A Randomized Phase 1 of of MZE001 in Healthy Volunteers

Phase 1

Completed

• Conditions: Healthy
• Interventions: Drug: MZE001; Drug: Placebo

• Registration Number: NCT05249621
• Lead Sponsor: Maze Therapeutics

Brief Summary

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

Detailed Description

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.

Study Timeline

• Study Start: 2022-02-07
• Study First Submitted: 2022-02-08
• Study First Submitted QC: 2022-02-17
• Study First Posted: 2022-02-21
• Primary Completion: 2022-12-21
• Study Completion: 2022-12-21
• Status Verified: 2023-02
• Last Update Submitted: 2023-02-02
• Last Update Posted: 2023-02-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 121

Inclusion Criteria

1. Male and female subjects, ages 18 - 55 years, inclusive;
2. Must provide written informed consent prior to any study assessments and be willing and able to comply with all study procedures;
3. Body mass index (BMI) between 18.0 and 32.0 kg/m2 inclusive;
4. Healthy as determined by the Investigator based on pre-study medical history, physical examination, and baseline safety laboratory studies.
5. Able to complete exercise treadmill test with no cardiac abnormalities detected
6. 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.
7. 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

1. 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;
2. Any history of coronary artery disease or cardiovascular disease;
3. History or presence of gastrointestinal, renal, or hepatic disease or any other condition known to interfere with absorption, distribution, metabolism, or excretion of drugs;
4. History of cancer within past 5 years, with the exception of non-melanoma skin cancer and treated / excised melanoma;
5. Any clinically important illness, medical/surgical procedure, or trauma within 4 weeks prior to the Screening visit;
6. Fridericia's corrected QT (QTcF) > 450 ms for male participants and > 470 ms for female participants or history of QT interval prolongation;
7. History or presence of an abnormal ECG that is clinically significant in the Investigator's opinion
8. Resting seated blood pressure < 90/40 mmHg or > 140/90 mmHg
9. Resting seated heart rate < 45 bpm or > 99 bpm
10. Poor peripheral venous access;
11. Have a history of drug hypersensitivity or anaphylaxis;
12. Current smoker or recent history of smoking within the last 3 months prior to the Screening visit;
13. Have a history of alcoholism or drug abuse or positive drug screen
14. Have used any prescription or non-prescription medicines or have been administered a vaccine within 14 days of admission,
15. Have received any investigational drug within 30 days or < 5 half-lives, whichever is longer, prior to the Screening visit;
16. Have donated or received any blood or blood products within the 3 months prior to the Screening visit.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
MZE001	MZE001	MZE001 is a small molecule inhibitor of muscle glycogen synthase for the potential treatment of Pompe disease.
Placebo	Placebo	Excipients containing no active ingredients.

Primary Outcome Measures

Name	Time	Method
Number of participants with adverse events as a measure of safety and tolerability of MZE001	14 days	Occurrence of adverse events, serious adverse events, adverse events of special interest

Secondary Outcome Measures

Name	Time	Method
AUC following multiple doses of MZE001	14 days	PK
Accumulation ratio following multiple doses of MZE001	14 days	PK
Maximum concentration following multiple doses of MZE001	14 days	PK

Trial Locations

Locations (1)

Orange County Research Center; 🇺🇸 Tustin, California, United States