Trial Outcomes & Findings for Neoadjuvant Cabozantinib in Treating Patients With Locally Advanced Kidney Cancer (NCT NCT04022343)
NCT ID: NCT04022343
Last Updated: 2026-01-05
Results Overview
Objective response rate will be evaluated using Response Evaluation Criteria in Solid Tumors 1.1 (RECIST 1.1) criteria. All tumor measurements must be recorded in centimeters. For target lesions, a complete response (CR) is defined as the disappearance of all target lesions. A partial response (PR) is defined as at least a 30% decrease in the sum of the longest diameter of target lesions, taking as reference the baseline sum longest diameter.
ACTIVE_NOT_RECRUITING
PHASE2
22 participants
At 12 weeks after cabozantinib dose
2026-01-05
Participant Flow
Participant milestones
| Measure |
Treatment (Cabozantinib)
Patients receive cabozantinib orally once daily for 12 weeks in the absence of disease progression or unacceptable toxicity. The assigned starting dose for cabozantinib is 60 mg/day. Two dose reduction levels of cabozantinib are permitted
Cabozantinib: Given PO
|
|---|---|
|
Overall Study
STARTED
|
22
|
|
Overall Study
COMPLETED
|
17
|
|
Overall Study
NOT COMPLETED
|
5
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Neoadjuvant Cabozantinib in Treating Patients With Locally Advanced Kidney Cancer
Baseline characteristics by cohort
| Measure |
Treatment (Cabozantinib)
n=17 Participants
Patients receive cabozantinib orally once daily for 12 weeks in the absence of disease progression or unacceptable toxicity. The assigned starting dose for cabozantinib is 60 mg/day. Two dose reduction levels of cabozantinib are permitted
Cabozantinib: Given PO
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=9667 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
13 Participants
n=9667 Participants
|
|
Age, Categorical
>=65 years
|
4 Participants
n=9667 Participants
|
|
Age, Continuous
|
57 years
n=9667 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=9667 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=9667 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=9667 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
17 Participants
n=9667 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9667 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=9667 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=9667 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=9667 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=9667 Participants
|
|
Race (NIH/OMB)
White
|
14 Participants
n=9667 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=9667 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9667 Participants
|
|
Region of Enrollment
United States
|
17 participants
n=9667 Participants
|
PRIMARY outcome
Timeframe: At 12 weeks after cabozantinib doseObjective response rate will be evaluated using Response Evaluation Criteria in Solid Tumors 1.1 (RECIST 1.1) criteria. All tumor measurements must be recorded in centimeters. For target lesions, a complete response (CR) is defined as the disappearance of all target lesions. A partial response (PR) is defined as at least a 30% decrease in the sum of the longest diameter of target lesions, taking as reference the baseline sum longest diameter.
Outcome measures
| Measure |
Treatment (cabozantinib)
n=17 Participants
Patients receive cabozantinib orally once daily for 12 weeks in the absence of disease progression or unacceptable toxicity. The assigned starting dose for cabozantinib is 60 mg/day. Two dose reduction levels of cabozantinib are permitted
Cabozantinib: Given PO
|
|---|---|
|
Objective Response Rate (ORR)
Partial Response
|
6 Participants
|
|
Objective Response Rate (ORR)
Stable Disease
|
11 Participants
|
SECONDARY outcome
Timeframe: From time of surgery to first tumor recurrence or death, assessed up to 3 yearsDisease-free survival will be defined as the interval between time of surgery and the first tumor recurrence or death. Patients will be censored at time of last follow-up. Disease-free survival will be estimated with the Kaplan-Meier method.
Outcome measures
| Measure |
Treatment (cabozantinib)
n=17 Participants
Patients receive cabozantinib orally once daily for 12 weeks in the absence of disease progression or unacceptable toxicity. The assigned starting dose for cabozantinib is 60 mg/day. Two dose reduction levels of cabozantinib are permitted
Cabozantinib: Given PO
|
|---|---|
|
Disease-free Survival (DFS)
12 month survival rate
|
82.4 percentage of participants
Interval 54.7 to 93.9
|
|
Disease-free Survival (DFS)
24 month survival rate
|
76.5 percentage of participants
Interval 48.8 to 90.4
|
SECONDARY outcome
Timeframe: From time of surgery to death from any cause, assessed up to 3 yearsFor overall survival, death from any cause will be defined as the event. Patients will be censored at time of last follow-up. Overall survival will be estimated with the Kaplan-Meier method.
Outcome measures
| Measure |
Treatment (cabozantinib)
n=17 Participants
Patients receive cabozantinib orally once daily for 12 weeks in the absence of disease progression or unacceptable toxicity. The assigned starting dose for cabozantinib is 60 mg/day. Two dose reduction levels of cabozantinib are permitted
Cabozantinib: Given PO
|
|---|---|
|
Overall Survival (OS)
12 month survival rate
|
94.1 percentage of participants
Interval 65.0 to 99.1
|
|
Overall Survival (OS)
24 month survival rate
|
88.2 percentage of participants
Interval 60.6 to 96.9
|
SECONDARY outcome
Timeframe: Baseline and weeks 6 and 12 after treatment initiationQuality of life will be studied using the Functional Assessment of Cancer Therapy-Kidney Specific Index-19 (FKSI-19) questionnaire. The questionnaire consists of 19 statements that patients answer on a 0-4 scale ("Not at all" to "Very much"). Per the instrument's official scoring guidelines, items are then reverse scored as needed and summed to create a final score ranging from 0-76, where 76 indicates the best quality of life and 0 indicates the worst.
Outcome measures
| Measure |
Treatment (cabozantinib)
n=17 Participants
Patients receive cabozantinib orally once daily for 12 weeks in the absence of disease progression or unacceptable toxicity. The assigned starting dose for cabozantinib is 60 mg/day. Two dose reduction levels of cabozantinib are permitted
Cabozantinib: Given PO
|
|---|---|
|
Quality of Life Assessment: Functional Assessment of Cancer Therapy-Kidney Specific Index-19 (FKSI-19) Questionnaire
Baseline
|
60 score on a scale
Interval 54.6 to 65.4
|
|
Quality of Life Assessment: Functional Assessment of Cancer Therapy-Kidney Specific Index-19 (FKSI-19) Questionnaire
Week 6
|
48.6 score on a scale
Interval 43.2 to 54.1
|
|
Quality of Life Assessment: Functional Assessment of Cancer Therapy-Kidney Specific Index-19 (FKSI-19) Questionnaire
Week 12
|
48.5 score on a scale
Interval 43.4 to 53.6
|
SECONDARY outcome
Timeframe: Baseline and weeks 6 and 12 after treatment initiationQuality of life will be studied using the Functional Assessment of Cancer Therapy-Kidney Specific Index-19 (FKSI-19) questionnaire. The questionnaire consists of 19 statements such as "I have a lack of energy" and "I have pain" which are answered on a scale of 0-4, with 0 being "Not at all" and 4 being "Very much." The scale is 0-76 (0 = worst QoL, 76 = best QoL).
Outcome measures
| Measure |
Treatment (cabozantinib)
n=17 Participants
Patients receive cabozantinib orally once daily for 12 weeks in the absence of disease progression or unacceptable toxicity. The assigned starting dose for cabozantinib is 60 mg/day. Two dose reduction levels of cabozantinib are permitted
Cabozantinib: Given PO
|
|---|---|
|
Quality of Life Assessment: Functional Assessment of Cancer Therapy-Kidney Specific Index-19 (FKSI-19) Questionnaire
Baseline
|
64 score on a scale
Interval 55.0 to 67.0
|
|
Quality of Life Assessment: Functional Assessment of Cancer Therapy-Kidney Specific Index-19 (FKSI-19) Questionnaire
Week 6
|
51 score on a scale
Interval 38.0 to 55.0
|
|
Quality of Life Assessment: Functional Assessment of Cancer Therapy-Kidney Specific Index-19 (FKSI-19) Questionnaire
Week 12
|
48 score on a scale
Interval 43.0 to 57.0
|
SECONDARY outcome
Timeframe: Baseline and weeks 6 and 12 after treatment initiationFrailty assessment will be studied using the Fried Frailty score. Domains to be assessed include shrinking, weakness, exhaustion, low activity, and slow walking speed. Scored on a total scale of 0-25 (from 5 variables, each 0-5). 0 = not frail, 25 = most frail.
Outcome measures
| Measure |
Treatment (cabozantinib)
n=17 Participants
Patients receive cabozantinib orally once daily for 12 weeks in the absence of disease progression or unacceptable toxicity. The assigned starting dose for cabozantinib is 60 mg/day. Two dose reduction levels of cabozantinib are permitted
Cabozantinib: Given PO
|
|---|---|
|
Frailty Assessment
Baseline
|
1.18 score on a scale
Standard Deviation 1.33
|
|
Frailty Assessment
Week 6
|
1.82 score on a scale
Standard Deviation 1.47
|
|
Frailty Assessment
Week 12
|
2.06 score on a scale
Standard Deviation 1.39
|
SECONDARY outcome
Timeframe: Baseline and week 12 after treatment initiationSarcopenia assessment will be done by using baseline and week 12 scans via SliceOmatic version 5.0 by TomoVision program. Cross-sectional skeletal muscle area (SMA) at the L3 vertebra will be segmented to calculate the Skeletal Muscle Index (SMI = SMA cm² / height m²). Sarcopenia is defined as an SMI at or below the 25th percentile of age- and sex-specific reference values from the Fintelmann et al. (2024) Framingham Heart Study cohort.
Outcome measures
| Measure |
Treatment (cabozantinib)
n=16 Participants
Patients receive cabozantinib orally once daily for 12 weeks in the absence of disease progression or unacceptable toxicity. The assigned starting dose for cabozantinib is 60 mg/day. Two dose reduction levels of cabozantinib are permitted
Cabozantinib: Given PO
|
|---|---|
|
Sarcopenia Assessment
Participants with Sarcopenia (baseline)
|
9 Participants
|
|
Sarcopenia Assessment
Participants with Sarcopenia (week12)
|
9 Participants
|
Adverse Events
Treatment (Cabozantinib)
Serious adverse events
| Measure |
Treatment (Cabozantinib)
n=17 participants at risk
Patients receive cabozantinib orally once daily for 12 weeks in the absence of disease progression or unacceptable toxicity. The assigned starting dose for cabozantinib is 60 mg/day. Two dose reduction levels of cabozantinib are permitted
Cabozantinib: Given PO
|
|---|---|
|
Vascular disorders
Vascular disorders - Other, specify
|
5.9%
1/17 • 3 years
|
|
Vascular disorders
Thromboembolic event
|
5.9%
1/17 • 3 years
|
|
Hepatobiliary disorders
Cholecystitis
|
5.9%
1/17 • 3 years
|
|
General disorders
Death
|
17.6%
3/17 • 3 years
|
Other adverse events
| Measure |
Treatment (Cabozantinib)
n=17 participants at risk
Patients receive cabozantinib orally once daily for 12 weeks in the absence of disease progression or unacceptable toxicity. The assigned starting dose for cabozantinib is 60 mg/day. Two dose reduction levels of cabozantinib are permitted
Cabozantinib: Given PO
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify
|
5.9%
1/17 • 3 years
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
5.9%
1/17 • 3 years
|
|
Hepatobiliary disorders
Hepatobiliary disorders - Other, specify
|
5.9%
1/17 • 3 years
|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders - Other, specify
|
5.9%
1/17 • 3 years
|
|
Investigations
GGT increased
|
5.9%
1/17 • 3 years
|
|
Renal and urinary disorders
Urinary frequency
|
5.9%
1/17 • 3 years
|
|
Psychiatric disorders
Insomnia
|
11.8%
2/17 • 3 years
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
5.9%
1/17 • 3 years
|
|
Investigations
Hemoglobin increased
|
5.9%
1/17 • 3 years
|
|
Blood and lymphatic system disorders
Anemia
|
5.9%
1/17 • 3 years
|
|
Gastrointestinal disorders
Bloating
|
5.9%
1/17 • 3 years
|
|
Nervous system disorders
Dysgeusia
|
17.6%
3/17 • 3 years
|
|
Gastrointestinal disorders
Hemorrhoids
|
5.9%
1/17 • 3 years
|
|
Gastrointestinal disorders
Dry mouth
|
5.9%
1/17 • 3 years
|
|
Hepatobiliary disorders
Cholecystitis
|
5.9%
1/17 • 3 years
|
|
Hepatobiliary disorders
Gallbladder perforation
|
5.9%
1/17 • 3 years
|
|
Psychiatric disorders
Depression
|
5.9%
1/17 • 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Voice alteration
|
11.8%
2/17 • 3 years
|
|
Gastrointestinal disorders
Abdominal pain
|
11.8%
2/17 • 3 years
|
|
Vascular disorders
Hypotension
|
5.9%
1/17 • 3 years
|
|
Investigations
Electrocardiogram QT corrected interval prolonged
|
5.9%
1/17 • 3 years
|
|
Nervous system disorders
Paresthesia
|
17.6%
3/17 • 3 years
|
|
Skin and subcutaneous tissue disorders
Skin hypopigmentation
|
5.9%
1/17 • 3 years
|
|
Cardiac disorders
Cardiac disorders - Other, specify
|
11.8%
2/17 • 3 years
|
|
General disorders
Edema limbs
|
5.9%
1/17 • 3 years
|
|
Gastrointestinal disorders
Oral pain
|
5.9%
1/17 • 3 years
|
|
Investigations
Investigations - Other, specify
|
5.9%
1/17 • 3 years
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
23.5%
4/17 • 3 years
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
17.6%
3/17 • 3 years
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
5.9%
1/17 • 3 years
|
|
Vascular disorders
Thromboembolic event
|
17.6%
3/17 • 3 years
|
|
Gastrointestinal disorders
Oral dysesthesia
|
11.8%
2/17 • 3 years
|
|
Investigations
Weight loss
|
11.8%
2/17 • 3 years
|
|
Metabolism and nutrition disorders
Hyperphosphatemia
|
5.9%
1/17 • 3 years
|
|
Infections and infestations
Papulopustular rash
|
11.8%
2/17 • 3 years
|
|
Nervous system disorders
Dizziness
|
11.8%
2/17 • 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
5.9%
1/17 • 3 years
|
|
Vascular disorders
Hot flashes
|
5.9%
1/17 • 3 years
|
|
Investigations
Creatinine increased
|
11.8%
2/17 • 3 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.9%
1/17 • 3 years
|
|
Psychiatric disorders
Anxiety
|
17.6%
3/17 • 3 years
|
|
Gastrointestinal disorders
Nausea
|
52.9%
9/17 • 3 years
|
|
General disorders
Fever
|
5.9%
1/17 • 3 years
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
23.5%
4/17 • 3 years
|
|
Gastrointestinal disorders
Fecal incontinence
|
5.9%
1/17 • 3 years
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
23.5%
4/17 • 3 years
|
|
General disorders
Gait disturbance
|
5.9%
1/17 • 3 years
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
17.6%
3/17 • 3 years
|
|
Nervous system disorders
Headache
|
23.5%
4/17 • 3 years
|
|
Gastrointestinal disorders
Diarrhea
|
58.8%
10/17 • 3 years
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
41.2%
7/17 • 3 years
|
|
Psychiatric disorders
Suicidal ideation
|
5.9%
1/17 • 3 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place