A Study to Evaluate the Safety and Efficacy of JNT-517 in Participants With Phenylketonuria (PKU)

Study Identifier:

JNT517-301

ClinicalTrials.gov Identifier:

NCT06971731

EudraCT Identifier:

N/A

Sponsor:

Otsuka Pharmaceutical Development & Commercialization, Inc.

Study Contact Information:

844-687-8522 or [email protected]

Will Be Recruiting

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Study Details

Medical Condition

Phenylketonuria (PKU)

Study Drug

Drug: JNT-517 Tablet
Drug: Placebo Tablet: BID

Date

Aug 2025 - Nov 2026

Phase 1/2

Phase 2/3

Phase 1

Phase 2

Phase 3

Phase 4

N/A

Patient Requirements

Sex: Female & Male

Age: 18+ years

Requirements Information

Protocol 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

Study Locations

No locations found.