MedDataX Logo

Trial Outcomes & Findings for Intranasal Bevacizumab for HHT-Related Epistaxis (NCT NCT02389959)

NCT ID: NCT02389959

Last Updated: 2021-09-01

Results Overview

ESS is a standardized and reproducible outcome measure for the control of epistaxis. It is composed of six factors that are independent predictors of self-described epistaxis severity. The range is 0 to 13. The higher the score worse is epistaxis severity.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

40 participants

Primary outcome timeframe

Baseline and month 1, month 2, month 4, month 6

Results posted on

2021-09-01

Participant Flow

Participant milestones

Participant milestones
Measure
Bevacizumab
Bevacizumab mixed by the Stanford Hospital Pharmacy to a total dose of 100mg in 4mL, and 50mg (2mL), injected into each side of the nose
Saline Control
Placebo (4mL of saline) mixed by the Stanford Hospital Pharmacy as a control.
Overall Study
STARTED
20
20
Overall Study
COMPLETED
18
19
Overall Study
NOT COMPLETED
2
1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Intranasal Bevacizumab for HHT-Related Epistaxis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Bevacizumab
n=19 Participants
Bevacizumab mixed by the Stanford Hospital Pharmacy to a total dose of 100mg in 4mL, and 50mg (2mL), injected into each side of the nose
Saline Control
n=20 Participants
Placebo (4mL of saline) mixed by the Stanford Hospital Pharmacy as a control.
Total
n=39 Participants
Total of all reporting groups
Age, Continuous
49.37 years
STANDARD_DEVIATION 15.4 • n=5 Participants
55.35 years
STANDARD_DEVIATION 11.75 • n=7 Participants
52.44 years
STANDARD_DEVIATION 13.82 • n=5 Participants
Sex: Female, Male
Female
8 Participants
n=5 Participants
10 Participants
n=7 Participants
18 Participants
n=5 Participants
Sex: Female, Male
Male
11 Participants
n=5 Participants
10 Participants
n=7 Participants
21 Participants
n=5 Participants
Race/Ethnicity, Customized
Asian
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Race/Ethnicity, Customized
Caucasian
15 Participants
n=5 Participants
18 Participants
n=7 Participants
33 Participants
n=5 Participants
Race/Ethnicity, Customized
Hispanic
3 Participants
n=5 Participants
2 Participants
n=7 Participants
5 Participants
n=5 Participants
Region of Enrollment
United States
19 Participants
n=5 Participants
20 Participants
n=7 Participants
39 Participants
n=5 Participants
Epistaxis Severity Score (ESS)
5.83 units on a scale
n=5 Participants
5.06 units on a scale
n=7 Participants
5.35 units on a scale
n=5 Participants
SF-12 Physical Component Summary (PCS) Score
33.92 units on a scale
n=5 Participants
38.32 units on a scale
n=7 Participants
36.48 units on a scale
n=5 Participants
SF-12 Mental Component Summary (MCS) Score
51.38 units on a scale
n=5 Participants
50.53 units on a scale
n=7 Participants
51.25 units on a scale
n=5 Participants

PRIMARY outcome

Timeframe: Baseline and month 1, month 2, month 4, month 6

Population: Participants with available data were included in the analysis.

ESS is a standardized and reproducible outcome measure for the control of epistaxis. It is composed of six factors that are independent predictors of self-described epistaxis severity. The range is 0 to 13. The higher the score worse is epistaxis severity.

Outcome measures

Outcome measures
Measure
Bevacizumab
n=19 Participants
Bevacizumab mixed by the Stanford Hospital Pharmacy to a total dose of 100mg in 4mL, and 50mg (2mL), injected into each side of the nose
Saline Control
n=20 Participants
Placebo (4mL of saline) mixed by the Stanford Hospital Pharmacy as a control.
Change in Epistaxis Severity Score (ESS)
Difference at 4 month
-2.00 score on a scale
Interval -3.3 to -0.71
-1.16 score on a scale
Interval -2.29 to -0.02
Change in Epistaxis Severity Score (ESS)
Difference at 1 month
-2.27 score on a scale
Interval -3.27 to -1.27
-1.16 score on a scale
Interval -2.09 to -0.24
Change in Epistaxis Severity Score (ESS)
Difference at 2 month
-2.27 score on a scale
Interval -3.47 to -1.07
-1.01 score on a scale
Interval -2.13 to 0.1
Change in Epistaxis Severity Score (ESS)
Difference at 6 month
-1.34 score on a scale
Interval -2.67 to -0.01
-0.87 score on a scale
Interval -2.05 to 0.31

SECONDARY outcome

Timeframe: Baseline and month 1, month 2, month 4, month 6

Population: Participants with available data were included in the analysis.

PCS of the SF-12 is a self-reported measure of mental health-related quality of life. PCS is calculated using the scores of twelve questions and range from 0 to 100, where a zero score indicates the lowest level of health measured by the scales and 100 indicates the highest level of health.

Outcome measures

Outcome measures
Measure
Bevacizumab
n=19 Participants
Bevacizumab mixed by the Stanford Hospital Pharmacy to a total dose of 100mg in 4mL, and 50mg (2mL), injected into each side of the nose
Saline Control
n=20 Participants
Placebo (4mL of saline) mixed by the Stanford Hospital Pharmacy as a control.
Short Form-12 (SF-12) Physical Component Summary (PCS) Score
Month 1
40.53 score on a scale
Standard Deviation 7.78
40.43 score on a scale
Standard Deviation 6.79
Short Form-12 (SF-12) Physical Component Summary (PCS) Score
Month 2
39.71 score on a scale
Standard Deviation 7.22
40.88 score on a scale
Standard Deviation 5.3
Short Form-12 (SF-12) Physical Component Summary (PCS) Score
Month 4
39.96 score on a scale
Standard Deviation 7.4
40.71 score on a scale
Standard Deviation 5.47
Short Form-12 (SF-12) Physical Component Summary (PCS) Score
Month 6
39.06 score on a scale
Standard Deviation 7.86
38.06 score on a scale
Standard Deviation 6.73

SECONDARY outcome

Timeframe: Baseline and month 1, month 2, month 4, month 6

Population: Participants with available data were included in the analysis.

MCS of the SF-12 is a self-reported measure of mental health-related quality of life. MCS is calculated using the scores of twelve questions and range from 0 to 100, where a zero score indicates the lowest level of health measured by the scales and 100 indicates the highest level of health.

Outcome measures

Outcome measures
Measure
Bevacizumab
n=19 Participants
Bevacizumab mixed by the Stanford Hospital Pharmacy to a total dose of 100mg in 4mL, and 50mg (2mL), injected into each side of the nose
Saline Control
n=20 Participants
Placebo (4mL of saline) mixed by the Stanford Hospital Pharmacy as a control.
Short Form-12 (SF-12) Mental Component Summary (MCS) Score
Mean at 1 month
51.76 score on a scale
Standard Deviation 9.91
54.22 score on a scale
Standard Deviation 6.89
Short Form-12 (SF-12) Mental Component Summary (MCS) Score
Mean at 2 month
54.48 score on a scale
Standard Deviation 7.99
50.96 score on a scale
Standard Deviation 8.68
Short Form-12 (SF-12) Mental Component Summary (MCS) Score
Mean at 4 month
51.09 score on a scale
Standard Deviation 10.61
53.61 score on a scale
Standard Deviation 7.84
Short Form-12 (SF-12) Mental Component Summary (MCS) Score
Mean at 6 month
48.39 score on a scale
Standard Deviation 12.97
55.18 score on a scale
Standard Deviation 8.03

SECONDARY outcome

Timeframe: Baseline, Month 2, Month 6

Population: Participants with available data are included in the analysis.

Evaluate the effect of bevacizumab injection on direct and indirect costs in USD associated with care and management of epistaxis as well as productivity lost after treatment. The cost of caring in USD for nasal bleeding was evaluated with two surveys, the Work Productivity and Activity Impairment Questionnaire, and the HHT Costing Data Sheet.

Outcome measures

Outcome measures
Measure
Bevacizumab
n=17 Participants
Bevacizumab mixed by the Stanford Hospital Pharmacy to a total dose of 100mg in 4mL, and 50mg (2mL), injected into each side of the nose
Saline Control
n=17 Participants
Placebo (4mL of saline) mixed by the Stanford Hospital Pharmacy as a control.
Reduction in Epistaxis-related Costs (Direct and Indirect)
Month 2
9 US Dollars
Interval 0.0 to 63.0
11 US Dollars
Interval 0.0 to 617.0
Reduction in Epistaxis-related Costs (Direct and Indirect)
Month 6
11 US Dollars
Interval 0.0 to 280.0
0 US Dollars
Interval 0.0 to 29.0
Reduction in Epistaxis-related Costs (Direct and Indirect)
Baseline
74 US Dollars
Interval 41.0 to 678.0
16 US Dollars
Interval 0.0 to 58.0

Adverse Events

Bevacizumab

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

Saline Control

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

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Peter H. Hwang, MD

Stanford University

Phone: (650) 723-5281

Results disclosure agreements

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