Phase 1 Study of Intratumoral Administration of VAX014 With Expansion in Combination With a Checkpoint Inhibitor in Subjects With Advanced Solid Tumors

NCT05901285 | Recruiting Phase 1 FDA Drug

• 1 other identifier: VX0120
• Study Type: interventional
• Target: 43
• Locations: 1 country
• Sites: 8

Brief 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. VAX014 is a targeted oncolytic agent designed to kill tumor cells following intratumoral injection into advanced solid tumors.

Conditions

Advanced Solid Tumor; Advanced Solid Tumors Appropriate for Treatment With Either Nivolumab or Pembrolizumab

Outcome Measures

Primary Outcomes (5)

• 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

  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

  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

  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

  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

  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]

  up to 1 week

• Anti-Drug Antibodies (Immunogenicity) [systemic ADA]

  Up to 20 weeks

• Overall Response Rate as VAX014 alone and in combination with either nivolumab or pembrolizumab

  Up to 20 weeks

Other Outcomes (6)

• Tumor Tissue (immune environment)

  up to 8 weeks

• Anti-Tumor T Cells

  Up to 20 weeks

• Cytokine levels

  Up to 20 weeks

Study Arms (2)

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.

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.

Drug: VAX014; Combination Product: Nivolumab or pembrolizumab

Interventions

VAX014 DRUG

Intratumorally administered oncolytic agent comprised of recombinant bacterial minicells. VAX014 is not infectious and is not capable of replication

VAX014 (Dose Escalation); VAX014 in Combination with Either Nivolumab or Pembrolizumab (Dose Expansion)

Nivolumab or pembrolizumab COMBINATION_PRODUCT

VAX014 will be given in combination with Investigator's choice of nivolumab or pembrolizumab.

VAX014 in Combination with Either Nivolumab or Pembrolizumab (Dose Expansion)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)
• 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:

You may not qualify if:

• 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 from prior anticancer therapy and C1D1 (e.g., chemotherapy, immunotherapy, intralesional therapy, irradiation therapy)
• Known CNS metastases or leptomeningeal carcinomatosis, unless adequately treated and clinically stable off steroids for ≥ 14 days from C1D1
• Severe infection requiring systemic antibiotic therapy or hospitalization for treatment of injection within 2 weeks of the first injection of VAX014
• Need for systemic immunosuppressive therapy (≤10 mg of prednisone equivalent, or one time pulse steroids excepted)
• Active autoimmune disease requiring systemic immunosuppressive therapy
• No active lung disease or pneumonitis
• No 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

Sponsors & Collaborators

• Vaxiion Therapeutics: lead

Study Sites (8)

University of Arizona Cancer Center

Tucson, Arizona, 85719, United States

RECRUITING

Sarah Cannon Research Institute at HealthONE

Denver, Colorado, 80218, United States

RECRUITING

George Washington University

Washington D.C., District of Columbia, 20052, United States

RECRUITING

University of Maryland

Baltimore, Maryland, 21201, United States

RECRUITING

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States

RECRUITING

Dartmouth Cancer Center

Lebanon, New Hampshire, 03756, United States

RECRUITING

Atlantic Health System

Morristown, New Jersey, 07960, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

MeSH Terms

Interventions

Nivolumab; pembrolizumab

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Central Study Contacts

Kirsten Dorr, IMBA

CONTACT

858-630-1959; kdorr@sciquus.com

Kate Peters, BA

CONTACT

619-614-3800; kpeters@sciquus.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 24, 2023
• First Posted: June 13, 2023
• Study Start: November 2, 2023
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): November 1, 2026
• Last Updated: November 25, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)