Advanced Solid TumorPhase 1NCT05901285
Phase 1 Study of Intratumoral Administration of VAX014 With Expansion in Combination With a Checkpoint Inhibitor in Subjects With Advanced Solid Tumors
A Phase 1 study testing VAX014 and Nivolumab or pembrolizumab in people with Advanced Solid Tumor and Advanced Solid Tumors Appropriate for Treatment With Either Nivolumab or Pembrolizumab, run by Vaxiion Therapeutics at 8 sites in United States.
Trial summary
The purpose of this research study is to evaluate the safety, tolerability and activity of VAX014 for intratumoral injections (VAX014) as a single agent as well as in combination with Investigator's choice of nivolumab or pembrolizumab in patients with advanced solid tumors. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Advanced Solid Tumor, Advanced Solid Tumors Appropriate for Treatment With Either Nivolumab or Pembrolizumab
- What is being tested?
- VAX014 and Nivolumab or pembrolizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 8 sitesUnited States
- Study dates
- 2 Nov 2023to Nov 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18+
- Informed consent
- Histological or cytopathological confirmed diagnosis of a locally advanced or metastatic solid tumor
- Progression following at least one prior standard treatment or intolerant of standard treatments.
- [Dose Escalation] Availability of archival or fresh tumor tissue
- [Expansion] Willing to undergo biopsy of the tumor to be injected prior to the initial VAX014 injection (may provide archival tissue instead if approved by Medical Monitor)
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- No available SOC therapy that would confer clinical benefit
- [Dose escalation] At least one cutaneous, subcutaneous, or nodal injectable tumor (between 1 and 10 cm in largest diameter) that can be injected by direct palpation or with the assistance of ultrasound without the need for interventional radiology
- [Expansion] At least one injectable tumor (>=0.5cm in largest diameter) that can be injected either with or without the need for interventional radiology
- [Expansion] Appropriate for treatment with either nivolumab or pembrolizumab
- [Expansion] Progression following at least one prior regimen containing PD-1 directed immune checkpoint blockade
- Measurable disease by RECIST v1.1
- ECOG Performance Status of 0, 1, or 2
- Resolution of any toxicity associated with prior therapy to ≤ Grade 1 (Residual toxicity of Grade 2 may be allowed following discussion with Medical Monitor)
- Adequate hematologic function defined as:
- Absolute neutrophil count >=1,500/uL
- Platelet count >=100,000/uL
- [Expansion] Hemoglobin >=9 gm/dL
- Adequate hepatic function defined as:
- Total bilirubin ≤ 1.5 x ULN
- Aspartate transaminase (AST) and alanine transaminase (ALT) ≤ 2.5 x ULN
- Adequate coagulation defined as:
- International normalized ratio (INR) ≤ 1.5 x ULN or prothrombin time (PT) ≤ 1.5 x ULN
- Partial thromboplastin time (PTT) or activated PTT (aPTT) ≤ 1.5 x ULN
- Serum creatinine ≤ 1.5 x ULN or estimated GFR ≥ 60 mL/min/1.73 m2 (per MDRD GFR formula)
- Women of childbearing potential must have a negative serum pregnancy test
- All subjects of childbearing potential must be willing to consent to using effective contraception (as determined by the Investigator) while on treatment and for 3 months after their participation in the study ends
Who cannot join
- Injectable tumor not sufficiently distanced from critical structures (e.g., major airway, neurovascular structure) where post injection swelling may place the subject at unacceptable risk
- ≤ 21 days or ≤ five half-lives (whichever is shorter) between any prior anticancer therapy (e.g., chemotherapy, immunotherapy, intralesional therapy, irradiation) and the first injection of VAX014
- Known CNS metastases or leptomeningeal carcinomatosis, unless adequately treated and clinically stable off steroids for ≥ 14 days from the first injection of VAX014
- Severe infection requiring systemic antibiotic therapy or hospitalization for treatment of injection
- Need for systemic immunosuppressive therapy (≤10 mg of prednisone equivalent, or one time pulse steroids excepted)
- Active autoimmune disease requiring systemic immunosuppressive therapy
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- Active lung disease or pneumonitis
- History of Grade 4 toxicity in response to prior PD-1 blockade
- Any other malignancy likely to require treatment in the next 2 years (exceptions include cancer such as basal or squamous cell skin cancers, noninvasive cancer of the cervix, and local prostate cancer)
- Known active infection with tuberculosis or HIV
- Active Hepatitis B or C
- [Females] pregnant or breastfeeding
- Clinically significant cardiovascular abnormalities including:
- ≤ 12 months from prior MI
- Unstable angina pectoris
- ≤ 6 months from NYHA classification >=3 CHF
- Electrocardiogram (ECG) with QTcF > 450 msec (males) and QTcF > 470 msec (females) based on an average of triplicate ECGs (triplicate ECGs performed only if initial QTcF exceeds male/female limits)
- Medical or psychological condition that places the subject at undue risk with study participation
Trial locations and contacts
8 sites in 1 country. Contact the study team or a site near you to ask about taking part.
8 sites in 1 country
United States8 sites, 8 recruiting
- Recruiting
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
DFCI Referral Team, 857-215-0180, dfcicctiexternalreferral@dfci.harvard.edu
- Recruiting
Cleveland Clinic
Cleveland, Ohio, 44195
Cancer Answer Line, 216-444-7923
- Recruiting
Melanoma & Skin Cancer Institute
Denver, Colorado, 80113
Tony Romero, MS, CCRC, 402-697-2229, tony@xcancer.com
- Recruiting
Sarah Cannon Research Institute at HealthONE
Denver, Colorado, 80218
General Contact, 720-754-2610, cann.ddudenvergeneral@sarahcannon.com
- Recruiting
Dartmouth Cancer Center
Lebanon, New Hampshire, 03756
General Contact, (603) 653-3615, cancer.research.nurse@hitchcock.org
- Recruiting
Atlantic Health System
Morristown, New Jersey, 07960
Maureen Nowakowski, 973-971-5569, Maureen.nowakowski@atlantichealth.org
- Recruiting
University of Arizona Cancer Center
Tucson, Arizona, 85719
Spencer Williams, 520-621-1508, spencerwilliams@arizona.edu
- Recruiting
George Washington University
Washington D.C., District of Columbia, 20052
Alyssa Barnett, 202-994-0246, alyssa.barnett@email.gwu.edu
About this study
The purpose of this research study is to evaluate the safety, tolerability and activity of VAX014 for intratumoral injections (VAX014) as a single agent as well as in combination with Investigator's choice of nivolumab or pembrolizumab in patients with advanced solid tumors. VAX014 is a targeted oncolytic agent designed to kill tumor cells following intratumoral injection into advanced solid tumors.
Read the detailed description
This study will evaluate the safety and tolerability of VAX014 using a 3+3 dose escalation design to determine a maximum tolerated dose (MTD) or maximum practical dose (MPD) of single agent VAX014. The DLT assessment period will be the initial 21-days of injections. Subjects will receive weekly injections for the initial 8 weeks. Up to six dose levels will be evaluated (i.e., [starting dose], [starting dose] x 3, [starting dose] x 10, [starting dose] x 30, [starting dose] x 100, [starting dose] x 300).
Subjects may continue on treatment following discussion between the Principal Investigator and Sponsor/Medical Monitor.
After the determination of a single agent RP2D by the SRC for single agent intratumoral VAX014, an Expansion Phase will be conducted combining intratumoral VAX014 with Investigator's choice of nivolumab or pembrolizumab. The SRC may adjust the RP2D of VAX014 used during the Expansion Phase based on accumulating safety data.
The Expansion Phase will consist of up to 25 subjects. For the first 3 subjects treated with the combination of VAX014 and either nivolumab or pembrolizumab, the initial 2 doses of intratumoral VAX014 will be reduced by one dose level from the VAX014 RP2D. If the initial 3 subjects are able to escalate to the RP2D for VAX014, all subsequent subjects will then receive VAX014 at the RP2D starting with the first dose of VAX014.
- Conditions
- Advanced Solid Tumor, Advanced Solid Tumors Appropriate for Treatment With Either Nivolumab or Pembrolizumab
- Being tested
- VAX014, Nivolumab or pembrolizumab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VAX014Drug
Intratumorally administered oncolytic agent comprised of recombinant bacterial minicells. VAX014 is not infectious and is not capable of replication
Nivolumab or pembrolizumabCombination product
VAX014 will be given in combination with Investigator's choice of nivolumab or pembrolizumab.
| Study group | Role | What they receive |
|---|---|---|
| VAX014 (Dose Escalation) | Experimental | Dose escalation of VAX014 [recombinant bacterial minicells (rBMCs)] intratumoral injections alone for subjects with solid tumors relapsed and/or refractory to standard treatment and appropriate for injection of a nodal, subcutaneous, or cutaneous tumor via palpation or with the assistance of ultrasound. Receives: Drug: VAX014 |
| VAX014 in Combination with Either Nivolumab or Pembrolizumab (Dose Expansion) | Experimental | Dose expansion of VAX014 [recombinant bacterial minicells (rBMCs)] intratumoral injections in combination with Investigator's choice of nivolumb or pembrolizumab for subjects with solid tumors relapsed and/or refractory to standard treatment and appropriate for injection of a nodal, subcutaneous, or cutaneous tumor via palpation, with the assistance of ultrasound, or interventional radiology. Receives: Drug: VAX014; Combination Product: Nivolumab or pembrolizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose (MTD) of VAX014
The MTD will be defined as the dose level at which at most one of six patients experiences a dose limiting toxicity (DLT) after 21 days of treatment have occurred, with the next higher dose having at least 2/3 or 2/6 patients experiencing a DLT
Measured: up to 21 days
Incidence of Treatment-Emergency Adverse Events (Safety and Tolerability)
Toxicities will be assessed in each subject by tracking the occurrence of graded Adverse Events (AEs). AEs will be graded according to the National Cancer Institute Common Terminology for Adverse Events (NCI CTCAE) v5.0
Measured: Through study completion, an average of 20 weeks
Recommended Phase 2 Dose (RP2D) for single agent intratumoral VAX014
The RP2D will be determined following the determination of the MTD and with agreement by the Safety Review Committee
Measured: up to 5 weeks
Acceptable dose of VAX014 in combination with PD-1 Inhibitor
Determine the acceptable dose of VAX014 when used in combination with Investigator's choice of nivolumab or pembrolizumab
Measured: 3 months
Evaluation of efficacy of VAX014 in combination with either nivolumab or pembrolizumab
Evaluate efficacy including overall response rate (ORR), response of injected tumor(s) of VAX014 in combination with Investigator's choice of nivolumab or pembrolizumab
Measured: 18 months
Secondary outcomes (3)
Characterize systemic exposure by evaluating pharmacokinetics of intratumoral VAX014 as monotherapy and (if appropriate) in combination with nivolumab or pembrolizumab [systemic PK]
Whole blood will be collected for determination of VAX014 levels. PK data may demonstrate limited exposure or lack of detectable VAX014 in blood.
Measured: up to 1 week
Anti-Drug Antibodies (Immunogenicity) [systemic ADA]
The presence or absence of anti-drug antibodies (ADA) in serum will be assessed by assay in patients receiving VAX014 along or in combination with Investigator's choice of nivolumab or pembrolizumab
Measured: Up to 20 weeks
Overall Response Rate as VAX014 alone and in combination with either nivolumab or pembrolizumab
Response rate will be evaluated for tumor lesions that will be assessed through CT, MRI, physical exam.
Measured: Up to 20 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jun 2023
- Study start2 Nov 2023
- Primary completionAug 2027 (estimated)
- Study completionNov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced Solid TumorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Vaxiion Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05901285
- Sponsor's protocol ID
- VX0120
- Status last verified
- Aug 2026
- First submitted
- 24 May 2023
- Last updated
- 24 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Study of Intratumoral Administration of VAX014 With Expansion in Combination With a Checkpoint Inhibitor in Subjects With Advanced Solid Tumors
- ClinicalTrials.gov ID
- NCT05901285
- Acronym / sponsor ID
- VX0120
- Conditions
- Advanced Solid Tumor, Advanced Solid Tumors Appropriate for Treatment With Either Nivolumab or Pembrolizumab
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Vaxiion TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 2 Nov 2023 (actual)
- Primary completion
- Aug 2027 (estimated)
- Study completion
- Nov 2027 (estimated)
- First posted
- 13 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this research study is to evaluate the safety, tolerability and activity of VAX014 for intratumoral injections (VAX014) as a single agent as well as in combination with Investigator's choice of nivolumab or pembrolizumab in patients with advanced solid tumors. VAX014 is a targeted oncolytic agent designed to kill tumor cells following intratumoral injection into advanced solid tumors.
Detailed description
This study will evaluate the safety and tolerability of VAX014 using a 3+3 dose escalation design to determine a maximum tolerated dose (MTD) or maximum practical dose (MPD) of single agent VAX014. The DLT assessment period will be the initial 21-days of injections. Subjects will receive weekly injections for the initial 8 weeks. Up to six dose levels will be evaluated (i.e., [starting dose], [starting dose] x 3, [starting dose] x 10, [starting dose] x 30, [starting dose] x 100, [starting dose] x 300).
Subjects may continue on treatment following discussion between the Principal Investigator and Sponsor/Medical Monitor.
After the determination of a single agent RP2D by the SRC for single agent intratumoral VAX014, an Expansion Phase will be conducted combining intratumoral VAX014 with Investigator's choice of nivolumab or pembrolizumab. The SRC may adjust the RP2D of VAX014 used during the Expansion Phase based on accumulating safety data.
The Expansion Phase will consist of up to 25 subjects. For the first 3 subjects treated with the combination of VAX014 and either nivolumab or pembrolizumab, the initial 2 doses of intratumoral VAX014 will be reduced by one dose level from the VAX014 RP2D. If the initial 3 subjects are able to escalate to the RP2D for VAX014, all subsequent subjects will then receive VAX014 at the RP2D starting with the first dose of VAX014.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| VAX014 (Dose Escalation) | Experimental | Drug: VAX014Dose escalation of VAX014 [recombinant bacterial minicells (rBMCs)] intratumoral injections alone for subjects with solid tumors relapsed and/or refractory to standard treatment and appropriate for injection of a nodal, subcutaneous, or cutaneous tumor via palpation or with the assistance of ultrasound. |
| VAX014 in Combination with Either Nivolumab or Pembrolizumab (Dose Expansion) | Experimental | Drug: VAX014, Combination Product: Nivolumab or pembrolizumabDose expansion of VAX014 [recombinant bacterial minicells (rBMCs)] intratumoral injections in combination with Investigator's choice of nivolumb or pembrolizumab for subjects with solid tumors relapsed and/or refractory to standard treatment and appropriate for injection of a nodal, subcutaneous, or cutaneous tumor via palpation, with the assistance of ultrasound, or interventional radiology. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (27 listed)
- Age 18+
- Informed consent
- Histological or cytopathological confirmed diagnosis of a locally advanced or metastatic solid tumor
- Progression following at least one prior standard treatment or intolerant of standard treatments.
- [Dose Escalation] Availability of archival or fresh tumor tissue
Key exclusion criteria (18 listed)
- Injectable tumor not sufficiently distanced from critical structures (e.g., major airway, neurovascular structure) where post injection swelling may place the subject at unacceptable risk
- ≤ 21 days or ≤ five half-lives (whichever is shorter) between any prior anticancer therapy (e.g., chemotherapy, immunotherapy, intralesional therapy, irradiation) and the first injection of VAX014
- Known CNS metastases or leptomeningeal carcinomatosis, unless adequately treated and clinically stable off steroids for ≥ 14 days from the first injection of VAX014
- Severe infection requiring systemic antibiotic therapy or hospitalization for treatment of injection
- Need for systemic immunosuppressive therapy (≤10 mg of prednisone equivalent, or one time pulse steroids excepted)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VAX014Intratumorally administered oncolytic agent comprised of recombinant bacterial minicells. VAX014 is not infectious and is not capable of replication | Drug | VAX014 (Dose Escalation), VAX014 in Combination with Either Nivolumab or Pembrolizumab (Dose Expansion) |
| Nivolumab or pembrolizumabVAX014 will be given in combination with Investigator's choice of nivolumab or pembrolizumab. | Combination product | VAX014 in Combination with Either Nivolumab or Pembrolizumab (Dose Expansion) |
Primary outcome
Maximum tolerated dose (MTD) of VAX014
Time frame: up to 21 days
The MTD will be defined as the dose level at which at most one of six patients experiences a dose limiting toxicity (DLT) after 21 days of treatment have occurred, with the next higher dose having at least 2/3 or 2/6 patients experiencing a DLT
Incidence of Treatment-Emergency Adverse Events (Safety and Tolerability)
Time frame: Through study completion, an average of 20 weeks
Toxicities will be assessed in each subject by tracking the occurrence of graded Adverse Events (AEs). AEs will be graded according to the National Cancer Institute Common Terminology for Adverse Events (NCI CTCAE) v5.0
Recommended Phase 2 Dose (RP2D) for single agent intratumoral VAX014
Time frame: up to 5 weeks
The RP2D will be determined following the determination of the MTD and with agreement by the Safety Review Committee
Acceptable dose of VAX014 in combination with PD-1 Inhibitor
Time frame: 3 months
Determine the acceptable dose of VAX014 when used in combination with Investigator's choice of nivolumab or pembrolizumab
Evaluation of efficacy of VAX014 in combination with either nivolumab or pembrolizumab
Time frame: 18 months
Evaluate efficacy including overall response rate (ORR), response of injected tumor(s) of VAX014 in combination with Investigator's choice of nivolumab or pembrolizumab
3 secondary outcomes
Characterize systemic exposure by evaluating pharmacokinetics of intratumoral VAX014 as monotherapy and (if appropriate) in combination with nivolumab or pembrolizumab [systemic PK]
Time frame: up to 1 week
Anti-Drug Antibodies (Immunogenicity) [systemic ADA]
Time frame: Up to 20 weeks
Overall Response Rate as VAX014 alone and in combination with either nivolumab or pembrolizumab
Time frame: Up to 20 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing VAX014 and Nivolumab or pembrolizumab in people with Advanced Solid Tumor and Advanced Solid Tumors Appropriate for Treatment With Either Nivolumab or Pembrolizumab, run by Vaxiion Therapeutics at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 27 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 24 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kirsten Dorr, IMBA (858-630-1959, kdorr@sciquus.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum tolerated dose (MTD) of VAX014 (time frame: up to 21 days). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Aug 2026.