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

PRIMARY OUTCOMES

Primary Outcome 1 - Measure:

Primary Outcome 1 - Timeframe: N/A

CONDITION

• Urothelial Carcinoma of the Urinary Bladder

ELIGIBILITY

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

OFFICIAL INFORMATION

Name: N/A

Role: N/A

Affiliation: N/A

Overall Contact

Name: N/A

Phone: N/A

Email: N/A

LOCATION