Trial Outcomes & Findings for Axitinib Monotherapy With Early Dynamic Contrast Enhanced Ultrasound Monitoring in Chemorefractory Third Line Metastatic Colorectal Cancer (NCT NCT04355156)

Results Overview

Difference in median overall survival between patients in the Axitinib arm who achieve a reduction in CEHPI index of at least 20% from baseline and those who do not achieve in both Axitinib and placebo arms

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

52 participants

Primary outcome timeframe

Through study completion, an average of 1 year

Results posted on

2026-04-16

Participant Flow

Participant milestones

Participant milestones
Measure	Axitinib small molecule multi-kinase inhibitor Axitinib: - All patients randomised to this arm will receive 3mg BD, and increased at 2 weeks to 5mg BD * Outpatient monitoring at 2 week intervals for a minimum of 8 weeks, with 2 weekly liver contrast ultrasound for assessment of differential blood supply (CEHPI) * At 8 weeks, after tumour CT RECIST assessments, responders will continue monotherapy. In non-responders, those with disease progression, monotherapy can be continued if patient chooses to continue and if tolerated	Placebo Placebo: - All patients receiving placebo will receive tablets to take BD \- Outpatient monitoring at 2 week intervals for a minimum of 8 weeks, with 2 weekly liver contrast ultrasound for assessment of differential blood supply (CEHPI)
Overall Study STARTED	34	17
Overall Study COMPLETED	34	17
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Axitinib n=34 Participants small molecule multi-kinase inhibitor Axitinib: - All patients randomised to this arm will receive 3mg BD, and increased at 2 weeks to 5mg BD * Outpatient monitoring at 2 week intervals for a minimum of 8 weeks, with 2 weekly liver contrast ultrasound for assessment of differential blood supply (CEHPI) * At 8 weeks, after tumour CT RECIST assessments, responders will continue monotherapy. In non-responders, those with disease progression, monotherapy can be continued if patient chooses to continue and if tolerated	Placebo n=17 Participants Placebo: - All patients receiving placebo will receive tablets to take BD \- Outpatient monitoring at 2 week intervals for a minimum of 8 weeks, with 2 weekly liver contrast ultrasound for assessment of differential blood supply (CEHPI)	Total n=51 Participants Total of all reporting groups
Region of Enrollment United Kingdom	34 participants n=34 Participants	17 participants n=17 Participants	51 participants n=51 Participants
Age, Categorical <=18 years	0 Participants n=34 Participants	0 Participants n=17 Participants	0 Participants n=51 Participants
Age, Categorical Between 18 and 65 years	24 Participants n=34 Participants	14 Participants n=17 Participants	38 Participants n=51 Participants
Age, Categorical >=65 years	10 Participants n=34 Participants	3 Participants n=17 Participants	13 Participants n=51 Participants
Sex: Female, Male Female	10 Participants n=34 Participants	8 Participants n=17 Participants	18 Participants n=51 Participants
Sex: Female, Male Male	24 Participants n=34 Participants	9 Participants n=17 Participants	33 Participants n=51 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.

PRIMARY outcome

Timeframe: Through study completion, an average of 1 year

Difference in median overall survival between patients in the Axitinib arm who achieve a reduction in CEHPI index of at least 20% from baseline and those who do not achieve in both Axitinib and placebo arms

Outcome measures

Outcome measures
Measure	Axitinib n=34 Participants small molecule multi-kinase inhibitor Axitinib: - All patients randomised to this arm will receive 3mg BD, and increased at 2 weeks to 5mg BD * Outpatient monitoring at 2 week intervals for a minimum of 8 weeks, with 2 weekly liver contrast ultrasound for assessment of differential blood supply (CEHPI) * At 8 weeks, after tumour CT RECIST assessments, responders will continue monotherapy. In non-responders, those with disease progression, monotherapy can be continued if patient chooses to continue and if tolerated	Placebo n=17 Participants Placebo: - All patients receiving placebo will receive tablets to take BD \- Outpatient monitoring at 2 week intervals for a minimum of 8 weeks, with 2 weekly liver contrast ultrasound for assessment of differential blood supply (CEHPI)
Overall Survival	175.0 Days Interval 20.0 to 893.0	169.0 Days Interval 36.0 to 875.0

SECONDARY outcome

Timeframe: Through study completion, an average of 1 year

Overall Survival of Contrast Enhanced Hepatic Perfusion Index (CEHPI) responders vs non-responders as assessed on ultrasound imaging and as defined by Response Evaluation Criteria in Solid Tumors (RECIST), independent of whether they were in the Axitinib or placebo group

Outcome measures

Outcome measures
Measure	Axitinib n=14 Participants small molecule multi-kinase inhibitor Axitinib: - All patients randomised to this arm will receive 3mg BD, and increased at 2 weeks to 5mg BD * Outpatient monitoring at 2 week intervals for a minimum of 8 weeks, with 2 weekly liver contrast ultrasound for assessment of differential blood supply (CEHPI) * At 8 weeks, after tumour CT RECIST assessments, responders will continue monotherapy. In non-responders, those with disease progression, monotherapy can be continued if patient chooses to continue and if tolerated	Placebo n=19 Participants Placebo: - All patients receiving placebo will receive tablets to take BD \- Outpatient monitoring at 2 week intervals for a minimum of 8 weeks, with 2 weekly liver contrast ultrasound for assessment of differential blood supply (CEHPI)
Hazard Ratio for Overall Survival Based on CEHPI Response	2.575 Hazard Ratio Interval 1.415 to 4.686	0.3884 Hazard Ratio Interval 0.2134 to 0.7068

SECONDARY outcome

Timeframe: Through study completion, an average of 1 year

Hazard ratio for Progression Free Survival between Axitinib and placebo groups

Outcome measures

Outcome measures
Measure	Axitinib n=32 Participants small molecule multi-kinase inhibitor Axitinib: - All patients randomised to this arm will receive 3mg BD, and increased at 2 weeks to 5mg BD * Outpatient monitoring at 2 week intervals for a minimum of 8 weeks, with 2 weekly liver contrast ultrasound for assessment of differential blood supply (CEHPI) * At 8 weeks, after tumour CT RECIST assessments, responders will continue monotherapy. In non-responders, those with disease progression, monotherapy can be continued if patient chooses to continue and if tolerated	Placebo n=16 Participants Placebo: - All patients receiving placebo will receive tablets to take BD \- Outpatient monitoring at 2 week intervals for a minimum of 8 weeks, with 2 weekly liver contrast ultrasound for assessment of differential blood supply (CEHPI)
Hazard Ratio for Progression Free Survival	1.828 Hazard Ratio Interval 1.021 to 3.272	0.5471 Hazard Ratio Interval 0.3056 to 0.9793

SECONDARY outcome

Timeframe: Through study completion, an average of 1 year

Hazard Ratio for Progression Free Survival between CEHPI responders and non-responders of patients in the Axitinib group

Outcome measures

Outcome measures
Measure	Axitinib n=14 Participants small molecule multi-kinase inhibitor Axitinib: - All patients randomised to this arm will receive 3mg BD, and increased at 2 weeks to 5mg BD * Outpatient monitoring at 2 week intervals for a minimum of 8 weeks, with 2 weekly liver contrast ultrasound for assessment of differential blood supply (CEHPI) * At 8 weeks, after tumour CT RECIST assessments, responders will continue monotherapy. In non-responders, those with disease progression, monotherapy can be continued if patient chooses to continue and if tolerated	Placebo n=19 Participants Placebo: - All patients receiving placebo will receive tablets to take BD \- Outpatient monitoring at 2 week intervals for a minimum of 8 weeks, with 2 weekly liver contrast ultrasound for assessment of differential blood supply (CEHPI)
Hazard Ratio for Progression Free Survival Between CEHPI Responders Compared to Non-Responders in Axitinib Group	1.783 Hazard Ratio Interval 0.8804 to 3.609	0.5610 Hazard Ratio Interval 0.2771 to 1.136

Adverse Events

Axitinib

Serious events: 19 serious events

Other events: 0 other events

Deaths: 31 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 14 deaths

Serious adverse events

Serious adverse events
Measure	Axitinib n=32 participants at risk small molecule multi-kinase inhibitor Axitinib: - All patients randomised to this arm will receive 3mg BD, and increased at 2 weeks to 5mg BD * Outpatient monitoring at 2 week intervals for a minimum of 8 weeks, with 2 weekly liver contrast ultrasound for assessment of differential blood supply (CEHPI) * At 8 weeks, after tumour CT RECIST assessments, responders will continue monotherapy. In non-responders, those with disease progression, monotherapy can be continued if patient chooses to continue and if tolerated	Placebo n=16 participants at risk Did not receive axitinib
General disorders Pain	3.1% 1/32 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.	0.00% 0/16 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.
Cardiac disorders Hypertension	6.2% 2/32 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.	0.00% 0/16 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.
General disorders Fatigue	6.2% 2/32 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.	0.00% 0/16 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.
General disorders Voice hoarseness	12.5% 4/32 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.	0.00% 0/16 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.
General disorders Anorexia	6.2% 2/32 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.	0.00% 0/16 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.
Gastrointestinal disorders Constipation	3.1% 1/32 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.	0.00% 0/16 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.
General disorders Mucositis	6.2% 2/32 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.	0.00% 0/16 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.
Gastrointestinal disorders Nausea	3.1% 1/32 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.	0.00% 0/16 • Adverse events were collected from Day 1 (first dose) until 28 days post-final dose, up to 49 months Regular list of expected side effects questionnaire and regular adverse event assessment by investigators on the study.

Other adverse events

Adverse event data not reported

Additional Information

Charlotte Wyard

Imperial College London

Phone: 02033131362

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place