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Brief Title: A Phase 1 Study of Intravesical VAX014 for Instillation in Subjects With Non-Muscle Invasive Bladder Cancer

Phase I Safety and Tolerability of Intravesical VAX014 for Instillation in Subjects With Non-Muscle Invasive Bladder Cancer (NMIBC)


  • Org Study ID: VX0116
  • Secondary ID: N/A
  • NCT ID: NCT03854721
  • Sponsor: Vaxiion Therapeutics


The purpose of this research study is to evaluate the safety, tolerability and activity of VAX014 for Instillation (VAX014) in patients with low-grade Non-Muscle Invasive Bladder Cancer (NMIBC). VAX014 is a targeted oncolytic agent designed to kill tumor cells following instillation into the urinary bladder.


This study will evaluate the safety and tolerability of VAX014 using a 3+3 dose escalation design to determine a maximum tolerated dose (MTD) followed by a dose expansion at the Recommended Phase 2 Dose (RP2D). Both phases of the study will use a Window of Opportunity study design where patients with a single, low-grade Ta lesion will receive VAX014 via a urinary catheter into the bladder, weekly for 6 weeks prior to undergoing Transurethral Resection of Bladder Tumor (TURBT) to assess antitumor activity against the mapped lesion.

Patients enrolled in this study must have low-grade (Ta) Non-Muscle Invasive Bladder Cancer. However, eligible patients may have up to 5 low-grade Ta lesions at screening, and all but a single mapped lesion will be resected prior to receiving VAX014. The mapped lesion is assessed for anti-tumor activity.

VAX014 is a formulation of recombinant bacterial minicells which is designed to selectively target two NMIBC-associated integrin heterodimers to de-stabilize tumor cell membranes, with the result being tumor cell lysis.

  • Overall Status
  • Start Date
    May 10, 2019
  • Phase
    Phase 1
  • Study Type


Primary Outcome 1 - Measure:

Primary Outcome 1 - Timeframe: N/A


  • Urothelial Carcinoma of the Urinary Bladder


Inclusion Criteria:
1. Signed, informed consent

- 2. Age 18 or more years

- 3. Pathologically confirmed low-grade Ta urothelial carcinoma (UC) of the urinary bladder

- 4. NMIBC with one solitary measurable tumor at the start of study, measuring ≥ 5 mm and ≤ 15 mm in greatest diameter (up to 4 additional low-grade Ta lesions, each measuring no more than 15 mm may be removed at screening provided a single lesion remains)

- 5. Treatment-naïve or failed one previous regimen of intravesical therapy (BCG or chemotherapy)

- 6. If recurrent disease, then more than 6 months from prior resection, more than 3 months from completion of last intravesical therapy with BCG, and more than 6 weeks from completion of last therapeutic intravesical therapy with chemotherapy

- 7. If previously treated, recovered from prior treatment-related toxicity to ≤ Grade 1

- 8. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 116

- 9. Absolute neutrophil count (ANC) ≥ 1,500/mm3

- 10. Platelet count ≥ 100,000/mm3

- 11. Total bilirubin ≤ 1.5 x upper limit of normal (ULN), or ≤ 3 x ULN in subjects with Gilberts disease

- 12. Serum creatinine ≤ 1.5 x ULN or creatinine clearance ≥ 30 mL/min

- 13. Serum aspartate transaminase (AST) and serum alanine transaminase (ALT) ≤ 2.5 x ULN

- 14. Willingness to participate in collection of pharmacokinetic samples

- 15. Women of childbearing potential must have a negative serum pregnancy test.

- 16. All subjects of childbearing potential must be willing to use effective contraception while on treatment and for 3 months after the last dose of VAX014
Exclusion Criteria:
1. Additional papillary disease at screening (in addition to the solitary low-grade Ta lesion detailed in the inclusion criteria) that
1. Consist of 6 or more lesions

- 2. Consists of any lesion with a maximal diameter of greater than 15 mm

- 2. Confirmed or suspected perforated bladder

- 3. History of difficult catheterization that in the opinion of the investigator will prevent administration of VAX014

- 4. Presence or history of any high-grade urothelial cancer (including CIS) or high-grade urine cytology

- 5. Intravesical chemo-or biological therapy within 6 months of first administration of VAX014

- 6. UC of the ureters or urethra

- 7. History of interstitial cystitis

- 8. History of radiation to the pelvis

- 9. History of vesicoureteral reflux or an indwelling urinary stent

- 10. Other known active cancer(s) likely to require treatment or interfere with study objectives over the next two (2) years

- 11. Active, uncontrolled bacterial, viral, or fungal infection(s) requiring systemic therapy

- 12. Known HIV, Hepatitis B, or Hepatitis C infection

- 13. Significant cardiovascular risk (e.g., coronary stenting within 8 weeks, myocardial infarction within 6 months)

- 14. Major surgery other than diagnostic surgery within 4 weeks of first administration of VAX014

- 15. Pregnant or currently breast-feeding

- 16. Psychiatric illness/social situations that would interfere with compliance with study requirements

- 17. Presence of any sessile appearing tumor suspected of being invasive or high-grade

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No


Name: N/A

Role: N/A

Affiliation: N/A

Overall Contact

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