Trial Outcomes & Findings for Codex: Study of Inodiftagene Vixteplasmid (BC-819) in Unresponsive NMIBC (NCT NCT03719300)
NCT ID: NCT03719300
Last Updated: 2020-08-19
Results Overview
Complete response is defined as at least one of the following: * Negative cystoscopy and negative (including atypical) urine cytology * Positive cystoscopy with biopsy-proven benign or low-grade NMIBC and negative cytology * Negative cystoscopy with malignant urine cytology if cancer is found in the upper tract or prostatic urethra and random bladder biopsies are negative The complete response in patients with CIS for this endpoint was documented on or after the Week 12 response assessment and on or prior to the Week 48 assessment. Duration of complete response in patients with CIS was calculated from the documented onset of the complete response to the assessment where the patient no longer met the definition of complete response.
TERMINATED
PHASE2
32 participants
12 weeks
2020-08-19
Participant Flow
Eligible patients were patients with High Risk Non Muscle Invasive Bladder Cancer whose disease was unresponsive to BCG. It was planned to enroll and treat 140 patients (N=140), of which 70 to 100 were estimated to have carcinoma in situ (CIS) (with or without papillary disease).
Participant milestones
| Measure |
Single Arm BC-819
The administration of BC-819 was separated into 2 phases, the induction phase, and the maintenance phase. During the induction phase, patients were to receive an intravesical instillation of BC-819 at a dose of 20 mg/50 mL aqueous solution once per week for 10 weeks according to the induction phase treatment schedule. Upon completion of the induction phase, patients transitioned to maintenance therapy of BC-819 every 3 weeks beginning at Week 12 (Visit 11) and continuing for the next 84 additional weeks until the end of the study, defined as completion of the 96-week visit (Visit 39).
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|---|---|
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Overall Study
STARTED
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32
|
|
Overall Study
COMPLETED
|
32
|
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Codex: Study of Inodiftagene Vixteplasmid (BC-819) in Unresponsive NMIBC
Baseline characteristics by cohort
| Measure |
Single Arm BC-819
n=32 Participants
The administration of BC-819 was separated into 2 phases, the induction phase, and the maintenance phase. During the induction phase, patients were to receive an intravesical instillation of BC-819 at a dose of 20 mg/50 mL aqueous solution once per week for 10 weeks according to the induction phase treatment schedule. Upon completion of the induction phase, patients transitioned to maintenance therapy of BC-819 every 3 weeks beginning at Week 12 (Visit 11) and continuing for the next 84 additional weeks until the end of the study, defined as completion of the 96-week visit (Visit 39).
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|---|---|
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Age, Continuous
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72.3 years
n=5 Participants
|
|
Sex: Female, Male
Female
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6 Participants
n=5 Participants
|
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Sex: Female, Male
Male
|
26 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
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0 Participants
n=5 Participants
|
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
32 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
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2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
30 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
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Weight
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104.4 kg
STANDARD_DEVIATION 36.8 • n=5 Participants
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Height
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172.0 cm
STANDARD_DEVIATION 21.3 • n=5 Participants
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BMI
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39.7 kg/m^2
STANDARD_DEVIATION 36.5 • n=5 Participants
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Baseline ECOG performance status
0: Fully active
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30 Participants
n=5 Participants
|
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Baseline ECOG performance status
1: Restricted activity but ambulatory
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0 Participants
n=5 Participants
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Baseline ECOG performance status
2: Ambulatory
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2 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: 12 weeksPopulation: 17 participants had efficacy assessment for this Outcome Measure.
Complete response is defined as at least one of the following: * Negative cystoscopy and negative (including atypical) urine cytology * Positive cystoscopy with biopsy-proven benign or low-grade NMIBC and negative cytology * Negative cystoscopy with malignant urine cytology if cancer is found in the upper tract or prostatic urethra and random bladder biopsies are negative The complete response in patients with CIS for this endpoint was documented on or after the Week 12 response assessment and on or prior to the Week 48 assessment. Duration of complete response in patients with CIS was calculated from the documented onset of the complete response to the assessment where the patient no longer met the definition of complete response.
Outcome measures
| Measure |
Single Arm BC-819
n=17 Participants
The administration of BC-819 was separated into 2 phases, the induction phase, and the maintenance phase. During the induction phase, patients were to receive an intravesical instillation of BC-819 at a dose of 20 mg/50 mL aqueous solution once per week for 10 weeks according to the induction phase treatment schedule. Upon completion of the induction phase, patients transitioned to maintenance therapy of BC-819 every 3 weeks beginning at Week 12 (Visit 11) and continuing for the next 84 additional weeks until the end of the study, defined as completion of the 96-week visit (Visit 39).
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|---|---|
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The Percentage of Patients With Baseline CIS That Achieve a Complete Response After Treatment With BC-819 (Measured at 12 Weeks)
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17.6 Percentage of participants
Interval 3.8 to 43.4
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SECONDARY outcome
Timeframe: 48 weeksPopulation: Data were not collected.
Time to recurrence (Kaplan-Meier plot) recurrence is defined as the reappearance or persistence of high-grade disease, or new high-grade disease. Recurrence must be biopsy proven. Persistence, appearance, or presence of lower grade disease was not considered to be a recurrence event
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 12, 24, 36, 72, and 96 weeksPopulation: Data were not collected.
Time to recurrence (Kaplan-Meier plot) recurrence is defined as the reappearance or persistence of high-grade disease, or new high-grade disease. Recurrence must be biopsy proven. Persistence, appearance, or presence of lower grade disease was not considered to be a recurrence event.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 48, 72, and 96 weeksPopulation: Data were not collected.
The incidence of PFS at 48, 72, and 96 weeks as well as time to progression estimated using Kaplan-Meir methods. Progression is defined as the development of T2 or greater disease. Sensitivity analyses was performed and included any of the following as progressions: * An increase in stage from Ta or CIS to T1, or * Development of T2 or greater, or * Lymph node disease, or * Distant metastasis
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 48, 72, and 96 weeksPopulation: Data were not collected.
Overall survival of patients enrolled in the study at 48, 72, and 96 weeks and survival time was estimated using Kaplan-Meier methods
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 48, 72, and 96 weeksPopulation: Data were not collected.
Measured by the The European Organization for Research and Treatment of Cancer quality of life questionnaire (EORTC QLQ-C30), a general questionnaire for assessing quality of life in cancer patients, and the Non-Muscle Invasive Bladder Cancer Questionnaire (QLQ-NMIBC24 ) for patients with NBIMC disease. EORTC QLQ-C30 include five functional scales , three symptom scales, a global health status/quality of life scale, and six single items. QLQ-NMIBC24 include five multi-item symptom scales, one multi-item functional scale, and five single-item measures.These scales range in score 0-100 scale and an for functional scales, a higher a higher score corresponds to greater function or quality of life. For symptom scales, a higher score corresponds to greater symptom burden.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 9 monthsPopulation: Data were not collected.
The safety was evaluated by assessment of AEs according to CTCAE version 5.0, regardless of relationship to study medication.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 12, 24, 36, 72, and 96 weeksPopulation: Data were not collected.
Time to recurrence (Kaplan-Meier plot) recurrence is defined as the reappearance or persistence of high-grade disease, or new high-grade disease. Recurrence must be biopsy proven. Persistence, appearance, or presence of lower grade disease was not considered to be a recurrence event
Outcome measures
Outcome data not reported
Adverse Events
Single Arm BC-819
Serious adverse events
| Measure |
Single Arm BC-819
n=32 participants at risk
The administration of BC-819 was separated into 2 phases, the induction phase, and the maintenance phase. During the induction phase, patients were to receive an intravesical instillation of BC-819 at a dose of 20 mg/50 mL aqueous solution once per week for 10 weeks according to the induction phase treatment schedule. Upon completion of the induction phase, patients transitioned to maintenance therapy of BC-819 every 3 weeks beginning at Week 12 (Visit 11) and continuing for the next 84 additional weeks until the end of the study, defined as completion of the 96-week visit (Visit 39).
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|---|---|
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Cardiac disorders
Acute left ventricular failure
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3.1%
1/32 • 9 months
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Nervous system disorders
Encephalopathy
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3.1%
1/32 • 9 months
|
|
Renal and urinary disorders
Haematuria
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3.1%
1/32 • 9 months
|
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Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
3.1%
1/32 • 9 months
|
Other adverse events
| Measure |
Single Arm BC-819
n=32 participants at risk
The administration of BC-819 was separated into 2 phases, the induction phase, and the maintenance phase. During the induction phase, patients were to receive an intravesical instillation of BC-819 at a dose of 20 mg/50 mL aqueous solution once per week for 10 weeks according to the induction phase treatment schedule. Upon completion of the induction phase, patients transitioned to maintenance therapy of BC-819 every 3 weeks beginning at Week 12 (Visit 11) and continuing for the next 84 additional weeks until the end of the study, defined as completion of the 96-week visit (Visit 39).
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|---|---|
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Gastrointestinal disorders
Diarrhoea
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6.2%
2/32 • 9 months
|
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General disorders
Fatigue
|
12.5%
4/32 • 9 months
|
|
General disorders
Chills
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6.2%
2/32 • 9 months
|
|
Infections and infestations
Urinary Tract Infection
|
12.5%
4/32 • 9 months
|
|
Infections and infestations
Nasopharyngitis
|
6.2%
2/32 • 9 months
|
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Musculoskeletal and connective tissue disorders
Back pain
|
6.2%
2/32 • 9 months
|
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Musculoskeletal and connective tissue disorders
Flank pain
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6.2%
2/32 • 9 months
|
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Nervous system disorders
Headache
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9.4%
3/32 • 9 months
|
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Psychiatric disorders
Insomnia
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6.2%
2/32 • 9 months
|
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Renal and urinary disorders
Micturition urgency
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21.9%
7/32 • 9 months
|
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Renal and urinary disorders
Dysuria
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18.8%
6/32 • 9 months
|
|
Renal and urinary disorders
Pollakiuria
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15.6%
5/32 • 9 months
|
|
Renal and urinary disorders
Urinary retention
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9.4%
3/32 • 9 months
|
|
Renal and urinary disorders
Bladder spasm
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6.2%
2/32 • 9 months
|
|
Renal and urinary disorders
Haematuria
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6.2%
2/32 • 9 months
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Additional Information
David Kerstein, M.D.,Chief Medical Officer
Anchiano Therapeutics, Israel Ltd.1 Kendall Square, Building 1400, Suite 105 Cambridge, MA 02139
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place