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Study identifier: NCT06971731 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

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

Condition: Phenylketonuria  ·  Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.

PhasePhase 3
Planned participants120
Who can joinAll sexes, 18 Years to no upper limit
Healthy volunteersNo

About this study

The goal of this Phase 3, randomized study is to assess the safety, efficacy, tolerability, and pharmacokinetics (PK) of oral JNT-517 in adults (18 years of age or older) with PKU. Participants will receive either JNT-517 or placebo and will be blinded to their treatment assignment. Participants will have a 2 in 3 (or approximately 67%) chance of receiving JNT-517 during the first part of the study which will last approximately six weeks. During the second part of the study every participant who continues in the study will receive one of two doses of JNT-517 for an additional 46 weeks. The study requires a screening period of up to 35 days to ensure dietary stabilization and amino acid levels required to meet study eligibility. In total, participation in the study could last for up to 400 days. Participants will: Take 75 mg JNT-517 or 150 mg JNT-517, or a placebo BID (2x per day) for approximately 365 days; Visit the clinic or have a mobile health nurse visit your home for checkups and tests; Collect urine sample at home and bring to clinic on specified days; Keep a food diary 3 days before each study visit

This description comes directly from the study's public registry record.

Talk to the study team

Otsuka Call Center  ·  844-687-8522  ·  otsuka-professionalservices@otsuka-us.com

Always discuss trial participation with your own doctor first.

Locations (26)

University of California Los Angeles (UCLA) School of MedicineLos Angeles, California, United StatesRecruiting
University of Florida (UF) Health Shands HospitalGainesville, Florida, United StatesRecruiting
University of South FloridaTampa, Florida, United StatesRecruiting
Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, Illinois, United StatesRecruiting
Oregon Health and Science UniversityPortland, Oregon, United StatesRecruiting
University of Pittsburgh Medical Center (UPMC) - Children's Hospital of PittsburghPittsburgh, Pennsylvania, United StatesRecruiting
Vanderbilt University Medical CenterNashville, Tennessee, United StatesRecruiting
University of Texas SouthwesternDallas, Texas, United StatesRecruiting
University of Texas Health (UTHealth) Science Center at HoustonHouston, Texas, United StatesRecruiting
Utah Health - The University of Utah HospitalSalt Lake City, Utah, United StatesRecruiting
Royal Adelaide HospitalAdelaide, AustraliaRecruiting
Royal Melbourne HospitalParkville, AustraliaRecruiting

+ 14 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT06971731