A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 10
- 试验地点
- 8
- 主要终点
- Number of participants with treatment-emergent adverse events (TEAEs)
研究概览
简要总结
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
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females 12 to less than 18 years of age, inclusive on Day
- •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/m
- •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.
排除标准
- •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
- •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.
研究组 & 干预措施
JNT-517 - 75 mg BID
Drug: JNT-517 Tablet
干预措施: JNT-517 Tablet (Drug)
Placebo - BID
Drug: Placebo Tablet
干预措施: Placebo (Other)
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.
干预措施: JNT-517 Tablet (Drug)
结局指标
主要结局
Number of participants with treatment-emergent adverse events (TEAEs)
时间窗: Screening to Study Completion (Average of 9 weeks)
Reported based on results of 12-lead electrocardiograms (ECGs), vital signs and clinical laboratory tests.
次要结局
- Plasma AUC (area under the concentration-time curve)(Screening to Study Completion (Average of 9 weeks))
- Cmax (maximum observed concentration)(Screening to Study Completion (Average of 9 weeks))
- Tmax (time to Cmax)(Screening to Study Completion (Average of 9 weeks))
- Change over time in plasma phenylalanine (Phe) and other amino acids(Screening to Study Completion (Average of 9 weeks))
- Change over time in urinary phenylalanine (Phe) and other amino acids(Screening to Study Completion (Average of 9 weeks))