A Study of JNT-517 for Phenylketonuria (PKU)

This study is testing a new oral medication called JNT-517 for adults with Phenylketonuria (PKU), a genetic condition where the body can't properly break down an amino acid called phenylalanine (Phe). Researchers want to see if JNT-517 is safe and effective at lowering Phe levels in the blood. You might be able to join if you are 18 or older, have a PKU diagnosis, and your blood Phe levels are at least 360 μmol/L. The main way they'll measure success is by looking at how much your blood Phe levels change after 6 weeks of treatment. The study is currently unclear on its recruitment status, but plans to enroll 120 participants.

Study design
This is a Phase 3, randomized study where you will either receive JNT-517 or a placebo (an inactive pill). You will not know which treatment you are receiving, and there's about a 67% chance you'll get JNT-517 in the first 6 weeks.
What's involved
You will have a screening period of up to 35 days to make sure your diet and amino acid levels are stable. The first part of the study lasts about six weeks, and if you continue, you'll receive JNT-517 for an additional 46 weeks.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint measures changes in phenylalanine levels from baseline to the end of Period 1 (Week 6).

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06971731

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

Recruiting
PHASE3Ages 18+InterventionalTreatment
Otsuka Pharmaceutical Development & Commercialization, Inc.
~120 participants
Updated 2026-07-08 on ClinicalTrials.gov
What's tested:JNT-517 TabletPlacebo Tablet: BID

At a glance

Recruiting sites
26 of 26 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Absolute Change in Plasma Phenylalanine (Phe) From Baseline to the Mean of Weeks 2, 4, and 6 in the JNT-517 150 mg BID Dose Group at End of Period 1
Measured over Baseline to End of Period 1 (Week 6)
Phenylketonuria
26 sites across 17 states
Australia3
Japan3
Florida2
Texas2
Germany2
Netherlands2
Spain2
California1

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  • Absolute Change in Plasma Phenylalanine (Phe) From Baseline to the Mean of Weeks 2, 4, and 6 in the JNT-517 150 mg BID Dose Group at End of Period 1Baseline to End of Period 1 (Week 6)