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Trial registered on ANZCTR


Registration number
ACTRN12625000163404
Ethics application status
Approved
Date submitted
6/01/2025
Date registered
12/02/2025
Date last updated
12/02/2025
Date data sharing statement initially provided
12/02/2025
Type of registration
Prospectively registered

Titles & IDs
Public title
A Phase 1, Open-label, First-in Human Study to Examine the Safety, Tolerability, Pharmacokinetic Profile, and Preliminary Efficacy of OZ-001 when Administered Orally in Adults with Solid Tumours with a Focus on Triple Negative Breast Cancer (Phase 1a)
Scientific title
A Phase 1, Open-label, First-in Human Study to Examine the Safety, Tolerability, Pharmacokinetic Profile, and Preliminary Efficacy of OZ-001 when Administered Orally in Adults with Solid Tumours with a Focus on Triple Negative Breast Cancer (Phase 1a)
Secondary ID [1] 313164 0
OZS001-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Neoplastic Disorders 335449 0
Condition category
Condition code
Cancer 332007 332007 0 0
Any cancer

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This is a Phase 1, first-in Human, open-label, multiple-dose study in adult patients with solid tumors who have failed or refused all available standard of care therapy. The maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) will also be identified in this study.

Dose Exploration (Phase 1a):
Up to 18 patients are planned to be enrolled to receive OZ-001 capsules at 1 of 3 dose escalation cohorts (up to 6 patients per cohort). Patients will receive once daily oral doses of OZ-001 in cycles of 28 days (Days 1 to 28) until unacceptable toxicity occurs or disease progression. The planned doses of OZ-001 are:
• Cohort 1a - Dose level 1: 280 mg
• Cohort 2a - Dose level 2: 560 mg
• Cohort 3a - Dose level 3: 840 mg

The decision to escalate between dose levels will be made by the SRC following review of safety and tolerability data.

Patients will be trained by study site personnel to self-administer the OZ-001 at home and will be documented in a study Diary for the monitoring of compliance. The patient will take the Diary, packaging of used OZ 001 and any unused OZ-001 to the study site visits where study site personnel will check compliance. Compliance will be assessed by Diary entries, capsule counts and the time taken to swallow all capsules per dose.
Intervention code [1] 329736 0
Treatment: Drugs
Comparator / control treatment
No Control Group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 339607 0
Composite Outcome: Phase 1a: To determine the safety and tolerability of OZ-001 when administered orally to patients with solid tumours.
Timepoint [1] 339607 0
Adverse Events will be graded using the most current version of the National Cancer Institute CTCAE 5-point scale and assessed continuously as they are reported or observed and reviewed daily from Day 1 until 28 days after last dose.
Secondary outcome [1] 440566 0
To determine the MTD of OZ-001 when administered orally to patients with solid tumours.
Timepoint [1] 440566 0
A DLT is defined as any of the treatment-emergent adverse events (TEAEs), as defined by the National Cancer Institute (NCI)-Common Terminology Criteria for Adverse Events (CTCAE) v5.0 and assessed continuously as they are reported or observed and reviewed daily from Day 1 until 28 days after last dose.
Secondary outcome [2] 440569 0
To determine the preliminary efficacy of OZ-001 when administered orally to patients with solid tumours.
Timepoint [2] 440569 0
Efficacy will be assessed by Standard Computed Tomography (CT) / magnetic resonance imaging (MRI) of the brain, chest, abdomen, and pelvis, which will be performed during screening, at the end of Cycle 1 (Day 28 ±1 day) and then every 8 weeks (2 Cycles) for the first 6 months and every 12 weeks thereafter until disease progression occurs or toxicity which ever one occurs first or 1 year post-intervention if neither disease progression nor toxicity is observed.
Secondary outcome [3] 440572 0
To determine the pharmacokinetic (PK) profile of OZ-001 when administered orally to patients with solid tumours.
Timepoint [3] 440572 0
Blood plasma samples will be assessed pre-dose Day 1 0.5, 1, 2, 3, 4, 6 and 8 hrs post-dose, and pre-dose Day 2 and 3. Urine samples will be collected pre-dose Day 1, between 0-4 hrs, 4-8 hrs and 8 - 24 hrs post-dose.
Secondary outcome [4] 444069 0
To determine the RP2D of OZ-001 when administered orally to patients with solid tumours.
Timepoint [4] 444069 0
A DLT is defined as any of the treatment-emergent adverse events (TEAEs), as defined by the National Cancer Institute (NCI)-Common Terminology Criteria for Adverse Events (CTCAE) v5.0 and assessed continuously as they are reported or observed and reviewed daily from Day 1 until 28 days after last dose.
Secondary outcome [5] 444070 0
To determine the preliminary efficacy of OZ-001 when administered orally to patients with solid tumours.
Timepoint [5] 444070 0
Efficacy will be assessed by Standard Computed Tomography (CT) / magnetic resonance imaging (MRI) of the brain, chest, abdomen, and pelvis, which will be performed during screening, at the end of Cycle 1 (Day 28 ±1 day) and then every 8 weeks (2 Cycles) for the first 6 months and every 12 weeks thereafter until disease progression occurs or toxicity which ever one occurs first or 1 year post-intervention if neither disease progression nor toxicity is observed.
Secondary outcome [6] 444071 0
To determine the preliminary efficacy of OZ-001 when administered orally to patients with solid tumours.
Timepoint [6] 444071 0
Efficacy will be assessed by Standard Computed Tomography (CT) / magnetic resonance imaging (MRI) of the brain, chest, abdomen, and pelvis, which will be performed during screening, at the end of Cycle 1 (Day 28 ±1 day) and then every 8 weeks (2 Cycles) for the first 6 months and every 12 weeks thereafter until disease progression occurs or toxicity which ever one occurs first or 1 year post-intervention if neither disease progression nor toxicity is observed.

Eligibility
Key inclusion criteria
1. Must have given written informed consent before any study related activities are performed and must be able to understand the full nature and purpose of the study, including possible risks and adverse effects.
2. Males and females (Phase 1a), greater than or equal to 18 years of age at the time of signing informed consent.
3. Body mass index (BMI) greater than or equal to 18.0 kg/m2, with a body weight greater than or equal to 60 kg at screening.
4. Phase 1a Only: Histopathologically or cytological diagnosis of a solid tumour that is advanced/metastatic. Patients must be considered refractory or intolerant to standard therapies or have refused standard therapy. Patients must have evaluable disease or measurable disease per RECIST1.1.
5. Eastern Cooperative Oncology Group (ECOG) performance status of less than or equal to 2.
6. Life expectancy greater than or equal to 4 months in the opinion of the PI (or delegate).
7. Evidence of adequate hepatic function, defined as:
a. AST or ALT less than or equal to 2.5 × upper limit of normal (ULN) (less than or equal to 5 × ULN if liver metastases are present);
b. Prothrombin time and international normalized ratio less than or equal to 1.5 × ULN, activated partial thromboplastin time (aPTT) less than or equal to 1.5 × ULN. If on chronic anticoagulation, the prothrombin time and aPTT must be within the therapeutic range.
and
c. Bilirubin less than or equal to 1.5 × ULN unless known to have Gilbert’s syndrome then bilirubin less than or equal to 3.0 × ULN.
8. Adequate hematology laboratory assessment defined as:
a. Absolute neutrophil count greater than or equal to 1.00 × 109/L;
b. Hemoglobin greater than or equal to 90 g/L (a transfusion and/or erythropoietin not permitted within 2 weeks prior to blood draw);
and
c. Platelet count greater than or equal to 100 × 109/L.
9. Evidence of adequate renal function, as defined by a calculated creatinine clearance greater than or equal to 60 mL/min as calculated using the Cockcroft-Gault formula.
10. No evidence of uncontrolled intercurrent illness; active bacterial, fungal, or viral infections requiring systemic therapy.
11. Able to swallow and tolerate oral medications.
12. Is able and willing to consume animal products of porcine and bovine origin.
13. Female volunteers:
a. Must be of non-childbearing potential i.e., surgically sterilized (hysterectomy, bilateral salpingectomy, bilateral oophorectomy) at least 6 weeks before the screening visit or post-menopausal (where post-menopausal is defined as no menses for 12 months without an alternative medical cause and a follicle stimulating hormone (FSH) level consistent with post-menopausal status, per local laboratory guidelines),or
b. If of childbearing potential, must:
i. Have a negative pregnancy test at the screening visit and on admission to the study site on Day-1.
ii. Agree not to attempt to become pregnant or donate ova from signing the consent form until at least 30 days after the last dose of OZ-001.
iii. Agree to use adequate contraception (defined as use of a condom by the male partner combined with use of a highly effective method of contraception) from one month prior to screening until at least 30 days after the last dose of OZ-001, if not exclusively in a same-sex relationship or abstinent as a committed lifestyle.
c. Male volunteers, must:
i. Agree not to donate sperm from signing the consent form until at least 90 days after the last dose of OZ-001.
ii. If engaging in sexual intercourse with a female partner who could become pregnant, agree to use adequate contraception (defined as use of a condom combined with use of a highly effective method of contraception) from signing the consent form until at least 90 days after the last dose of OZ-001.
iii. If engaging in sexual intercourse with a female partner who is not of child-bearing potential or a same-sex partner, agree to use a condom from signing the consent form until at least 90 days after the last dose of OZ-001.
14. Have suitable venous access for blood sampling.
15. Willing and able to comply with all study assessments and adhere to the protocol schedule and restrictions.
16. Patients must have available archival tissue (at least 10 formalin-fixed, paraffin-embedded [FFPE] slides or 1 FFPE block). Patients without any archival tissue or who refuse to provide archival tissue may be approved to enrol on a case-by-case basis in discussion with Sponsor.
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Any major surgery or any therapy within the last 4 weeks and/or not recovered from prior therapy/surgery.
2. Receiving anticancer therapy (chemotherapy, surgery, or radiotherapy, etc.) within 28 days of OZ-001 administration. (Note that patients receiving localized palliative radiation can be allowed on the study after discussion with the Sponsor).
3. Patients with unresolved toxicities from prior anticancer therapies not resolved to baseline or Grade 1. Patients with residual AEs >Grade 1 considered NCS may be allowed on a case-by-case basis after consultation with the Sponsor.
4. Confirmed brain metastases, with the exception of stable brain metastases defined as:
a. The patient’s brain metastases have been treated or do not require urgent local treatment at the time of study enrolment or are unlikely to require treatment during the study.
b. The patient has been off dexamethasone or is on a stable dose of dexamethasone of less than or equal to 2 mg daily (or an alternate steroid equivalent) for at least 2 weeks prior to first OZ-001 administration.
c. Patients are stable (no evidence of progression or new enlarging brain metastases) by imaging; same imaging modality (magnetic resonance imaging [MRI] or computed tomography [CT] scan must be used for each assessment) for at least 28 days prior to the first dose of OZ-001.
Note: Patients with a history of carcinomatous meningitis may not participate even if stable clinically.
5. Liver cirrhosis, a history of or positive test results for human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV) antibodies at the screening visit. Patients positive for HCV antibody, negative for viral load by polymerase chain reaction (PCR) and treated may be eligible after discussion with Sponsor.
6. Clinically significant interstitial pulmonary disease.
7. History of risk factors for torsade de pointes (including a family history of long QT syndrome or sudden cardiac death), a known arrythmia, or receiving drugs that prolong the QTc interval.
8. Evidence of abnormal cardiac function, as defined by the following:
a. Presence of New York Heart Association Class III or IV congestive heart failure;
b. Hypertension that cannot be controlled by medications (>160/100 mmHg despite optimal medical therapy);
c. Presence of myocardial infarction in the previous 6 months;
d. Presence of clinically significant bradycardia, or other uncontrolled cardiac arrhythmia defined as Grade 3 or 4 according to NCI-CTCAE Version 5.0 unless the patient has a pacemaker;
e. Prolongation of HR corrected QT interval by Fridericia’s method (QTcF) at rest, where the mean QTcF interval is >470 ms based on ECG. If QTcF exceeds 470 ms the ECG should be repeated 2 more times and the average of the 3 QTcF values should be used to determine the patient’s eligibility.
9. Acute or subacute intestinal obstruction or inflammatory bowel disease within 6 months of study enrolment.
10. Participation in another clinical study of an investigational drug or investigational device within 30 days or 5 half-lives of the investigational drug (whichever is longer) prior to screening.
11. Known hypersensitivity to any of the OZ-001 ingredients.
12. History of anaphylaxis or other significant allergy which, in the opinion of the PI (or delegate), would interfere with the volunteer’s ability to participate in the study.
13. History of second primary malignancy within 3 years prior to starting study treatment (excluding completely resected cervical cancer or surgically resected skin squamous cell or basal cell carcinoma).
14. Major psychiatric disorder and/or history of suicide ideation. Any other psychiatric illness that, in the opinion of the PI (or delegate), may affect compliance with scheduled visits.
15. Female who is pregnant or breastfeeding, or who plans to become pregnant and/or breastfeed.
16. Presence or having sequelae of gastrointestinal, liver, kidney, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs.
17. History of known alcohol or substance abuse and/or a known psychiatric illness/social situation that would limit compliance with study requirements.
18. Use of live vaccinations within 30 days prior to screening.
19. Any thromboembolic events (e.g., deep vein thrombosis, pulmonary embolism, or symptomatic cerebrovascular events); within 6 months.
20. Any medications or food that interact with cytochromes or receiving medications that are strong inhibitors or inducers of cytochrome P450 (CYP)3A4 including grapefruit, grapefruit juice, bitter orange [Seville orange], pomegranate, or star fruit.
21. Patients must not be using immunosuppressive medication >10 mg prednisolone per day or equivalent within 14 days prior to the first dose of the OZ-001, with the exception of local (topical, nasal, or inhaled) steroid use.
Note: Use of immunosuppressive medications as prophylaxis in patients with contrast allergies is acceptable.
22. Any other condition, medical illness, or prior therapy that in the opinion of the PI (or delegate) would make the volunteer unsuitable for this study, including inability to cooperate fully with the requirements of the study protocol or likelihood of noncompliance with any study requirements.

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
Who is / are masked / blinded?



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
24/03/2025
Actual
Date of last participant enrolment
Anticipated
30/06/2026
Actual
Date of last data collection
Anticipated
29/01/2027
Actual
Sample size
Target
18
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 27356 0
The Border Cancer Hospital - Albury
Recruitment postcode(s) [1] 43446 0
2640 - Albury

Funding & Sponsors
Funding source category [1] 317606 0
Commercial sector/Industry
Name [1] 317606 0
Oncozen Co., Ltd
Country [1] 317606 0
Korea, Republic Of
Primary sponsor type
Commercial sector/Industry
Name
Oncozen Co., Ltd
Address
Country
Korea, Republic Of
Secondary sponsor category [1] 319915 0
Commercial sector/Industry
Name [1] 319915 0
Avance Clinical Pty Ltd
Address [1] 319915 0
Country [1] 319915 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 316311 0
Bellberry Human Research Ethics Committee H
Ethics committee address [1] 316311 0
Ethics committee country [1] 316311 0
Australia
Date submitted for ethics approval [1] 316311 0
27/11/2024
Approval date [1] 316311 0
29/01/2025
Ethics approval number [1] 316311 0
2024-11-1943

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

Contacts
Principal investigator
Name 137482 0
Dr Brett Hamilton
Address 137482 0
Albury Wodonga Cancer Care Centre, 477 Wilson Street, Albury, NSW, 2640
Country 137482 0
Australia
Phone 137482 0
+61 428185041
Fax 137482 0
Email 137482 0
bhamilton@alburywodongacancercare.com
Contact person for public queries
Name 137483 0
Brett Hamilton
Address 137483 0
Albury Wodonga Cancer Care Centre, 477 Wilson Street, Albury, NSW, 2640
Country 137483 0
Australia
Phone 137483 0
+61 428185041
Fax 137483 0
Email 137483 0
bhamilton@alburywodongacancercare.com
Contact person for scientific queries
Name 137484 0
Brett Hamilton
Address 137484 0
Albury Wodonga Cancer Care Centre, 477 Wilson Street, Albury, NSW, 2640
Country 137484 0
Australia
Phone 137484 0
+61 428185041
Fax 137484 0
Email 137484 0
bhamilton@alburywodongacancercare.com

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


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.