New shot could free teens with PKU from strict diet

NCT ID NCT05270837

Ongoing Disease control Sponsor: BioMarin Pharmaceutical Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jul 22, 2026 · Updated 3 times

Summary

This study tests an injectable drug called pegvaliase in 55 teenagers (ages 12-17) with phenylketonuria (PKU) who have high blood Phe levels despite diet management. Half get the drug, half stick to diet only. The goal is to see if pegvaliase safely lowers Phe levels and reduces the need for a strict low-protein diet.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: pegvaliase
What this could lead to: If it works, this could offer a new treatment option for teens with PKU who struggle to manage their condition with diet alone.
What could go wrong: This is a relatively small Phase 3 trial, and pegvaliase may cause side effects like allergic reactions. It's not a cure, and long-term safety is still being studied.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

phenylketonuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PHENYLKETONURIA (PKU)

Contacts and locations

Locations

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

Chicago, Illinois, 60611, United States
Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States
Boston Children's Hospital

Boston, Massachusetts, 02115, United States
Charité - Universitätsmedizin Berlin

Berlin, Germany
Children's Hospital of Colorado

Aurora, Colorado, 80045, United States
Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States
IU Health University Hospital

Indianapolis, Indiana, 46202, United States
McGovern Medical School, University of Texas Health Science Center at Houston (UTHealth)

Houston, Texas, 77030, United States
Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States
Universitat Mainz

Mainz, Germany
University of Kentucky College of Medicine

Lexington, Kentucky, 40506, United States
University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15224, United States
University of South Florida

Tampa, Florida, 33606, United States
University of Utah Medical Center

Salt Lake City, Utah, 84132, United States
University of Virginia School of Medicine

Charlottesville, Virginia, 22903, United States
Universitätsklinikum Hamburg-Eppendorf

Hamburg, Germany

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