An Open-Label Study to Evaluate the Long-Term Safety of JNT-517 in Participants With Phenylketonuria

Otsuka Pharmaceutical Development & Commercialization, Inc.12 个研究点分布在 2 个国家目标入组 240 人2025年8月11日

适应症Phenylketonuria (PKU)

干预措施JNT-517

概览

阶段: 3 期
干预措施: JNT-517
疾病 / 适应症: Phenylketonuria (PKU)
发起方: Otsuka Pharmaceutical Development & Commercialization, Inc.
入组人数: 240
试验地点: 12
主要终点: Number of participants with treatment-emergent adverse events (TEAEs)
状态: 招募中
最后更新: 昨天

概览研究者入排标准研究组 & 干预措施结局指标研究点相似试验

概览

简要总结

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.

注册库

clinicaltrials.gov

开始日期

2025年8月11日

结束日期

2028年2月1日

最后更新

昨天

研究类型

Interventional

研究设计

Single Group

性别

All

研究者

发起方

Otsuka Pharmaceutical Development & Commercialization, Inc.

责任方

Sponsor

入排标准

入选标准

•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:
•Must have a negative serum pregnancy test at Screening and a negative urine pregnancy test by Day
•Must practice sexual abstinence, or if involved in any sexual intercourse that could lead to pregnancy, must agree to use 2 different contraceptive methods, where at least 1 method must be highly effective, from Screening until at least 30 days after the last study drug administration.

排除标准

•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.
•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).
•History of drug or alcohol abuse in the last year.
•Use of any medication that are inhibitors or inducers of cytochrome P450 (CYP)3A4 or inhibitors of the transporter P glycoprotein (P-gp) within 4 weeks prior to the first dose of study drug and unwilling and/or unable to avoid these medications throughout the treatment duration.

研究组 & 干预措施

JNT-517

干预措施: JNT-517

结局指标

主要结局

Number of participants with treatment-emergent adverse events (TEAEs)

时间窗: Screening to +2 weeks from last dose

Reported based on results of 12-lead electrocardiograms (ECGs), vital signs, clinical laboratory tests, and other medical assessments.

Number of Participants with Treatment-emergent Adverse Events (TEAEs)

时间窗: Screening to +2 weeks from last dose

Reported based on results of 12-lead electrocardiograms (ECGs), vital signs, clinical laboratory tests, and other medical assessments.

次要结局

Change from baseline in plasma Phe and other amino acids(Baseline to +2 weeks from last dose)
Change from baseline in urinary Phe and other amino acids(Baseline to +2 weeks from last dose)
Change from baseline in dietary Phe and protein intake(Baseline to +2 weeks from last dose)
Percentage of Participants with Plasma Phe ≤360 µM Over Time in Participants with Baseline Phe >360 µM(Baseline to +2 weeks from last dose)
Absolute Change from Baseline in Plasma Phe(Baseline to +2 weeks from last dose)
Percent Change from Baseline Over Time in Plasma Phe(Baseline to +2 weeks from last dose)
Percentage of Participants with Plasma Phe <600 micromoles (µM) Over Time in Participants with Baseline Phe >600 µM(Baseline to +2 weeks from last dose)
Percentage of Participants with Plasma Phe ≥120 µM Over Time in Participants with Baseline Phe >120 µM(Baseline to +2 weeks from last dose)
Change from Baseline Over Time in Urinary Phe and Other Amino Acids(Baseline to +2 weeks from last dose)
Absolute Change from Baseline Over Time in Dietary Phe Intake(Baseline to +2 weeks from last dose)
Absolute Change from Baseline Over Time in Dietary Intact Protein Intake (grams per kilogram per day [g/kg/day]) for Participants Achieving Plasma Phe ≤360 μM(Baseline to +2 weeks from last dose)
Percentage of Participants who Increase Phe Intake Over Time While Maintaining Plasma Phe ≤360 μM(Baseline to +2 weeks from last dose)
Absolute Change from Baseline Over Time in the Attention-Deficit/Hyperactivity Disorder Rating Scale Version 5 (ADHD-RS-5) in Pediatric Participants who Previously Participated in JNT517-301(Month 6, Month 12, and yearly thereafter up to approximately 5 years)
Plasma Concentrations Over Time in de novo Participants Aged 4 to 11 Years(Day 1 (1-hour postdose), and predose and 1-hour postdose on Day 7 and Day 14)