NCT06628128

A Study to Evaluate the Long-Term Safety and Efficacy of JNT-517 in Participants With Phenylketonuria

An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of JNT-517 in Participants With Phenylketonuria

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 45 (estimated)
• Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The goal of this Phase 3, open-label extension study is to evaluate the long-term safety and efficacy of JNT-517 in participants with Phenylketonuria (PKU) after completion of either Study JNT517-101 or JNT517-201. In this long-term extension (LTE) study, all adults (aged ≥18 years) who complete Study JNT517-101 will be randomized 1:1 to receive JNT-517 at 75 mg twice daily (BID) or 150 mg BID, regardless of their previous dose. Adolescent participants who complete Study JNT517-201 will receive the same JNT-517 dose of the cohort they were initially assigned to, either 75 mg BID or 150 mg BID.

Conditions

• Phenylketonuria (PKU)

Interventions

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

Timeline

• Start date: 2025-06-01
• Primary completion: 2027-11-01
• Completion: 2028-02-01
• First posted: 2024-10-04
• Last updated: 2025-06-03

Regulatory

• FDA-regulated drug study