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First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria

NCT05781399 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 111

Last updated 2026-05-06

No results posted yet for this study

Summary

The goal of Parts A and B of this Phase 1/2, first-in-human, randomized study is to assess the safety, tolerability, and pharmacokinetics (PK) of single (SAD) and multiple (MAD) ascending doses of oral JNT-517 in healthy participants. In Part C, the goal is to evaluate the differences in bioavailability between a tablet and suspension formulation of JNT-517 and the food effect in healthy volunteers. All participants in Part C will receive JNT-517. The goal of Part D is to assess the safety, tolerability, PK, and effect on urinary Phe and other amino acids of JNT-517 in participants with phenylketonuria (PKU). Participants in Part D will receive either JNT-517 or placebo and will be blinded to their treatment assignment.

The study consists of 6 parts:

* Part A: SAD in healthy participants -randomized, double-blind, placebo-controlled
* Part B: MAD in healthy participants (14 days)-randomized, double-blind, placebo-controlled
* Part C: Relative bioavailability of 2 formulations and food effect in healthy participants-randomized, open-label
* Part D: Phase 2 in participants with PKU (4 weeks)-randomized, double-blind, placebo-controlled
* Part E: Phase 2 in participants with PKU (4 weeks) open label
* Part F: SAD Phase 1 in healthy participants, randomized, double-blind, placebo-controlled

In each part, participants will complete a Screening Period, a Treatment Period, and a Follow-up Period for safety.

Conditions

Interventions

DRUG

JNT-517 Suspension

JNT-517 in on-site compounded suspension

DRUG

Placebo Suspension

On-site compounded placebo suspension

DRUG

JNT-517 Tablet

JNT-517 tablets, 25 mg and 75 mg

DRUG

Placebo Tablet

Matching film-coated placebo tablet

Sponsors & Collaborators

  • Otsuka Pharmaceutical Development & Commercialization, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2022-10-31
Primary Completion
2025-07-14
Completion
2025-07-14
FDA Drug
Yes

Countries

  • United States
  • Australia

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 NCT05781399 on ClinicalTrials.gov