A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria
Otsuka Pharmaceutical Development & Commercialization, Inc.
10 participants
Jul 16, 2025
INTERVENTIONAL
Conditions
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
Eligibility
Inclusion Criteria7
- Males and females 12 to less than 18 years of age, inclusive on Day 1.
- Clinical diagnosis of PKU.
- Ability to swallow tablets.
- Average of 2 plasma Phe levels during the Screening period greater than 360 μM and no plasma Phe level less than 300 μM.
- Body weight equal or greater than 45 kg and body mass index less than 40 kg/m2.
- Females of childbearing potential must practice sexual abstinence or agree to use 2 highly effective contraceptive methods.
- Capable of giving signed informed consent (emancipated minors) or parent/legal guardian to provide informed consent and the participant to give assent and confirm ability to comply with study procedures.
Exclusion Criteria13
- 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.
- Positive for hepatitis B or C or human immunodeficiency virus.
- Any history of malignancy in the last 5 years, excluding nonmelanoma skin cancer.
- Any history of liver disease.
- Any history of cataracts or more than minimal cataracts observed during the Screening ophthalmologic examination.
- Any surgical or medical conditions that may affect study drug absorption, distribution, metabolism, or excretion.
- Creatinine clearance less than 90 mL/min by Cockcroft-Gault formula.
- History of drug or alcohol abuse in the last year
- Current, recent, or suspected infection within 14 days of Screening of SARS CoV 2/COVID 19.
- Participation in another investigational drug trial within 30 days or, if known 5 half-lives of investigational drug (whichever is longer).
- Unable to tolerate oral medication.
- Allergy to JNT-517 or any component of the investigational product.
- Received greater than 50 mL of blood or plasma within 30 days of Screening or greater than 500 mL of blood or plasma within 60 days of Screening.
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Interventions
JNT-517: 75 mg BID
Placebo Tablet: BID
JNT-517: 150 mg BID
Locations(7)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06637514