A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria
NCT ID: NCT06637514
Last Updated: 2025-08-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
10 participants
INTERVENTIONAL
2025-07-16
2026-02-28
Brief Summary
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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
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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JNT-517 - 75 mg BID
Drug: JNT-517 Tablet
JNT-517 Tablet
JNT-517: 75 mg BID
Placebo - BID
Drug: Placebo Tablet
Placebo
Placebo Tablet: BID
JNT-517 - 150 mg BID
Drug: JNT-517 Tablet - 150 mg BID is a potential dose that may be used in the study. After safety, efficacy and pharmacokinetic data (PK) data have been reviewed for the first 5 participants assigned in the study, a decision will be made to increase dosage for additional participants.
JNT-517 Tablet
JNT-517: 150 mg BID
Interventions
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JNT-517 Tablet
JNT-517: 75 mg BID
Placebo
Placebo Tablet: BID
JNT-517 Tablet
JNT-517: 150 mg BID
Eligibility Criteria
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Inclusion Criteria
2. Clinical diagnosis of PKU.
3. Ability to swallow tablets.
4. Average of 2 plasma Phe levels during the Screening period greater than 360 μM and no plasma Phe level less than 300 μM.
5. Body weight equal or greater than 45 kg and body mass index less than 40 kg/m2.
6. Females of childbearing potential must practice sexual abstinence or agree to use 2 highly effective contraceptive methods.
7. 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 Criteria
2. Positive for hepatitis B or C or human immunodeficiency virus.
3. Any history of malignancy in the last 5 years, excluding nonmelanoma skin cancer.
4. Any history of liver disease.
5. Any history of cataracts or more than minimal cataracts observed during the Screening ophthalmologic examination.
6. Any surgical or medical conditions that may affect study drug absorption, distribution, metabolism, or excretion.
7. Creatinine clearance less than 90 mL/min by Cockcroft-Gault formula.
8. History of drug or alcohol abuse in the last year
9. Current, recent, or suspected infection within 14 days of Screening of SARS CoV 2/COVID 19.
10. Participation in another investigational drug trial within 30 days or, if known 5 half-lives of investigational drug (whichever is longer).
11. Unable to tolerate oral medication.
12. Allergy to JNT-517 or any component of the investigational product.
13. 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.
12 Years
17 Years
ALL
No
Sponsors
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Otsuka Pharmaceutical Development & Commercialization, Inc.
INDUSTRY
Responsible Party
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Locations
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University of Texas Southwestern Medical Center
Dallas, Texas, United States
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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JNT517-201
Identifier Type: -
Identifier Source: org_study_id
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