HealthyPhase 1NCT05249621
A Randomized Phase 1 of of MZE001 in Healthy Volunteers
A Phase 1 study testing MZE001 in people with Healthy, run by Maze Therapeutics at 1 site in United States.
Trial summary
This is a Phase 1, randomized, double-blind, placebo-controlled, single (SAD) / multiple (MAD) ascending dose and food effect study. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy
- What is being tested?
- MZE001
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 121 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Feb 2022to 21 Dec 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Orange County Research Center
Tustin, California, 92780
About this study
Official title: 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
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.
- Conditions
- Healthy
- Being tested
- MZE001, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MZE001Drug
Also called MZ1449348
Small molecule inhibitor of muscle glycogen synthase
PlaceboDrug
Also called mannitol, silicified microcrystalline cellulose, magnesium stearate
Product containing excipients with no active ingredients
| Study group | Role | What they receive |
|---|---|---|
| MZE001 | Experimental | MZE001 is a small molecule inhibitor of muscle glycogen synthase for the potential treatment of Pompe disease. Receives: Drug: MZE001 |
| Placebo | Placebo comparator | Excipients containing no active ingredients. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 14 days
Secondary outcomes (3)
Maximum concentration following multiple doses of MZE001
PK
Measured: 14 days
AUC following multiple doses of MZE001
PK
Measured: 14 days
Accumulation ratio following multiple doses of MZE001
PK
Measured: 14 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2022
- Study start7 Feb 2022
- Primary completion21 Dec 2022
- Study completion21 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HealthyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 121 participants (actual)
Sponsor and investigators
- Lead sponsor
- Maze Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sarah Noonberg, MD, Study director, Maze Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05249621
- Sponsor's protocol ID
- MZE001-01
- Status last verified
- Feb 2023
- First submitted
- 8 Feb 2022
- Last updated
- 6 Feb 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05249621
- Acronym / sponsor ID
- MZE001-01
- Conditions
- Healthy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 121 participants (actual)
- Lead sponsor
- Maze TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 7 Feb 2022 (actual)
- Primary completion
- 21 Dec 2022 (actual)
- Study completion
- 21 Dec 2022 (actual)
- First posted
- 21 Feb 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MZE001 | Experimental | Drug: MZE001MZE001 is a small molecule inhibitor of muscle glycogen synthase for the potential treatment of Pompe disease. |
| Placebo | Placebo comparator | Drug: PlaceboExcipients containing no active ingredients. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (16 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MZE001Also: MZ1449348Small molecule inhibitor of muscle glycogen synthase | Drug | MZE001 |
| PlaceboAlso: mannitol, silicified microcrystalline cellulose, magnesium stearateProduct containing excipients with no active ingredients | Drug | Placebo |
Primary outcome
Number of participants with adverse events as a measure of safety and tolerability of MZE001
Time frame: 14 days
Occurrence of adverse events, serious adverse events, adverse events of special interest
3 secondary outcomes
Maximum concentration following multiple doses of MZE001
Time frame: 14 days
AUC following multiple doses of MZE001
Time frame: 14 days
Accumulation ratio following multiple doses of MZE001
Time frame: 14 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sarah Noonberg, MD study director
Maze Therapeutics
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MZE001 in people with Healthy, run by Maze Therapeutics at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with adverse events as a measure of safety and tolerability of MZE001 (time frame: 14 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Feb 2023.