CClinicalTrials.gg
CompletedNCT05249621Updated Feb 6, 2023

A Randomized Phase 1 of of MZE001 in Healthy Volunteers

A Phase 1 interventional study of MZE001 and Placebo in Healthy, sponsored by Maze Therapeutics. Completed at 1 site in United States. Open to participants aged 18 Years to 55 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2023-02-06.

Sponsored by Maze Therapeutics · Phase 1, Interventional, and Treatment

From the registry’s dates

  • Primary completion was Dec 2022, 3 years 9 months ago, and no results have been posted to the registry.
Phase
Phase 1
Study type
Interventional
Enrollment
121
Allocation
Randomized
Ages
18 Years to 55 Years
Sex
All
01

Study summary

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

Read the 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.

02

Conditions studied

  • Healthy
03

In context

Lead sponsor

Maze Therapeutics is the lead sponsor of 3 studies on the registry; 2 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 55 Years
Sexes eligible
All
Accepts healthy volunteers
Yes

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

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.
05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Sequential assignment
Masking
Triple (Participant, Investigator, Outcomes assessor)
Enrollment
121 participants (actual)

Study arms

  • Experimental
    MZE001

    MZE001 is a small molecule inhibitor of muscle glycogen synthase for the potential treatment of Pompe disease.

    Drug: MZE001

  • Placebo comparator
    Placebo

    Excipients containing no active ingredients.

    Drug: Placebo

Interventions

  • DrugMZE001

    Small molecule inhibitor of muscle glycogen synthase

    Also known as: MZ1449348

  • DrugPlacebo

    Product containing excipients with no active ingredients

    Also known as: mannitol, silicified microcrystalline cellulose, magnesium stearate

06

What researchers measure

Primary outcomes

  1. 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

    Time frame: 14 days

Secondary outcomes

  1. Maximum concentration following multiple doses of MZE001

    PK

    Time frame: 14 days

  2. AUC following multiple doses of MZE001

    PK

    Time frame: 14 days

  3. Accumulation ratio following multiple doses of MZE001

    PK

    Time frame: 14 days

07

Study locations

1 site
  • Orange County Research Center
    Tustin, California 92780, United States
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 6, 2023, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT05249621
Lead sponsor
Maze Therapeutics
Responsible party
Sponsor
First posted
Feb 21, 2022
Start date
Feb 7, 2022
Primary completion
Dec 21, 2022
Completion
Dec 21, 2022
Last update
Feb 6, 2023

Study contacts

Sarah Noonberg, MD
study director · Maze Therapeutics

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Feb 2023. You cannot join it, but the record below documents what was studied.

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