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New PKU pill shows promise in small teen trial

NCT ID NCT06637514

First seen Apr 24, 2026 · Last updated May 21, 2026 · Updated 4 times

Summary

This study tested an experimental drug called JNT-517 in 14 teenagers with phenylketonuria (PKU), a genetic condition that causes harmful buildup of an amino acid. Participants took either the drug or a placebo twice daily for 28 days to check safety and how the body processes the drug. The goal was to see if JNT-517 could lower phenylalanine levels, but the main focus was on safety and drug levels in the blood.

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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

Conditions

Explore the condition pages connected to this study.