Trial Outcomes & Findings for Window Study of Nivolumab With or Without Ipilimumab in Squamous Cell Carcinoma of the Oral Cavity (NCT NCT02919683)

NCT ID: NCT02919683

Last Updated: 2026-01-28

Results Overview

Response rate to window treatment with single agent nivolumab or nivolumab combined with ipilimumab is determined using bidirectional measurements (product of longest 2 diameters of lesions) of primary and nodal lesions to be removed at the time of surgery. Responders will have demonstrated any reduction in overall tumor volume as determined by the product of the longest perpendicular bidirectional tumor measurements.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

30 participants

Primary outcome timeframe

At time of surgery

Results posted on

2026-01-28

Participant Flow

Participant milestones

Participant milestones
Measure	Nivolumab With Ipilimumab * Nivolumab to be delivered at a pre-determine dose for two weeks * Ipilimumab to be delivered at a pre-determine dose for one week * Blood Sample Collected * Standard of Care Surgery Nivolumab Ipilimumab Standard of Care Surgery	Nivolumab * Nivolumab to be delivered at a pre-determine dose for two weeks * Blood Sample Collected * Standard of Care Surgery Nivolumab Standard of Care Surgery
Pre-Surgery Intervention STARTED	15	15
Pre-Surgery Intervention COMPLETED	15	15
Pre-Surgery Intervention NOT COMPLETED	0	0
Post-Surgery Follow-Up STARTED	15	15
Post-Surgery Follow-Up COMPLETED	12	13
Post-Surgery Follow-Up NOT COMPLETED	3	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Nivolumab With Ipilimumab * Nivolumab to be delivered at a pre-determine dose for two weeks * Ipilimumab to be delivered at a pre-determine dose for one week * Blood Sample Collected * Standard of Care Surgery Nivolumab Ipilimumab Standard of Care Surgery	Nivolumab * Nivolumab to be delivered at a pre-determine dose for two weeks * Blood Sample Collected * Standard of Care Surgery Nivolumab Standard of Care Surgery
Post-Surgery Follow-Up Death	2	2
Post-Surgery Follow-Up Patient is still completing follow-ups	1	0

Baseline Characteristics

Window Study of Nivolumab With or Without Ipilimumab in Squamous Cell Carcinoma of the Oral Cavity

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Nivolumab With Ipilimumab n=15 Participants * Nivolumab to be delivered at a pre-determine dose for two weeks * Ipilimumab to be delivered at a pre-determine dose for one week * Blood Sample Collected * Standard of Care Surgery Nivolumab Ipilimumab Standard of Care Surgery	Nivolumab n=15 Participants * Nivolumab to be delivered at a pre-determine dose for two weeks * Blood Sample Collected * Standard of Care Surgery Nivolumab Standard of Care Surgery	Total n=30 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=41 Participants	7 Participants n=1581 Participants	14 Participants n=4626 Participants
Age, Categorical >=65 years	8 Participants n=41 Participants	8 Participants n=1581 Participants	16 Participants n=4626 Participants
Sex: Female, Male Female	7 Participants n=41 Participants	5 Participants n=1581 Participants	12 Participants n=4626 Participants
Sex: Female, Male Male	8 Participants n=41 Participants	10 Participants n=1581 Participants	18 Participants n=4626 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants
Race (NIH/OMB) Asian	1 Participants n=41 Participants	0 Participants n=1581 Participants	1 Participants n=4626 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants
Race (NIH/OMB) Black or African American	0 Participants n=41 Participants	1 Participants n=1581 Participants	1 Participants n=4626 Participants
Race (NIH/OMB) White	14 Participants n=41 Participants	14 Participants n=1581 Participants	28 Participants n=4626 Participants
Race (NIH/OMB) More than one race	0 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants

PRIMARY outcome

Timeframe: At time of surgery

Population: Thirty patients were treated from 2016 to 2019. One patient was excluded from efficacy analyses as she was ineligible because of evidence of distant metastatic disease at baseline.

Outcome measures

Outcome measures
Measure	Nivolumab With Ipilimumab n=15 Participants * Nivolumab to be delivered at a pre-determine dose for two weeks * Ipilimumab to be delivered at a pre-determine dose for one week * Blood Sample Collected * Standard of Care Surgery Nivolumab Ipilimumab Standard of Care Surgery	Nivolumab n=14 Participants * Nivolumab to be delivered at a pre-determine dose for two weeks * Blood Sample Collected * Standard of Care Surgery Nivolumab Standard of Care Surgery
Percentage of Participants With a Volumetric Response Rate to Treatment	53 percentage of participants Interval 34.2 to 71.8	50 percentage of participants Interval 30.5 to 69.5

PRIMARY outcome

Timeframe: At the time of surgery

Outcome measure includes number of participants with treatment-related adverse events as assessed by CTCAE v4.0, number of dose-limiting toxicities in safety run-ins following a 3 + 3 design, and delays to surgery.

Outcome measures

Outcome measures
Measure	Nivolumab With Ipilimumab n=15 Participants * Nivolumab to be delivered at a pre-determine dose for two weeks * Ipilimumab to be delivered at a pre-determine dose for one week * Blood Sample Collected * Standard of Care Surgery Nivolumab Ipilimumab Standard of Care Surgery	Nivolumab n=15 Participants * Nivolumab to be delivered at a pre-determine dose for two weeks * Blood Sample Collected * Standard of Care Surgery Nivolumab Standard of Care Surgery
Safety and Tolerability of Protocol Treatment Delays to Surgery	0 instances	0 instances
Safety and Tolerability of Protocol Treatment Dose-Limiting Toxic Effects during Safety Run-In	0 instances	0 instances
Safety and Tolerability of Protocol Treatment Grade 3-4 Events At Least Possibly Related to Protocol Treatment	5 instances	2 instances

SECONDARY outcome

Timeframe: At time of surgery

Population: For this analysis, patients with no radiographically measurable lesions on imaging review were excluded. Thirty patients were treated from 2016 to 2019. One patient was excluded from efficacy analyses as she was ineligible because of evidence of distant metastatic disease at baseline.

Determining the radiologic response rate following the window treatment as determined by RECIST v1.1.

Outcome measures

Outcome measures
Measure	Nivolumab With Ipilimumab n=13 Participants * Nivolumab to be delivered at a pre-determine dose for two weeks * Ipilimumab to be delivered at a pre-determine dose for one week * Blood Sample Collected * Standard of Care Surgery Nivolumab Ipilimumab Standard of Care Surgery	Nivolumab n=14 Participants * Nivolumab to be delivered at a pre-determine dose for two weeks * Blood Sample Collected * Standard of Care Surgery Nivolumab Standard of Care Surgery
Percentage of Participants Demonstrating Objective Response Using RECIST Criteria	38 percentage of participants	13 percentage of participants

SECONDARY outcome

Timeframe: At time of surgery

Population: Thirty patients were treated from 2016 to 2019. One patient was excluded from efficacy analyses as she was ineligible because of evidence of distant metastatic disease at baseline.

Pathologic response in the primary tumor was assessed using a quantitative grading scheme: pathologic tumor response \[nonviable tumor\] PTR0 = no or \<10% response PTR1 = ≥10% PTR2 = ≥50%

Outcome measures

Outcome measures
Measure	Nivolumab With Ipilimumab n=15 Participants * Nivolumab to be delivered at a pre-determine dose for two weeks * Ipilimumab to be delivered at a pre-determine dose for one week * Blood Sample Collected * Standard of Care Surgery Nivolumab Ipilimumab Standard of Care Surgery	Nivolumab n=14 Participants * Nivolumab to be delivered at a pre-determine dose for two weeks * Blood Sample Collected * Standard of Care Surgery Nivolumab Standard of Care Surgery
Percentage of Participants Demonstrating Pathological Response PTR1	40 percentage of participants	38 percentage of participants
Percentage of Participants Demonstrating Pathological Response PTR2	33 percentage of participants	15 percentage of participants

SECONDARY outcome

Timeframe: 1 year

Population: Thirty patients were treated from 2016 to 2019. One patient was excluded from efficacy analyses as she was ineligible because of evidence of distant metastatic disease at baseline.

Progression-free survival is defined as the time between first study treatment and either recurrent disease or death. Recurrent disease includes a local failure, regional failure, or distant metastasis.

Outcome measures

Outcome measures
Measure	Nivolumab With Ipilimumab n=29 Participants * Nivolumab to be delivered at a pre-determine dose for two weeks * Ipilimumab to be delivered at a pre-determine dose for one week * Blood Sample Collected * Standard of Care Surgery Nivolumab Ipilimumab Standard of Care Surgery	Nivolumab * Nivolumab to be delivered at a pre-determine dose for two weeks * Blood Sample Collected * Standard of Care Surgery Nivolumab Standard of Care Surgery
Participant One Year Progression-Free Survival Percentage	85 percentage of participants Interval 72.4 to 99.7	—

SECONDARY outcome

Timeframe: Data Cutoff (14.2 Months Median Follow Up)

Population: Thirty patients were treated from 2016 to 2019. One patient was excluded from efficacy analyses as she was ineligible because of evidence of distant metastatic disease at baseline.

Overall survival is defined as the time between first study treatment and death.

Outcome measures

Outcome measures
Measure	Nivolumab With Ipilimumab n=29 Participants * Nivolumab to be delivered at a pre-determine dose for two weeks * Ipilimumab to be delivered at a pre-determine dose for one week * Blood Sample Collected * Standard of Care Surgery Nivolumab Ipilimumab Standard of Care Surgery	Nivolumab * Nivolumab to be delivered at a pre-determine dose for two weeks * Blood Sample Collected * Standard of Care Surgery Nivolumab Standard of Care Surgery
Participant Overall Survival Percentage	89 percentage of participants Interval 78.3 to 100.0	—

Adverse Events

Nivolumab With Ipilimumab

Serious events: 2 serious events

Other events: 12 other events

Deaths: 2 deaths

Nivolumab

Serious events: 3 serious events

Other events: 11 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Nivolumab With Ipilimumab n=15 participants at risk * Nivolumab to be delivered at a pre-determine dose for two weeks * Ipilimumab to be delivered at a pre-determine dose for one week * Blood Sample Collected * Standard of Care Surgery Nivolumab Ipilimumab Standard of Care Surgery	Nivolumab n=15 participants at risk * Nivolumab to be delivered at a pre-determine dose for two weeks * Blood Sample Collected * Standard of Care Surgery Nivolumab Standard of Care Surgery
Vascular disorders Thromboembolic event	0.00% 0/15 • All adverse events experienced by participants were collected from the time of the first dose of study treatment, through the study and until the final study visit 1 year after enrollment. The descriptions and grading scales found in the CTEP Active Version of the NCI Common Terminology Criteria for Adverse Events (CTCAE version 4.0) were utilized for AE reporting.	6.7% 1/15 • All adverse events experienced by participants were collected from the time of the first dose of study treatment, through the study and until the final study visit 1 year after enrollment. The descriptions and grading scales found in the CTEP Active Version of the NCI Common Terminology Criteria for Adverse Events (CTCAE version 4.0) were utilized for AE reporting.
Musculoskeletal and connective tissue disorders Right knee effusion and fever	6.7% 1/15 • All adverse events experienced by participants were collected from the time of the first dose of study treatment, through the study and until the final study visit 1 year after enrollment. The descriptions and grading scales found in the CTEP Active Version of the NCI Common Terminology Criteria for Adverse Events (CTCAE version 4.0) were utilized for AE reporting.	0.00% 0/15 • All adverse events experienced by participants were collected from the time of the first dose of study treatment, through the study and until the final study visit 1 year after enrollment. The descriptions and grading scales found in the CTEP Active Version of the NCI Common Terminology Criteria for Adverse Events (CTCAE version 4.0) were utilized for AE reporting.
Infections and infestations Infection	0.00% 0/15 • All adverse events experienced by participants were collected from the time of the first dose of study treatment, through the study and until the final study visit 1 year after enrollment. The descriptions and grading scales found in the CTEP Active Version of the NCI Common Terminology Criteria for Adverse Events (CTCAE version 4.0) were utilized for AE reporting.	6.7% 1/15 • All adverse events experienced by participants were collected from the time of the first dose of study treatment, through the study and until the final study visit 1 year after enrollment. The descriptions and grading scales found in the CTEP Active Version of the NCI Common Terminology Criteria for Adverse Events (CTCAE version 4.0) were utilized for AE reporting.
Surgical and medical procedures Wound complication - flap thrombosis and failure	6.7% 1/15 • All adverse events experienced by participants were collected from the time of the first dose of study treatment, through the study and until the final study visit 1 year after enrollment. The descriptions and grading scales found in the CTEP Active Version of the NCI Common Terminology Criteria for Adverse Events (CTCAE version 4.0) were utilized for AE reporting.	0.00% 0/15 • All adverse events experienced by participants were collected from the time of the first dose of study treatment, through the study and until the final study visit 1 year after enrollment. The descriptions and grading scales found in the CTEP Active Version of the NCI Common Terminology Criteria for Adverse Events (CTCAE version 4.0) were utilized for AE reporting.
Vascular disorders Stroke	6.7% 1/15 • All adverse events experienced by participants were collected from the time of the first dose of study treatment, through the study and until the final study visit 1 year after enrollment. The descriptions and grading scales found in the CTEP Active Version of the NCI Common Terminology Criteria for Adverse Events (CTCAE version 4.0) were utilized for AE reporting.	0.00% 0/15 • All adverse events experienced by participants were collected from the time of the first dose of study treatment, through the study and until the final study visit 1 year after enrollment. The descriptions and grading scales found in the CTEP Active Version of the NCI Common Terminology Criteria for Adverse Events (CTCAE version 4.0) were utilized for AE reporting.
Endocrine disorders hyperglycemia and diabetic ketoacidosis	0.00% 0/15 • All adverse events experienced by participants were collected from the time of the first dose of study treatment, through the study and until the final study visit 1 year after enrollment. The descriptions and grading scales found in the CTEP Active Version of the NCI Common Terminology Criteria for Adverse Events (CTCAE version 4.0) were utilized for AE reporting.	6.7% 1/15 • All adverse events experienced by participants were collected from the time of the first dose of study treatment, through the study and until the final study visit 1 year after enrollment. The descriptions and grading scales found in the CTEP Active Version of the NCI Common Terminology Criteria for Adverse Events (CTCAE version 4.0) were utilized for AE reporting.

Other adverse events

Additional Information

Jonathan Schoenfeld, M.D. M.P.H.

Dana-Farber Cancer Institute

Phone: 617-632-3591

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place