NCT07694440招募中2 期
A Phase 2, Randomized, Double-blind, Placebo-controlled, Dose-ranging Study to Evaluate the Safety, Tolerability, and Efficacy of MZE782 in Adults With Phenylketonuria (PKU)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 38
- 试验地点
- 6
- 主要终点
- Safety and tolerability based on incidence of adverse events (AEs)
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, and efficacy of MZE782 in adults with Phenylketonuria (PKU).
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
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females 18 to 75 years old
- •Clinical diagnosis of PKU
- •Willing and able to keep diet and protein intake consistent during the study
- •For some study groups, is willing and able to stop certain PKU medications before starting study treatment
- •For some study groups, may need to meet additional blood phenylalanine level or background-treatment requirements
- •Meets minimum blood phenylalanine level requirements for study group
排除标准
- •Is pregnant, breastfeeding, or planning to become pregnant or conceive a child during the study or within 90 days after the last dose
- •Medical conditions or abnormal test results that could make participation unsafe or make it difficult to complete study procedures or interpret study results
- •Has recently received another investigational drug
- •Is unable to swallow tablets or tolerate oral medication
- •Other inclusion/exclusion criteria defined in protocol apply.
研究组 & 干预措施
MZE782 High Dose (Cohort 2)
Experimental
干预措施: MZE782 (Drug)
Placebo (Cohort 1A)
Placebo Comparator
干预措施: Placebo (Other)
Placebo (Cohort 2)
Placebo Comparator
干预措施: Placebo (Other)
MZE782 Low Dose (Cohort 1A)
Experimental
干预措施: MZE782 (Drug)
Placebo (Cohort 1B)
Placebo Comparator
干预措施: Placebo (Other)
MZE782 High Dose (Cohort 1B)
Experimental
干预措施: MZE782 (Drug)
结局指标
主要结局
Safety and tolerability based on incidence of adverse events (AEs)
时间窗: Week 0 to Week 17
For Cohorts 1A and 1B: Absolute change in plasma Phe levels from Baseline to the mean of plasma Phe levels
时间窗: Baseline and Weeks 2, 3, and 4
次要结局
- For Cohorts 1A and 1B, analyzed separately: Geometric mean plasma concentrations of MZE782(Weeks 1, 2, 3, 4, 8, and 13)
- For Cohorts 1A and 1B, analyzed separately: Absolute and Percent change in plasma Phe levels from Baseline to the mean of plasma Phe levels(Baseline up to Week 13)
- For Cohorts 1A and 1B, analyzed separately: Proportion of participants with mean plasma Phe levels ≤600 μMol/L, ≤360 μMol/L, and ≤120 μMol/L(Baseline and Weeks 2, 3, 4, 8, 10, and 13)
研究者
研究点 (6)
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