New drug aims to supercharge immune system against Hard-to-Treat cancers
NCT ID NCT06302426
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial is testing a new drug called INI-4001, which is designed to activate the immune system to fight advanced solid tumors. About 50 adults with cancers that have spread or cannot be removed will receive the drug alone or combined with standard immunotherapy drugs like nivolumab or pembrolizumab. The main goal is to find the safest dose and check for side effects.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- INI-4001 (a drug that activates the immune system to attack tumors), given alone or with nivolumab or pembrolizumab
- What this could lead to
- If it works, this could point toward a new treatment option for advanced solid tumors by boosting the body's immune response.
- What could go wrong
- This is a very early Phase 1 trial with only 50 people, so it may not work or could have side effects. The main goal is just to find a safe dose, not to prove effectiveness.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 50 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jul 2024
- Expected to finish
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Mar 2027
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Patient has locally advanced or metastatic cancer (all solid tumours allowed except primary brain/CNS tumour or untreated spinal cord compression) 2. Patient has at least one extracranial measurable disease lesion per RECIST 1.1/ iRECIST criteria. 3. Patients with known brain metastases are eligible if they meet all the following criteria: 1. Patient has received definitive treatment of brain metastases with stereotactic body radiation therapy (SBRT) or surgery provided that the brain lesions are stable (without evidence of progression by imaging for at least 4 weeks before the first dose of study treatment) 2. Patient is neurologically stable and has had no persistent side effects / complications from prior treatment. 3. Patient has no evidence of new or enlarging brain metastases (confirmed by repeat imaging) and has not required steroids for at least 14 days prior to first dose administration on Day 1. 4. Female patients must be of non-child-bearing potential i.e., surgically sterilised at least 6 weeks before the screening visit or postmenopausal Exclusion Criteria: 1. Prior therapy with a TLR7 and/or TLR8 agonist, unless first approved by the medical monitor. 2. Has primary brain/CNS tumour or untreated spinal cord compression. 3. Has known active, uncontrolled brain or CNS metastases and/or carcinomatous meningitis. 4. Evidence of abnormal cardiac function 5. Clinically significant active infection within 2 weeks prior to commencement of treatment, or unexplained fever (temperature \> 38.1°C) within 7 days prior to first dose administration on Cycle 1 Day 1. 6. Known active human immunodeficiency virus (HIV-1 or HIV-2), hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV) antibodies at the screening visit. 7. History of other malignancy not meeting inclusion criterion #1 within the past 2 years 8. Major surgery within 28 days of Cycle 1, Day 1, or minor surgical procedures within 7 days of Cycle 1, Day 1. 9. Received cancer-directed therapy 10. A history of autoimmune diseases that has caused terminal organ damage or required systemic immunosuppression / systemic disease modulating drugs within the past 2 years. 11. Chronic use of immune-suppressive drugs (i.e., systemic corticosteroids used in the management of cancer or non-cancer related illnesses, (e.g., COPD) in dosing exceeding 10 mg daily of prednisone equivalent). Inhaled steroids are allowed. 12. History of prior organ allograft. 13. Known hypersensitivity to the study drug or its inactive ingredients.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
3 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Cabrini Hospital
RECRUITINGMalvern, Victoria, 3144, Australia
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Southern Oncology Clinical Research Unit
RECRUITINGBedford Park, South Australia, 5042, Australia
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The Border Cancer Hospital
RECRUITINGAlbury, New South Wales, 2640, Australia
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Oral drug that targets two cancer drivers tested in first human study
- Mitochondria-Targeting drug gamitrinib tested for first time in humans
- Can a Two-Drug combo outsmart advanced cancer?
- Can an experimental pill boost cancer immunotherapy? early trial puts HG146 to the test
- New injectable drug tested for safety in Hard-to-Treat solid tumors
- New antibody drug conjugate tested against advanced cancers