First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria
Completed · Phase 1/Phase 2 · Has a placebo group
Conditions studied: Phenylketonuria
In brief
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.
Key facts
- Study ID
- NCT05781399
- Run by
- Otsuka Pharmaceutical Development & Commercialization, Inc.
- People needed
- 163
- Starts
- 2022-10-31
- Expected to finish
- 2025-07-29
- Last updated by the study team
- 2026-07-16
Who can join
Age: 18 and older, up to 65. Sex: any. Healthy volunteers: accepted.
Where it is running
- University of Florida College of Medicine — Gainesville, Florida, United States
- University of South Florida — Tampa, Florida, United States
- Rare Disease Research — Atlanta, Georgia, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- University of Nebraska Medical Center — Omaha, Nebraska, United States
- Oregon Health & Sciences University — Portland, Oregon, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
- UT Southwestern Medical Center — Dallas, Texas, United States
- University of Texas Health Science Center at Houston — Houston, Texas, United States
- Utah Health - The University of Utah Hospital — Salt Lake City, Utah, United States
- Nucleus Network Melbourne — Melbourne, Melbourne VIC, Australia
- Westmead Hospital — Westmead, New South Wales, Australia
- Mater Misericordia Ltd — South Brisbane, Queensland, Australia
- Royal Adelaide Hospital — Adelaide, South Australia, Australia
Full record on ClinicalTrials.gov
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