Trial Outcomes & Findings for Pembrolizumab & Cabozantinib in Patients With Head and Neck Squamous Cell Cancer (NCT NCT03468218)
NCT ID: NCT03468218
Last Updated: 2026-01-23
Results Overview
Will assess the proportion of subjects with partial response or complete response as defined by Response Evaluation Criteria in Solid Tumors version 1.1 response criteria. ORR will be calculated with 95% confidence interval by binomial distribution. The ability of biomarkers to predict ORR will be estimated by chi-square test and/or logistic regression model.
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE2
Target enrollment
36 participants
Primary outcome timeframe
Up to 2 years after treatment start
Results posted on
2026-01-23
Participant Flow
Participant milestones
| Measure |
Treatment (Pembrolizumab, Cabozantinib)
Patients receive pembrolizumab IV over 30 minutes on day 1 and cabozantinib PO QD on days 1-21. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity.
Cabozantinib: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
Overall Study
STARTED
|
36
|
|
Overall Study
COMPLETED
|
11
|
|
Overall Study
NOT COMPLETED
|
25
|
Reasons for withdrawal
| Measure |
Treatment (Pembrolizumab, Cabozantinib)
Patients receive pembrolizumab IV over 30 minutes on day 1 and cabozantinib PO QD on days 1-21. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity.
Cabozantinib: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
Overall Study
Adverse Event
|
5
|
|
Overall Study
Progressive Disease
|
16
|
|
Overall Study
Death
|
1
|
|
Overall Study
Alternate therapy
|
1
|
|
Overall Study
Withdrawal by Subject
|
2
|
Baseline Characteristics
Pembrolizumab & Cabozantinib in Patients With Head and Neck Squamous Cell Cancer
Baseline characteristics by cohort
| Measure |
Treatment (Pembrolizumab, Cabozantinib)
n=36 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1 and cabozantinib PO QD on days 1-21. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity.
Cabozantinib: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=270 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
23 Participants
n=270 Participants
|
|
Age, Categorical
>=65 years
|
13 Participants
n=270 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=270 Participants
|
|
Sex: Female, Male
Male
|
30 Participants
n=270 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=270 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
34 Participants
n=270 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=270 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=270 Participants
|
|
Race (NIH/OMB)
Asian
|
5 Participants
n=270 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=270 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=270 Participants
|
|
Race (NIH/OMB)
White
|
27 Participants
n=270 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=270 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=270 Participants
|
|
Region of Enrollment
United States
|
36 Participants
n=270 Participants
|
|
Smoking Status
Current Smoker
|
9 Participants
n=270 Participants
|
|
Smoking Status
Former Smoker
|
8 Participants
n=270 Participants
|
|
Smoking Status
Never Smoker
|
19 Participants
n=270 Participants
|
PRIMARY outcome
Timeframe: Up to 2 years after treatment startWill assess the proportion of subjects with partial response or complete response as defined by Response Evaluation Criteria in Solid Tumors version 1.1 response criteria. ORR will be calculated with 95% confidence interval by binomial distribution. The ability of biomarkers to predict ORR will be estimated by chi-square test and/or logistic regression model.
Outcome measures
| Measure |
Treatment (Pembrolizumab, Cabozantinib)
n=36 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1 and cabozantinib PO QD on days 1-21. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity.
Cabozantinib: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
Overall Response Rate (ORR)
|
52 percentage of participants
|
SECONDARY outcome
Timeframe: Duration from date of treatment start to the date of objectively documented progression or death due to any cause, whichever status is recorded first, assessed up to 2 yearsThe median PFS will be estimated by Kaplan-Meier method along with 95% confidence interval. The biomarker association with PFS will be assessed by the Kaplan-Meier method, log-rank test, and Cox model.
Outcome measures
| Measure |
Treatment (Pembrolizumab, Cabozantinib)
n=36 Participants
Patients receive pembrolizumab IV over 30 minutes on day 1 and cabozantinib PO QD on days 1-21. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity.
Cabozantinib: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
Progression Free Survival (PFS)
|
54 percentage of participants
Interval 31.5 to 72.0
|
Adverse Events
Treatment (Pembrolizumab, Cabozantinib)
Serious events: 18 serious events
Other events: 24 other events
Deaths: 7 deaths
Serious adverse events
| Measure |
Treatment (Pembrolizumab, Cabozantinib)
n=36 participants at risk
Patients receive pembrolizumab IV over 30 minutes on day 1 and cabozantinib PO QD on days 1-21. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity.
Cabozantinib: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
Gastrointestinal disorders
Hospitalization-Transanitis
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Vascular disorders
Hospitalization- Low blood pressure
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Investigations
Hospitalization-hyponatremia
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Respiratory, thoracic and mediastinal disorders
Hospiltaization- acute respiratory failure
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Hospitalization-drug induced hepatitis
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
General disorders
Death
|
19.4%
7/36 • 2 years
CTCAE Version 4
|
|
Cardiac disorders
Hospitalization- Atrial Fibrillation
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Vascular disorders
Hospitalization-hypotension
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Hospitalization- Aspiration
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Hospitalization- Ulcer in right oropharynx
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Investigations
Hospitalization- Hypercalcemia
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Hospitalizaiton- Pancreatitis
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Respiratory, thoracic and mediastinal disorders
Hospitalization- lower lung consolidation/ NV
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Infections and infestations
Hospitalzation- Leukocytosis, sepsis bowel obstruction
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Blood and lymphatic system disorders
Hospitalizaiton- Anemia
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Musculoskeletal and connective tissue disorders
Hospitalization- Back Pain
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Cardiac disorders
Hospitalization- Tachycardia
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Hospitalization- Dysphagia
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Infections and infestations
Hospitalization- Port infection
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
Other adverse events
| Measure |
Treatment (Pembrolizumab, Cabozantinib)
n=36 participants at risk
Patients receive pembrolizumab IV over 30 minutes on day 1 and cabozantinib PO QD on days 1-21. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity.
Cabozantinib: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
General disorders
Fatigue
|
44.4%
16/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Diarrhea
|
36.1%
13/36 • 2 years
CTCAE Version 4
|
|
Endocrine disorders
Hypothyroidism
|
33.3%
12/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Constipation
|
30.6%
11/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Dry Mouth
|
27.8%
10/36 • 2 years
CTCAE Version 4
|
|
Metabolism and nutrition disorders
Anorexia
|
27.8%
10/36 • 2 years
CTCAE Version 4
|
|
Nervous system disorders
Headache
|
27.8%
10/36 • 2 years
CTCAE Version 4
|
|
Vascular disorders
Hypertension
|
33.3%
12/36 • 2 years
CTCAE Version 4
|
|
Metabolism and nutrition disorders
Hyponatremia
|
27.8%
10/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Oral Mucositis
|
25.0%
9/36 • 2 years
CTCAE Version 4
|
|
Metabolism and nutrition disorders
hypomagnesemia
|
22.2%
8/36 • 2 years
CTCAE Version 4
|
|
General disorders
Pain
|
25.0%
9/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Dysphagia
|
27.8%
10/36 • 2 years
CTCAE Version 4
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
22.2%
8/36 • 2 years
CTCAE Version 4
|
|
Skin and subcutaneous tissue disorders
Palmer-Plantar erythrodysesthesia syndrome
|
19.4%
7/36 • 2 years
CTCAE Version 4
|
|
Respiratory, thoracic and mediastinal disorders
Sore Throat
|
22.2%
8/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Abdominal pain
|
16.7%
6/36 • 2 years
CTCAE Version 4
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
22.2%
8/36 • 2 years
CTCAE Version 4
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
19.4%
7/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Nausea
|
19.4%
7/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Oral Pain
|
19.4%
7/36 • 2 years
CTCAE Version 4
|
|
Skin and subcutaneous tissue disorders
Maculopapular rash
|
16.7%
6/36 • 2 years
CTCAE Version 4
|
|
Blood and lymphatic system disorders
Decreased white blood count
|
16.7%
6/36 • 2 years
CTCAE Version 4
|
|
Blood and lymphatic system disorders
Anemia
|
16.7%
6/36 • 2 years
CTCAE Version 4
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
13.9%
5/36 • 2 years
CTCAE Version 4
|
|
Nervous system disorders
Dysgeusia
|
13.9%
5/36 • 2 years
CTCAE Version 4
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders-Other, specify
|
13.9%
5/36 • 2 years
CTCAE Version 4
|
|
Metabolism and nutrition disorders
Increased alanine aminotransferase
|
13.9%
5/36 • 2 years
CTCAE Version 4
|
|
Metabolism and nutrition disorders
Increased AST
|
16.7%
6/36 • 2 years
CTCAE Version 4
|
|
Nervous system disorders
Dizziness
|
11.1%
4/36 • 2 years
CTCAE Version 4
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
13.9%
5/36 • 2 years
CTCAE Version 4
|
|
Vascular disorders
Hypotension
|
16.7%
6/36 • 2 years
CTCAE Version 4
|
|
Investigations
Investigations- other
|
11.1%
4/36 • 2 years
CTCAE Version 4
|
|
Blood and lymphatic system disorders
Decreased Platelet Count
|
13.9%
5/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Vomiting
|
13.9%
5/36 • 2 years
CTCAE Version 4
|
|
Metabolism and nutrition disorders
Dehydration
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Hepatobiliary disorders
Increased gamma-glutamyl transferase
|
11.1%
4/36 • 2 years
CTCAE Version 4
|
|
General disorders
General disorders and administration site conditions- Other, specify
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Renal and urinary disorders
Hematuria
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Hepatobiliary disorders
Hepatobiliary disorders-Other, specify
|
11.1%
4/36 • 2 years
CTCAE Version 4
|
|
Renal and urinary disorders
Hypoalbuminemia
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Metabolism and nutrition disorders
Hypokalemia
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Investigations
Increased Lipase
|
11.1%
4/36 • 2 years
CTCAE Version 4
|
|
General disorders
Pain in Extremity
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Nervous system disorders
Paresthesia
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Skin and subcutaneous tissue disorders
acneiform rash
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Metabolism and nutrition disorders
Increased serum amylase
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Skin and subcutaneous tissue disorders
skin and subcutaneous tissue disorders-Other, specify
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Weight loss
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Investigations
Increased alkaline phosphate
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Psychiatric disorders
Anxiety
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Investigations
Increased blood bilirubin
|
8.3%
3/36 • 2 years
CTCAE Version 4
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Dyspepsia
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Ear and labyrinth disorders
Ear pain
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Infections and infestations
Infections and infestations-Other
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Blood and lymphatic system disorders
Leukocytosis
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Investigations
Decreased lymphocyte count
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Oral dysesthesia
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Hepatobiliary disorders
Pancreatitis
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Infections and infestations
Sinusitis
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Stomach pain
|
5.6%
2/36 • 2 years
CTCAE Version 4
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Cardiac disorders
Myocardial infarction
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
gastric obstruction
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
|
Gastrointestinal disorders
Pancreatitis
|
2.8%
1/36 • 2 years
CTCAE Version 4
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place