Illinois

  • Ann and Robert H. Lurie Children's Hospital of Chicago - Chicago

Indiana

  • IU Health University Hospital - Indianapolis

Kentucky

  • University of Kentucky College of Medicine - Lexington

Trial Details

FieldValue
Enrollment Target 55 participants
Start Date 2022-06-17
Est. Completion 2027-10
Phase Phase 3

Sponsor

BioMarin Pharmaceutical

80 total trials

What the Registry Record Tells You About NCT05270837

The ClinicalTrials.gov registry entry for NCT05270837 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 55 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 173-participant average among 6 other Phenylketonuria (PKU) trials with a reported enrollment target (68% lower). 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 2 interventions - of which Pegvaliase is the first listed.

NCT05270837 reports 16 study locations spanning 14 distinct geographic areas - top geographies include Other, Arizona, Arkansas.

Frequently Asked Questions

What is clinical trial NCT05270837 about?

NCT05270837 is a clinical study titled "Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria". This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.

What is the current status of trial NCT05270837?

This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 55 participants. The study started on 2022-06-17. Estimated completion is 2027-10.

What conditions does trial NCT05270837 study?

This clinical trial studies the following conditions: Phenylketonuria (PKU).

What interventions are being tested in trial NCT05270837?

The interventions under investigation include: Pegvaliase (DRUG), Diet Only (OTHER).

Who is sponsoring clinical trial NCT05270837?

This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT05270837 being conducted?

This trial has 16 study locations across Arizona, Arkansas, Colorado, Florida, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

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.