Trial Outcomes & Findings for Serial Ultrasound in Metastatic Renal Cell Carcinoma (mRCC) (NCT NCT04508725)

NCT ID: NCT04508725

Last Updated: 2025-07-25

Results Overview

Initial objective response was defined as having either Complete Response (CR) or Partial Response (PR) per RECIST v1.1 at first on-treatment response evaluation 8-16 weeks after initiating treatment.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

22 participants

Primary outcome timeframe

12 weeks

Results posted on

2025-07-25

Participant Flow

A total of 22 participants were enrolled in the study. Twelve participants started in Arm 1 and five participants started in Arm 2. Additional two participants consecutively participated in both arms; these participants are reported in Arm 3. Three participants were enrolled but did not start treatment and are not included in any arm.

Participant milestones

Participant milestones
Measure
ARM 1: Tyrosine Kinase Inhibitor (TKI) Plus Immune Checkpoint Inhibitor (ICI)
Patients were treated with vascular endothelial growth factor 2 (VEGFR2) tyrosine kinase inhibitor (TKI) plus immune checkpoint inhibitor (ICI).
ARM 2: Non-ICI Therapy
Patients were treated with non-ICI therapy.
ARM 3: Enrolled Consecutively in Both Arms
Patients were enrolled consecutively in both arms.
Overall Study
STARTED
12
5
2
Overall Study
COMPLETED
10
5
2
Overall Study
NOT COMPLETED
2
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Serial Ultrasound in Metastatic Renal Cell Carcinoma (mRCC)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
ARM 1: Tyrosine Kinase Inhibitor (TKI) Plus Immune Checkpoint Inhibitor (ICI)
n=12 Participants
Patients are planned to be treated with vascular endothelial growth factor 2 (VEGFR2) tyrosine kinase inhibitor (TKI) plus immune checkpoint inhibitor (ICI) Doppler Ultrasound: Power Doppler measurements will be made. SIEMENS S3000 and Verasonics Vantage 256: Vantage 256 used for power Doppler ultrasound, manufactured by Verasonics.
ARM 2: Non-ICI Therapy
n=5 Participants
Patients are planned to be treated with non-ICI therapy Doppler Ultrasound: Power Doppler measurements will be made. SIEMENS S3000 and Verasonics Vantage 256: Vantage 256 used for power Doppler ultrasound, manufactured by Verasonics.
ARM 3: Enrolled Consecutively in Both Arms
n=2 Participants
Patients were enrolled consecutively in both arms.
Total
n=19 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
0 Participants
n=4 Participants
Age, Categorical
Between 18 and 65 years
9 Participants
n=5 Participants
1 Participants
n=7 Participants
2 Participants
n=5 Participants
12 Participants
n=4 Participants
Age, Categorical
>=65 years
3 Participants
n=5 Participants
4 Participants
n=7 Participants
0 Participants
n=5 Participants
7 Participants
n=4 Participants
Sex: Female, Male
Female
1 Participants
n=5 Participants
1 Participants
n=7 Participants
1 Participants
n=5 Participants
3 Participants
n=4 Participants
Sex: Female, Male
Male
11 Participants
n=5 Participants
4 Participants
n=7 Participants
1 Participants
n=5 Participants
16 Participants
n=4 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
4 Participants
n=5 Participants
1 Participants
n=7 Participants
2 Participants
n=5 Participants
7 Participants
n=4 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
8 Participants
n=5 Participants
4 Participants
n=7 Participants
0 Participants
n=5 Participants
12 Participants
n=4 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
0 Participants
n=4 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
0 Participants
n=4 Participants
Race (NIH/OMB)
Asian
1 Participants
n=5 Participants
2 Participants
n=7 Participants
0 Participants
n=5 Participants
3 Participants
n=4 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
0 Participants
n=4 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
0 Participants
n=4 Participants
Race (NIH/OMB)
White
6 Participants
n=5 Participants
3 Participants
n=7 Participants
1 Participants
n=5 Participants
10 Participants
n=4 Participants
Race (NIH/OMB)
More than one race
1 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
1 Participants
n=4 Participants
Race (NIH/OMB)
Unknown or Not Reported
4 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
5 Participants
n=4 Participants
Region of Enrollment
United States
12 participants
n=5 Participants
5 participants
n=7 Participants
2 participants
n=5 Participants
19 participants
n=4 Participants

PRIMARY outcome

Timeframe: 12 weeks

Population: Two of fourteen participants in Arm 1 were not evaluable because they were removed from the study during active participation without undergoing any response evaluation.

Initial objective response was defined as having either Complete Response (CR) or Partial Response (PR) per RECIST v1.1 at first on-treatment response evaluation 8-16 weeks after initiating treatment.

Outcome measures

Outcome measures
Measure
ARM 1: Tyrosine Kinase Inhibitor (TKI) Plus Immune Checkpoint Inhibitor (ICI)
n=12 Participants
Patients were treated with vascular endothelial growth factor 2 (VEGFR2) tyrosine kinase inhibitor (TKI) plus immune checkpoint inhibitor (ICI).
ARM 2: Non-ICI Therapy
n=5 Participants
Patients were planned to be treated with non-ICI therapy.
Initial Objective Response- First Participation
Yes (CR or PR)
5 Participants
1 Participants
Initial Objective Response- First Participation
No (SD or PD)
7 Participants
4 Participants

PRIMARY outcome

Timeframe: 12 weeks

Population: This measure reflects outcome data from the second participation of two individuals enrolled in both Arm 1 and Arm 2. For their second participation, these individuals are grouped under Arm 3. Each participant is counted once in this outcome based on the second treatment period. For one of the two participants the outcome measure was not defined for their second treatment period.

Initial objective response is defined as having either Complete Response (CR) or Partial Response (PR) per RECIST v1.1 at first on-treatment response evaluation 8-16 weeks after initiating treatment.

Outcome measures

Outcome measures
Measure
ARM 1: Tyrosine Kinase Inhibitor (TKI) Plus Immune Checkpoint Inhibitor (ICI)
n=1 Participants
Patients were treated with vascular endothelial growth factor 2 (VEGFR2) tyrosine kinase inhibitor (TKI) plus immune checkpoint inhibitor (ICI).
ARM 2: Non-ICI Therapy
Patients were planned to be treated with non-ICI therapy.
Initial Objective Response- Second Participation
Yes (CR or PR)
0 Participants
Initial Objective Response- Second Participation
No (SD or PD)
1 Participants

SECONDARY outcome

Timeframe: 8-16 weeks after the start of treatment

Population: Two of fourteen participants in Arm 1 were not evaluable because they were removed from the study during active participation without undergoing any response evaluation.

Tumor burden was assessed as the sum of all tumor diameters at baseline compared to the first on-treatment response evaluation (8-16 weeks after the start of treatment) using RECIST v1.1 criteria

Outcome measures

Outcome measures
Measure
ARM 1: Tyrosine Kinase Inhibitor (TKI) Plus Immune Checkpoint Inhibitor (ICI)
n=12 Participants
Patients were treated with vascular endothelial growth factor 2 (VEGFR2) tyrosine kinase inhibitor (TKI) plus immune checkpoint inhibitor (ICI).
ARM 2: Non-ICI Therapy
n=5 Participants
Patients were planned to be treated with non-ICI therapy.
Initial Relative Change in Tumor Burden Compared to Baseline - First Participation
-16.0 Percentage change from baseline
Standard Deviation 70.3
43.7 Percentage change from baseline
Standard Deviation 94.8

SECONDARY outcome

Timeframe: 8-16 weeks after the start of treatment

Population: This measure reflects outcome data from the second participation of two individuals enrolled in both Arm 1 and Arm 2. For their second participation, these individuals are grouped under Arm 3. Each participant is counted once in this outcome based on the second treatment period. For one of the two participants the outcome measure was not defined for their second treatment period.

Tumor burden was assessed as the sum of all tumor diameters at baseline compared to the first on-treatment response evaluation (8-16 weeks after the start of treatment) using RECIST v1.1 criteria

Outcome measures

Outcome measures
Measure
ARM 1: Tyrosine Kinase Inhibitor (TKI) Plus Immune Checkpoint Inhibitor (ICI)
n=1 Participants
Patients were treated with vascular endothelial growth factor 2 (VEGFR2) tyrosine kinase inhibitor (TKI) plus immune checkpoint inhibitor (ICI).
ARM 2: Non-ICI Therapy
Patients were planned to be treated with non-ICI therapy.
Initial Relative Change in Tumor Burden Compared to Baseline - Second Participation
-20.7 Percentage change from baseline
Standard Deviation 0

SECONDARY outcome

Timeframe: 12 weeks

Population: Two of fourteen participants in Arm 1 were not evaluable because they were removed from the study during active participation without undergoing any response evaluation.

The relative change in tumor diameter of a single lesion between treatment 'baseline' and the first on-treatment response evaluation 8-16 weeks after the start of treatment, using RECIST v1.1 for tumor diameter measurements. This was measured as percent change and reported as mean ± standard deviation.

Outcome measures

Outcome measures
Measure
ARM 1: Tyrosine Kinase Inhibitor (TKI) Plus Immune Checkpoint Inhibitor (ICI)
n=18 lesions
Patients were treated with vascular endothelial growth factor 2 (VEGFR2) tyrosine kinase inhibitor (TKI) plus immune checkpoint inhibitor (ICI).
ARM 2: Non-ICI Therapy
n=5 lesions
Patients were planned to be treated with non-ICI therapy.
Initial Per-Lesion Response Compared To Baseline - First Participation
-28.0 Percentage change from baseline
Standard Deviation 23.0
-4.6 Percentage change from baseline
Standard Deviation 19.0

SECONDARY outcome

Timeframe: 12 weeks

Population: The total number of participants analyzed for this outcome measure reflects only those who were evaluable. Two participants in Arm 1 were not evaluable because they were removed from the study during active participation without undergoing any response evaluation. One participant in Arm 3 received a treatment during the second participation, for which the outcome measure was not defined.

The relative change in tumor diameter of a single lesion between treatment 'baseline' and the first on-treatment response evaluation 8-16 weeks after the start of treatment, using RECIST v1.1 for tumor diameter measurements. This was measured as percent change and reported as mean ± standard deviation.

Outcome measures

Outcome measures
Measure
ARM 1: Tyrosine Kinase Inhibitor (TKI) Plus Immune Checkpoint Inhibitor (ICI)
n=2 lesions
Patients were treated with vascular endothelial growth factor 2 (VEGFR2) tyrosine kinase inhibitor (TKI) plus immune checkpoint inhibitor (ICI).
ARM 2: Non-ICI Therapy
Patients were planned to be treated with non-ICI therapy.
Initial Per-Lesion Response Compared To Baseline - Second Participation
-6.3 Percentage change from baseline
Standard Deviation 18.7

SECONDARY outcome

Timeframe: 12 months

Population: Three of fourteen patients in arm 1 were not evaluable.

PFS was defined as not having experienced any progressive disease (PD) per RECIST v1.1 within the first 12 months after initiating treatment (day 1 will be treatment start date).

Outcome measures

Outcome measures
Measure
ARM 1: Tyrosine Kinase Inhibitor (TKI) Plus Immune Checkpoint Inhibitor (ICI)
n=11 Participants
Patients were treated with vascular endothelial growth factor 2 (VEGFR2) tyrosine kinase inhibitor (TKI) plus immune checkpoint inhibitor (ICI).
ARM 2: Non-ICI Therapy
n=5 Participants
Patients were planned to be treated with non-ICI therapy.
12-month Progression Free Survival (PFS)- First Participation
Yes (No PD and no death experienced)
7 Participants
3 Participants
12-month Progression Free Survival (PFS)- First Participation
No (PD or death experienced)
4 Participants
2 Participants

SECONDARY outcome

Timeframe: 12 months

Population: This outcome measure reflects 12-month progression-free survival for two participants who enrolled a second time after initial treatment. These participants are grouped under Arm 3, and each is counted once based on survival status from their second participation. For one participant the outcome measure was not defined for their second participation.

PFS is defined as not having experienced any progressive disease (PD) per RECIST v1.1 within the first 12 months after initiating treatment (day 1 will be treatment start date), as a number and proportion without dispersion.

Outcome measures

Outcome measures
Measure
ARM 1: Tyrosine Kinase Inhibitor (TKI) Plus Immune Checkpoint Inhibitor (ICI)
n=1 Participants
Patients were treated with vascular endothelial growth factor 2 (VEGFR2) tyrosine kinase inhibitor (TKI) plus immune checkpoint inhibitor (ICI).
ARM 2: Non-ICI Therapy
Patients were planned to be treated with non-ICI therapy.
12-month Progression Free Survival (PFS)- Second Participation
Yes (No PD and no death experienced)
0 Participants
12-month Progression Free Survival (PFS)- Second Participation
No (PD or death experienced)
1 Participants

Adverse Events

ARM 1: Tyrosine Kinase Inhibitor (TKI) Plus Immune Checkpoint Inhibitor (ICI)

Serious events: 0 serious events
Other events: 2 other events
Deaths: 1 deaths

ARM 2: Non-ICI Therapy

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

ARM 3: Enrolled Consecutively in Both Arms

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
ARM 1: Tyrosine Kinase Inhibitor (TKI) Plus Immune Checkpoint Inhibitor (ICI)
n=12 participants at risk
Patients were treated with vascular endothelial growth factor 2 (VEGFR2) tyrosine kinase inhibitor (TKI) plus immune checkpoint inhibitor (ICI).
ARM 2: Non-ICI Therapy
n=5 participants at risk
Patients were planned to be treated with non-ICI therapy
ARM 3: Enrolled Consecutively in Both Arms
n=2 participants at risk
Patients were enrolled consecutively in both arms.
Gastrointestinal disorders
Abdominal Pain
16.7%
2/12 • Number of events 2 • First study ultrasound exam through 12 weeks after starting treatment.
Only Adverse Events were recorded for this study that were definitely, probably, or possibly related to study procedures.
0.00%
0/5 • First study ultrasound exam through 12 weeks after starting treatment.
Only Adverse Events were recorded for this study that were definitely, probably, or possibly related to study procedures.
0.00%
0/2 • First study ultrasound exam through 12 weeks after starting treatment.
Only Adverse Events were recorded for this study that were definitely, probably, or possibly related to study procedures.

Additional Information

Dr. Alice Fan

Stanford University

Phone: (650) 498-6000

Results disclosure agreements

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