Trial Review

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Trial registered on ANZCTR


Registration number
Help: Registration number
ACTRN12626000289314
Ethics application status
Help: Ethics application status
Approved
Date submitted
Help: Date submitted
22/12/2025
Date registered
Help: Date registered
5/03/2026
Date last updated
Help: Date last updated
14/10/2026
Date data sharing statement initially provided
Help: Data sharing statement date
5/03/2026
Type of registration
Help: Type of registration
Retrospectively registered

Titles & IDs
Public title
A Phase 1b study evaluating the safety and preliminary efficacy of AXA-042 monotherapy in patients with advanced solid tumours.
Scientific title
Part C: A Phase 1b, open-label, non-randomised, multicentre dose-expansion study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of AXA-042 monotherapy in subjects with advanced solid tumours.
Additional trial identifiers [1] 316209 0
None
Universal Trial Number (UTN)
Trial acronym
Related trial records
This registration if for Part C of the same study with registration number ACTRN12622000993796 (Part A) and ACTRN12625000863437 (Part B).

Health condition
Health condition(s) studied:
Advanced solid tumours 340206 0
Condition category
Condition code
Cancer 336403 336403 0 0
Kidney
Cancer 336405 336405 0 0
Other cancer types

Intervention/exposure
Study type
Interventional
Description of the intervention or exposure
Participants will receive AXA-042 as monotherapy administered by intravenous infusion once every 3 weeks (21-day treatment cycle). The dose administered will be 0.0003 mg/kg, every 3 weeks. Pending evaluation of efficacy and safety by the Safety Monitoring Committee, the dose may be increased to 0.0004 mg/kg, every 3 weeks.
Treatment duration: treatment will continue until disease progression, unacceptable toxicity, withdrawal of consent, investigator decision, or end of study.
Intervention code [1] 332833 0
Treatment: Drugs
Description of the comparator or control
None
Control group
Uncontrolled

Outcomes
Primary outcome [1] 343919 0
Determine the incidence of adverse events, serious adverse events, adverse events of special interest and dose-limiting toxicities. This will be assessed as a composite outcome.
Timepoint [1] 343919 0
- Continuously from first dose (Cycle 1 Day 1) until end of treatment and 30 days after last dose. - Dose limiting toxicities assessed during the first treatment cycle (Cycle 1 Day 1 to Day 21). - Safety assessments (laboratory parameters, vital signs, ECG, ECOG performance status, physical examination) conducted at screening, pre-dose on each treatment cycle, during treatment visits, at end-of-treatment, and during safety follow-up.
Secondary outcome [1] 456105 0
Concentrations of AXA-042 in plasma, including the concentration-time profile and PK parameters such as AUC0-336h, AUC0-504h, AUC0-last, AUC0-inf, t½, MRT in vivo, Vd, CL, Cmax, and tmax.
Timepoint [1] 456105 0
Blood samples collected at Cycle 1 Day 1 pre-dose and post-dose at 0.25, 1, 2, 4 and 6 hours.
Secondary outcome [2] 456106 0
Incidence of immunogenicity (anti-drug antibodies) against AXA-042 in serum.
Timepoint [2] 456106 0
Immunogenicity assessments at Cycle 1, Cycle 5 and Cycle 10 only.
Secondary outcome [3] 456107 0
Pharmacodynamic (PD) parameters evaluated in blood including cytokines and immune related gene expression.
Timepoint [3] 456107 0
PD assessed pre-dose on C1 Day 1 and post dose 1h, 4h, C1 Day 2, C2 D1 pre dose and 4h post dose, C3 D1 pre dose and 4h post dose. PD samples will be collected on Cycle 3 Day 1 and for every odd cycle after Cycle 3 (e.g., Cycle 5, Cycle 7…) at the same time points indicated for Cycle 3. These samples are required until such time as sufficient PD data has accrued to satisfactorily characterize the effect of the AXA-042. The decision to forgo the need for these samples to be taken in one or more cohorts will be made by Axelia Oncology in consultation with the Safety Monitoring Committee.
Secondary outcome [4] 456111 0
Tumour response assessed using RECIST v1.1, iRECIST 1.1 and PERCIST 1.0.
Timepoint [4] 456111 0
Tumour assessments will be conducted at Screening, at Cycle 3 Day 1, Cycle 5 Day 1, and thereafter, every 3 cycles (i.e. Cycle 8 Day 1, Cycle 11 Day 1, etc.).
Secondary outcome [5] 456113 0
Quality of life assessment using a validated questionnaire.
Timepoint [5] 456113 0
Quality of life parameters will be assessed at Screening, at Cycle 3 Day 1, Cycle 5 Day 1, and thereafter, every 3 cycles
Secondary outcome [6] 456116 0
Pharmacodynamic (PD) parameters evaluated in tumour tissue including histology, gene expression and target engagement biomarkers.
Timepoint [6] 456116 0
At screening (pre-dose). For a subgroup of participants at Screening and Cycle 1 Day 15.

Eligibility
Key inclusion criteria
1. Diagnosis of a histologically or cytologically confirmed locally advanced or metastatic cancer (all solid tumours). Subjects must be considered refractory or intolerant to the standard of care therapies or have refused standard therapy.
2. Age greater or equal to 18 years old at the time of Screening (signing the Informed Consent Form [ICF])
3. Eastern Cooperative Oncology Group (ECOG)9 performance status 0 to 1.
4. The estimated life expectancy of at least 3 months as per the Investigator’s judgment.
5. At least one measurable disease tumour lesion by Response Evaluation Criteria in Solid Tumours Version 1.1 (RECIST v1.1) criteria.10 For suitable subjects, such as subjects that may not have measurable lesions, positron emission tomography (PET) may be implemented in addition to, or in place of, RECIST v1.1, to monitor disease response (PERCIST; PET response criteria in solid tumours version 1.0) on discussion and approval by the sponsor’s medical monitor.
6. Subjects who have undergone treatment with an anti-PD-1, anti-PD-L1, or anti-CTLA-4 antibody must have a gap of at least 4 weeks from the last dose of antibody and evidence of disease progression per the Investigator's assessment before enrolment.
7. Subjects who have previously received an immune CPI prior to enrolment must have any immune- related toxicities resolved less than or equal to Grade 1 or baseline (prior to the CPI) with the exception of toxicities not considered a safety risk (e.g., hypothyroidism, alopecia, neuropathy, or asymptomatic laboratory abnormalities).
8. Adequate organ function based on laboratory assessments at Screening, as defined by:
a. Haemoglobin greater than or equal to 90 g/L (subjects may be transfused more than 2 weeks before Screening, but should not be transfusion -dependent)
b. Platelets greater than or equal to 100 × 109/L
c. Absolute neutrophil count greater than or equal to 1.5 × 109/L
d. Serum creatinine less than or equal to 1.5 × upper limit of normal (ULN); or a calculated creatinine clearance (Cockcroft-Gault method) greater than or equal to 50 mL/minute if serum creatinine greater than 1.5 × ULN. Lower calculated creatinine clearance values may be allowed at the Investigator’s discretion and in consultation with the Medical Monitor and Sponsor.
e. Total bilirubin less than or equal to ULN; or conjugated bilirubin less than or equal to ULN and total bilirubin less than or equal to 1.5 × ULN (less than 3.0 ×ULN for subjects with liver metastases or Gilbert’s syndrome)
f. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) less than or equal to 3 × ULN (AST and ALT less than or equal to 5 × ULN if liver metastases present)
g. International normalized ratio and activated partial thromboplastin time less than or equal to 1.5 × UL.
9. Available archived tumour tissue sample (block of formalin-fixed paraffin-embedded [FFPE] tissues) to allow for exploratory biomarker studies. In the setting where archival material is unavailable or unsuitable for use (e.g., recently diagnosed subjects or diagnosed with fine -needle aspiration), the subject will have an option to consent for and undergo fresh tumour biopsy (at acceptable risk as judged by the Investigator). The requirement for fresh biopsy collected from a given subject could be waived after the discussion with the Medical Monitor if the tumour tissues are not safely accessible as determined by the Investigator or the tumour biopsies have to be obtained from sites that require significant risk procedures. For Part C, subjects may be advised by their study doctor to undergo mandatory pretreatment (during screening period) biopsies and 2 weeks post first treatment cycle.
10. Female subjects must not be pregnant, or must be of non- childbearing potential as defined or if of childbearing potential, must agree to use highly effective birth control methods during the study treatment period and for at least 90 days after the last dose of the study treatment.
11. Non-sterilized male subjects must agree to use contraception as detailed in Appendix 6 of this protocol during the treatment period and for at least 90 days after the last dose of the study treatment for Part A and C and 4 months after the last dose of the study treatment for Part B.
12. For women of childbearing potential (WOCBP) only: A negative serum pregnancy test during Screening and a negative serum or urine pregnancy test within 24 hours of the first dose of study treatment.
13. Voluntarily agrees to participate by giving written informed consent and is willing and able to comply with this protocol and scheduled visits.

Additional eligibility requirements for participants enrolled in each tumour-specific cohort in Part C are described below:
Renal Cell Carcinoma (RCC) Cohort:
1. Histologically or cytologically proven clear cell renal cell carcinoma. Participants with mixed clear cell and other histology may be approved on a case-by-case basis in discussion with the study Sponsor.
2. Must have progressed on or is intolerant to immune checkpoint inhibitor therapy.
3. Must have received no more than 3 prior lines of systemic anti-cancer therapy in the advanced or metastatic disease setting.
4. Neoadjuvant and adjuvant not counted as a line of therapy in the advanced disease setting.
5. Maintenance therapy is considered the same line as / counted as one with the preceding systemic treatment regimen.
6. On a case-by-case basis in discussion with the study Sponsor, therapy change due to toxicity may be considered the same line as / counted as one with the preceding systemic treatment regimen. Participants who have received more than 3 prior lines of systemic anti-cancer therapy may be approved on a case-by-case basis in discussion with the study Sponsor.

BRCA1/BRCA2/PALB2-mutant Ovarian Cancer Cohort:
1. Histologically or cytologically proven high-grade serous epithelial ovarian, fallopian tube or primary peritoneal cancer with known somatic or germline BRCA1/BRCA2/PALB2 pathological aberrancy.
2. Must have progressed within 6 months of or is intolerant to platinum-based chemotherapy (platinum-resistant disease) and have progressed on or is intolerant to a PARP inhibitor therapy.
3. Must have received no more than 3 prior lines of systemic anti-cancer therapy in the advanced or metastatic disease setting.
4. Neoadjuvant and adjuvant not counted as a line of therapy in the advanced disease setting.
5. Maintenance therapy, e.g., bevacizumab, PARP inhibitor or hormonal therapy, is considered the same line as / counted as one with the preceding systemic treatment regimen.
6. Hormonal therapy is considered its own line of systemic treatment unless given as maintenance.
7. On a case-by-case basis in discussion with the study Sponsor, therapy change due to toxicity may be considered the same line as / counted as one with the preceding systemic treatment regimen. Participants who have received more than 3 prior lines of systemic anti-cancer therapy may be approved on a case-by-case basis in discussion with the study Sponsor.

Triple Negative Breast Cancer (TNBC) Cohort:
1. Histologically or cytologically proven triple negative breast cancer with known somatic or germline BRCA1/BRCA2/PALB2 pathological aberrancy.
2. Must have received no more than 3 prior lines of systemic anti-cancer therapy in the advanced or metastatic disease setting.
3. Neoadjuvant and adjuvant not counted as a line of therapy in the advanced disease setting.
4. Maintenance therapy is considered the same line as / counted as one with the preceding systemic treatment regimen.
5. On a case-by-case basis in discussion with the study Sponsor, therapy change due to toxicity may be considered the same line as / counted as one with the preceding systemic treatment regimen.
6. Participants who have received more than 3 prior lines of systemic anti-cancer therapy may be approved on a case-by-case basis in discussion with the study Sponsor.
Additional eligibility requirements for other tumour types will be described in a future protocol amendment.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Healthy volunteers
No
Key exclusion criteria
1. Subjects diagnosed with glioblastoma, other brain tumours, and haematological cancer. In the case of rare cancers, there may be some exceptions at the Investigator’s discretion in consultation with the Medical Monitor.
2. Prior malignancies active within previous 3 years, except the cancer for which the subject is enrolled in the current study or locally curable cancers that have been cured (e.g., basal cell skin cancer, squamous cell carcinoma, or carcinoma in situ of the cervix or breast).
3. Known active central nervous system metastases or carcinomatous meningitis.
4. Active or documented history of autoimmune disease that has caused terminal organ damage or required systemic immunosuppression and/or systemic disease modulating drugs within the past 2 years, or participant is immunocompromised for any other reason (as determined by the Investigator/Medical Monitor). Note that subjects with active or document history of autoimmune disease that has caused terminal organ damage or required systemic immunosuppression and/or systemic disease modulating drugs within the past 2 years or participant is immunocompromised for any other reason may be allowed on case-by-case basis with the approval of the study Sponsor if it is deemed not to put subject at an increased risk of treatment-related toxicities and/or interfere with study data integrity, e.g., hypothyroidism on adequate thyroid hormone replacement therapy.
5. Active or a history of clinically significant atopy (severe or multiple allergic manifestations). Subjects with asthma requiring the administration of regular inhaled steroids, or a history of asthma requiring hospitalisation will be excluded. Note that subjects with active mild asthma requiring intermittent bronchodilator administration and/or short-term inhaled steroids may be allowed on Medical Monitor/Sponsor approval.
6. Current or known history of rheumatoid arthritis, ankylosing spondylitis, or any other joint inflammatory conditions that have systemic and/or organ involvement, and/or involve disease flares affecting joint function. Osteoarthritis alone is not exclusionary. For uncertain cases, such as those with arthritis of undiagnosed aetiology and elevated titres of rheumatoid factor, anti-citrullinated antibody or anti-nuclear antibody, final determination can be made as per the discretion of the PI in consultation with the Medical Monitor.
7. Active systemic infection requiring treatment with intravenous antibiotics or a significant infection (minor superficial skin infections or urinary tract infections are not exclusionary).
8. History of drug-induced severe hypersensitivity reaction.
9. History of recurrent or clinically significant syncope.1
10. Have uncontrolled pleural effusion(s), pericardial effusion, or ascites.
11. Evidence of abnormal cardiac function as defined by any of the following:
11a. Myocardial infarction within 6 months of Cycle 1 Day 1.
11b. Symptomatic congestive heart failure (New York Heart Association Class II or greater).
11c. Unstable angina.
12. Received chemotherapy or other anticancer therapy within 4 weeks (or 5 half-lives for biologic/non-cytotoxic agents, whichever is shorter) prior to the first dose of study treatment. Exceptions include concurrent standard of care (SOC) therapies such as the use of hormones for noncancer-related conditions (e.g., insulin for diabetes), hormone deprivation therapy (e.g., androgen deprivation therapy for prostate cancer), antiresorptive therapy (e.g., bisphosphonates, denosumab for prevention of osteoporosis, bone disease etc.), and local treatment of isolated lesions for palliative intent (e.g., by local surgery or local radiotherapy). Subjects on SOC therapy are required to be on a stable dose for a period of at least 1 month prior to enrolment without any foreseeable changes in dose during the duration of this trial. Palliative subjects who have received radiotherapy treatment are allowed after 7 days of their last treatment. Any other concurrent SOC therapies not included here are to be discussed and approved by the MM prior to screening of the subject
13. Received prior TLR agonist therapy.
14. Currently taking chronic systemic glucocorticoid therapy in excess of replacement doses (i.e., greater than 10 mg prednisone/day or equivalent) or other systemic immunosuppressive treatment.
15. Have undergone major surgery in the 4 weeks prior to Screening or minor surgical procedures less than or equal to 7 days (no waiting required following port-a-cath placement or for venous access). For any minor surgical procedures related to the biopsy collection, a less than or equal to 7-day washout may be considered sufficient in discussion with study medical monitor.
16. Received investigational therapy or used an investigational device within 4 weeks prior to the first dose of study treatment.
17. Received a live vaccine within 30 days prior to the first dose of study treatment. Administration of an approved coronavirus disease 2019 (COVID-19) vaccine (both the doses of a 2-dose vaccine or a booster shot) greater than or equal to 14 days prior to the first dose of study treatment is allowed. There can be exceptions on a case-by-case basis as approved by the Medical Monitor.
18. Pregnant or breastfeeding or expecting to conceive a child during the study or within 90 days after the last dose of study treatment.
19. Known history of human immunodeficiency virus infection or active infection with hepatitis B or C.
20. Have a psychiatric or substance use disorder that would interfere with cooperation with the requirements of the study.
21. History of any condition or treatment which could confound the results of the study, interfere with the subject’s participation in the study, or make study participation not in the subject’s best interests, in the opinion of the Investigator.
22. Known clinically significant, uncontrolled hypertension. Exception can be made based on MM approval if BP increase is transient due to other reasons like the white coat syndrome or deemed not to put participant at a higher risk of treatment-related toxicity and/or interfere with the integrity of study data.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Safety
Statistical methods / analysis
Given the exploratory nature of this Phase 1b dose-expansion study, the sample size is based on a Simon’s two-stage design rather than formal hypothesis testing. Up to 31 participants may be enrolled across selected tumour indications. An interim futility analysis will be conducted after completion of Stage 1, with continuation to Stage 2 dependent on predefined efficacy criteria.
All analyses, summaries, and listings will be performed using SAS software (version 9.4 or higher). In general data will be presented by AXA-042 dose level and tumour cohort, where applicable. Data for all study subjects combined will also be presented. Individual subject data will be presented in listings. Data will be summarised using descriptive statistics (mean, median, standard deviation, minimum and maximum) or frequency counts and percentages, as appropriate to the type of data.

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
20/01/2026
Actual
20/01/2026
Date of last participant enrolment
Anticipated
30/11/2026
Actual
Date of last data collection
Anticipated
29/10/2027
Actual
Sample size
Target
12
Accrual to date
4
Final
Recruitment in Australia
Recruitment state(s)
NSW,SA,WA,VIC
Recruitment hospital [1] 29010 0
Linear Clinical Research - Nedlands
Recruitment hospital [2] 29011 0
Peter MacCallum Cancer Centre - Melbourne
Recruitment hospital [3] 29012 0
Dandenong Hospital- Monash Health - Dandenong
Recruitment hospital [4] 29013 0
Scientia Clinical Research - Randwick
Recruitment hospital [5] 29014 0
The Chris O’Brien Lifehouse - Camperdown
Recruitment hospital [6] 29015 0
Calvary Mater Newcastle - Waratah
Recruitment hospital [7] 29242 0
Macquarie University Hospital - Macquarie Park
Recruitment hospital [8] 30340 0
Wollongong Hospital - Wollongong
Recruitment hospital [9] 30341 0
Icon Cancer Care Adelaide - Kurralta Park
Recruitment postcode(s) [1] 45290 0
6009 - Nedlands
Recruitment postcode(s) [2] 45291 0
3000 - Melbourne
Recruitment postcode(s) [3] 45292 0
3175 - Dandenong
Recruitment postcode(s) [4] 45293 0
2031 - Randwick
Recruitment postcode(s) [5] 45294 0
2050 - Camperdown
Recruitment postcode(s) [6] 45295 0
2298 - Waratah
Recruitment postcode(s) [7] 45550 0
2109 - Macquarie Park
Recruitment postcode(s) [8] 46757 0
2500 - Wollongong
Recruitment postcode(s) [9] 46758 0
5037 - Kurralta Park
Recruitment outside Australia
Country [1] 28641 0
New Zealand
State/province [1] 28641 0
Auckland City Hospital

Funding & Sponsors
Funding source category [1] 320805 0
Commercial sector/Industry
Name [1] 320805 0
Axelia Oncology Pty Ltd
Country [1] 320805 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Axelia Oncology Pty Ltd
Address
Country
Australia
Secondary sponsor category [1] 323472 0
None
Name [1] 323472 0
Address [1] 323472 0
Country [1] 323472 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 319338 0
Bellberry Human Research Ethics Committee A
Ethics committee address [1] 319338 0
Ethics committee country [1] 319338 0
Australia
Date submitted for ethics approval [1] 319338 0
01/08/2025
Approval date [1] 319338 0
08/09/2025
Ethics approval number [1] 319338 0
2022-04-349-A-23
Ethics committee name [2] 319359 0
The Royal Melbourne Hospital Human Research Ethics Committee
Ethics committee address [2] 319359 0
Ethics committee country [2] 319359 0
Australia
Date submitted for ethics approval [2] 319359 0
29/08/2025
Approval date [2] 319359 0
22/10/2025
Ethics approval number [2] 319359 0

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

Contacts
Principal investigator
Name 147026 0
Prof Michael Millward
Address 147026 0
Ground Floor, B block, QEII Medical Centre, Hospital Avenue, Nedlands WA 6009
Country 147026 0
Australia
Phone 147026 0
+61 8 6373 6201
Fax 147026 0
Email 147026 0
[email protected]
Contact person for public queries
Name 147027 0
Ardian Latifi
Address 147027 0
Southern Clinical Development Consulting (SCDC) Level 17, 60 City Road, Southbank, VIC 3006
Country 147027 0
Australia
Phone 147027 0
+61 421814712
Fax 147027 0
Email 147027 0
[email protected]
Contact person for scientific queries
Name 147028 0
Anna Galkin
Address 147028 0
Axelia Oncology, 31 Queen Street, Melbourne VIC 3000
Country 147028 0
Australia
Phone 147028 0
+1 310 422 5495
Fax 147028 0
Email 147028 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.