Trial Outcomes & Findings for Cabozantinib and Pembrolizumab for the First-Line Treatment of Advanced Liver Cancer (NCT NCT04442581)
NCT ID: NCT04442581
Last Updated: 2023-04-05
Results Overview
Will be assessed per Response Evaluation Criteria in Solid Tumors (RECIST) version (v)1.1 (by blinded central review). Assessment of objective response per Response Evaluation Criteria in Solid Tumors version 1.1 of target lesions by cross sectional imaging (either CT or MRI), defined as the following: 1.) Complete Response (CR): Disappearance of all target lesions, Partial Response (PR): At least a 30% decrease in the sum of the longest diameter (LD) of target lesions, taking as reference the baseline sum LD, Progressive Disease (PD): At least a 20% increase in the sum of the LD of target lesions, Stable Disease (SD): neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. Overall Response Rate is defined as percentage of CR + PR.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
2 participants
Primary outcome timeframe
Assessed at baseline, every 2 cycles until cycle 4, then every 3 cycles until disease progression, up to 10 months 16 days.
Results posted on
2023-04-05
Participant Flow
Participant milestones
| Measure |
Treatment (Cabozantinib S-malate, Pembrolizumab)
Patients receive cabozantinib S-malate PO QD on days 1-21 and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
Stage 1
STARTED
|
2
|
|
Stage 1
COMPLETED
|
2
|
|
Stage 1
NOT COMPLETED
|
0
|
|
Stage 2
STARTED
|
0
|
|
Stage 2
COMPLETED
|
0
|
|
Stage 2
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Cabozantinib and Pembrolizumab for the First-Line Treatment of Advanced Liver Cancer
Baseline characteristics by cohort
| Measure |
Treatment (Cabozantinib S-malate, Pembrolizumab)
n=2 Participants
Patients receive cabozantinib S-malate PO QD on days 1-21 and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=93 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=93 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=93 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=93 Participants
|
PRIMARY outcome
Timeframe: Assessed at baseline, every 2 cycles until cycle 4, then every 3 cycles until disease progression, up to 10 months 16 days.Will be assessed per Response Evaluation Criteria in Solid Tumors (RECIST) version (v)1.1 (by blinded central review). Assessment of objective response per Response Evaluation Criteria in Solid Tumors version 1.1 of target lesions by cross sectional imaging (either CT or MRI), defined as the following: 1.) Complete Response (CR): Disappearance of all target lesions, Partial Response (PR): At least a 30% decrease in the sum of the longest diameter (LD) of target lesions, taking as reference the baseline sum LD, Progressive Disease (PD): At least a 20% increase in the sum of the LD of target lesions, Stable Disease (SD): neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. Overall Response Rate is defined as percentage of CR + PR.
Outcome measures
| Measure |
Treatment (Cabozantinib S-malate, Pembrolizumab)
n=2 Participants
Patients receive cabozantinib S-malate PO QD on days 1-21 and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
Number of Participants With Objective Response (Complete or Partial Response)
|
0 Participants
|
SECONDARY outcome
Timeframe: Assessed at baseline, every 2 cycles until cycle 4, then every 3 cycles until disease progression, up to 10 months 16 days.Will be assessed per RECIST v1.1 and iRECIST. Assessment of objective response per Response Evaluation Criteria in Solid Tumors version 1.1 of target lesions by cross sectional imaging (either CT or MRI), defined as the following: 1.) Complete Response (CR): Disappearance of all target lesions, Partial Response (PR): At least a 30% decrease in the sum of the longest diameter (LD) of target lesions, taking as reference the baseline sum LD, Progressive Disease (PD): At least a 20% increase in the sum of the LD of target lesions, Stable Disease (SD): neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. Overall Response Rate is defined as percentage of CR + PR.
Outcome measures
| Measure |
Treatment (Cabozantinib S-malate, Pembrolizumab)
n=2 Participants
Patients receive cabozantinib S-malate PO QD on days 1-21 and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
Number of Participants With Disease Control (Complete + Partial Response + Stable Disease)
|
2 Participants
|
SECONDARY outcome
Timeframe: Assessed at baseline, every 2 cycles until cycle 4, then every 3 cycles until disease progression, up to 10 months 16 days.Defined as time from study registration to radiographic progression per RECIST v1.1 (blinded central assessment), clinical progression, or death of any cause.
Outcome measures
| Measure |
Treatment (Cabozantinib S-malate, Pembrolizumab)
n=2 Participants
Patients receive cabozantinib S-malate PO QD on days 1-21 and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
Time in Months With Progression-free Survival
|
7.26 months
Interval 4.0 to 10.52
|
SECONDARY outcome
Timeframe: Survival assessed from study registration through study completion, 10 months 16 days.Defined as time from study registration to death of any cause.
Outcome measures
| Measure |
Treatment (Cabozantinib S-malate, Pembrolizumab)
n=2 Participants
Patients receive cabozantinib S-malate PO QD on days 1-21 and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
Overall Survival Time in Months
|
9.26 months
Interval 8.0 to 10.52
|
SECONDARY outcome
Timeframe: Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 daysWill be evaluated according to National Cancer Institute Common Terminology Criteria for Adverse Events v5.
Outcome measures
| Measure |
Treatment (Cabozantinib S-malate, Pembrolizumab)
n=2 Participants
Patients receive cabozantinib S-malate PO QD on days 1-21 and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
Number of Participants With Adverse Events
|
2 Participants
|
Adverse Events
Treatment (Cabozantinib S-malate, Pembrolizumab)
Serious events: 2 serious events
Other events: 2 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Treatment (Cabozantinib S-malate, Pembrolizumab)
n=2 participants at risk
Patients receive cabozantinib S-malate PO QD on days 1-21 and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
General disorders
General Weakness/Poor Performance
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Infections and infestations
Necrotizing fasciitis
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
Other adverse events
| Measure |
Treatment (Cabozantinib S-malate, Pembrolizumab)
n=2 participants at risk
Patients receive cabozantinib S-malate PO QD on days 1-21 and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
Pembrolizumab: Given IV
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
General disorders
Dry Mouth
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Hepatobiliary disorders
Alanine transaminase Increased
|
50.0%
1/2 • Number of events 3 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Hepatobiliary disorders
Aspartate aminotransferase Increased
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Blood and lymphatic system disorders
Neutrophil Count Decreased
|
50.0%
1/2 • Number of events 3 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Blood and lymphatic system disorders
White Blood Cell Count Decreased
|
50.0%
1/2 • Number of events 3 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
General disorders
Fatigue
|
100.0%
2/2 • Number of events 2 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Gastrointestinal disorders
Nausea
|
100.0%
2/2 • Number of events 2 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
General disorders
Abdominal Pain
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Gastrointestinal disorders
Vomiting
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
General disorders
Glossitis
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Gastrointestinal disorders
Anorexia
|
50.0%
1/2 • Number of events 2 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Renal and urinary disorders
Creatinine Increase
|
50.0%
1/2 • Number of events 2 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Cardiac disorders
Hypertension
|
50.0%
1/2 • Number of events 5 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Psychiatric disorders
Depression
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Gastrointestinal disorders
Constipation
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Skin and subcutaneous tissue disorders
Rash
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Blood and lymphatic system disorders
Blood Lactate Dehydrogenase Increase
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
|
Endocrine disorders
Thyroid Stimulating Hormone Increase
|
50.0%
1/2 • Number of events 1 • Adverse events monitored from time of informed consent through the last follow-up visit or 30 days after the date of the last dose of cabozantinib or pembrolizumab treatment, whichever occurs last, up to 10 months 16 days.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place