AI Cure is a research tool showing open data. Not medical advice. Discuss with your doctor. Terms

← Trials
🧪

A Study of JNT-517 in Participants With Phenylketonuria (PKU)

RecruitingPhase 3Ages 4 Years - anyAllOtsuka Pharmaceutical Development & Commercializa…
💬

In plain words

What the trial is, and whether it could suit you

This Phase 3 study will evaluate the safety, effectiveness, tolerability, and how the body processes JNT-517 in children, adolescents, and adults with phenylketonuria. Participants will be randomly assigned to receive either JNT-517 or placebo during an initial 6-week treatment period. After this period, eligible participants will receive JNT-517 for an additional 46 weeks.

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
Inclusion Criteria: 1. Male or female participants aged 4 years or older. 2. Diagnosed with phenylketonuria. 3. Have phenylalanine levels that meet study requirements. 4. Meet study-specific requirements regarding current treatment for phenylketonuria. 5. Willing and able to follow study dietary requirements. 6. Weigh at least 12.5 kilograms. 7. If able to become pregnant or father a child, willing to follow the study's contraception requirements. 8. Any ongoing mental health condition must be stable before entering the study. 9. Able to provide informed consent (or have consent provided by a parent or legal guardian, when applicable) and comply with study procedures. Exclusion Criteria: Participants will be excluded from the study if any of the following criteria are met: 1. Significant medical condition that, in the investigator's opinion, could make participation unsafe or interfere with study participation. 2. Significant liver disease or impaired kidney function. 3. Certain eye conditions, including significant cataracts. 4. Active infection requiring medical evaluation. 5. History of certain cancers within the past 5 years. 6. Allergy or hypersensitivity to JNT-517 or its ingredients. 7. Prior treatment with JNT-517. 8. Recent participation in another investigational study or receipt of an investigational treatment that does not meet study requirements. 9. Use of medications that are not permitted during the study. 10. Abnormal laboratory or heart test results that would make study participation inappropriate. 11. Conditions that could affect the absorption of oral study medication. 12. History of recent substance or alcohol abuse. 13. Unwillingness to follow study requirements, including restrictions on intentional weight loss.
💊

What it tests

  • JNT-517
  • JNT-517 Matching Placebo
📍

Where

26 sites
University of California Los Angeles (UCLA) School of Medicine
Los Angeles, United States
University of Florida (UF) Health Shands Hospital
Gainesville, United States
University of South Florida
Tampa, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States
Oregon Health and Science University
Portland, United States
University of Pittsburgh Medical Center (UPMC) - Children's Hospital of Pittsburgh
Pittsburgh, United States
Vanderbilt University Medical Center
Nashville, United States
University of Texas Southwestern
Dallas, United States

…and 18 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
🛡️

AI Cure shows public evidence (Open Targets, ClinicalTrials.gov, PubMed, openFDA, DepMap, AlphaFold) with its source, and AI summaries that cite it — not medical advice and not a cure. Treatment decisions belong with your doctor. Research ideas are shared only with approved researchers and labs, who can test them. In an emergency call your local emergency number (112 in India and Europe, 911 in the US). AI Cure Terms of Use · Privacy