NCT05249621已完成1 期
A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Study of MZE001 to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics in Healthy Subjects
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 121
- 试验地点
- 1
- 主要终点
- Number of participants with adverse events as a measure of safety and tolerability of MZE001
研究概览
简要总结
This is a Phase 1, randomized, double-blind, placebo-controlled, single (SAD) / multiple (MAD) ascending dose and food effect study.
详细描述
Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of orally administered MZE001 in healthy subjects, in fasted and fed states. Each SAD and MAD cohort will enroll 8 subjects randomized 6 active: 2 placebo to receive single or multiple doses of MZE001.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 55 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Male and female subjects, ages 18 - 55 years, inclusive;
- •Must provide written informed consent prior to any study assessments and be willing and able to comply with all study procedures;
- •Body mass index (BMI) between 18.0 and 32.0 kg/m2 inclusive;
- •Healthy as determined by the Investigator based on pre-study medical history, physical examination, and baseline safety laboratory studies.
- •Able to complete exercise treadmill test with no cardiac abnormalities detected
- •Females of childbearing potential who are sexually active with a non-sterilized partner must use a highly effective method of contraception during study participation and for 30 days after last administration of study drug.
- •Males of childbearing potential must use a highly effective method of birth control during study participation and for 90 days after last administration of study drug.
排除标准
- •Any concurrent condition that in the opinion of the Investigator would interfere with the evaluation of the investigational product or lead to increased risk of harm;
- •Any history of coronary artery disease or cardiovascular disease;
- •History or presence of gastrointestinal, renal, or hepatic disease or any other condition known to interfere with absorption, distribution, metabolism, or excretion of drugs;
- •History of cancer within past 5 years, with the exception of non-melanoma skin cancer and treated / excised melanoma;
- •Any clinically important illness, medical/surgical procedure, or trauma within 4 weeks prior to the Screening visit;
- •Fridericia's corrected QT (QTcF) > 450 ms for male participants and > 470 ms for female participants or history of QT interval prolongation;
- •History or presence of an abnormal ECG that is clinically significant in the Investigator's opinion
- •Resting seated blood pressure < 90/40 mmHg or > 140/90 mmHg
- •Resting seated heart rate < 45 bpm or > 99 bpm
- •Poor peripheral venous access;
- •Have a history of drug hypersensitivity or anaphylaxis;
- •Current smoker or recent history of smoking within the last 3 months prior to the Screening visit;
- •Have a history of alcoholism or drug abuse or positive drug screen
- •Have used any prescription or non-prescription medicines or have been administered a vaccine within 14 days of admission,
- •Have received any investigational drug within 30 days or < 5 half-lives, whichever is longer, prior to the Screening visit;
- •Have donated or received any blood or blood products within the 3 months prior to the Screening visit.
研究组 & 干预措施
MZE001
Experimental
MZE001 is a small molecule inhibitor of muscle glycogen synthase for the potential treatment of Pompe disease.
干预措施: MZE001 (Drug)
Placebo
Placebo Comparator
Excipients containing no active ingredients.
干预措施: Placebo (Drug)
结局指标
主要结局
Number of participants with adverse events as a measure of safety and tolerability of MZE001
时间窗: 14 days
Occurrence of adverse events, serious adverse events, adverse events of special interest
次要结局
- AUC following multiple doses of MZE001(14 days)
- Accumulation ratio following multiple doses of MZE001(14 days)
- Maximum concentration following multiple doses of MZE001(14 days)
研究者
研究点 (1)
Loading locations...
相似试验
尚未招募
1 期
A Study to Evaluate SGB-3403 in Healthy Volunteers and Subjects With Elevated Low-Density Lipoprotein Cholesterol (LDL-C)HyperlipidemiasNCT06239714Suzhou Sanegene Bio Inc.64
已完成
1 期
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL151 in Healthy VolunteersHealthy VolunteersNCT04557800Denali Therapeutics Inc.186
已完成
1 期
A Study of NP-011 in Healthy VolunteersMyocardial InfarctionNCT05387499Nexel Co., Ltd.64
已完成
1 期
Study to Assess the Safety, Tolerability, Pharmacokinetics and Immunogenicity of AK006 in Healthy Subjects and Subjects With Chronic Spontaneous UrticariaHealthy ParticipantsChronic Spontaneous UrticariaNCT06072157Allakos Inc.136
招募中
1 期
JX09 SAD/MAD in Healthy ParticipantsResistant HypertensionNCT06183671Ji Xing Pharmaceuticals Australia Pty Ltd92