Trial Outcomes & Findings for Regorafenib, With Cetuximab or Panitumumab, for the Treatment of Unresectable, Locally Advanced, or Metastatic Colorectal Cancer (NCT NCT04117945)

NCT ID: NCT04117945

Last Updated: 2024-09-27

Results Overview

The median OS and 95% confidence intervals in each arm will be reported.

• Recruitment status: ACTIVE_NOT_RECRUITING
• Study phase: PHASE2
• Target enrollment: 22 participants
• Primary outcome timeframe: 20 months
• Results posted on: 2024-09-27

Participant Flow

Participant milestones

Measure	Arm A (Regorafenib) Patients receive regorafenib PO QD on days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion. \> \> Regorafenib: Given PO	Arm B (Cetuximab, Panitumumab, Irinotecan) Patients receive cetuximab or panitumumab IV over 30-90 minutes on days 1 and 15. Patients may also receive irinotecan IV on days 1 and 15 as determined by the study doctor. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion. \> \> Cetuximab: Given IV \> \> Irinotecan: Given IV \> \> Panitumumab: Given IV
Overall Study STARTED	12	10
Overall Study Crossed Over to Other Treatment	4	8
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	12	10

Reasons for withdrawal

Measure	Arm A (Regorafenib) Patients receive regorafenib PO QD on days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion. \> \> Regorafenib: Given PO	Arm B (Cetuximab, Panitumumab, Irinotecan) Patients receive cetuximab or panitumumab IV over 30-90 minutes on days 1 and 15. Patients may also receive irinotecan IV on days 1 and 15 as determined by the study doctor. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion. \> \> Cetuximab: Given IV \> \> Irinotecan: Given IV \> \> Panitumumab: Given IV
Overall Study Death	1	0
Overall Study Withdrawal by Subject	3	1
Overall Study Disease Progression	0	2
Overall Study Ineligible	8	7

Baseline Characteristics

Regorafenib, With Cetuximab or Panitumumab, for the Treatment of Unresectable, Locally Advanced, or Metastatic Colorectal Cancer

Baseline characteristics by cohort

Measure	Arm A (Regorafenib) n=4 Participants Patients receive regorafenib PO QD on days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Regorafenib: Given PO	Arm B (Cetuximab, Panitumumab, Irinotecan) n=3 Participants Patients receive cetuximab or panitumumab IV over 30-90 minutes on days 1 and 15. Patients may also receive irinotecan IV on days 1 and 15 as determined by the study doctor. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Cetuximab: Given IV\> \> Irinotecan: Given IV\> \> Panitumumab: Given IV	Total n=7 Participants Total of all reporting groups
Age, Continuous	70.5 years STANDARD_DEVIATION 8.19 • n=5 Participants	54.0 years STANDARD_DEVIATION 19.16 • n=7 Participants	63.4 years STANDARD_DEVIATION 15.28 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants	3 Participants n=7 Participants	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants	3 Participants n=7 Participants	7 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants	3 participants n=7 Participants	7 participants n=5 Participants
BMI	22.6 kg/m^2 STANDARD_DEVIATION 0.92 • n=5 Participants	27.0 kg/m^2 STANDARD_DEVIATION 3.52 • n=7 Participants	24.5 kg/m^2 STANDARD_DEVIATION 3.20 • n=5 Participants
Primary Tumor Site Right	4 Participants n=5 Participants	3 Participants n=7 Participants	7 Participants n=5 Participants
Primary Tumor Site Left	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
ECOG Performance Status 0	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
ECOG Performance Status 1	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: 20 months

Population: Only eligible patients were included in analysis

The median OS and 95% confidence intervals in each arm will be reported.

Outcome measures

Measure	Arm A (Regorafenib) n=4 Participants Patients receive regorafenib PO QD on days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Regorafenib: Given PO	Arm B (Cetuximab, Panitumumab, Irinotecan) n=3 Participants Patients receive cetuximab or panitumumab IV over 30-90 minutes on days 1 and 15. Patients may also receive irinotecan IV on days 1 and 15 as determined by the study doctor. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Cetuximab: Given IV\> \> Irinotecan: Given IV\> \> Panitumumab: Given IV
Overall Survival (OS)	3.6 months Interval 0.7 to The upper limit of the confidence interval was not able to be estimated due to a low number of events.	13.3 months Interval 7.9 to The upper limit of the confidence interval was not able to be estimated due to a low number of events.

SECONDARY outcome

Timeframe: 11 months

Population: Only eligible patients were included in analysis

The median first PFS and 95% confidence intervals in each arm will be reported.

Outcome measures

Measure	Arm A (Regorafenib) n=4 Participants Patients receive regorafenib PO QD on days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Regorafenib: Given PO	Arm B (Cetuximab, Panitumumab, Irinotecan) n=3 Participants Patients receive cetuximab or panitumumab IV over 30-90 minutes on days 1 and 15. Patients may also receive irinotecan IV on days 1 and 15 as determined by the study doctor. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Cetuximab: Given IV\> \> Irinotecan: Given IV\> \> Panitumumab: Given IV
First Progression-free Survival (PFS)	1.2 months Interval 0.7 to The upper limit of the confidence interval was not able to be estimated due to a low number of events.	7.0 months Interval 3.9 to The upper limit of the confidence interval was not able to be estimated due to a low number of events.

SECONDARY outcome

Timeframe: 3 months

Population: Only patients that were eligible and experienced at least one progression were included in this analysis

The median second PFS and 95% confidence intervals in each arm will be reported.

Outcome measures

Measure	Arm A (Regorafenib) n=2 Participants Patients receive regorafenib PO QD on days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Regorafenib: Given PO	Arm B (Cetuximab, Panitumumab, Irinotecan) n=2 Participants Patients receive cetuximab or panitumumab IV over 30-90 minutes on days 1 and 15. Patients may also receive irinotecan IV on days 1 and 15 as determined by the study doctor. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Cetuximab: Given IV\> \> Irinotecan: Given IV\> \> Panitumumab: Given IV
Second PFS	2.3 months The limits of the confidence interval were not able to be estimated due to a low number of events.	1.8 months Interval 1.8 to The upper limit of the confidence interval was not able to be estimated due to a low number of events.

SECONDARY outcome

Timeframe: 12 months

Population: Only eligible patients were included in analysis

The median PFS while on sequential treatment and 95% confidence intervals in each arm will be reported.

Outcome measures

Measure	Arm A (Regorafenib) n=4 Participants Patients receive regorafenib PO QD on days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Regorafenib: Given PO	Arm B (Cetuximab, Panitumumab, Irinotecan) n=3 Participants Patients receive cetuximab or panitumumab IV over 30-90 minutes on days 1 and 15. Patients may also receive irinotecan IV on days 1 and 15 as determined by the study doctor. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Cetuximab: Given IV\> \> Irinotecan: Given IV\> \> Panitumumab: Given IV
Sequential Treatment PFS	3.6 months Interval 0.7 to The upper limit of the confidence interval was not able to be estimated due to a low number of events.	8.9 months Interval 5.9 to The upper limit of the confidence interval was not able to be estimated due to a low number of events.

SECONDARY outcome

Timeframe: 20 months

Population: Only eligible patients were included in analysis

Point estimates and the corresponding 95% confidence intervals for the true success proportions in each arm will be reported.

Outcome measures

Measure	Arm A (Regorafenib) n=4 Participants Patients receive regorafenib PO QD on days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Regorafenib: Given PO	Arm B (Cetuximab, Panitumumab, Irinotecan) n=3 Participants Patients receive cetuximab or panitumumab IV over 30-90 minutes on days 1 and 15. Patients may also receive irinotecan IV on days 1 and 15 as determined by the study doctor. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Cetuximab: Given IV\> \> Irinotecan: Given IV\> \> Panitumumab: Given IV
Objective Response Rate	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 20 months

Population: Only patients that started sequential treatment were included in this analysis

Point estimates and the corresponding 95% confidence intervals for the true success proportions will be reported.

Outcome measures

Measure	Arm A (Regorafenib) n=2 Participants Patients receive regorafenib PO QD on days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Regorafenib: Given PO	Arm B (Cetuximab, Panitumumab, Irinotecan) n=2 Participants Patients receive cetuximab or panitumumab IV over 30-90 minutes on days 1 and 15. Patients may also receive irinotecan IV on days 1 and 15 as determined by the study doctor. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Cetuximab: Given IV\> \> Irinotecan: Given IV\> \> Panitumumab: Given IV
Sequential Treatment Objective Response Rate	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 20 months

Population: Only eligible patients were included in analysis

The number of patients who experience a grade 3 or higher adverse event (Common Terminology Criteria for Adverse Events [CTCAE] version [v.] 5.0), regardless of attribution, will be reported by arm.

Outcome measures

Measure	Arm A (Regorafenib) n=4 Participants Patients receive regorafenib PO QD on days 1-21. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Regorafenib: Given PO	Arm B (Cetuximab, Panitumumab, Irinotecan) n=3 Participants Patients receive cetuximab or panitumumab IV over 30-90 minutes on days 1 and 15. Patients may also receive irinotecan IV on days 1 and 15 as determined by the study doctor. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. If received as initial treatment, patients who experience disease progression may switch over to the other treatment regimen, per treating physician discretion.\> \> Cetuximab: Given IV\> \> Irinotecan: Given IV\> \> Panitumumab: Given IV
Number of Patients Experiencing Adverse Events	4 Participants	2 Participants

Adverse Events

Arm A (Regorafenib)

Serious events: 6 serious events

Other events: 12 other events

Deaths: 4 deaths

Arm B (Cetuximab, Panitumumab, Irinotecan)

Serious events: 4 serious events

Other events: 9 other events

Deaths: 6 deaths

Crossover From Arm A

Serious events: 1 serious events

Other events: 4 other events

Deaths: 2 deaths

Crossover From Arm B

Serious events: 1 serious events

Other events: 7 other events

Deaths: 6 deaths

Serious adverse events

Measure	Arm A (Regorafenib) n=12 participants at risk Regorafenib: Given PO	Arm B (Cetuximab, Panitumumab, Irinotecan) n=10 participants at risk Panitumumab: Given IV	Crossover From Arm A n=4 participants at risk Regorafenib followed by anti-EGFR mAb	Crossover From Arm B n=8 participants at risk Anti-EGFR mAb followed by regorafenib
Blood and lymphatic system disorders Blood and lymph sys disorders - Oth Spec	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Blood and lymphatic system disorders Leukocytosis	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Abdominal pain	8.3% 1/12 • Number of events 1 • 20 months	10.0% 1/10 • Number of events 2 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Colonic hemorrhage	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Diarrhea	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	12.5% 1/8 • Number of events 2 • 20 months
Gastrointestinal disorders Dry mouth	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Enterovesical fistula	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Gastrointestinal disorders - Oth spec	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Nausea	8.3% 1/12 • Number of events 1 • 20 months	10.0% 1/10 • Number of events 2 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Vomiting	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
General disorders Chills	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
General disorders Death NOS	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
General disorders Disease progression	0.00% 0/12 • 20 months	0.00% 0/10 • 20 months	25.0% 1/4 • Number of events 1 • 20 months	0.00% 0/8 • 20 months
General disorders Fatigue	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
General disorders Gen disord and admin site conds-Oth spec	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Hepatobiliary disorders Hepatobiliary disorders - Other, specify	8.3% 1/12 • Number of events 1 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Infections and infestations Sepsis	8.3% 1/12 • Number of events 1 • 20 months	10.0% 1/10 • Number of events 2 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Infections and infestations Skin infection	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 2 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Injury, poisoning and procedural complications Fall	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 2 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Investigations Blood bilirubin increased	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Metabolism and nutrition disorders Anorexia	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Metabolism and nutrition disorders Hypocalcemia	0.00% 0/12 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
Metabolism and nutrition disorders Hypomagnesemia	0.00% 0/12 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
Musculoskeletal and connective tissue disorders Back pain	8.3% 1/12 • Number of events 2 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Musculoskeletal and connective tissue disorders Flank pain	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, mal, uncpec - Oth spec	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Nervous system disorders Headache	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Renal and urinary disorders Bladder perforation	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Skin and subcutaneous tissue disorders Alopecia	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Skin and subcutaneous tissue disorders Palmar-plantar erythrodysesthesia syndrm	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months

Other adverse events

Measure	Arm A (Regorafenib) n=12 participants at risk Regorafenib: Given PO	Arm B (Cetuximab, Panitumumab, Irinotecan) n=10 participants at risk Panitumumab: Given IV	Crossover From Arm A n=4 participants at risk Regorafenib followed by anti-EGFR mAb	Crossover From Arm B n=8 participants at risk Anti-EGFR mAb followed by regorafenib
Blood and lymphatic system disorders Anemia	8.3% 1/12 • Number of events 1 • 20 months	50.0% 5/10 • Number of events 13 • 20 months	75.0% 3/4 • Number of events 5 • 20 months	37.5% 3/8 • Number of events 3 • 20 months
Blood and lymphatic system disorders Blood and lymph sys disorders - Oth Spec	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	25.0% 1/4 • Number of events 1 • 20 months	0.00% 0/8 • 20 months
Blood and lymphatic system disorders Leukocytosis	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Eye disorders Dry eye	0.00% 0/12 • 20 months	0.00% 0/10 • 20 months	25.0% 1/4 • Number of events 2 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Ascites	0.00% 0/12 • 20 months	0.00% 0/10 • 20 months	25.0% 1/4 • Number of events 1 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Bloating	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Constipation	16.7% 2/12 • Number of events 2 • 20 months	20.0% 2/10 • Number of events 2 • 20 months	25.0% 1/4 • Number of events 1 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Diarrhea	41.7% 5/12 • Number of events 7 • 20 months	80.0% 8/10 • Number of events 35 • 20 months	50.0% 2/4 • Number of events 4 • 20 months	62.5% 5/8 • Number of events 11 • 20 months
Gastrointestinal disorders Enterocolitis	0.00% 0/12 • 20 months	0.00% 0/10 • 20 months	25.0% 1/4 • Number of events 1 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Flatulence	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Gastrointestinal disorders - Oth spec	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Mucositis oral	8.3% 1/12 • Number of events 2 • 20 months	20.0% 2/10 • Number of events 4 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Nausea	50.0% 6/12 • Number of events 8 • 20 months	50.0% 5/10 • Number of events 16 • 20 months	25.0% 1/4 • Number of events 1 • 20 months	37.5% 3/8 • Number of events 6 • 20 months
Gastrointestinal disorders Stomach pain	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Gastrointestinal disorders Vomiting	8.3% 1/12 • Number of events 1 • 20 months	40.0% 4/10 • Number of events 9 • 20 months	25.0% 1/4 • Number of events 1 • 20 months	25.0% 2/8 • Number of events 2 • 20 months
General disorders Fatigue	83.3% 10/12 • Number of events 16 • 20 months	80.0% 8/10 • Number of events 40 • 20 months	75.0% 3/4 • Number of events 6 • 20 months	62.5% 5/8 • Number of events 13 • 20 months
General disorders Gen disord and admin site conds-Oth spec	8.3% 1/12 • Number of events 2 • 20 months	0.00% 0/10 • 20 months	25.0% 1/4 • Number of events 1 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
General disorders Malaise	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
General disorders Pain	0.00% 0/12 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
Infections and infestations Nail infection	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Infections and infestations Paronychia	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 2 • 20 months	0.00% 0/4 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
Infections and infestations Peritoneal infection	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Infections and infestations Sepsis	0.00% 0/12 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
Infections and infestations Upper respiratory infection	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
Injury, poisoning and procedural complications Infusion related reaction	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Investigations Activated partial throm time prolonged	0.00% 0/12 • 20 months	0.00% 0/10 • 20 months	25.0% 1/4 • Number of events 1 • 20 months	0.00% 0/8 • 20 months
Investigations Alanine aminotransferase increased	41.7% 5/12 • Number of events 11 • 20 months	70.0% 7/10 • Number of events 13 • 20 months	50.0% 2/4 • Number of events 3 • 20 months	37.5% 3/8 • Number of events 4 • 20 months
Investigations Alkaline phosphatase increased	25.0% 3/12 • Number of events 4 • 20 months	60.0% 6/10 • Number of events 16 • 20 months	25.0% 1/4 • Number of events 9 • 20 months	37.5% 3/8 • Number of events 3 • 20 months
Investigations Aspartate aminotransferase increased	58.3% 7/12 • Number of events 13 • 20 months	60.0% 6/10 • Number of events 17 • 20 months	50.0% 2/4 • Number of events 3 • 20 months	50.0% 4/8 • Number of events 4 • 20 months
Investigations Blood bilirubin increased	25.0% 3/12 • Number of events 4 • 20 months	20.0% 2/10 • Number of events 3 • 20 months	0.00% 0/4 • 20 months	25.0% 2/8 • Number of events 2 • 20 months
Investigations Creatinine increased	8.3% 1/12 • Number of events 1 • 20 months	10.0% 1/10 • Number of events 3 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Investigations Investigations - Other, specify	8.3% 1/12 • Number of events 4 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Investigations Lymphocyte count decreased	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Investigations Neutrophil count decreased	0.00% 0/12 • 20 months	20.0% 2/10 • Number of events 2 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Investigations Platelet count decreased	16.7% 2/12 • Number of events 2 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Investigations Weight loss	0.00% 0/12 • 20 months	30.0% 3/10 • Number of events 9 • 20 months	25.0% 1/4 • Number of events 1 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
Metabolism and nutrition disorders Anorexia	8.3% 1/12 • Number of events 3 • 20 months	10.0% 1/10 • Number of events 3 • 20 months	0.00% 0/4 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
Metabolism and nutrition disorders Dehydration	8.3% 1/12 • Number of events 1 • 20 months	10.0% 1/10 • Number of events 3 • 20 months	0.00% 0/4 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
Metabolism and nutrition disorders Hyperglycemia	8.3% 1/12 • Number of events 2 • 20 months	20.0% 2/10 • Number of events 4 • 20 months	0.00% 0/4 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
Metabolism and nutrition disorders Hyperkalemia	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Metabolism and nutrition disorders Hypermagnesemia	8.3% 1/12 • Number of events 1 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Metabolism and nutrition disorders Hypoalbuminemia	8.3% 1/12 • Number of events 2 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Metabolism and nutrition disorders Hypocalcemia	8.3% 1/12 • Number of events 2 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	25.0% 1/4 • Number of events 1 • 20 months	0.00% 0/8 • 20 months
Metabolism and nutrition disorders Hypokalemia	8.3% 1/12 • Number of events 2 • 20 months	40.0% 4/10 • Number of events 7 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Metabolism and nutrition disorders Hypomagnesemia	0.00% 0/12 • 20 months	60.0% 6/10 • Number of events 23 • 20 months	25.0% 1/4 • Number of events 8 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
Metabolism and nutrition disorders Hyponatremia	8.3% 1/12 • Number of events 2 • 20 months	10.0% 1/10 • Number of events 2 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Musculoskeletal and connective tissue disorders Arthralgia	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Musculoskeletal and connective tissue disorders Myalgia	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Musculoskeletal and connective tissue disorders Pain in extremity	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Nervous system disorders Cognitive disturbance	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Nervous system disorders Dizziness	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Nervous system disorders Dysgeusia	8.3% 1/12 • Number of events 1 • 20 months	10.0% 1/10 • Number of events 3 • 20 months	0.00% 0/4 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
Nervous system disorders Headache	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Nervous system disorders Peripheral sensory neuropathy	16.7% 2/12 • Number of events 2 • 20 months	20.0% 2/10 • Number of events 3 • 20 months	0.00% 0/4 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
Nervous system disorders Syncope	0.00% 0/12 • 20 months	0.00% 0/10 • 20 months	25.0% 1/4 • Number of events 1 • 20 months	0.00% 0/8 • 20 months
Renal and urinary disorders Hematuria	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Renal and urinary disorders Renal and urinary disorders - Oth spec	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Renal and urinary disorders Urinary tract obstruction	16.7% 2/12 • Number of events 2 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Respiratory, thoracic and mediastinal disorders Cough	16.7% 2/12 • Number of events 2 • 20 months	10.0% 1/10 • Number of events 2 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Respiratory, thoracic and mediastinal disorders Dyspnea	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	12.5% 1/8 • Number of events 1 • 20 months
Respiratory, thoracic and mediastinal disorders Hoarseness	25.0% 3/12 • Number of events 4 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Respiratory, thoracic and mediastinal disorders Resp, thoracic, mediastinal - Oth spec	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Respiratory, thoracic and mediastinal disorders Voice alteration	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Skin and subcutaneous tissue disorders Alopecia	0.00% 0/12 • 20 months	30.0% 3/10 • Number of events 9 • 20 months	25.0% 1/4 • Number of events 1 • 20 months	25.0% 2/8 • Number of events 2 • 20 months
Skin and subcutaneous tissue disorders Dry skin	0.00% 0/12 • 20 months	20.0% 2/10 • Number of events 6 • 20 months	50.0% 2/4 • Number of events 2 • 20 months	0.00% 0/8 • 20 months
Skin and subcutaneous tissue disorders Nail changes	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 5 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Skin and subcutaneous tissue disorders Palmar-plantar erythrodysesthesia syndrm	58.3% 7/12 • Number of events 16 • 20 months	40.0% 4/10 • Number of events 12 • 20 months	50.0% 2/4 • Number of events 17 • 20 months	62.5% 5/8 • Number of events 12 • 20 months
Skin and subcutaneous tissue disorders Photosensitivity	0.00% 0/12 • 20 months	10.0% 1/10 • Number of events 1 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Skin and subcutaneous tissue disorders Pruritus	8.3% 1/12 • Number of events 1 • 20 months	20.0% 2/10 • Number of events 5 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Skin and subcutaneous tissue disorders Rash acneiform	0.00% 0/12 • 20 months	40.0% 4/10 • Number of events 4 • 20 months	50.0% 2/4 • Number of events 10 • 20 months	0.00% 0/8 • 20 months
Skin and subcutaneous tissue disorders Rash maculo-papular	25.0% 3/12 • Number of events 6 • 20 months	60.0% 6/10 • Number of events 29 • 20 months	50.0% 2/4 • Number of events 19 • 20 months	50.0% 4/8 • Number of events 8 • 20 months
Skin and subcutaneous tissue disorders Skin and subcut tissue disord - Oth spec	8.3% 1/12 • Number of events 1 • 20 months	30.0% 3/10 • Number of events 11 • 20 months	0.00% 0/4 • 20 months	25.0% 2/8 • Number of events 2 • 20 months
Vascular disorders Hot flashes	8.3% 1/12 • Number of events 1 • 20 months	0.00% 0/10 • 20 months	0.00% 0/4 • 20 months	0.00% 0/8 • 20 months
Vascular disorders Hypertension	66.7% 8/12 • Number of events 17 • 20 months	60.0% 6/10 • Number of events 21 • 20 months	50.0% 2/4 • Number of events 8 • 20 months	62.5% 5/8 • Number of events 11 • 20 months

Additional Information

Daniel H. Ahn, D.O.

Mayo Clinic

Phone: (480) 342-4800

Email: Ahn.Daniel@mayo.edu

Results disclosure agreements

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