A Long-Term Study of JNT-517 in Participants With Phenylketonuria (NCT06628128) | Clinical Trial Compass
RecruitingPhase 3
A Long-Term Study of JNT-517 in Participants With Phenylketonuria
United States240 participantsStarted 2025-08-11
Plain-language summary
The goal of this Phase 3, open-label study is to evaluate the long-term safety of JNT-517 in pediatric and adult participants with Phenylketonuria (PKU) after completion of either Study JNT517-101 (NCT05781399) or JNT517-201 (NCT06637514) as well as participants who have not participated in a prior JNT-517 study. In this trial, all participants will receive JNT-517 using age- and weight-banded dosing as outlined in the protocol, regardless of any dose received in a previous study.
Who can participate
Age range4 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Diagnosis of phenylketonuria (ie, PAH deficiency) by either molecular testing or biochemical criteria consistent with the applicable regional guidelines.
✓. Participants 4 years of age and older, inclusive, at time of Screening.
✓. Not on pegvaliase within 4 weeks of Screening.
✓. Not on sepiapterin within 2 weeks of Screening.
✓. If on sapropterin or large neutral amino acids at Screening, must be on a stable dose for 4 weeks prior to Screening.
✓. Willing and able to maintain a diet consistent in Phe content from the Screening period through the duration of the study, unless otherwise directed by a dietician as allowed in the protocol.
✓. Body weight ≥ 12.5 kg.
✓. If female of childbearing potential:
Exclusion criteria
✕. Participation in this study is not considered safe and/or feasible in the opinion of the Investigator.
✕. Participants have not completed a previous JNT-517 study and are eligible for another active JNT-517 trial at the site, unless approval is obtained from the medical monitor.
✕. 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.
What they're measuring
1
Number of Participants with Treatment-emergent Adverse Events (TEAEs)
Timeframe: Screening to +2 weeks from last dose
Trial details
NCT IDNCT06628128
SponsorOtsuka Pharmaceutical Development & Commercialization, Inc.
. Positive for hepatitis B or C or human immunodeficiency virus.
✕. Any history of significant 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.
✕. Estimated glomerular filtration rate \< 60 milliliters per minute per 1.73 square meters (mL/min/1.73 m\^2) by 2021 Chronic Kidney Disease Epidemiology Collaboration formula (participants aged 17 years or greater) or by Schwartz formula (participants aged 4 to 16 years of age).