A Randomized Phase 1 of of MZE001 in Healthy Volunteers

Study Overview
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Brief Summary
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This is a Phase 1, randomized, double-blind, placebo-controlled, single (SAD) / multiple (MAD) ascending dose and food effect study.

Detailed Description
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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.

Official Title
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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

Conditions
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Healthy

Intervention / Treatment
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* Drug: MZE001
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Exclusion Criteria: 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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 55 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: MZE001<br>MZE001 is a small molecule inhibitor of muscle glycogen synthase for the potential treatment of Pompe disease. | Drug: MZE001<br>* Small molecule inhibitor of muscle glycogen synthase<br>* Other names: MZ1449348;|
| Placebo Comparator: Placebo<br>Excipients containing no active ingredients. | Drug: Placebo<br>* Product containing excipients with no active ingredients<br>* Other names: mannitol, silicified microcrystalline cellulose, magnesium stearate;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with adverse events as a measure of safety and tolerability of MZE001 | Occurrence of adverse events, serious adverse events, adverse events of special interest | 14 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Maximum concentration following multiple doses of MZE001 | PK | 14 days | 
| AUC following multiple doses of MZE001 | PK | 14 days | 
| Accumulation ratio following multiple doses of MZE001 | PK | 14 days |