Trial Outcomes & Findings for Cabozantinib in Patients With Hepatocellular Carcinoma (ACTION) (NCT NCT04316182)

NCT ID: NCT04316182

Last Updated: 2025-03-30

Results Overview

Percentage of patients with Grade 3 AEs in relation with total number of treated patients

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 24 participants
• Primary outcome timeframe: Up to 18 months
• Results posted on: 2025-03-30

Participant Flow

Participant milestones

Measure	Cabozantinib Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
Overall Study STARTED	24
Overall Study COMPLETED	24
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Cabozantinib in Patients With Hepatocellular Carcinoma (ACTION)

Baseline characteristics by cohort

Measure	Cabozantinib n=24 Participants Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=93 Participants
Age, Categorical >=65 years	17 Participants n=93 Participants
Age, Continuous	69.67 years STANDARD_DEVIATION 10.37 • n=93 Participants
Sex: Female, Male Female	9 Participants n=93 Participants
Sex: Female, Male Male	15 Participants n=93 Participants
Race/Ethnicity, Customized Black or African American	1 Participants n=93 Participants
Race/Ethnicity, Customized Latin or Hispanic	1 Participants n=93 Participants
Race/Ethnicity, Customized White	22 Participants n=93 Participants
Tumor burden Extrahepatic spread	3 Participants n=93 Participants
Tumor burden Multinodular	14 Participants n=93 Participants
Tumor burden Portal invasion	4 Participants n=93 Participants
Tumor burden Single or up to 3 nodules >= 3cm	3 Participants n=93 Participants
Cirrhosis	19 Participants n=93 Participants
Vascular Invasion	8 Participants n=93 Participants
ECOG ECOG 0: Fully active, able to carry on all pre-disease performance without restriction	20 Participants n=93 Participants
ECOG ECOG 1: Restricted in strenuous activity but ambulatory/able to carry out work of light nature	4 Participants n=93 Participants
First line treatment Atezolizumab+bevacizumab	3 Participants n=93 Participants
First line treatment Lenvatinib	1 Participants n=93 Participants
First line treatment Nivolumab+ipilimumab	1 Participants n=93 Participants
First line treatment Sorafenib	18 Participants n=93 Participants
First line treatment Tislelizumab	1 Participants n=93 Participants
Worst type of progression Extrahepatic growth	2 Participants n=93 Participants
Worst type of progression Intrahepatic growth	8 Participants n=93 Participants
Worst type of progression New extrahepatic lesion	1 Participants n=93 Participants
Worst type of progression New intrahepatic lesion	11 Participants n=93 Participants
Worst type of progression Not available	2 Participants n=93 Participants
Worst first progression pattrern Extrahepatic growth	2 Participants n=93 Participants
Worst first progression pattrern Intrahepatic growth	7 Participants n=93 Participants
Worst first progression pattrern New extrahepatic lesion	3 Participants n=93 Participants
Worst first progression pattrern New intrahepatic lesion	10 Participants n=93 Participants
Worst first progression pattrern Not available	2 Participants n=93 Participants

PRIMARY outcome

Timeframe: Up to 18 months

Percentage of patients with Grade 3 AEs in relation with total number of treated patients

Outcome measures

Measure	Cabozantinib n=24 Participants Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
Rate of Adverse Events (AE) ≥ Grade 3 (CTCAE 5.0) Excluding Palmar-plantar Erythrodysesthesia	16 Participants

PRIMARY outcome

Timeframe: Up to 18 months

Percentage of patients with AEs in relation with total number of treated patients

Outcome measures

Measure	Cabozantinib n=24 Participants Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
Rate of Adverse Events	24 Participants

PRIMARY outcome

Timeframe: Up to 18 months

Percentage of patients with related AEs in relation with total number of treated patients

Outcome measures

Measure	Cabozantinib n=24 Participants Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
Rate of Related-AEs	24 Participants

PRIMARY outcome

Timeframe: Up to 18 months

Percentage of patients who die during treatment due to adverse events in relation with total number of treated patients

Outcome measures

Measure	Cabozantinib n=24 Participants Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
Rate of Death Due to Adverse Events	0 Participants

PRIMARY outcome

Timeframe: Up to 18 months

Percentage of patients with AEs leading to treatment discontinuation in relation with total number of treated patients

Outcome measures

Measure	Cabozantinib n=24 Participants Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
Rate of AEs Leading to Treatment Discontinuation	3 Participants

SECONDARY outcome

Timeframe: Up to 18 months

Time from the date of start of treatment until the date of objective disease progression or death

Outcome measures

Measure	Cabozantinib n=24 Participants Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
Time to Progression (TTP)	6 months Interval 3.0 to 8.0

SECONDARY outcome

Timeframe: Up to 18 months

ORR is defined as the number of subjects with a best overall response of a complete response (CR) or partial response (PR) divided by the number of included patients

Outcome measures

Measure	Cabozantinib n=24 Participants Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
Objective Response Rate (ORR)	8.3 percentage of patients Interval 1.0 to 27.0

SECONDARY outcome

Timeframe: Up to 18 months

Population: They are included patients with available radiological progression (18 patients). One patient did not progress and 5 patients died due to progression without having a corresponding radiological evaluation.

Type of progression divided by number of patients

Outcome measures

Measure	Cabozantinib n=18 Participants Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
Pattern of Progression Intrahepatic growth (IHG)	44.4 percentage of patients
Pattern of Progression New intrahepatic lesion (NIH)	22.2 percentage of patients
Pattern of Progression Extrahepatic growth (EHG)	11.1 percentage of patients
Pattern of Progression New extrahepatic lesion (NEH)	22.2 percentage of patients

SECONDARY outcome

Timeframe: Up to 18 months

Time from the date of start of treatment until the date of death

Outcome measures

Measure	Cabozantinib n=24 Participants Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
Overall Survival (OS)	11 months Interval 8.0 to 20.0

SECONDARY outcome

Timeframe: Up to 18 months

Time from the date of disease progression until the date of death

Outcome measures

Measure	Cabozantinib n=24 Participants Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
Post-progression Survival (PPS)	5 months Interval 2.0 to The statistical program does not provide the upper limit of the interval for this outcome due to insufficient number of participants with the event.

SECONDARY outcome

Timeframe: Up to 18 months

Number of subjects who develop new extra-hepatic spread divided by number of included patients

Outcome measures

Measure	Cabozantinib n=24 Participants Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
Rate of Patients Who Develop New Extra-hepatic Spread	22 percentage of patients

Adverse Events

Cabozantinib

Serious events: 8 serious events

Other events: 24 other events

Deaths: 15 deaths

Serious adverse events

Measure	Cabozantinib n=24 participants at risk Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
General disorders Pyrexia	4.2% 1/24 • Number of events 1 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Gastrointestinal disorders Upper gastrointestinal haemorrhage	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Gastrointestinal disorders Rectal haemorrhage	4.2% 1/24 • Number of events 1 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Gastrointestinal disorders Intestinal ischaemia	4.2% 1/24 • Number of events 1 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Hepatobiliary disorders Cholecystitis acute	4.2% 1/24 • Number of events 1 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Musculoskeletal and connective tissue disorders Back pain	4.2% 1/24 • Number of events 1 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Infections and infestations Peritonitis bacterial	4.2% 1/24 • Number of events 1 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Infections and infestations Skin infection	4.2% 1/24 • Number of events 1 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Infections and infestations Pneumonia	4.2% 1/24 • Number of events 1 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Infections and infestations COVID-19	4.2% 1/24 • Number of events 1 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.

Other adverse events

Measure	Cabozantinib n=24 participants at risk Cabozantinib at 60 mg/day in monotherapy until symptomatic tumor progression, unacceptable adverse events, patient decision or death Cabozantinib: Cabozantinib 60 mg/day. Cabozantinib dose will be modified upon development of adverse events.
Vascular disorders Hypertension	66.7% 16/24 • Number of events 27 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
General disorders Asthenia	25.0% 6/24 • Number of events 21 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
General disorders Discomfort	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
General disorders Fatigue	50.0% 12/24 • Number of events 17 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
General disorders Mucosal inflammation	12.5% 3/24 • Number of events 3 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
General disorders Oedema	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
General disorders Oedema peripheral	8.3% 2/24 • Number of events 3 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
General disorders Pyrexia	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
General disorders Decreased appetite	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Psychiatric disorders Confusional state	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Investigations Alanine aminotransferase increased	20.8% 5/24 • Number of events 16 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Investigations Aspartate aminotransferase increased	25.0% 6/24 • Number of events 13 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Investigations Blood bilirubin increased	8.3% 2/24 • Number of events 3 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Investigations Blood lactate dehydrogenase increased	16.7% 4/24 • Number of events 4 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Investigations Blood thyroid stimulating hormone increased	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Investigations Platelet count decreased	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Investigations Weight decreased	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Investigations Blood alkaline phosphatase increased	8.3% 2/24 • Number of events 5 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Injury, poisoning and procedural complications Joint injury	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Nervous system disorders Dizziness	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Headache	8.3% 2/24 • Number of events 3 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Nervous system disorders Tension headache	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Gastrointestinal disorders Abdominal discomfort	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Gastrointestinal disorders Abdominal pain	16.7% 4/24 • Number of events 6 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Gastrointestinal disorders Abdominal pain upper	20.8% 5/24 • Number of events 6 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Gastrointestinal disorders Ascites	12.5% 3/24 • Number of events 3 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Gastrointestinal disorders Constipation	25.0% 6/24 • Number of events 7 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Gastrointestinal disorders Diarrhoea	50.0% 12/24 • Number of events 31 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Gastrointestinal disorders Dyspepsia	20.8% 5/24 • Number of events 6 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Gastrointestinal disorders Nausea	20.8% 5/24 • Number of events 6 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Gastrointestinal disorders Oesophagitis	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Gastrointestinal disorders Rectal haemorrhage	8.3% 2/24 • Number of events 4 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Gastrointestinal disorders Vomiting	12.5% 3/24 • Number of events 5 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Hepatobiliary disorders Jaundice	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Skin and subcutaneous tissue disorders Hyperkeratosis	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Skin and subcutaneous tissue disorders Palmar-plantar erythrodysaesthesia syndrome	62.5% 15/24 • Number of events 46 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Skin and subcutaneous tissue disorders Pruritus	16.7% 4/24 • Number of events 7 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Skin and subcutaneous tissue disorders Rash	12.5% 3/24 • Number of events 3 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Skin and subcutaneous tissue disorders Skin lesion	8.3% 2/24 • Number of events 3 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Endocrine disorders Hypothyroidism	16.7% 4/24 • Number of events 4 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Musculoskeletal and connective tissue disorders Arthralgia	12.5% 3/24 • Number of events 3 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Musculoskeletal and connective tissue disorders Back pain	12.5% 3/24 • Number of events 7 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Musculoskeletal and connective tissue disorders Muscle spasms	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Musculoskeletal and connective tissue disorders Myalgia	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Musculoskeletal and connective tissue disorders Pain in extremity	8.3% 2/24 • Number of events 2 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Infections and infestations COVID-19	16.7% 4/24 • Number of events 4 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Metabolism and nutrition disorders Hyponatraemia	8.3% 2/24 • Number of events 3 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Metabolism and nutrition disorders Hypophosphataemia	8.3% 2/24 • Number of events 3 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.
Metabolism and nutrition disorders Decreased appetite	12.5% 3/24 • Number of events 8 • Adverse events were evaluated at each study visit through treatment completion and 30 days after the last dose, an average of 34 weeks.

Additional Information

Juan Luis Sanz (MW)

APICES

Phone: +34 918166804

Email: juanluis.sanz@apices.es

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place