Trial Outcomes & Findings for A Study of Vascular Endothelial Growth Factor (VEGF) Inhibition in Patients With Unilateral Upper Extremity Lymphedema Following Treatment for Cancer (NCT NCT00827372)
NCT ID: NCT00827372
Last Updated: 2023-10-16
Results Overview
The primary endpoint will be change in excess arm volume (affected arm volume minus unaffected arm volume) compared to baseline. This will be done at Cycle 2 (29 days) and Cycle 6 (174 days).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
Baseline through Cycle 6, Day 1
Results posted on
2023-10-16
Participant Flow
This study was to enroll 14-15 patients. Due to toxicity and enough patients met the definition of response , this study was halted early, leading to a smaller number of subjects analyzed. There were 10 patients at baseline, 7 patients at Cycle 2, Day 1 and 2 patients who completed the 6 cycles of planned treatment.
Participant milestones
| Measure |
Overall Study
All patients who were treated
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
8
|
Reasons for withdrawal
| Measure |
Overall Study
All patients who were treated
|
|---|---|
|
Overall Study
Adverse Event
|
5
|
|
Overall Study
Lack of Efficacy
|
3
|
Baseline Characteristics
A Study of Vascular Endothelial Growth Factor (VEGF) Inhibition in Patients With Unilateral Upper Extremity Lymphedema Following Treatment for Cancer
Baseline characteristics by cohort
| Measure |
Overall Study
n=10 Participants
All patients who were treated
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
9 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
|
Age, Continuous
|
56.3 Years
STANDARD_DEVIATION 11.92 • n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline through Cycle 6, Day 1Population: All patients with non-missing results. There were 10 patients at baseline, 7 patients at Cycle 2, Day 1, and 2 patients who completed the 6 cycles of treatment.
The primary endpoint will be change in excess arm volume (affected arm volume minus unaffected arm volume) compared to baseline. This will be done at Cycle 2 (29 days) and Cycle 6 (174 days).
Outcome measures
| Measure |
Overall Study
n=10 Participants
All patients who were treated
|
|---|---|
|
Change in Volume Ipsilateral Lymphedema in Arm
Change from Baseline to Cycle 2, Day 1
|
-161.0 mL
Standard Deviation 189.7 • Interval 0.0 to 0.0
|
|
Change in Volume Ipsilateral Lymphedema in Arm
Change from Baseline to Cycle 6, Day 1
|
-372.8 mL
Standard Deviation 233.2
|
SECONDARY outcome
Timeframe: First 24 hours after drug was administeredPopulation: All patients with non-missing results at both baseline and at 24 hours.
Interstitial fluid pressure was reported at 24 hours. This is the difference in the last-first reading, affected arm. To assess the degree of improvement in arm edema as measured by changes in interstitial fluid pressure (ECF volume using an automated device lymphometer)
Outcome measures
| Measure |
Overall Study
n=7 Participants
All patients who were treated
|
|---|---|
|
Changes in Interstitial Fluid Pressure (ECF Volume) in the Arm
|
-0.76 mm Hg
Standard Deviation 0.28
|
SECONDARY outcome
Timeframe: Baseline, and Cycle 2, Day 1Population: All patients with non-missing results. This includes 8 patients at the first baseline, 10 patients at the second baseline, and 7 patients at Cycle 2, Day 1.
Arm impedance was reported at two baseline readings and for Cycle 2, Day 1. To assess the degree of improvement in arm edema as measured by changes in arm impedance (ECF volume using an automated device lymphometer). Data reported is the ratio of the impedance in the affected versus unaffected arm
Outcome measures
| Measure |
Overall Study
n=10 Participants
All patients who were treated
|
|---|---|
|
Change in Impedance or ECF Volume in the Arm
Impedence Baseline 1
|
1.39 ratio
Standard Deviation 0.34
|
|
Change in Impedance or ECF Volume in the Arm
Impedence Baseline 2
|
1.43 ratio
Standard Deviation 0.31
|
|
Change in Impedance or ECF Volume in the Arm
Impedence Cycle 2, Day 1
|
1.46 ratio
Standard Deviation 0.25
|
SECONDARY outcome
Timeframe: End of TreatmentPopulation: All treated patients
This is the number of patients who had greater than or equal to Grade 2 Adverse Events related to treatment. This also includes the number of patients who had treatment related Grade 2 or greater Adverse Events that lasted more than 2 weeks (14 days) and excluded events of hypertension (labeled as 'special').
Outcome measures
| Measure |
Overall Study
n=10 Participants
All patients who were treated
|
|---|---|
|
Number of Patients With Trt Related Grade 2+ AEs
Patients with Trt Related Grade 2+ AEs
|
9 participants
|
|
Number of Patients With Trt Related Grade 2+ AEs
Patients with Trt Related Grade 2+ AEs (special)
|
7 participants
|
SECONDARY outcome
Timeframe: Baseline through Cycle 6, Day 1Population: All patients with non-missing results. There were 10 patients at baseline/Cycle 1, Day 1, 7 patients at Cycle 2, Day 1, and 2 patients who completed the 6 cycles of treatment.
The quality of life questionnaire (FACT-B+4 lymphedema questions) was given at various timepoints during the study. The values for the subscales are given for baseline, Cycle 1:Day 1, Cycle 2:Day 1, and Cycle 6:Day 1. Physical Well-Being (PWB; sum of 7 items, point range 0-28) Social /Family Well-Being (SWB, sum of 7-items, point range 0-28) Emotional Well-Being (EWB; sum of 6-items, point range 0-24) Functional Well-Being (FWB; sum of 7-items, point range 0-28) Additional Concerns (BCS; sum of 9-items, point range 0-36) Arm subscale (AS; sum of 5-items, point range 0-20) -- This was not collected in Cycle 1 or 2. Fact-B+4 score=Sum of PWB, SWB, EWB, FWB, BCS, AS, point range 0-164 Trial Outcome Index=Sum of PWB, FWB, BCS, point range 0-92 Fact-G score=sum of PWB, SWB, EWB, FWB, point range 0-108 Fact-B score=sum of PWB, SWB, EWB, FWB, BCS, point range 0-144 Note: The higher the score, the better the outcome
Outcome measures
| Measure |
Overall Study
n=10 Participants
All patients who were treated
|
|---|---|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 1, Day 1 Physical Well Being
|
25.3 Units on a scale
Standard Deviation 2.8
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 1, Day 1 Social Well Being
|
24.9 Units on a scale
Standard Deviation 2.1
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cylce 1, Day 1 Functional Well Being
|
22.9 Units on a scale
Standard Deviation 3.9
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cylce 1, Day 1 Emotional Well Being
|
21.2 Units on a scale
Standard Deviation 2.5
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 1, Day 1 Additional Concerns
|
25.2 Units on a scale
Standard Deviation 5.5
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 1, Day 1 FACT-B+4 Score
|
119.5 Units on a scale
Standard Deviation 11.5
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 1, Day 1 Trial Outcome Index
|
73.4 Units on a scale
Standard Deviation 9.2
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 1, Day 1 FACT G Score
|
94.3 Units on a scale
Standard Deviation 7.6
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 1, Day 1 FACT B Score
|
119.5 Units on a scale
Standard Deviation 11.5
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 2, Day 1 Physical Well Being
|
22.0 Units on a scale
Standard Deviation 3.8
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 2, Day 1 Social Well Being
|
25.8 Units on a scale
Standard Deviation 2.1
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 2, Day 1 Functional Well Being
|
21.1 Units on a scale
Standard Deviation 4.5
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 2, Day 1 Emotional Well Being
|
20.8 Units on a scale
Standard Deviation 3.4
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 2, Day 1 Additional Concerns
|
29.8 Units on a scale
Standard Deviation 3.7
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 2, Day 1 FACT-B+4 Score
|
119.6 Units on a scale
Standard Deviation 11.5
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 2, Day 1 Trial Outcome Index
|
73.0 Units on a scale
Standard Deviation 9.1
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 2, Day 1 FACT G Score
|
89.8 Units on a scale
Standard Deviation 9.2
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 2, Day 1 FACT B Score
|
119.6 Units on a scale
Standard Deviation 11.5
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 6, Day 1 Physical Well Being
|
24.5 Units on a scale
Standard Deviation 0.7
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 6, Day 1 Social Well Being
|
27.4 Units on a scale
Standard Deviation 0.8
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cylce 6, Day 1 Functional Well Being
|
25.5 Units on a scale
Standard Deviation 3.5
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cylce 6, Day 1 Emotional Well Being
|
23.0 Units on a scale
Standard Deviation 1.4
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 6, Day 1 Additional Concerns
|
30.9 Units on a scale
Standard Deviation 2.4
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 6, Day 1 FACT-B+4 Score
|
140.9 Units on a scale
Standard Deviation 20.9
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 6, Day 1 Trial Outcome Index
|
80.9 Units on a scale
Standard Deviation 5.2
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 6, Day 1 FACT G Score
|
100.4 Units on a scale
Standard Deviation 5.1
|
|
Clinical Benefit as Assessed by Quality of Life Questionnaire (FACT-B+4 Lymphedema Questions)
Cycle 6, Day 1 FACT B Score
|
131.4 Units on a scale
Standard Deviation 7.5
|
Adverse Events
Overall
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Overall
n=10 participants at risk
All patients who were treated
|
|---|---|
|
Eye disorders
VISION-BLURRED VISION
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Gastrointestinal disorders
MUCOSITIS/STOMATITIS (CLINICAL EXAM) - ORAL CAVITY
|
50.0%
5/10 • Number of events 5 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Gastrointestinal disorders
CONSTIPATION
|
20.0%
2/10 • Number of events 2 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Gastrointestinal disorders
DIARRHEA
|
60.0%
6/10 • Number of events 6 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Gastrointestinal disorders
FLATULENCE
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Gastrointestinal disorders
HEARTBURN/DYSPEPSIA
|
40.0%
4/10 • Number of events 4 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Gastrointestinal disorders
NAUSEA
|
40.0%
4/10 • Number of events 4 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Gastrointestinal disorders
PAIN - ABDOMEN NOS
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Gastrointestinal disorders
PAIN - ORAL-GUMS
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Gastrointestinal disorders
TASTE ALTERATION (DYSGEUSIA)
|
60.0%
6/10 • Number of events 6 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Gastrointestinal disorders
VOMITING
|
20.0%
2/10 • Number of events 2 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
General disorders
FATIGUE (ASTHENIA, LETHARGY, MALAISE)
|
80.0%
8/10 • Number of events 8 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
General disorders
FEVER (IN THE ABSENCE OF NEUTROPENIA, WHERE NEUTROPENIA IS DEFINED AS ANC <1.0 X 10E9/L)
|
20.0%
2/10 • Number of events 2 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
General disorders
SWEATING (DIAPHORESIS)
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Investigations
ALKALINE PHOSPHATASE
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Metabolism and nutrition disorders
ANOREXIA
|
70.0%
7/10 • Number of events 7 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Musculoskeletal and connective tissue disorders
INFECTION WITH NORMAL ANC OR GRADE 1 OR 2 NEUTROPHILS - NECK NOS
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Musculoskeletal and connective tissue disorders
INFECTION WITH NORMAL ANC OR GRADE 1 OR 2 NEUTROPHILS - SKIN (CELLULITIS)
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Musculoskeletal and connective tissue disorders
PAIN - EXTREMITY-LIMB
|
20.0%
2/10 • Number of events 2 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Musculoskeletal and connective tissue disorders
PAIN - JOINT
|
30.0%
3/10 • Number of events 3 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Musculoskeletal and connective tissue disorders
PAIN - NECK
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Musculoskeletal and connective tissue disorders
PAIN - BACK
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Nervous system disorders
NEUROPATHY: SENSORY
|
20.0%
2/10 • Number of events 2 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Nervous system disorders
PAIN - HEAD/HEADACHE
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Nervous system disorders
PAIN - NEURALGIA/PERIPHERAL NERVE
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Psychiatric disorders
INSOMNIA
|
40.0%
4/10 • Number of events 4 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Psychiatric disorders
MOOD ALTERATION - DEPRESSION
|
30.0%
3/10 • Number of events 3 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
30.0%
3/10 • Number of events 3 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNEA (SHORTNESS OF BREATH)
|
20.0%
2/10 • Number of events 2 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Respiratory, thoracic and mediastinal disorders
INFECTION WITH NORMAL ANC OR GRADE 1 OR 2 NEUTROPHILS - SINUS
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Skin and subcutaneous tissue disorders
DERMATOLOGY/SKIN - OTHER (SPECIFY, __)
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Skin and subcutaneous tissue disorders
HAIR LOSS/ALOPECIA (SCALP OR BODY)
|
70.0%
7/10 • Number of events 7 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Skin and subcutaneous tissue disorders
HYPOPIGMENTATION
|
20.0%
2/10 • Number of events 2 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Skin and subcutaneous tissue disorders
PRURITUS/ITCHING
|
30.0%
3/10 • Number of events 3 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Skin and subcutaneous tissue disorders
RASH/DESQUAMATION
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Skin and subcutaneous tissue disorders
RASH: ACNE/ACNEIFORM
|
30.0%
3/10 • Number of events 3 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Skin and subcutaneous tissue disorders
RASH: ERYTHEMA MULTIFORME (E.G., STEVENS-JOHNSON SYNDROME, TOXIC EPIDERMAL NECROLYSIS)
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Vascular disorders
HOT FLASHES/FLUSHES
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
|
Vascular disorders
HYPERTENSION
|
10.0%
1/10 • Number of events 1 • Adverse Events are reported from the beginning of study until the end of the study, up to 28 weeks.
There were no serious adverse events entered in this trial.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place