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

NCT06971731 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 120

Last updated 2026-05-04

No results posted yet for this study

Summary

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

Conditions

Interventions

DRUG

JNT-517 Tablet

JNT-517: 75 mg BID

DRUG

JNT-517 Tablet

JNT-517: 150 mg BID

DRUG

Placebo Tablet: BID

Placebo Tablet: BID

Sponsors & Collaborators

  • Otsuka Pharmaceutical Development & Commercialization, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-10-20
Primary Completion
2028-03-01
Completion
2028-04-01
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • Czechia
  • France
  • Germany
  • Japan
  • Netherlands
  • Poland
  • Spain

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06971731 on ClinicalTrials.gov