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Urothelial Carcinoma of the Urinary BladderPhase 1NCT03854721

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

A Phase 1 study testing VAX014 in people with Urothelial Carcinoma of the Urinary Bladder, run by Vaxiion Therapeutics at 5 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
VAX014
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
5 sitesUnited States
Study dates
10 May 2019to 1 Mar 2023
Phase
Phase 1
Are there results?
None posted in the registry

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

  • 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
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  • 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.
  • 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

Who cannot join

  • Additional papillary disease at screening (in addition to the solitary low-grade Ta lesion detailed in the inclusion criteria) that
  • 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
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  • 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
  • Pregnant or currently breast-feeding
  • Psychiatric illness/social situations that would interfere with compliance with study requirements
  • Presence of any sessile appearing tumor suspected of being invasive or high-grade

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

United States5 sites

  • The Urology Center of Colorado

    Denver, Colorado, 80211

  • New Jersey Urology, LLC.

    Edison, New Jersey, 08837

  • Baylor College of Medicine

    Houston, Texas, 77030

  • University of Iowa Hospital and Clinics

    Iowa City, Iowa, 52242

  • Carolina Urologic Research Center

    Myrtle Beach, South Carolina, 29572

Other trials in United States

About this study

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

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.

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) 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.

Being tested
VAX014
Keywords
Non-Muscle Invasive Bladder Cancer
Medical subject headings
Non-Muscle Invasive Bladder Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VAX014Drug

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

Study groups in NCT03854721
Study groupRoleWhat they receive
VAX014ExperimentalIntravesical VAX014 (dose: 3.33x10^10 - 9.0x10^11 recombinant bacterial minicells (rBMCs)), given once per week for Weeks 1-6
Receives: Drug: VAX014

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    Measured: up to 28 days

  2. 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

    Measured: Through study completion, an average of 20 weeks

Secondary outcomes (10)

  1. Recommended Phase 2 Dose (RP2D) of intravesical VAX014

    The RP2D will be determined following the determination of the MTD and an overall assessment of safety as determined by the Safety Committee

    Measured: up to 5 weeks

  2. Peak Plasma Concentration (Cmax)

    The peak plasma concentration (Cmax) will be measured as part of pharmacokinetic (PK) testing. In the event early analysis determines that VAX014 is not detectable in whole blood, PK sampling and analysis will be suspended.

    Measured: Day 1

  3. Trough Plasma Concentration (Cmin)

    The trough plasma concentration (Cmin) will be measured as part of pharmacokinetic (PK) testing. In the event early analysis determines that VAX014 is not detectable in whole blood, PK sampling and analysis will be suspended.

    Measured: Day 1

  4. Time to Peak Plasma Concentration (Tmax)

    The time to peak plasma concentration (Tmax) will be measured as part of pharmacokinetic (PK) testing. In the event early analysis determines that VAX014 is not detectable in whole blood, PK sampling and analysis will be suspended.

    Measured: Day 1

  5. Volume and Distribution (Vd)

    The volume and distribution (Vd) will be measured as part of pharmacokinetic (PK) testing. In the event early analysis determines that VAX014 is not detectable in whole blood, PK sampling and analysis will be suspended.

    Measured: Day 1

  6. Half Life (t[1/2])

    The half life (t[1/2]) will be measured as part of pharmacokinetic (PK) testing. In the event early analysis determines that VAX014 is not detectable in whole blood, PK sampling and analysis will be suspended.

    Measured: Day 1

  7. Area Under Curve (AUC)

    The area under the plasma concentration versus time curve (AUC) will be measured as part of pharmacokinetic (PK) testing. In the event early analysis determines that VAX014 is not detectable in whole blood, PK sampling and analysis will be suspended.

    Measured: Day 1

  8. Clearance (Cl)

    The clearance (Cl) will be measured as part of pharmacokinetic (PK) testing. In the event early analysis determines that VAX014 is not detectable in whole blood, PK sampling and analysis will be suspended.

    Measured: Day 1

  9. Overall Response Rate

    Response rate will be evaluated for low-grade Ta lesions. Lesions will be assessed with cystoscopy and change in tumor size will be recorded.

    Measured: Up to 20 weeks

  10. Anti-Drug Antibodies (Immunogenicity)

    The presence or absence of anti-drug antibodies (ADA) in serum will be assessed by assay.

    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.

  1. First posted26 Feb 2019
  2. Study start10 May 2019
  3. Primary completion1 Mar 2023
  4. Study completion1 Mar 2023

Resources

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03854721
Sponsor's protocol ID
VX0116
Status last verified
Mar 2023
First submitted
21 Feb 2019
Last updated
3 Mar 2023
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing VAX014 in people with Urothelial Carcinoma of the Urinary Bladder, run by Vaxiion Therapeutics at 5 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 16 inclusion and 19 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: finished, as of the sponsor's last update on 3 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maximum tolerated dose (MTD) of VAX014 (time frame: up to 28 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03854721.

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 3 Mar 2023.

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