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

NCT03854721 | Completed Phase 1 FDA Drug

• 1 other identifier: VX0116
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 5

Brief Summary

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.

Conditions

Urothelial Carcinoma of the Urinary Bladder

Keywords

Non-Muscle Invasive Bladder Cancer

Outcome Measures

Primary Outcomes (2)

• 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 28 days of treatment have occurred, with the next higher dose having at least 2/3 or 2/6 patients experiencing a DLT

  up to 28 days

• Incidence of Treatment-Emergence 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

Secondary Outcomes (10)

• Recommended Phase 2 Dose (RP2D) of intravesical VAX014

  up to 5 weeks

• Peak Plasma Concentration (Cmax)

  Day 1

• Trough Plasma Concentration (Cmin)

  Day 1

• Time to Peak Plasma Concentration (Tmax)

  Day 1

• Volume and Distribution (Vd)

  Day 1

Study Arms (1)

VAX014

EXPERIMENTAL

Intravesical VAX014 (dose: 3.33x10\^10 - 9.0x10\^11 recombinant bacterial minicells (rBMCs)), given once per week for Weeks 1-6

Drug: VAX014

Interventions

VAX014 DRUG

Solution for intravesical infusion, 3.33x10\^10 rBMCs per vial

VAX014

Eligibility Criteria

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

You may qualify if:

• Signed, informed consent
• Age 18 or more years
• Pathologically confirmed low-grade Ta urothelial carcinoma (UC) of the urinary bladder
• 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)
• Treatment-naïve or failed one previous regimen of intravesical therapy (BCG or chemotherapy)
• 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
• If previously treated, recovered from prior treatment-related toxicity to ≤ Grade 1
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 116
• Absolute neutrophil count (ANC) ≥ 1,500/mm3
• Platelet count ≥ 100,000/mm3
• Total bilirubin ≤ 1.5 x upper limit of normal (ULN), or ≤ 3 x ULN in subjects with Gilberts disease
• Serum creatinine ≤ 1.5 x ULN or creatinine clearance ≥ 30 mL/min
• Serum aspartate transaminase (AST) and serum alanine transaminase (ALT) ≤ 2.5 x ULN
• Willingness to participate in collection of pharmacokinetic samples
• Women of childbearing potential must have a negative serum pregnancy test.

You may not qualify if:

• Consist of 6 or more lesions
• Consists of any lesion with a maximal diameter of greater than 15 mm
• Confirmed or suspected perforated bladder
• History of difficult catheterization that in the opinion of the investigator will prevent administration of VAX014
• Presence or history of any high-grade urothelial cancer (including CIS) or high-grade urine cytology
• Intravesical chemo-or biological therapy within 6 months of first administration of VAX014
• UC of the ureters or urethra
• History of interstitial cystitis
• History of radiation to the pelvis
• History of vesicoureteral reflux or an indwelling urinary stent
• Other known active cancer(s) likely to require treatment or interfere with study objectives over the next two (2) years
• Active, uncontrolled bacterial, viral, or fungal infection(s) requiring systemic therapy
• Known HIV, Hepatitis B, or Hepatitis C infection
• Significant cardiovascular risk (e.g., coronary stenting within 8 weeks, myocardial infarction within 6 months)
• Major surgery other than diagnostic surgery within 4 weeks of first administration of VAX014

Sponsors & Collaborators

• Vaxiion Therapeutics: lead

Study Sites (5)

The Urology Center of Colorado

Denver, Colorado, 80211, United States

Location

University of Iowa Hospital and Clinics

Iowa City, Iowa, 52242, United States

Location

New Jersey Urology, LLC.

Edison, New Jersey, 08837, United States

Location

Carolina Urologic Research Center

Myrtle Beach, South Carolina, 29572, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Non-Muscle Invasive Bladder Neoplasms

Condition Hierarchy (Ancestors)

Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Urinary Bladder Neoplasms; Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Urinary Bladder Diseases; Urologic Diseases; Male Urogenital Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 21, 2019
• First Posted: February 26, 2019
• Study Start: May 10, 2019
• Primary Completion: March 1, 2023
• Study Completion: March 1, 2023
• Last Updated: March 3, 2023

Record last verified: 2023-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)