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New pill for PKU shows promise in early human testing

NCT ID NCT05781399

Disease control Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 17, 2026 · Updated 4 times

Summary

This early-stage study tested an experimental oral drug called JNT-517 in healthy volunteers and people with phenylketonuria (PKU), a genetic condition that causes harmful buildup of the amino acid phenylalanine. The main goals were to check safety and how the body processes the drug. In the PKU part, participants received either JNT-517 or a placebo for 4 weeks to see if the drug could lower phenylalanine levels.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

163 people

The number who actually took part.

Started

Oct 2022

Finished

Jul 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 65 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: Parts A, B, C, and F: 1. Males and females 18 to 55 years of age. 2. Medically healthy with no clinically significant medical history. 3. Body mass index (BMI) of 18-40 kg/m2 and total body weight \>50 kg (110 lbs). 4. Non-smoker for at least 2 weeks prior to dosing and willing to abstain during the study. Part D and E: 5. Males and females 18 to 65 years of age, inclusive. 6. Diagnosis of PKU with a confirmed genotype. 7. At least 2 plasma Phe levels \>600 μM over the past 12 months. 8. BMI of 18-40 kg/m2. All Parts: 9. Females of childbearing potential must agree to use 2 highly effective contraceptive methods. 10. Capable of giving signed informed consent and able to comply with study procedures. Key Exclusion Criteria: All Parts: 1. Any acute or chronic medical condition that would prevent the participant from complying with the procedures or place the participant at risk if they participate in the study. 2. Positive for hepatitis B or C or human immunodeficiency virus. 3. Any history of malignancy in the last 5 years, excluding non-melanoma skin cancer. 4. Any history of liver disease. 5. Any surgical or medical conditions that may affect study drug absorption, distribution, metabolism, or excretion. 6. Participation in another investigational drug trial within 30 days or, if known, 5 half-lives of the investigational drug (whichever is longer). 7. History of drug/alcohol abuse in the last year. 8. Current, recent, or suspected infection within 4 weeks of Screening of SARS-CoV-2/COVID-19. 9. Received a vaccine for SARS-CoV-2/COVID-19 within 14 days of Screening. 10. Unable to tolerate oral medication. 11. Allergy to JNT-517 or any component of the investigational product. 12. Received \>50 mL of blood or plasma within 30 days of Screening or \>500 mL of blood or plasma within 60 days of Screening.

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Conditions

The condition(s) this trial relates to.

phenylketonuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Phenylketonuria

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Mater Misericordia Ltd

    South Brisbane, Queensland, 4101, Australia

  • Nucleus Network Melbourne

    Melbourne, Melbourne VIC, 3004, Australia

  • Oregon Health & Sciences University

    Portland, Oregon, 97239, United States

  • Rare Disease Research

    Atlanta, Georgia, 30329, United States

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000, Australia

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • UT Southwestern Medical Center

    Dallas, Texas, 75390, United States

  • University of Florida College of Medicine

    Gainesville, Florida, 32610, United States

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68198, United States

  • University of South Florida

    Tampa, Florida, 33620, United States

  • University of Texas Health Science Center at Houston

    Houston, Texas, 77030, United States

  • Utah Health - The University of Utah Hospital

    Salt Lake City, Utah, 84112, United States

  • Westmead Hospital

    Westmead, New South Wales, 2145, Australia

More trials for these conditions

Other studies related to the condition(s) this trial covers.