New pill for PKU shows promise in small teen trial

NCT ID NCT06637514

Completed 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 study tested a new drug called JNT-517 in 14 teenagers with phenylketonuria (PKU). Participants took either JNT-517 or a placebo twice daily for 28 days. The main goal was to check safety and how the drug moves through the body, while also measuring changes in 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 (a tablet taken twice daily)
What this could lead to: If successful, this could lead to a new oral treatment option to help control phenylalanine levels in teenagers with PKU.
What could go wrong: This is a small, early-phase trial with only 14 participants, so results may not apply to everyone. The main goal is safety, not yet proof of effectiveness.

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 Medical Center Dallas

Dallas, Texas, 75235, United States
Murdoch Children's Research Institute

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

Portland, Oregon, 97239, United States
University of Florida (UF) Health Shands Hospital

Gainesville, Florida, 32608, United States
University of South Florida

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

Houston, Texas, 77030, United States
Utah Health - The University of Utah Hospital

Salt Lake City, Utah, 84112, United States

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