New PKU drug JNT-517 moves to Long-Term safety trial

NCT ID NCT06628128

Recruiting now Disease control Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This Phase 3 study is testing the long-term safety of an oral drug called JNT-517 in 240 children and adults with phenylketonuria (PKU). All participants will receive the drug twice daily, with doses based on age and weight. The goal is to see if JNT-517 is safe to use over time and how it affects blood phenylalanine levels.

What this could mean

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

Active substance: JNT-517 (oral drug taken twice daily)
What this could lead to: If successful, this could confirm JNT-517 as a safe long-term treatment to help control phenylalanine levels in people with PKU.
What could go wrong: This is an open-label study with no placebo, so results may be biased. Long-term safety and effectiveness are still being evaluated, and side effects are possible.

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

Children's Health Queensland - Queensland Children's Hospital
RECRUITING

South Brisbane, Queensland, 4101, Australia
Children's Medical Center Dallas
RECRUITING

Dallas, Texas, 75235, United States
Mater Health - Mater Hospital Brisbane
RECRUITING

South Brisbane, Queensland, 4101, Australia
Murdoch Children's Research Institute
RECRUITING

Parkville, Victoria, 3052, Australia
Oregon Health and Science University
RECRUITING

Portland, Oregon, 97239, United States
Royal Adelaide Hospital
RECRUITING

Adelaide, South Australia, 5000, Australia
University of Florida (UF) Health Shands Hospital
RECRUITING

Gainesville, Florida, 32608, United States
University of Pittsburgh Medical Center (UPMC) - Children's Hospital of Pittsburgh
RECRUITING

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

Tampa, Florida, 33606, United States
University of Texas Health (UTHealth) Science Center at Houston
RECRUITING

Houston, Texas, 77030, United States
University of Texas Southwestern Medical Center
RECRUITING

Dallas, Texas, 75390, United States
Utah Health - The University of Utah Hospital
RECRUITING

Salt Lake City, Utah, 84112, United States

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