A Randomized Phase 1 of of MZE001 in Healthy Volunteers

NCT05249621 | Completed Phase 1 FDA Drug

• 1 other identifier: MZE001-01
• Study Type: interventional
• Target: 121
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Healthy

Outcome Measures

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

  14 days

Secondary Outcomes (3)

• Maximum concentration following multiple doses of MZE001

  14 days

• AUC following multiple doses of MZE001

  14 days

• Accumulation ratio following multiple doses of MZE001

  14 days

Study Arms (2)

MZE001

EXPERIMENTAL

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

Drug: MZE001

Placebo

PLACEBO COMPARATOR

Excipients containing no active ingredients.

Drug: Placebo

Interventions

MZE001 DRUG

Small molecule inhibitor of muscle glycogen synthase

Also known as: MZ1449348

MZE001

Placebo DRUG

Product containing excipients with no active ingredients

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

Placebo

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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;

Sponsors & Collaborators

• Maze Therapeutics: lead

Study Sites (1)

Orange County Research Center

Tustin, California, 92780, United States

Location

MeSH Terms

Interventions

Mannitol; stearic acid

Intervention Hierarchy (Ancestors)

Sugar Alcohols; Alcohols; Organic Chemicals; Carbohydrates

Study Officials

• Sarah Noonberg, MD

  Maze Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 8, 2022
• First Posted: February 21, 2022
• Study Start: February 7, 2022
• Primary Completion: December 21, 2022
• Study Completion: December 21, 2022
• Last Updated: February 6, 2023

Record last verified: 2023-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)