New pill for PKU shows promise in early human testing

NCT ID NCT05781399

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

First seen Jun 27, 2026 · Last updated Jul 17, 2026 · Updated 4 times

Summary

This early-stage study tested an experimental oral drug called JNT-517 in healthy volunteers and people with phenylketonuria (PKU), a genetic condition that causes harmful buildup of the amino acid phenylalanine. The main goals were to check safety and how the body processes the drug. In the PKU part, participants received either JNT-517 or a placebo for 4 weeks to see if the drug could lower phenylalanine levels.

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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

Contacts and locations

Locations

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

Chicago, Illinois, 60611, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Mater Misericordia Ltd

South Brisbane, Queensland, 4101, Australia
Nucleus Network Melbourne

Melbourne, Melbourne VIC, 3004, Australia
Oregon Health & Sciences University

Portland, Oregon, 97239, United States
Rare Disease Research

Atlanta, Georgia, 30329, United States
Royal Adelaide Hospital

Adelaide, South Australia, 5000, Australia
UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States
UT Southwestern Medical Center

Dallas, Texas, 75390, United States
University of Florida College of Medicine

Gainesville, Florida, 32610, United States
University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States
University of South Florida

Tampa, Florida, 33620, United States
University of Texas Health Science Center at Houston

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

Salt Lake City, Utah, 84112, United States
Westmead Hospital

Westmead, New South Wales, 2145, Australia

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