NCT06637514

A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. · Industry
• Sex: All
• Age: 12 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

The goal of this Phase 2, randomized study is to assess the safety, tolerability, and pharmacokinetics (PK) of oral JNT-517 in adolescents (12 to less than 18 years of age) with PKU. Participants will receive either JNT-517 or placebo and will be blinded to their treatment assignment. Participants will have a 4 in 5 (or 80%) chance of receiving JNT-517. The study will last for up to 63 days including a Screening period, Treatment period and Follow-up period for safety. Participants will: * Take 75 mg JNT-517 or a placebo BID (2x per day) for 28 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

• Phenylketonuria (PKU)

Interventions

Type	Name	Description
DRUG	JNT-517 Tablet	JNT-517: 75 mg BID
OTHER	Placebo	Placebo Tablet: BID
DRUG	JNT-517 Tablet	JNT-517: 150 mg BID

Timeline

• Start date: 2025-07-16
• Primary completion: 2025-12-01
• Completion: 2026-02-01
• First posted: 2024-10-15
• Last updated: 2025-08-19

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study