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Trial registered on ANZCTR


Registration number
Help: Registration number
ACTRN12626000328370
Ethics application status
Help: Ethics application status
Approved
Date submitted
Help: Date submitted
20/02/2026
Date registered
Help: Date registered
16/03/2026
Date last updated
Help: Date last updated
14/08/2026
Date data sharing statement initially provided
Help: Data sharing statement date
16/03/2026
Type of registration
Help: Type of registration
Prospectively registered

Titles & IDs
Public title
A Randomised, Double-Blind, Placebo-Controlled, First-in-Human Study of Orally Administered LW-1017 to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single and Multiple Ascending Doses of LW-1017 in Healthy Volunteers
Scientific title
A Randomised, Double-Blind, Placebo-Controlled, First-in-Human Study of Orally Administered LW-1017 to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single and Multiple Ascending Doses of LW-1017 in Healthy Volunteers
Additional trial identifiers [1] 316481 0
LW-1017-101
Universal Trial Number (UTN)
Trial acronym
Related trial records

Health condition
Health condition(s) studied:
Neurodegenerative diseases 340604 0
Condition category
Condition code
Neurological 336800 336800 0 0
Alzheimer's disease
Neurological 336801 336801 0 0
Parkinson's disease

Intervention/exposure
Study type
Interventional
Description of the intervention or exposure
This is a double-blind, randomised, placebo-controlled study evaluating the safety, tolerability, and pharmacokinetics (PK) and pharmacodynamics (PD) of oral doses of LW-1017. The study will be conducted in 2 parts: Part A Single Ascending Dose (SAD) and Part B Multiple Ascending Dose (MAD).

Part A (SAD):
Up to 40 participants will be enrolled in up to 5 cohorts (S1–S5). Each cohort will include up to 8 participants (6 LW-1017; 2 placebo). Participants will receive a single oral dose of LW-1017 tablets or matching placebo on Day 1. The starting dose will be 10 mg, with dose escalation up to a maximum of 500 mg. Dose levels for each cohort will be determined by the Safety Review Committee (SRC) based on review of safety and PK data from preceding cohorts.

Study drug administration will be supervised by site staff and documented in study records to ensure compliance.

Part B (MAD):
Up to 40 participants will be enrolled across 5 cohorts (M1–M4 and one healthy elderly cohort). Each cohort will include up to 8 participants (6 LW-1017; 2 placebo).

Participants will receive once-daily (QD) oral LW-1017 tablets or matching placebo for 10 consecutive days (Days 1–10; total of 10 doses). The planned starting dose for Cohort M1 is estimated at 50 mg; however, the final starting dose may be adjusted based on review of Part A (SAD) safety and PK data. Subsequent dose levels (up to a protocol-defined maximum dose) will be determined by the Safety Review Committee (SRC) based on safety and PK data from preceding cohorts and the predicted minimum effective dose (MED).

A healthy elderly cohort (65–80 years) will commence only after acceptable safety and PK data are observed in younger adults (18–50 years) following at least 5 days of dosing at the same or higher exposure.

Study drug administration will be supervised by site staff and documented in study records to ensure adherence and compliance.
Intervention code [1] 333145 0
Treatment: Drugs
Description of the comparator or control
Oral tablets of matching appearance to the investigational product but containing no active drug (placebo), will be administered as placebo in this study.
Control group
Placebo

Outcomes
Primary outcome [1] 344323 0
To assess the safety and tolerability of single oral doses of LW-1017 in healthy adult volunteers. Safety and tolerability will be assessed as a composite primary outcome.
Timepoint [1] 344323 0
Adverse event (AE) severity will be assessed continuously as observed and reviewed daily from Screening until Day 8 End of Study/Early Termination Visit (EoS/ETV). Body weight assessed at Screening, Day -1 then Day 8 End of Study/Early Termination Visit (EoS/ETV). Vital signs will be assessed from Screening, Day -1, pre-dose Day 1 then 0.05, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8 and 12 hrs post-dose, Day 2 24 hrs post-dose, Day 3 48 hrs post-dose, Day 4 72 hrs post-dose then Day 8 End of Study/Early Termination Visit (EoS/ETV). Clinical laboratory blood and urine samples will be collected from Screening, Day -1, Day 1 6 hrs post-dose, Day 2 24 hrs post-dose, Day 4 72 hrs post-dose then Day 8 End of Study/Early Termination Visit (EoS/ETV). Triplicate ECG's will be conducted at Screening, Day -1, pre-dose Day 1 6 hrs post-dose, Day 2 24 hrs post-dose, Day 4 72 hrs post-dose then Day 8 End of Study/Early Termination Visit (EoS/ETV). Columbia-Suicide Severity Rating Scale (C-SSRS) will be assessed at Screening, Day 1, Day 4 then Day 8 End of Study/Early Termination Visit (EoS/ETV).
Primary outcome [2] 344324 0
To assess the safety and tolerability of multiple oral doses of LW-1017 in healthy adult volunteers. Safety and tolerability will be assessed as a composite primary outcome.
Timepoint [2] 344324 0
Adverse event (AE) severity will be assessed continuously as observed and reviewed daily from Screening until Day 17 End of Study/Early Termination Visit (EoS/ETV). Body weight assessed at Screening, Day -1 then Day 17 End of Study/Early Termination Visit (EoS/ETV). Vital signs will be assessed from Screening, Day -1, pre-dose Day 1 then 0.5, 1, 1.5, 2, 3, 4, 6, 8 and 12 hrs post-dose, Day 2 to Day 9 pre-dose then 2 and 6 hrs post-dose, pre-dose Day 10 then 0.5, 1, 1.5, 2, 3, 4, 6, 8 and 12 hrs post-dose, Day 11 24 hrs post-Day 10 dose, Day 12 48 hrs post-Day 10 dose, Day 13 72 hrs post-Day 10 dose then Day 17 End of Study/Early Termination Visit (EoS/ETV). Clinical laboratory blood and urine samples will be collected from Screening, Day -1, Day 1 6 hrs post-dose, Day 2 24 hrs post-Day 1 dose, Day 5 post-dose, Day 10 6 hrs post-dose, Day 11 24 hrs post-Day 10 dose then Day 17 End of Study/Early Termination Visit (EoS/ETV). Triplicate ECG's will be conducted at Screening, Day -1, pre-dose Day 1 6 hrs post-dose, Day 2 24 hrs post-Day 1 dose, Day 5 and Day 6 6 hrs post-dose, pre-dose Day 10 then 6 hrs post-dose, Day 11 24 hrs post-Day 10 dose, Day 12 48 hrs post-Day 10 dose, Day 13 72 hrs post-Day 10 dose and Day 17 End of Study/Early Termination Visit (EoS/ETV). Columbia-Suicide Severity Rating Scale (C-SSRS) will be assessed at Screening, Day 1, Day 13 then Day 17 End of Study/Early Termination Visit (EoS/ETV).
Secondary outcome [1] 457468 0
To characterize the PK profile of LW 1017 in plasma following administration of single oral doses in healthy adult volunteers.
Timepoint [1] 457468 0
Blood plasma samples will be collected pre-dose Day 1 - 0.5, 1, 2, 3, 4, 5, 6, 8 & 12 hrs post-dose, Day 2 24 & 36 hrs post-dose, Day 3 48 hrs post-dose, Day 4 72 hrs post-dose then End of Study/Early Termination Visit (EoS/ETV)
Secondary outcome [2] 457469 0
To characterize the PK profile of LW 1017 in plasma following administration of multiple oral doses in healthy adult volunteers.
Timepoint [2] 457469 0
Blood plasma samples will be collected pre-dose Day 1 then 0.5, 1, 2, 3, 4, 5, 6, 8 & 12 hrs post-dose, Day 2 24 post-Day 1 dose, pre-dose Day 3, pre-dose Day 5, pre-dose Day 7, pre-dose Day 10 then 0.5, 1, 2, 4, 6, 8 & 12 hrs post-dose, Day 11 24 and 36 hrs post-Day 10 dose, Day 12 48 hrs post-Day 10 dose, Day 13 72 hrs post-Day 10 dose then Day 17 End of Study/Early Termination Visit (EoS/ETV).
Secondary outcome [3] 457471 0
To evaluate the concentration of LW-1017 in cerebral spinal fluid (CSF) following administration of multiple oral doses of LW-1017 in healthy adult volunteers (Part B, 1 selected cohort only)
Timepoint [3] 457471 0
CSF samples collected and assessed at Screening and Day 9 or Day 10.

Eligibility
Key inclusion criteria
1. Able to provide written informed consent and comply with study requirements.
2. Healthy adult males and females aged 18–55 years (SAD/MAD cohorts); healthy elderly adults aged 65–80 years (elderly cohort, if applicable).
3. Body mass index (BMI) 18.0–32.0 kg/m² and body weight greater than or equal to 45 kg.
4. Medically healthy based on medical history, physical examination, vital signs, ECG, and laboratory tests, without clinically significant abnormalities.
5. Females must be of non-childbearing potential or agree to use highly effective contraception and have negative pregnancy tests prior to dosing.
6. Males must agree to use appropriate contraception and refrain from sperm donation for the protocol-specified period after dosing.
7. Suitable venous access for blood sampling and willingness to comply with study procedures.
8. For MAD cohorts involving CSF collection: willing and medically eligible to undergo lumbar puncture.
Minimum age
18 Years
Maximum age
80 Years
Sex
Both males and females
Healthy volunteers
Yes
Key exclusion criteria
1. Known hypersensitivity to the study drug or its components.
2. Clinically significant medical conditions (including cardiovascular, hepatic, renal, neurological, psychiatric, or other systemic diseases) that may interfere with study participation or safety.
3. Significant ECG abnormalities, history of clinically relevant arrhythmia, or risk factors for QT prolongation.
4. Clinically significant laboratory abnormalities, including impaired liver or renal function.
5. History of malignancy within the past 5 years (except adequately treated non-melanoma skin cancer).
6. Clinically relevant immunodeficiency or use of immunosuppressive therapy.
7. Positive screening tests for HIV, hepatitis B, or hepatitis C.
8. Positive drug or alcohol screening at admission.
9. Excessive alcohol consumption or significant tobacco/nicotine use.
10. Pregnant or breastfeeding females.
11. Use of medications or substances that may interact with CYP3A4 or interfere with study assessments within protocol-defined timeframes.
12. Recent participation in another clinical trial, recent blood donation, or recent vaccination within protocol-defined timeframes.
13. Active suicidal ideation or behaviour, or significant psychiatric risk as assessed by C-SSRS or clinical evaluation.
14. Any other condition that, in the opinion of the Investigator, makes the participant unsuitable for the study.

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)
All study participants who sign an informed consent form (ICF) will receive a unique sequential number (i.e., a screening number).
Participants who meet the study eligibility criteria will be assigned a randomisation number, which corresponds to a study treatment (LW-1017 or placebo), in accordance with the randomisation schedule. The randomisation schedule will be prepared by unblinded statistician(s) and will be maintained under controlled access. Blinded study staff will not have access to the randomisation schedule until after database lock.
As the study is double-blinded, sealed participant-specific code break envelopes will be produced by the unblinded statistician(s) and will be retained at the study site in a secure, accessible location.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
The allocation to LW-1017 or placebo will be performed using a block randomisation algorithm.
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
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
2/04/2026
Actual
5/05/2026
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
80
Accrual to date
16
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 29109 0
Nucleus Network - Melbourne
Recruitment postcode(s) [1] 45412 0
3004 - Melbourne

Funding & Sponsors
Funding source category [1] 321092 0
Commercial sector/Industry
Name [1] 321092 0
Lysoway Therapeutics Pty Ltd
Country [1] 321092 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Lysoway Therapeutics Pty Ltd
Address
Country
Australia
Secondary sponsor category [1] 323799 0
Commercial sector/Industry
Name [1] 323799 0
Avance Clinical Pty Ltd
Address [1] 323799 0
Country [1] 323799 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 319627 0
Alfred Hospital Ethics Committee
Ethics committee address [1] 319627 0
Ethics committee country [1] 319627 0
Australia
Date submitted for ethics approval [1] 319627 0
04/03/2026
Approval date [1] 319627 0
30/03/2026
Ethics approval number [1] 319627 0
HREC/126769-Alfred-2026

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

Contacts
Principal investigator
Name 147910 0
Dr Ofer Gonen
Address 147910 0
Nucleus Network, Level 5, Burnet Tower, 89 Commercial Road, Melbourne, VIC, 3004
Country 147910 0
Australia
Phone 147910 0
+61 0385 939 801
Fax 147910 0
Email 147910 0
[email protected]
Contact person for public queries
Name 147911 0
Nucleus Network
Address 147911 0
Level 5, Burnet Tower, 89 Commercial Road, Melbourne, VIC, 3004
Country 147911 0
Australia
Phone 147911 0
+61 1800 243 733
Fax 147911 0
Email 147911 0
[email protected]
Contact person for scientific queries
Name 147912 0
Yongchang Qiu, Ph.D.
Address 147912 0
Lysoway Therapeutics, Inc., 245 1st St, Ste 1800 Cambridge, MA 02142, USA
Country 147912 0
United States of America
Phone 147912 0
+1 781 608 8206
Fax 147912 0
Email 147912 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.