Codex: Study of Inodiftagene Vixteplasmid (BC-819) in Unresponsive NMIBC

INTRODUCTION

Org Study ID: BC-819-18-204
Secondary ID: N/A
NTC ID: NCT03719300
Sponsor: Anchiano Therapeutics Israel Ltd.

This study, BC-819-18-204, is a Phase 2, open-label, monotherapy, single-arm, multicenter
clinical trial of BC-819 (inodiftagene vixteplasmid) in patients with NMIBC adequately
treated with Bacillus Calmette-Guerin (BCG) whose disease is BCG unresponsive according to
the US Food and Drug Administration (FDA) guidance.

BC-819 (inodiftagene vixteplasmid) is a recombinant DNA plasmid that directs the expression
of a potent toxin specifically in malignant cells but not in normal tissue. It has been
designed to exploit the established biology of the H19 gene, which is upregulated and
expressed at high levels only in malignant cells, to produce bacterial diphtheria toxin only
in bladder cancer tissue. BC-819 is administered directly into the bladder to enable maximal
topical exposure to target bladder cancer cells.

Overall Status
Recruiting
Start Date
December 11, 2018
Phase
Phase 2
Study Type
Interventional

Primary Outcome 1 - Measure: Complete response rate

Primary Outcome 1 - Timeframe: 12-48 weeks

Non-muscle Invasive Bladder Cancer (NMIBC)

Inclusion Criteria:

1. Male or female patients ≥18 years of age at the time of consent

2. Patient must have been adequately treated with BCG defined as at least one of the
following (FDA 2018):

1. At least five of six doses of an initial induction course plus at least two of
three doses of maintenance therapy

2. At least five of six doses of an initial induction course plus at least two of
six doses of a second induction course

3. A single course of induction BCG can qualify if the patient has T1 high-grade
disease at first evaluation (see 3c)

3. Patient must be BCG-unresponsive defined as at least one of the following (FDA 2018):

1. Persistent or recurrent CIS alone or with recurrent Ta/T1 disease within 12
months of completion of adequate BCG therapy. An assessment within 15 months can
also qualify when no assessment was done 12 months after completion of adequate
BCG therapy.

2. Recurrent high-grade Ta/T1 disease within 6 months of completion of adequate BCG
therapy. An assessment within 9 months can also qualify when no assessment was
done 6 months after completion of adequate BCG therapy.

3. T1 high-grade disease at the first evaluation following a single course of
induction BCG qualifies (Lerner et al. 2015, Steinberg et al. 2016)

4. Patient must have, at study entry, NMIBC indicated by 1 or more of the following:

1. Ta or T1 high-grade disease

2. CIS disease

5. Patient must have no known evidence of concomitant upper tract urothelial carcinoma or
urothelial carcinoma within the prostatic urethra within 6 months of enrollment

6. Patient must have an Eastern Cooperative Oncology Group (ECOG) performance status ≤2

7. Patient must have adequate hematologic function, as demonstrated by the following:

1. Hemoglobin level ≥10 g/dL

2. Absolute neutrophil count ≥1.5 x 109/L

3. Platelet count ≥100 x 109/L

8. Patient must have adequate liver and renal function as demonstrated by the following:

1. Aspartate aminotransferase and alanine aminotransferase each ≤3.0 x upper limit
of normal

2. Total bilirubin ≤1.5 x upper limit of normal, unless prior documentation of
Gilbert's syndrome in which case, 3.0 mg/dL is allowed

3. Serum creatinine ≤1.5 x upper limit of normal or measured or calculated
creatinine clearance ≥30 mL/min

9. Female patients of childbearing potential must use maximally effective birth control
during the period of therapy and for 1 month after the last study drug infusion

10. Male patients who are sexually active must be willing to use a double barrier
contraceptive method upon study enrollment, during the course of the study, and for 1
month after the last study drug infusion

Exclusion Criteria:

1. Patient has current or previous evidence of muscle invasive (muscularis propria) or
metastatic bladder cancer disease

2. Patient has received prior investigational therapy for NMIBC

3. Patient has received any therapy for NMIBC within 10 weeks before the start of study
treatment other than surgical resection, 1 dose of chemotherapy, and previous BCG

4. Patient is intolerant to previous BCG treatment in the absence of meeting other
criteria for BCG unresponsiveness and adequate BCG therapy

5. Patient has received external beam radiation therapy for bladder cancer at any time or
for any other condition

6. Patient has an active infection, including urinary tract infection (viral, bacterial,
or fungal) and cystitis

7. Patient has urinary tract signs or symptoms that preclude retention of drug in the
bladder; this does not include anticholinergic drugs

8. Patient is known to have tested positive for human immunodeficiency virus (HIV). No
HIV testing is required if patient is not known have tested positive

9. Patient is female and is pregnant or breastfeeding

10. Patient has a known presence or history of malignancy of other organ system within the
5 years before study start, with the exception of non-melanoma skin cancer; very low
or low-risk prostate cancer; or patients who have been disease free for at least 2
years following stage 1 or 2 cancer

Gender: All

Minimum Age: N/A

Maximum Age: 18 Years

Healthy Volunteers: No

Name: N/A

Role: N/A

Affiliation: N/A

Name: N/A

Phone: 857-277-5577

Email: sean.daly@anchiano.com

Facility	Status	Contact
Facility: Alaska Urological Institute Anchorage, Alaska 99503 United States	Status: Recruiting	Contact: N/A
Facility: Banner MD Anderson Cancer Center Gilbert, Arizona 85234 United States	Status: Recruiting	Contact: N/A
Facility: Mayo Clinic Arizona Phoenix, Arizona 85054 United States	Status: Recruiting	Contact: N/A
Facility: Urological Associates of Southern Arizona Tucson, Arizona 85715 United States	Status: Recruiting	Contact: N/A
Facility: American Institute of Research Los Angeles, California 90017 United States	Status: Recruiting	Contact: N/A
Facility: UC Davis Medical Center Sacramento, California 95817 United States	Status: Recruiting	Contact: N/A
Facility: The Urology Center of Colorado Denver, Colorado 80211 United States	Status: Recruiting	Contact: N/A
Facility: Mayo Clinic Florida Jacksonville, Florida 32224 United States	Status: Recruiting	Contact: N/A
Facility: University of Florida Health Jacksonville, Shands Hospital Jacksonville, Florida 33209 United States	Status: Recruiting	Contact: N/A
Facility: Emory University Atlanta, Georgia 30322 United States	Status: Recruiting	Contact: N/A
Facility: North Idaho Urology Coeur d'Alene, Idaho 83814 United States	Status: Recruiting	Contact: N/A
Facility: Idaho Urologic Institute, PA Meridian, Idaho 83642 United States	Status: Recruiting	Contact: N/A
Facility: University of Illinois Hospital and Health Systems (Outpatient Care Center) Chicago, Illinois 60612 United States	Status: Recruiting	Contact: N/A
Facility: The University of Kansas Cancer Center Westwood, Kansas 66205 United States	Status: Recruiting	Contact: N/A
Facility: Wichita Urology Group Wichita, Kansas 67226 United States	Status: Recruiting	Contact: N/A
Facility: Tulane University School of Medicine New Orleans, Louisiana 70114 United States	Status: Recruiting	Contact: N/A
Facility: Ochsner Clinical Foundation New Orleans, Louisiana 70121 United States	Status: Recruiting	Contact: N/A
Facility: Johns Hopkins Medical Institution Baltimore, Maryland 21287 United States	Status: Recruiting	Contact: N/A
Facility: Spectrum Health Medical Group Grand Rapids, Michigan 49546 United States	Status: Recruiting	Contact: N/A
Facility: Mayo Clinic Rochester, Minnesota 55905 United States	Status: Recruiting	Contact: N/A
Facility: New Jersey Urology, LLC Belleville, New Jersey 08043 United States	Status: Recruiting	Contact: N/A
Facility: MD Anderson Cancer Center at Cooper Voorhees, New Jersey 08103 United States	Status: Recruiting	Contact: N/A
Facility: Montefiore Medical Center Bronx, New York 10461 United States	Status: Recruiting	Contact: N/A
Facility: Weill Cornell Medical College - NY Presbyterian Hospital New York, New York 10065 United States	Status: Recruiting	Contact: N/A
Facility: UNC Chapel Hill Hospital, Urology Clinic Chapel Hill, North Carolina 27514 United States	Status: Recruiting	Contact: N/A
Facility: Alliance Urology Specialists, PA Greensboro, North Carolina 27403 United States	Status: Recruiting	Contact: N/A
Facility: Carolina Urology Partners, PLLC Huntersville, North Carolina 28078 United States	Status: Recruiting	Contact: N/A
Facility: University of Toledo, Dept. of Urology and Kidney Transplant Toledo, Ohio 43614 United States	Status: Recruiting	Contact: N/A
Facility: University of Oklahoma Health Sciences Center Oklahoma City, Oklahoma 73104 United States	Status: Recruiting	Contact: N/A
Facility: MidLantic Urology Bala-Cynwyd, Pennsylvania 19004 United States	Status: Recruiting	Contact: N/A
Facility: The Hospital of the University of Pennsylvania Philadelphia, Pennsylvania 19104 United States	Status: Recruiting	Contact: N/A
Facility: Medical University South Carolina Charleston, South Carolina 29425 United States	Status: Recruiting	Contact: N/A
Facility: Regional Urology Greenville, South Carolina 29605 United States	Status: Recruiting	Contact: N/A
Facility: Urology Associates, P.C. Nashville, Tennessee 37209 United States	Status: Recruiting	Contact: N/A
Facility: University of Texas Southwestern Medical Center Dallas, Texas 75390 United States	Status: Recruiting	Contact: N/A
Facility: The Methodist Hospital d/b/a Houston Methodist Hospital Houston, Texas 77030 United States	Status: Recruiting	Contact: N/A
Facility: The University of Texas MD Anderson Cancer Center Houston, Texas 77030 United States	Status: Recruiting	Contact: N/A
Facility: Virginia Urology Richmond, Virginia 23235 United States	Status: Recruiting	Contact: N/A
Facility: Urology of Virginia Virginia Beach, Virginia 23462 United States	Status: Recruiting	Contact: N/A
Facility: West Virginia University Cancer Institute Morgantown, West Virginia 26506 United States	Status: Recruiting	Contact: N/A

Brief Title: Codex: Study of Inodiftagene Vixteplasmid (BC-819) in Unresponsive NMIBC

A Phase 2 Study of BC-819 in Patients With Non-Muscle Invasive Bladder Cancer Whose Disease is Unresponsive to Bacillus Calmette-Guerin

INTRODUCTION

BRIEF SUMMARY

DETAILED DESCRIPTION

PRIMARY OUTCOMES

CONDITION

ELIGIBILITY

OFFICIAL INFORMATION

Overall Contact

LOCATION

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