Trial Outcomes & Findings for Efficacy and Safety of BC-819 and Gemcitabine in Patients With Locally Advanced Pancreatic Adenocarcinoma (NCT NCT01413087)

Last Updated: 2019-11-01

Results Overview

To compare the effect of intratumoral endoscopic ultrasound injection of BC-819 administered with intravenous gemcitabine on progression-free survival. PFS was defined as the time from the date of consent until objective tumor progression or death. Median PFS by Kaplan-Meier analysis was used for evaluation. The target tumor lesion was identified and the longest diameter of the target lesion was measured according to Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 guidelines. For disease evaluations after treatment, scans conducted at baseline that were used for tumor measurements were repeated.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

24 months

Results posted on

2019-11-01

Participant Flow

Participant milestones

Participant milestones
Measure	8 mg BC-819 and Gemcitabine gemcitabine dose of 1000mg/m2 + 8 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine gemcitabine dose of 1000mg/m2 + 12 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819
Overall Study STARTED	6	6
Overall Study COMPLETED	6	6
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Efficacy and Safety of BC-819 and Gemcitabine in Patients With Locally Advanced Pancreatic Adenocarcinoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	8 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 8 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 12 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819	Total n=12 Participants Total of all reporting groups
Age, Continuous	72.2 years STANDARD_DEVIATION 6.7 • n=93 Participants	62.2 years STANDARD_DEVIATION 13.0 • n=4 Participants	67.4 years STANDARD_DEVIATION 11.3 • n=27 Participants
Sex: Female, Male Female	4 Participants n=93 Participants	1 Participants n=4 Participants	5 Participants n=27 Participants
Sex: Female, Male Male	2 Participants n=93 Participants	5 Participants n=4 Participants	7 Participants n=27 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Black or African American	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants
Race (NIH/OMB) White	5 Participants n=93 Participants	6 Participants n=4 Participants	11 Participants n=27 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants

PRIMARY outcome

Timeframe: 24 months

Population: ITT comprised of all subjects who received any treatment with either gemcitabine or BC-819 and whose tumors were positive for H19. All 12 patients were included in the ITT population.

Outcome measures

Outcome measures
Measure	8 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 8 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 12 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819
Progression-free Survival (PFS)	9.3 months Interval 2.6 to 26.3	7.6 months Interval 6.3 to 10.7

SECONDARY outcome

Timeframe: 24 months

Population: ITT population comprised all subjects who received any treatment with either gemcitabine or BC-819 and whose tumors were positive for H19. All 12 patients were included in the ITT population

OS was calculated from the date of consent until death due to any cause.

Outcome measures

Outcome measures
Measure	8 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 8 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 12 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819
Overall Survival (OS)	9.9 years Interval 5.4 to 26.4	8.5 years Interval 6.8 to Upper limit of OS not reached

SECONDARY outcome

Timeframe: 8 weeks

Population: ITT population comprised of all subjects who received any treatment with either gemcitabine or BC-819 and whose tumors were positive for H19. All 12 patients were included in the ITT population

Response rate will be assessed both for the primary target tumor lesion alone and overall, including development of metastases. Target tumor lesions are identified and the longest diameter of the target lesion is measured by the Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 guidelines. Complete response: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. Partial Response: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. Progressive Disease: At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum must also show an increase of at least 5 mm. Stable Disease: Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.

Outcome measures

Outcome measures
Measure	8 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 8 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 12 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819
Response Rate of Target Lesion Complete Response	0 Participants	0 Participants
Response Rate of Target Lesion Partial Response	1 Participants	0 Participants
Response Rate of Target Lesion Stable Disease	2 Participants	6 Participants
Response Rate of Target Lesion Progressive Disease	1 Participants	0 Participants
Response Rate of Target Lesion Inevaluable - Missing Postbaseline Scan	2 Participants	0 Participants

SECONDARY outcome

Timeframe: an average of 16 weeks

Resectability of the target tumor lesion was determined by CT/MRI as defined in the National Comprehensive Cancer network (NCCN) guidelines. Resectable tumors have no arterial tumor contact (celiac axis \[CA\], superior mesenteric artery \[SMA\], or common hepatic artery \[CHA\]) and no tumor contact with the superior mesenteric vein (SMV) or portal vein (PV) or ≤180° contact without vein contour irregularity. \*Not Applicable refers to another clinically significant abnormality that interfered with resectability determination of the target tumor lesion.

Outcome measures

Outcome measures
Measure	8 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 8 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 12 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819
Resectability of the Target Tumor Lesion Not Resectable	5 Participants	6 Participants
Resectability of the Target Tumor Lesion Not Applicable*	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Screening, Visit 4 (post gemcitabine induction), Visit 9 (5 weeks), Visit 13 (9 weeks)

Quality of life will be assessed by the The Karnofsky Performance Status (KPS) Index. KPS scores over time will be compared to those at baseline and changes from baseline will be presented. KPS scores are on a scale (0, 10, 20, 30, 40, 50, 60, 70, 80, 90, 100) of 0 (dead) to 100 (Normal no complaints; no evidence of disease).

Outcome measures

Outcome measures
Measure	8 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 8 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 12 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819
Quality of Life (QoL) Assessed by Karnofsky Performance Status (KPS) Screening	90 score on a scale Interval 80.0 to 100.0	90 score on a scale Interval 80.0 to 100.0
Quality of Life (QoL) Assessed by Karnofsky Performance Status (KPS) Visit 4 (pre-randomization)	90 score on a scale Interval 90.0 to 90.0	85 score on a scale Interval 80.0 to 100.0
Quality of Life (QoL) Assessed by Karnofsky Performance Status (KPS) Visit 9 (Week 5)	80 score on a scale Interval 80.0 to 90.0	90 score on a scale Interval 80.0 to 90.0
Quality of Life (QoL) Assessed by Karnofsky Performance Status (KPS) Visit 13 (Week 9)	80 score on a scale Interval 80.0 to 90.0	90 score on a scale Interval 80.0 to 90.0

SECONDARY outcome

Timeframe: Screening, Visit 4 (post gemcitabine induction), Visit 9 (5 weeks), Visit 13 (9 weeks), every 6 months after Visit 13

The FACT-G is a 27-item compilation of general questions divided into 4 primary QoL domains: physical well-being, social/family well-being, emotional well-being and functional well-being. The total FACT-G scores will be summarized by descriptive statistics (e.g., n, mean, median, standard deviation and range). The individual FACT-G score will be presented by frequency and percentage. Scores range from 0-108. High total scores represent better general QoL.

Outcome measures

Outcome measures
Measure	8 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 8 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 12 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819
Quality of Life Using the Functional Assessment of Cancer Therapy-General (FACT-G) Questionnaire Screening	71.8 units on a scale Standard Deviation 11.8	81.9 units on a scale Standard Deviation 14.0
Quality of Life Using the Functional Assessment of Cancer Therapy-General (FACT-G) Questionnaire Max change from baseline	-1.3 units on a scale Standard Deviation 18.5	-2.5 units on a scale Standard Deviation 18.8

SECONDARY outcome

Timeframe: 24 months

Serum was collected for quantitative measurement of CA 19-9.

Outcome measures

Outcome measures
Measure	8 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 8 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 12 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819
Serological Tumor Marker: CA 19-9 Screening	1025.9 U/mL Standard Deviation 1090.6	587.7 U/mL Standard Deviation 340.1
Serological Tumor Marker: CA 19-9 Nadir value	369.8 U/mL Standard Deviation 511.7	149.1 U/mL Standard Deviation 97.0
Serological Tumor Marker: CA 19-9 Change at Nadir	-656.1 U/mL Standard Deviation 887.7	-438.6 U/mL Standard Deviation 252.2

SECONDARY outcome

Timeframe: 24 months

Measured by the average and median number of exposure of the patients to BC-819 and gemcitabine.

Outcome measures

Outcome measures
Measure	8 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 8 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 12 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819
Extent of Exposure - Gemcitabine Total Exposure (g)	20.1 g Standard Deviation 9.5	21.3 g Standard Deviation 11.9

SECONDARY outcome

Timeframe: 24 months

Measured by the average and median number of treatments of the patients to BC-819 and gemcitabine.

Outcome measures

Outcome measures
Measure	8 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 8 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 12 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819
Extent of Exposure - Gemcitabine Total Number of Treatments	17.3 number of treatments Standard Deviation 14.9	15.3 number of treatments Standard Deviation 12.7

SECONDARY outcome

Timeframe: 24 months

Measured by the average and median exposure of the patients to BC-819 and gemcitabine.

Outcome measures

Outcome measures
Measure	8 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 8 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 12 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819
Extent of Exposure - BC-819 Total Exposure (mg)	68.0 mg Standard Deviation 29.8	118.0 mg Standard Deviation 41.2

SECONDARY outcome

Timeframe: 24 months

Measured by the average and median number of treatments of the patients to BC-819 and gemcitabine.

Outcome measures

Outcome measures
Measure	8 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 8 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine n=6 Participants gemcitabine dose of 1000mg/m2 + 12 mg of BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive 6 weekly IV infusions of gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819
Extent of Exposure - BC-819 Total Number of Treatments	10.7 number of treatments Standard Deviation 8.4	11.2 number of treatments Standard Deviation 6.0

Adverse Events

8 mg BC-819 and Gemcitabine

Serious events: 5 serious events

Other events: 5 other events

Deaths: 0 deaths

12 mg BC-819 and Gemcitabine

Serious events: 5 serious events

Other events: 5 other events

Deaths: 1 deaths

Serious adverse events

Other adverse events

Additional Information

Dr. David Kerstein

Anchiano Therapeutics Israel Ltd.

Phone: 857-259-4626

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60

Measure	8 mg BC-819 and Gemcitabine n=6 participants at risk gemcitabine dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine n=6 participants at risk gemcitabine dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819
Infections and infestations Pneumonia	16.7% 1/6 • Number of events 1 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
General disorders Generalized Edema	16.7% 1/6 • Number of events 1 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Gastrointestinal disorders Gastrointestinal obstruction	16.7% 1/6 • Number of events 1 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Infections and infestations Clostridium difficile colitis	16.7% 1/6 • Number of events 1 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Metabolism and nutrition disorders Dehydration	16.7% 1/6 • Number of events 1 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Gastrointestinal disorders Epigastric pain	16.7% 1/6 • Number of events 1 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Gastrointestinal disorders Frequent bowel movement	16.7% 1/6 • Number of events 1 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Hepatobiliary disorders Bile duct obstruction	16.7% 1/6 • Number of events 1 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	33.3% 2/6 • Number of events 3 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Gastrointestinal disorders Duodenitis	16.7% 1/6 • Number of events 2 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Product Issues Stent malfunction	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • Number of events 2 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Gastrointestinal disorders Melena	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • Number of events 1 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Vascular disorders Deep vein thrombosis	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • Number of events 1 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Respiratory, thoracic and mediastinal disorders Bilateral pulmonary embolism	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • Number of events 1 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Injury, poisoning and procedural complications Injection site abscess	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • Number of events 1 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Investigations Blood bilirubin increased	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • Number of events 1 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Infections and infestations Urinary tract infection	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • Number of events 1 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.

Measure	8 mg BC-819 and Gemcitabine n=6 participants at risk gemcitabine dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 8 mg BC-819	12 mg BC-819 and Gemcitabine n=6 participants at risk gemcitabine dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819 Biological/Vaccine: BC-819: Post screening and prior to randomization, all patients will receive gemcitabine by IV infusion at a dose of 1000mg/m2 once weekly for four weeks. They may also enter the study if they received up to 4 doses of gemcitabine before entering the study. In this case, they will be randomized immediately provided there is no evidence of disease progression. After randomization patients will receive gemcitabine at a dose of 1000mg/m2 + 7 intratumoral injections of 12 mg BC-819
Gastrointestinal disorders Nausea	50.0% 3/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	50.0% 3/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Gastrointestinal disorders Vomiting	33.3% 2/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Gastrointestinal disorders Constipation	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Gastrointestinal disorders Abdominal pain	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Gastrointestinal disorders Diarrhea	33.3% 2/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
General disorders Fatigue	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
General disorders Asthenia	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Eye disorders Peripheral edema	33.3% 2/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
General disorders Pyrexia	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Investigations Increased blood bilirubin	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	50.0% 3/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Investigations Increased gamma glatamyl transferase	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	33.3% 2/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Investigations Decreased neutrophil count	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	33.3% 2/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Investigations Increased alanine aminotransferase	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	33.3% 2/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Investigations Increased aspartate aminotransferase	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	33.3% 2/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Investigations Increased blood alkaline phosphatase	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	33.3% 2/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Investigations Decreased haemoglobin	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Investigations Abnormal liver function tests	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Investigations Decreased platelet count	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Investigations Decreased weight	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Blood and lymphatic system disorders Anemia	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Blood and lymphatic system disorders Neutropenia	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Blood and lymphatic system disorders Thrombocytopenia	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Metabolism and nutrition disorders Decreased appetite	33.3% 2/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Hepatobiliary disorders Bile duct obstruction	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	33.3% 2/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
General disorders Chills	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Gastrointestinal disorders Diahrrea	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Gastrointestinal disorders Stomatitis	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
General disorders Peripheral edema	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Infections and infestations Cellulitis	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Investigations Abnormal liver function test	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Blood and lymphatic system disorders Decreased platelet count	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Skin and subcutaneous tissue disorders Swelling face	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.
Skin and subcutaneous tissue disorders Alopecia	0.00% 0/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.	16.7% 1/6 • 24 months Adverse Events were only assessed after randomization to either dose level of BC-819.