Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12625000511437
Ethics application status
Approved
Date submitted
12/02/2025
Date registered
23/05/2025
Date last updated
23/05/2025
Date data sharing statement initially provided
23/05/2025
Type of registration
Prospectively registered

Titles & IDs
Public title
Study to Evaluate the Safety and Pharmacokinetics of M102 in Healthy Participants
Scientific title
A Two-Part, Double-Blind, Placebo-Controlled, Phase 1 Study of the Safety and Pharmacokinetics of Single and Multiple Ascending Doses of M102 in Healthy Volunteers
Secondary ID [1] 313564 0
M102-C01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic lateral sclerosis (ALS) 336074 0
Condition category
Condition code
Neurological 332622 332622 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This is a double-blind, placebo-controlled, ascending dose, multi-cohort trial. The study will be conducted in 2 phases: a single ascending dose (SAD) phase Part A and a multiple ascending dose (MAD) phase Part B in healthy volunteers.

In Part A, participants will receive a single-dose of either study drug (M102) or placebo. In Part B, participants will receive daily doses of M102 or placebo for 14 days. The first Part B cohort (Cohort 1) will be dosed for 7 days. In Part A and Part B, sequential cohorts will be exposed to increasing doses of M102 in order to identify an optimal therapeutic dose for future studies.

Part A- This consists of 6 cohorts where participants will receive a single-dose of either study drug (M102) or placebo on Day 1. The planned doses of IP from A1 to A5 will be 70mg, 140mg, 280mg, 560mg and 1120 mg which will be conducted in fasted state (no food for 8 hours prior to dosing). Based upon review of the safety and PK data from Part A, a cohort (1 of Cohort A1 to A5) will be invited to return for a crossover food effect study (Cohort A6). All cohorts will consent to this adaptive design upon study enrollment. The food effect study (Cohort A6 at target therapeutic dose) may be run concurrently with other cohorts.

Please note:
Food effect cohort participants will receive the same treatment they previously received during their initial dosing
The washout period for the crossover food effect cohort will be at least 7 days between the two treatment periods.

Part B- This part consists of 5 cohorts where participants will receive either study drug or placebo on a daily basis for 7 days (cohort 1) or 14 days (cohort 2-5). The planned dose of IP fromB1 to B3 are as follows- 140mg, 280 and 560mg. Doses for B4 and B5 will be based on the review of safety, tolerability and pharmacokinetic (PK) of the previous cohorts.

Following ongoing review of safety and available PK data from Part A, as well as modeling of expected repeated-dose steady state exposures with daily dosing, the multiple ascending dose Part B will commence in a minimum of 3 cohorts and up to 5 cohorts. Part B may commence prior to completion of all cohorts in Part A.

Adherence to the intervention will be ensured by participant confinement during the dosing period.
Intervention code [1] 330158 0
Treatment: Drugs
Comparator / control treatment
Placebo will be an oral capsule matching with study drug with no active ingredients.
Control group
Placebo

Outcomes
Primary outcome [1] 340198 0
Composite outcome assessing the safety and tolerability profile of a single oral dose of M102 over a range of dose exposures in healthy volunteers.
Timepoint [1] 340198 0
- Adverse events monitored from screening to Day 8 post first dose administration for Part A and Day 14 post dose first dose administration for Part B. - Safety Lab parameters from screening to Day 8 post first dose administration for Part A and Day 14 post dose first dose administration for Part B. - Vital signs will be assessed from screening to Day 8 post first dose administration for Part A and Day 14 post dose first dose administration for Part B. All measures will be assessed as a composite outcome
Primary outcome [2] 340199 0
Composite outcome assessing the safety and tolerability profile (including cardiovascular safety) of up to 14 days of once-daily (QD) or twice-daily (BID, Cohort B5) oral doses with M102 over a range of dose exposures in healthy volunteers.
Timepoint [2] 340199 0
- Adverse events monitored from screening to Day 8 post first dose administration for Part A and Day 14 post dose first dose administration for Part B. - Safety Lab parameters from screening to Day 8 post first dose administration for Part A and Day 14 post dose first dose administration for Part B. - Vital signs will be assessed from screening to Day 8 post first dose administration for Part A and Day 14 post dose first dose administration for Part B. - ECG will be monitored from baseline (-Day 1) Day 8 post first dose administration for Part A and Day 14 post dose first dose administration for Part B
Secondary outcome [1] 443236 0
To establish the pharmacokinetic (PK) profile of M102 in blood in healthy volunteers, following single doses and following repeated dosing and attainment of steady state exposures.
Timepoint [1] 443236 0
-Part A (SAD): Predose on Day 1 (within 60 minutes of dose), and 0.25, 0.5, 1, 2, 4, 6, 8, 12, 24 (Day 2), 36, and 48 (Day 3) hours post dose. -Part B (MAD B1): Day 1 and Day 7 Predose (within 60 minutes of dose), and 0.25, 0.5, 1, 2, 4, 6, 8, 12, and 24 (Day 2 and Day 8) and 48 (Day 9 only) hours post dose, as well as Predose on Day 5 and Day 6. -Part B (MAD B2 through B4): Day 1 and Day 14 Predose and 0.25, 0.5, 1, 2, 4, 6, 8, 12, 24 (Day 2 and Day 15) and 48 (Day 16 only) hours post dose, as well as predose on Day 12 and Day 13. -Part B (MAD, Cohort B5): Predose on Day 1 and 0.25, 0.5, 1, 2, 4, 6, 8, and 12 hours post dose, as well as Predose on Day 12 and Day 13 (within 60 minutes of dose), and Predose on Day 14, and 0.25, 0.5, 1, 2, 4, 6, 8, 12, 24 (Day 15), and 48 (Day 16) hours post-final dose.
Secondary outcome [2] 443237 0
Composite outcome to determine if repeated dosing and steady state exposures alter the clearance parameters of M102.
Timepoint [2] 443237 0
-Part A (SAD): Predose on Day 1 (within 60 minutes of dose), and 0.25, 0.5, 1, 2, 4, 6, 8, 12, 24 (Day 2), 36, and 48 (Day 3) hours post dose. - Part B (MAD B1): Day 1 and Day 7 Predose (within 60 minutes of dose), and 0.25, 0.5, 1, 2, 4, 6, 8, 12, and 24 (Day 2 and Day 8) and 48 (Day 9 only) hours post dose, as well as Predose on Day 5 and Day 6. -Part B (MAD B2 through B4): Day 1 and Day 14 Predose and 0.25, 0.5, 1, 2, 4, 6, 8, 12, 24 (Day 2 and Day 15) and 48 (Day 16 only) hours post dose, as well as Predose on Day 12 and Day 13. -Part B (MAD, Cohort B5): Predose on Day 1 and 0.25, 0.5, 1, 2, 4, 6, 8, and 12 hours post dose, as well as Predose on Day 12 and Day 13 (within 60 minutes of dose), and Predose on Day 14, and 0.25, 0.5, 1, 2, 4, 6, 8, 12, 24 (Day 15), and 48 (Day 16) hours post-final dose.
Secondary outcome [3] 443238 0
To investigate the urinary excretion of M102 following single doses
Timepoint [3] 443238 0
-Part A (SAD): predose on Day 1, 0 to 4 hours, 4 to 8 hours, and 8 to 24 hours (Day 2) postdose. -Part B (MAD, Cohorts B1 through B4): Day 1 and Day 7 (Cohort B1) or Day 14 (Cohorts B2 through B4): predose on Day 1 and Day 7 or Day 14, and 0 to 4 hours, 4 to 8 hours, and 8 to 24 hours postdose (Day 2) (or post-final dose, Day 8 or Day 15). -Part B (MAD, Cohort B5): Day 1 and Day 14: predose on Day 1 and Day 14, and 0 to 4 hours, 4 to 8 hours, 8 to 12 hours, as well as 12 to 24 (Day 15) hours post-final dose only
Secondary outcome [4] 443239 0
To determine the food effect of M102 following single oral dosing.
Timepoint [4] 443239 0
-Part A (SAD): Predose on Day 1 (within 60 minutes of dose), and 0.25, 0.5, 1, 2, 4, 6, 8, 12, 24 (Day 2), 36, and 48 (Day 3) hours post dose. - Part B (MAD B1): Day 1 and Day 7 Predose (within 60 minutes of dose), and 0.25, 0.5, 1, 2, 4, 6, 8, 12, and 24 (Day 2 and Day 8) and 48 (Day 9 only) hours post dose, as well as Predose on Day 5 and Day 6. -Part B (MAD B2 through B4): Day 1 and Day 14 Predose and 0.25, 0.5, 1, 2, 4, 6, 8, 12, 24 (Day 2 and Day 15) and 48 (Day 16 only) hours post dose, as well as Predose on Day 12 and Day 13. -Part B (MAD, Cohort B5): Predose on Day 1 and 0.25, 0.5, 1, 2, 4, 6, 8, and 12 hours post dose, as well as Predose on Day 12 and Day 13 (within 60 minutes of dose), and Predose on Day 14, and 0.25, 0.5, 1, 2, 4, 6, 8, 12, 24 (Day 15), and 48 (Day 16) hours post-final dose.
Secondary outcome [5] 443240 0
To investigate cerebrospinal fluid (CSF) penetration of M102 following up to 14 days of QD or BID (Cohort B5) oral doses.
Timepoint [5] 443240 0
Part B (MAD) Cohorts B2 through B5: postdose (approximately at Tmax) on Day 14 (single sample).

Eligibility
Key inclusion criteria
1. Aged 18 to 64 years (inclusive) at the time of informed consent.
2. At the discretion of the PI or designee, in good general health, with no significant medical history, and have no clinically significant abnormalities on physical examination at Screening and/or before the first administration of IP.
3. BMI between more than equal to 18.0 and less than equal to 32.0 kg/m2 and weight more than equal to 50 kg.
4. Non-smokers and casual smokers who smoke less than equal to 5 cigarettes or equivalent (eg, cigars, vaping, nicotine patches) per week can be included in the study at the discretion of the PI or designee, provided they can abstain from smoking from time of Screening until EOS.
5. Clinical laboratory values within normal range as specified by the testing laboratory, unless deemed not clinically significant by the PI or designee.
6. Not pregnant or breastfeeding, or willing to cease breastfeeding.
7. Woman of childbearing potential or fertile man agrees to use an acceptable method of contraception from the start of Screening until 90 days after the last dose of IP.
Notes:
a. Males must be congenitally sterile (with supportive medical documentation), or surgically sterile (> 30 days since vasectomy with no viable sperm) or, if engaged in sexual relations with a WOCBP, must agree to use an acceptable contraceptive method.
b. Females or males with same-sex partners (abstinence from penile-vaginal intercourse) or who are abstinent from heterosexual intercourse are not required to use contraception when this is their preferred and usual lifestyle.
c. Males must not donate sperm from the first dose of IP until at least 90 days after the last dose of IP.
d. Females are required to abstain from ova/egg donation during the study
8. For Cohort A6 only, be willing to consume a standardized, high-fat, high-calorie meal on the morning of dosing.
9. Participant agrees to avoid cruciferous vegetables (ie, broccoli, cauliflower, brussels sprouts, artichoke, kale, Chinese cabbage, radish, wasabi, horseradish, turnip) from at least 14 days prior to first dose of the study drug until 7 days post-final dose.
10. Participant is normotensive as defined by systolic blood pressure (BP) less than equal to 140 mmHg and diastolic less than equal to 90 mmHg.
11. Able and willing to attend the study site for the full confinement period.
12. Able and willing to provide written informed consent after the nature of the study has been explained and prior to the commencement of any study procedures.
Minimum age
18 Years
Maximum age
64 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1. Underlying physical or psychological medical condition that, in the opinion of the PI or designee, would make the participant unlikely to comply with the protocol or complete the study per-protocol.
2. Recent history (within 12 months of Screening) of moderate to severe depressive disorder.
3. Blood or plasma donation or had significant blood loss (more than equal to 500 mL) within 30 days prior to the first administration of IP.
4. Fever (body temperature >38°C) or symptomatic viral or bacterial infection within 2 weeks prior to Screening or Day -1.
5. Poor pill swallowing ability
6. Positive test for hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), human immunodeficiency virus (HIV)-1/ -2 antibody.
7. Infections requiring parenteral antibiotics within 6 months prior to Screening.
8. History of life-threatening infection (eg, meningitis).
9. Vaccination with a live vaccine within 4 weeks prior to the first administration of IP.
10. History of severe allergic or anaphylactic reactions, or sensitivity to the IP or its constituents.
11. History of malignancy, except for non-melanoma skin cancer, excised more than 2 years ago and cervical intraepithelial neoplasia that has been successfully cured more than 5 years prior to Screening.
12. Abnormal ECG findings at Screening or Day -1 that are considered by the PI or designee to be clinically significant. The following abnormalities should be excluded:
a. Participant has left ventricular hypertrophy defined as the combination of the following ECG criteria (both i. and ii. must be met):
i. Voltage criteria (both criteria must be met):
- S in V1 + R in V5 or V6 (whichever is larger) more than equal to 35 mm; and
- R in aVL more than equal to 11 mm.
ii. Repolarization abnormalities (at least one criterion needs to be met):
- At least 1 mm ST depression (horizontal or down-sloping); or
- Abnormal T wave inversions.
b. Participant has other significant ECG abnormalities that might interfere with ECG analysis including evidence of a previous myocardial infarction, flat T waves (particularly in the inferior leads) or more than minor non-specific ST-T wave changes or:
i. QRS > 120 milliseconds (msec).
ii. QT interval corrected using Fridericia’s formula (QTcF) more than equal to 450 msec (men) and more than equal to 460 msec women).
iii. PR interval > 220 msec.
iv. Heart rate < 45 beats per minute (bpm) or > 90 bpm.
v. Complete right bundle branch block or left bundle branch block.
vi. History of Congenital long QT syndrome
13. Participant is regularly using any restricted mediations including 5HT3 antagonists, antihypertensive medications, vasodilators, drugs that may prolong QTc interval, drugs known to modulate CYP2C8 or CYP3A4, or known substrates of CYP1A2.
14. Participant exhibits any signs of dementia or cognitive impairment.
15. Alkaline phosphatase (ALP), aspartate aminotransferase (AST), and alanine aminotransferase (ALT) > 1.5 × upper limit of normal (ULN) at Screening. Repeat testing at Screening is acceptable for out-of-range values following approval by the PI or designee.
16. Positive toxicology screening panel (urine test including qualitative identification of tetrahydrocannabinol (THC), cocaine, amphetamines, barbiturates, benzodiazepines, opiates, methadone, methamphetamines, methylenedioxymethamphetamine (MDMA), and phencyclidine (PCP)], or alcohol breath test.
17. History of substance abuse or dependency or history of recreational intravenous (IV) drug use over the last 2 years (by self-declaration).
18. Regular alcohol consumption defined as > 10 standard drinks per week (where 1 standard drink equal to 360 mL of beer, 45 mL of 40% spirit or a 150 mL glass of wine) or > 4 standard drinks on any single day.
19. Unwilling to abstain from alcohol from 48 hours prior to admission to the study site until EOS.
20. Use of any investigational medical device or investigational drug within 30 days or 5 half-lives of the investigational drug (whichever is longer) prior to the first administration of the IP.
21. Use of (or anticipated use of) any prescription drugs (other than hormone replacement therapy or hormonal contraception; oral contraceptive pills, long-acting implantable hormones, injectable hormones, a vaginal ring, or an intrauterine device [IUD]), over-the-counter medication, herbal remedies, supplements or vitamins within 7 days prior to the first administration of IP (or 14 days for remedies or supplements known to modulate CYP3A4 such as St John’s Wort) and during the course of the study without prior approval of the PI and MM. Simple analgesia (paracetamol, nonsteroidal anti-inflammatory drug [NSAID]) may be permitted at the discretion of the PI.
22. Unwilling to refrain from strenuous exercise (including weightlifting) from 5 days prior to admission to the study site until EOS.
23. Anything that the PI considers would jeopardize the safety of the participant, prevent complete participation in the study, or compromise interpretation of study data.

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)
sealed opaque envelopes
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Simple randomisation using a randomisation table created by computer software (i.e. computerised sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

Intervention assignment
Other design features
Phase
Phase 1
Type of endpoint/s
Safety
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
1/08/2025
Actual
Date of last participant enrolment
Anticipated
15/02/2026
Actual
Date of last data collection
Anticipated
3/03/2026
Actual
Sample size
Target
80
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 27432 0
Nucleus Network - Melbourne
Recruitment postcode(s) [1] 43544 0
3004 - Melbourne

Funding & Sponsors
Funding source category [1] 318027 0
Commercial sector/Industry
Name [1] 318027 0
Aclipse One, Inc.
Country [1] 318027 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
Aclipse One, Inc.
Address
Country
United States of America
Secondary sponsor category [1] 320371 0
None
Name [1] 320371 0
Address [1] 320371 0
Country [1] 320371 0
Other collaborator category [1] 283327 0
Commercial sector/Industry
Name [1] 283327 0
Novotech (Australia) Pty Limited
Address [1] 283327 0
Country [1] 283327 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 316685 0
Alfred Hospital Ethics Committee
Ethics committee address [1] 316685 0
Ethics committee country [1] 316685 0
Australia
Date submitted for ethics approval [1] 316685 0
08/01/2025
Approval date [1] 316685 0
31/01/2025
Ethics approval number [1] 316685 0

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

Contacts
Principal investigator
Name 138662 0
Dr Ofer Gonen
Address 138662 0
Nucleus Network, Level 5, Burnet Tower, 89 Commercial Road, Melbourne Victoria 3004.
Country 138662 0
Australia
Phone 138662 0
+61 03 8593 9801
Fax 138662 0
Email 138662 0
[email protected]
Contact person for public queries
Name 138663 0
Nucleus Network Melbourne
Address 138663 0
Nucleus Network, Level 5, Burnet Tower, 89 Commercial Road, Melbourne Victoria 3004.
Country 138663 0
Australia
Phone 138663 0
+61 1800 243 733
Fax 138663 0
Email 138663 0
[email protected]
Contact person for scientific queries
Name 138664 0
Ira N. Kalfus MD
Address 138664 0
Aclipse One, Inc. 411 Swedeland Rd suite 23 1080, King of Prussia, PA 19406 United States
Country 138664 0
United States of America
Phone 138664 0
+19178177517
Fax 138664 0
Email 138664 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.

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No additional documents have been identified.