Trial Outcomes & Findings for A Trial of NIS793 With FOLFIRINOX in Pancreatic Cancer (NCT NCT05546411)

Last Updated: 2024-12-17

Results Overview

Major pathological response (MPR) defined as \<5% residual tumor cells visible in the pancreatic resection specimen. The major pathological response rate will be analyzed among all patients who start protocol therapy, including those not resected due to early progression, death or off study.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

8 participants

Primary outcome timeframe

Pathology review is done in surgery assessments, once within 14 days prior to the operation.

Results posted on

2024-12-17

Participant Flow

Participant milestones

Participant milestones
Measure	mFOLFIRINOX + NIS793 Participants will be randomly assigned to receive: * FOLFIRINOX + NIS793 on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion NIS793: Given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor	mFOLFIRINOX Participants will be randomly assigned to receive: * FOLFIRINOX on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor
Overall Study STARTED	6	2
Overall Study COMPLETED	4	1
Overall Study NOT COMPLETED	2	1

Reasons for withdrawal

Reasons for withdrawal
Measure	mFOLFIRINOX + NIS793 Participants will be randomly assigned to receive: * FOLFIRINOX + NIS793 on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion NIS793: Given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor	mFOLFIRINOX Participants will be randomly assigned to receive: * FOLFIRINOX on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor
Overall Study Progressive Disease	1	0
Overall Study Adverse Event	0	1
Overall Study Metastatic disease	1	0

Baseline Characteristics

A Trial of NIS793 With FOLFIRINOX in Pancreatic Cancer

Baseline characteristics by cohort

PRIMARY outcome

Timeframe: Pathology review is done in surgery assessments, once within 14 days prior to the operation.

Population: This study was closed prematurely by the drug manufacturer, Novartis, due to safety concerns. At the time of study closure, 8 patients had received any protocol therapy. Out of the 8 participants who received treatment, only 5 underwent surgery before study closure.

Outcome measures

Outcome measures
Measure	mFOLFIRINOX + NIS793 n=6 Participants Participants will be randomly assigned to receive: * FOLFIRINOX + NIS793 on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion NIS793: Given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor	mFOLFIRINOX n=2 Participants Participants will be randomly assigned to receive: * FOLFIRINOX on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor
Major Pathological Response Rate (MPR)	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Cycle 1 Day 1 through 30 days post last treatment date, up to 9 months.

Population: This study was closed prematurely by the drug manufacturer, Novartis, due to safety concerns. At the time of study closure, 6 patients had received mFOLFIRINIX + NIS793 (Arm A) protocol therapy and 2 received mFOLFIRINOX (Arm B).

All adverse events (AE) with treatment attribution of possibly, probably or definite based on CTCAEv5 as reported on case report forms will be counted. Rate is the proportion of treated participants experiencing at least one treatment-related AE of any type during the time of observation.

Outcome measures

Outcome measures
Measure	mFOLFIRINOX + NIS793 n=6 Participants Participants will be randomly assigned to receive: * FOLFIRINOX + NIS793 on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion NIS793: Given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor	mFOLFIRINOX n=2 Participants Participants will be randomly assigned to receive: * FOLFIRINOX on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor
Treatment-Related Toxicity Rate	5 Participants	0 Participants

SECONDARY outcome

Timeframe: From randomization (or registration) to the earlier of progression or death due to any cause, up to 9 months. Participants alive without disease progression are censored at date of last disease evaluation.

Population: 4 participants received scans for PFS analysis in Arm A and 1 participant received a scan for PFS analysis in Arm B.

Progression-free survival based on the Kaplan-Meier method is defined as the duration of time from study entry to documented disease progression (PD) or death. Per RECIST 1.1 criteria: progressive disease (PD) is at least a 20% increase in the sum of longest diameter (LD) of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. PD for the evaluation of non-target lesions is the appearance of one or more new lesions and/or unequivocal progression of non-target lesions.

Outcome measures

Outcome measures
Measure	mFOLFIRINOX + NIS793 n=4 Participants Participants will be randomly assigned to receive: * FOLFIRINOX + NIS793 on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion NIS793: Given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor	mFOLFIRINOX n=1 Participants Participants will be randomly assigned to receive: * FOLFIRINOX on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor
Median Progression-free Survival (PFS)	114 days Interval 108.0 to 118.0	122 days Interval 122.0 to 122.0

SECONDARY outcome

Timeframe: Post treatment follow up done at least once every 12 weeks (+/- 28 days), up to 9 months.

Population: All participants were analyzed from the date of study entry until the date the participant came off study. No deaths were recorded while the participants were on study. All dates reflect the date the study was terminated or the date a subject began a new therapy and therefore transitioned off study.

OS is based on the Kaplan-Meier method, defined as the time from study entry to death or censored at date last known alive.

Outcome measures

Outcome measures
Measure	mFOLFIRINOX + NIS793 n=6 Participants Participants will be randomly assigned to receive: * FOLFIRINOX + NIS793 on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion NIS793: Given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor	mFOLFIRINOX n=2 Participants Participants will be randomly assigned to receive: * FOLFIRINOX on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor
Median Overall Survival (OS)	184 days Interval 43.0 to 254.0	155.5 days Interval 85.0 to 226.0

Adverse Events

mFOLFIRINOX + NIS793

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

mFOLFIRINOX

Serious events: 2 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	mFOLFIRINOX + NIS793 n=6 participants at risk Participants will be randomly assigned to receive: * FOLFIRINOX + NIS793 on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion NIS793: Given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor	mFOLFIRINOX n=2 participants at risk Participants will be randomly assigned to receive: * FOLFIRINOX on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor
Blood and lymphatic system disorders Febrile neutropenia	0.00% 0/6 • Adverse events were reviewed and reported after the initial dose of study treatment, during treatment, or within 30 days of the last dose of treatment. Adverse events were recorded between the first patient treated (February 2023) and 30 days out from the last patient ending treatment (October 2023), equaling a collection time of 9 months.	50.0% 1/2 • Adverse events were reviewed and reported after the initial dose of study treatment, during treatment, or within 30 days of the last dose of treatment. Adverse events were recorded between the first patient treated (February 2023) and 30 days out from the last patient ending treatment (October 2023), equaling a collection time of 9 months.
Gastrointestinal disorders Nausea	0.00% 0/6 • Adverse events were reviewed and reported after the initial dose of study treatment, during treatment, or within 30 days of the last dose of treatment. Adverse events were recorded between the first patient treated (February 2023) and 30 days out from the last patient ending treatment (October 2023), equaling a collection time of 9 months.	50.0% 1/2 • Adverse events were reviewed and reported after the initial dose of study treatment, during treatment, or within 30 days of the last dose of treatment. Adverse events were recorded between the first patient treated (February 2023) and 30 days out from the last patient ending treatment (October 2023), equaling a collection time of 9 months.

Other adverse events

Additional Information

Kimberly Perez, MD

Dana Farber Cancer Institute

Phone: 617-632-5370

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	mFOLFIRINOX + NIS793 n=6 Participants Participants will be randomly assigned to receive: * FOLFIRINOX + NIS793 on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion NIS793: Given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor	mFOLFIRINOX n=2 Participants Participants will be randomly assigned to receive: * FOLFIRINOX on day 1 of each 14 day cycle up to 8 cycles/16 weeks * Cycles 9+: Based on study team determination, some participants may be offered chemoradiation following completion of chemotherapy and/or surgery following either completion of chemotherapy or chemoradiation mFOLFIRINOX: Combination of the drugs 5-Fluorouracil (5-FU), Oxaliplatin, Irinotecan, and Leucovorin given by intravenous infusion 5-Fluorouracil (5-FU): Part of the FOLFIRINOX drug combination, given by intravenous infusion Oxaliplatin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Irinotecan: Part of the FOLFIRINOX drug combination, given by intravenous infusion Leucovorin: Part of the FOLFIRINOX drug combination, given by intravenous infusion Chemoradiation: Combination of Chemo (Capecitabine) and Radiation Therapy Surgery: Surgical removal of tumor	Total n=8 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants	1 Participants n=7 Participants	5 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants	1 Participants n=7 Participants	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=5 Participants	2 Participants n=7 Participants	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants	2 Participants n=7 Participants	7 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants