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Trial registered on ANZCTR


Registration number
ACTRN12625000488404p
Ethics application status
Submitted, not yet approved
Date submitted
9/05/2025
Date registered
20/05/2025
Date last updated
20/05/2025
Date data sharing statement initially provided
20/05/2025
Type of registration
Prospectively registered

Titles & IDs
Public title
A Phase 1 Randomized, Double-blind, Placebo-controlled Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of MZE782 in Healthy Adults
Scientific title
A Phase 1 Randomized, Double-blind, Placebo-controlled Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of MZE782 in Healthy Adults
Secondary ID [1] 314299 0
MZE782-103
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Chronic Kidney Disease 337242 0
Phenylketonuria 337396 0
Condition category
Condition code
Renal and Urogenital 333650 333650 0 0
Kidney disease
Human Genetics and Inherited Disorders 333775 333775 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 333888 333888 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This is a Phase 1, randomized, double-blinded, placebo-controlled, multiple dose escalation (MAD) study designed to assess the safety, tolerability, and pharmacokinetics (PK) of MZE782 in healthy adult volunteers.

Up to three cohorts of 8 participants each (6 active and 2 placebo) will be enrolled.

On Day 1, participants will be randomized to receive an oral administration of MZE782 or placebo tablets under fasted conditions.

The proposed starting dose to be evaluated will be 720 mg MZE782 or placebo tablets daily for 7 days.

Following review of safety and PK data by the Safety Review Committee (SRC) and determination of adequate safety and tolerability in Cohort 1, dose escalation will occur.

The proposed dose for Cohort 2 is 960 mg MZE782 or placebo tablets daily for 7 days.

The dose of Cohort 3 has not been determined.

Participants will be discharged from the Clinical Research Unit (CRU) on Day 10, at approximately 72 hours after the last dose.

Participants will be administered study drug in the CRU under the direct medical supervision of the investigator or designee.
Intervention code [1] 330914 0
Treatment: Drugs
Comparator / control treatment
Placebo tablet identical in appearance to study drug and containing microcrystalline cellulose.
Control group
Placebo

Outcomes
Primary outcome [1] 341236 0
To determine the safety and tolerability of multiple MZE782 doses in healthy participants
Timepoint [1] 341236 0
Adverse event (AE) severity will be coded by system organ class and preferred terms using the most current Medical Dictionary for Regulatory Activities (MedDRA) and assessed continuously as observed and reviewed daily from Day 1 until Day 14 Safety Follow Up Visit. Vital signs (blood pressure assessed using digital sphygmomanometer, heart rate and respiratory rate assessed using pulse oximeter and temperature assessed using digital thermometer) will be assessed from Screening, pre-dose Day 1 then 1, 2, 4, 6, 8 and 12 hrs post-first dose and then pre-dose every 12 hrs on Days 2, 3, 4, 5, 6 and Day 7, then on morning of Day 8, Day 9, Day 10 and Day 14 Safety Follow Up Visit. Single ECG's will be conducted at Screening, Day -1, pre-dose Day 1 then 2, 4, 8 and 12 hrs post-first dose and in the morning on other days, as indicated, except on Days 3, 4 and 7. On Days 3, 4 and 7, ECG performed 5 hrs after morning dose. Full physical exam - General appearance, skin, lungs, neurological system, and heart conducted at Screening, pre-dose Day 1, Day 10 and Day 14 Safety Follow Up Visit. Clinical laboratory and blood and urine samples will be collected from Screening, Day -1, pre-dose Day 1, then Days 2, 4, 6, 8, 10 and Day 14 Safety Follow Up Visit.
Secondary outcome [1] 446870 0
To determine the plasma pharmacokinetics (PK) of multiple MZE782 doses
Timepoint [1] 446870 0
Blood plasma will be collected and assessed at the following timepoints: predose and at 0.5, 1, 2, 3, 4, 6, 8, 12 hours after the first dose on Day 1 and first dose on Day 7. A pre dose sample will be collected on Days 2 to 6 prior to the morning dose. Single PK samples will also be collected 24 hours (Day 8) and 48 hours (Day 9) after the first dose on Day 7.

Eligibility
Key inclusion criteria
1. Age is 18 to 60 years (inclusive) at the time of signing the informed consent.
2. BMI is within 18.5 to 32.0 kg/m2 (inclusive) and weight is at least 55 kg.
3. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
4. Overtly healthy, as determined by the investigator through medical history, physical examination, vital signs, ECGs, and laboratory safety tests performed at the screening visit and prior to administration of initial dose of study drug. Slight excursions outside the limits of normal may be acceptable if deemed to be clinically not significant by the investigator.
5. Negative tests for, HBsAg, anti-HCV, and HIV antibody at the screening visit (positive anti-HCV antibody allowed if HCV PCR is negative).
6. Biological female participants must have a negative pregnancy test at screening and at check-in. Not required of post-menopausal female participants if greater than or equal to 12 months without menses and follicle-stimulating hormone (FSH) documented in post-menopausal range (greater than or equal to 40 IU/L).
7. Use of highly effective contraception is required as follows:
i. Female participants of childbearing potential (ie, ovulating, premenopausal, and not surgically sterile;) must use highly effective methods of birth control starting 2 weeks prior to admission to the CRU until 33 days after the last administration of study drug. Highly effective methods of birth control are defined as those with 99% or greater efficacy (Trussell, 2004). Hormonal contraceptives are not prohibited but cannot be considered highly effective pending additional drug interaction investigations. Acceptable methods of birth control include:
a. Complete abstinence from sexual intercourse if this is the participant’s usual and preferred lifestyle.
b. Dual method of contraception including: i) condom in conjunction with a intrauterine device (hormonal or nonhormonal); ii) condom and tubal ligation/ occlusion; or iii) condom and vasectomy (with documented azoospermia 90 days post procedure).
Participants must agree to abstain from egg donation through 33 days after administration of the last dose of study drug.
ii. All male participants with sexual partners of childbearing potential must use highly effective methods of birth control from time of dosing until 93 days after the last administration of study drug, specifically double barrier contraception or complete abstinence if this is part of the participants usual lifestyle. Acceptable methods of double barrier contraception are: i) condom and hormonal contraception in female partner with hormonal contraception started at least 30 days prior; ii) condom and IUD; iii) or condom and tubal ligation/occlusion or iv) condom and vasectomy with documented azoospermia at least 90 days post procedure. Participants must agree to abstain from sperm donation through 93 days after administration of the last dose of study drug. Female partners of male participants should use a dual method of contraception (as described for female participants) and includes hormonal contraception as long as it is part of the dual method.
Participants must agree to abstain from sperm donation through 93 days.
iii. Participants with solely same-sex partners are not required to use contraception.
8. Post-menopausal female participants or those with a history of hysterectomy or bilateral salpingectomy and/or bilateral oophorectomy do not require contraception if greater than or equal to 12 months without menses and follicle-stimulating hormone (FSH) documented at screening in post-menopausal range (greater than or equal to 40 IU/L).
Minimum age
18 Years
Maximum age
60 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key 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 (s/p appendectomy is allowed).
2. Any chronic medical condition requiring ongoing treatment.
3. History of cancer within the past 3 years, except for treated non-melanoma skin cancer.
4. Any clinically significant illness, medical/surgical procedure, or trauma within 4 weeks prior to the screening visit, as per investigator judgement. Additionally, any major surgery (in the opinion of the investigator within 3 months prior to screening visit.
5. Clinically significant abnormal ECG, including but not limited to QTc >450 ms at screening and/or check-in or history of QT interval prolongation or known history of cardiac arrhythmia.
Note: ECGs may be repeated up to 3 times as per investigator discretion at any timepoint to obtain a clinically reliable result.
6. Vital sign outside the normal ranges, specifically for blood pressure and heart rate taken in seated or supine position, as well as for tympanic temperature:
i. Systolic blood pressure 140 mg Hg, inclusive
ii. Diastolic blood pressure 90 mg Hg, inclusive
iii. Heart rate 99 bpm, inclusive
iv. Body temperature (tympanic) outside of the range 35.5 - 37.5 °C
Note: assessments for vital signs may be repeated as per investigator discretion at any timepoint to obtain a clinically reliable result.
7. History of drug hypersensitivity (based on investigator judgement) or anaphylaxis.
8. Use of any investigational drug within 30 days or <5 half-lives, whichever is longer, prior to first dose of study drug.
9. Consumption of food and/or beverages containing caffeine- or xanthine-containing products (eg, coffee, tea, cola drinks, and chocolate), red wine or other alcohol, Seville oranges or Seville orange juice, grapefruit or grapefruit juice, or poppy seeds within 48 hours prior to check-in.
10. Known sensitivity to any of the study drug formulation components.
11. More than social/casual use of any tobacco or nicotine products (including e-cigarettes, vaping, or dipping) within the last 2 months before the screening visit (social/casual use is up to 2 cigarettes/day or up to 5-10/week) and any use of tobacco or nicotine products within 72 hours before the screening visit (less than 2 cigarettes/day or up to 5-10/week within the 2 months before screening is not exclusionary as long as there has been no use in the 72 hours prior to screening). Testing for cotinine will be performed at screening and check-in as per the SOA and must be negative for inclusion. Testing for cotinine may be repeated at the discretion of the investigator should the investigator suspect that a false-positive result has occurred.
12. History of alcohol or drug abuse within the past 12 months or positive drug screen (including marijuana) at the time of screening or check-in visit. Consumption of more than 14 drinks per week by males or more than 10 per week by females would be considered alcohol abuse.
13. Donation of any blood or blood products at a blood bank or donation center within the 30 days prior to the screening visit or after the end of study or receipt of blood or blood products within 3 months prior to screening visit.
14. Strenuous exercise within 48 hours of check-in visit.
15. Pregnant, breastfeeding, or planning to become pregnant (either self or partner).
16. In the investigator’s judgement, the participant should not participate.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Participants will be randomised to study treatment using a central Interactive Web Response System (IWRS). Before the study is initiated, login information and directions for the IWRS (including directions for emergency unblinding) will be provided to the study site. Participants who meet the study eligibility criteria will be assigned a unique randomisation number in ascending numerical order. The randomisation number encodes the participant’s assignment to receive either MZE782 or placebo, according to the randomization schedule generated prior to dosing. Each participant will be administered blinded study drug, labeled with their unique randomization number, throughout the study. The randomisation schedule will be kept in a secure area which is restricted to unblinded study personnel only.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
The allocation to MZE782 or placebo will be performed using a block randomisation algorithm and will be documented in the study randomisation schedule.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis
A formal statistical analysis plan will be finalized prior to database lock. Statistical analyses will be performed using Statistical Analysis Software (SAS) version 9.4 or higher. Statistical analysis of disposition, demographics/baseline data, treatment compliance and exposure data will be outlined in a Statistical Analysis Plan.

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
1/07/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
24
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 27846 0
Nucleus Network - Melbourne
Recruitment postcode(s) [1] 44041 0
3004 - Melbourne

Funding & Sponsors
Funding source category [1] 318820 0
Commercial sector/Industry
Name [1] 318820 0
Maze Therapeutics
Country [1] 318820 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
Maze Therapeutics
Address
Country
United States of America
Secondary sponsor category [1] 321265 0
Commercial sector/Industry
Name [1] 321265 0
Avance Clinical Pty Ltd
Address [1] 321265 0
Country [1] 321265 0
Australia

Ethics approval
Ethics application status
Submitted, not yet approved
Ethics committee name [1] 317435 0
Alfred Hospital Ethics Committee
Ethics committee address [1] 317435 0
Ethics committee country [1] 317435 0
Australia
Date submitted for ethics approval [1] 317435 0
07/05/2025
Approval date [1] 317435 0
Ethics approval number [1] 317435 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 141022 0
Dr Philip Ryan
Address 141022 0
Nucleus Network Pty Ltd, Level 5, Burnet Tower, 89 Commercial Rd, Melbourne, VIC, 3004
Country 141022 0
Australia
Phone 141022 0
+61 0385 939 801
Fax 141022 0
Email 141022 0
[email protected]
Contact person for public queries
Name 141023 0
Philip Ryan
Address 141023 0
Nucleus Network Pty Ltd, Level 5, Burnet Tower, 89 Commercial Rd, Melbourne, VIC, 3004
Country 141023 0
Australia
Phone 141023 0
+61 0385 939 801
Fax 141023 0
Email 141023 0
[email protected]
Contact person for scientific queries
Name 141024 0
Philip Ryan
Address 141024 0
Nucleus Network Pty Ltd, Level 5, Burnet Tower, 89 Commercial Rd, Melbourne, VIC, 3004
Country 141024 0
Australia
Phone 141024 0
+61 0385 939 801
Fax 141024 0
Email 141024 0
[email protected]

Data sharing statement
Will the study consider sharing individual participant data?
No


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.