Trial Outcomes & Findings for Bevacizumab for Symptomatic Vestibular Schwannoma in Neurofibromatosis Type 2 (NF2) (NCT NCT01207687)
NCT ID: NCT01207687
Last Updated: 2018-08-27
Results Overview
A hearing response was defined as increased word recognition score above the 95% critical threshold that is maintained across two sequential evaluation time points. The word recognition score (WRS) is the percentage of phonetically-balanced, monosyllabic words that a patient can accurately repeat presented at either most comfortable level or most intelligible level.The proportion of patients with hearing response in the target ear was estimated using a binomial distribution along with 95% confidence intervals.
COMPLETED
PHASE2
14 participants
Baseline to 12 months
2018-08-27
Participant Flow
Participant milestones
| Measure |
Treatment (Bevacizumab) - All Participants
Bevacizumab IV over 30-90 minutes once every 3 weeks. Courses repeat every 6 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Overall Study
STARTED
|
14
|
|
Overall Study
COMPLETED
|
12
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Treatment (Bevacizumab) - All Participants
Bevacizumab IV over 30-90 minutes once every 3 weeks. Courses repeat every 6 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
Bevacizumab for Symptomatic Vestibular Schwannoma in Neurofibromatosis Type 2 (NF2)
Baseline characteristics by cohort
| Measure |
Treatment (Bevacizumab)
n=14 Participants
People with NF2, not eligible for surgery with progressive vestibular schwannoma
|
|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
11 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
|
Age, Continuous
|
30.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
11 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
14 Participants
n=5 Participants
|
|
Karnofsky performance status scale
|
80 units on a scale
n=5 Participants
|
|
word recognition score of target ear
|
60.5 units on a scale
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to 12 monthsPopulation: All people who underwent treatment were analyzed. Two patients stopped treatment early. One due to toxicity at week 25 and one due to need for medical care not permitted while on treatment at week 49.
A hearing response was defined as increased word recognition score above the 95% critical threshold that is maintained across two sequential evaluation time points. The word recognition score (WRS) is the percentage of phonetically-balanced, monosyllabic words that a patient can accurately repeat presented at either most comfortable level or most intelligible level.The proportion of patients with hearing response in the target ear was estimated using a binomial distribution along with 95% confidence intervals.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=14 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
n=2 Participants
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Proportion of Patients With Hearing Response
|
0.36 Proportion with hearing response
Interval 0.13 to 0.65
|
0.50 Proportion with hearing response
Interval 0.01 to 0.99
|
SECONDARY outcome
Timeframe: Up to 6 months post-treatmentPopulation: Participants evaluated for serious or life threatening toxicities
The number of patients with serious or life threatening toxicities (CTCAE grade 3 or above)
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=14 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
n=2 Participants
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Incidence of Serious or Life Threatening Toxicities
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline to 6 months post-treatmentThe proportion of participants with radiographic response as measured by a \>/= 20% reduction in tumor volume from baseline on MRI imaging will be estimated using a binomial distribution.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=14 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
n=2 Participants
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Radiographic Response
|
0.43 participants
Interval 0.18 to 0.71
|
0.50 participants
Interval 0.13 to 0.99
|
SECONDARY outcome
Timeframe: Baseline to 12 monthsThe median percent change in the volume of the target vestibular schwannoma using volumetric MRI
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=14 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
n=2 Participants
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Median Percent Change in Target Vestibular Schwannoma Volume Using Volumetric MRI
|
0.5 percentage of change in tumor volume
Interval -29.0 to 41.0
|
10.8 percentage of change in tumor volume
Interval 3.4 to 18.2
|
SECONDARY outcome
Timeframe: Baseline to 6 months post-treatmentPopulation: Distortion product optoacoustic emissions (DPOEs) were only obtained for participants at the NCI site; thus, data from 5 participants were analyzed.
The primary distortion product optoacoustic emissions (DPOAE) measurement will be treated non-parametrically (present or absent across time) DPOAE's will be considered present at the frequency of F2 when the distortion product is 6dB above the noise floor. Variables will be analyzed for differences using t-tests if the effects and sample sizes warrant, but this may not be advisable given the small numbers to be accrued.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=5 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Number of Participants With Changes in Function of the Auditory System
Present
|
3 Participants
|
—
|
|
Number of Participants With Changes in Function of the Auditory System
Absent
|
2 Participants
|
—
|
SECONDARY outcome
Timeframe: Baseline to week 72Correlation assessment were planned for imaging parameters and hearing response based on the estimated changes in Ktrans: a MRI measure of vascular permeability. Only 1/14 participants had complete Ktrans data that was amenable to analysis at baseline and week 72. Hence, these statistical analyses were not possible.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=1 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Percent Change in Median Vascular Permeability (Ktrans)
|
-20 percent change in median Ktrans
|
—
|
SECONDARY outcome
Timeframe: BaselineThe SF-36 is a patient reported outcome. SF-36 (v.1) was administered and was scored according to instructions found on the RAND website. The range of scores is 0 - 100, with a higher value indicating a more favorable health state. The total score is the median of the all the individual items.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=14 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Total Score
|
77.85 units on a scale
Interval 27.36 to 98.19
|
—
|
SECONDARY outcome
Timeframe: BaselineThe SF-36 is a patient reported outcome. SF-36 (v.1) was administered and was scored according to instructions found on the RAND website. The range of scores is 0 - 100, with a higher value indicating a more favorable health state. Scores are commonly reported as a physical component summary (PCS) and mental component summary (MCS). The PCS and MCS score have been transformed to the T-score metric, which has a mean of 50 and a standard deviation of 10 for the U.S. general population.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=14 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores
Physical Component Summary (PCS)
|
50.67 T-score
Interval 22.8 to 61.05
|
—
|
|
Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores
Mental Component Summary
|
50.79 T-score
Interval 33.01 to 56.58
|
—
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: One participant was off study at this point secondary to an adverse event and did not complete the questionnaire. Hence, data from 13 of the 14 participants were available for analysis at this time point.
The SF-36 is a patient reported outcome. SF-36 (v.1) was administered and was scored according to instructions found on the RAND website. The range of scores is 1 - 100, with a higher value indicating a more favorable health state. The total score is the median of the all the individual items.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=13 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Total Score
|
80.56 units on a scale
Interval 26.25 to 95.97
|
—
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: One participant was off study at this point secondary to an adverse event and did not complete the questionnaire. Hence, data from 13 of the 14 participants were available for analysis at this time point.
The SF-36 is a patient reported outcome. SF-36 (v.1) was administered and was scored according to instructions found on the RAND website. The range of scores is 0 - 100, with a higher value indicating a more favorable health state. Scores are commonly reported as a physical component summary (PCS) and mental component summary (MCS). The PCS and MCS score have been transformed to the T-score metric, which has a mean of 50 and a standard deviation of 10 for the U.S. general population.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=13 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores
Physical Component Score (PCS)
|
52.23 T-score
Interval 22.73 to 60.45
|
—
|
|
Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores
Mental component Summary (MCS)
|
54.30 T-score
Interval 36.22 to 59.22
|
—
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: One participant was off study at this point secondary to an adverse event and did not complete the questionnaire. Hence, data from 13 of the 14 participants were available for analysis at this time point.
The SF-36 is a patient reported outcome. SF-36 (v.1) was administered and was scored according to instructions found on the RAND website. The range of scores is 0 - 100, with a higher value indicating a more favorable health state. The total score is the median of the all the individual items.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=13 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Total Score
|
75.97 units on a scale
Interval 35.42 to 95.42
|
—
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: One participant was off study at this point secondary to an adverse event and did not complete the questionnaire. Hence, data from 13 of the 14 participants were available for analysis at this time point.
The SF-36 is a patient reported outcome. SF-36 (v.1) was administered and was scored according to instructions found on the RAND website. The range of scores is 0 - 100, with a higher value indicating a more favorable health state. Scores are commonly reported as a physical component summary (PCS) and mental component summary (MCS). The PCS and MCS score have been transformed to the T-score metric, which has a mean of 50 and a standard deviation of 10 for the U.S. general population.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=13 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores
Physical Component Summary (PCS)
|
47.88 T-score
Interval 19.98 to 62.34
|
—
|
|
Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores
Mental Component Summary (MCS)
|
56.62 T-score
Interval 21.1 to 60.94
|
—
|
SECONDARY outcome
Timeframe: 18 monthsPopulation: One participant had withdrawn consent from the study and did not complete the questionnaire. Hence, data from 13 of the 14 participants were available for analysis at this time point.
The SF-36 is a patient reported outcome. SF-36 (v.1) was administered and was scored according to instructions found on the RAND website. The range of scores is 0 - 100, with a higher value indicating a more favorable health state. The total score is the median of the all the individual items.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=13 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Total Score
|
75.28 units on a scale
Interval 51.67 to 92.64
|
—
|
SECONDARY outcome
Timeframe: 18 monthsPopulation: One participant had withdrawn consent from the study and did not complete the questionnaire. Hence, data from 13 of the 14 participants were available for analysis at this time point.
The SF-36 is a patient reported outcome. SF-36 (v.1) was administered and was scored according to instructions found on the RAND website. The range of scores is 0 - 100, with a higher value indicating a more favorable health state. Scores are commonly reported as a physical component summary (PCS) and mental component summary (MCS). The PCS and MCS score have been transformed to the T-score metric, which has a mean of 50 and a standard deviation of 10 for the U.S. general population.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=13 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores
Physical Component Summary (PCS)
|
51.73 T-score
Interval 21.07 to 60.02
|
—
|
|
Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores
Mental Component Summary (MCS)
|
51.51 T-score
Interval 26.94 to 67.78
|
—
|
SECONDARY outcome
Timeframe: baselineThe SSQ is a 49 item patient-reported outcome with three subscales: speech understanding (14 questions), spatial location of sounds (17 questions), and the qualities of sounds (18 questions) as they appear to the patient with hearing impairment. Each response is recorded on an 11 point scale (0 - 10), with the anchor points "not at all" (= 0) and "perfectly" (=10). A higher score indicates better hearing. The median score is reported for each subscale. The minimum and maximum scores for the median of each subscale would be 0 and 10, respectively.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=14 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ)
Speech Understanding
|
3.2 units on a scale
Interval 0.9 to 6.9
|
—
|
|
Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ)
Spatial Location of Sounds
|
3.2 units on a scale
Interval 0.3 to 8.6
|
—
|
|
Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ)
Qualities of Sounds
|
4.7 units on a scale
Interval 3.1 to 8.9
|
—
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: At this time point, one participant was off study due to adverse events and did not complete the questionnaire. Three participants did not complete the questionnaire at this time point. Hence, data from 10 participants were available for analysis.
The SSQ is a 49 item patient-reported outcome with three subscales: speech understanding (14 questions), spatial location of sounds (17 questions), and the qualities of sounds (18 questions) as they appear to the patient with hearing impairment. Responses are recorded on a scale from 0 - 10, with the anchor points "not at all" (= 0) and "perfectly" (=10). A higher score indicates better hearing. The median score for each subscale is reported. The minimum and maximum scores for the median of each subscale would be 0 and 10, respectively.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=10 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ)
Speech Understanding
|
4.2 units on a scale
Interval 1.3 to 6.8
|
—
|
|
Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ)
Spatial Location of Sounds
|
4.0 units on a scale
Interval 0.9 to 7.2
|
—
|
|
Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ)
Qualities of Sounds
|
6.8 units on a scale
Interval 3.1 to 8.2
|
—
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: At this time point, one participant was off study due to an adverse event and did not complete the questionnaire. Hence, data from 13 of the 14 participants were available for analysis at this time point.
The SSQ is a 49 item patient-reported outcome with three subscales: speech understanding (14 questions), spatial location of sounds (17 questions), and the qualities of sounds (18 questions) as they appear to the patient with hearing impairment. Responses are recorded on a scale from 0 - 10, with the anchor points "not at all" (= 0) and "perfectly" (=10). A higher score indicates better hearing. The median score for each subscale is reported. The minimum and maximum scores for the median of each subscale would be 0 and 10, respectively.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=13 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ)
Speech Understanding
|
5.4 units on a scale
Interval 2.9 to 8.1
|
—
|
|
Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ)
Spatial Location of Sounds
|
4.8 units on a scale
Interval 1.6 to 9.4
|
—
|
|
Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ)
Qualities of Sounds
|
6.9 units on a scale
Interval 3.1 to 9.7
|
—
|
SECONDARY outcome
Timeframe: 18 monthsPopulation: One participant had withdrawn consent from the study and did not complete the questionnaire. Hence, data from 13 of the 14 participants were available for analysis at this time point.
The SSQ is a 49 item patient-reported outcome with three subscales: speech understanding (14 questions), spatial location of sounds (17 questions), and the qualities of sounds (18 questions) as they appear to the patient with hearing impairment. Responses are recorded on a scale from 0 - 10, with the anchor points "not at all" (= 0) and "perfectly" (=10). A higher score indicates better hearing. The median score for each subscale is reported. The minimum and maximum scores for the median of each subscale would be 0 and 10, respectively.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=13 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ)
Speech Understanding
|
4.1 units on a scale
Interval 2.1 to 7.4
|
—
|
|
Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ)
Spatial Location of Sounds
|
3.6 units on a scale
Interval 0.2 to 9.4
|
—
|
|
Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ)
Qualities of Sounds
|
6.3 units on a scale
Interval 3.2 to 9.6
|
—
|
SECONDARY outcome
Timeframe: baselineThe TRQ is a 26 item patient reported outcome. It is scored using a 5 point Likert scale (0-4). The responses are summed, resulting in a range of 0 - 104 with higher scores indicating more distress related to tinnitus.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=14 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life Assessed by the Tinnitus Reaction Questionnaire (TRQ)
|
18 units on a scale
Interval 0.0 to 81.0
|
—
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: At this time point, one participant was off study due to an adverse event and did not complete the questionnaire. One participant did not complete this questionnaire at this time point. Hence, data from 12 of the 14 participants were available for analysis at this time point.
The TRQ is a 26 item patient reported outcome. It is scored using a 5 point Likert scale (0-4). The responses are summed, resulting in a range of 0 - 104 with higher scores indicating more distress related to tinnitus.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=12 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life Assessed by the Tinnitus Reaction Questionnaire (TRQ)
|
18 units on a scale
Interval 0.0 to 43.0
|
—
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: At this time point, one participant was off study due to an adverse event and did not complete the questionnaire. Hence, data from 13 of the 14 participants were available for analysis at this time point.
The TRQ is a 26 item patient reported outcome. It is scored using a 5 point Likert scale (0-4). The responses are summed, resulting in a range of 0 - 104 with higher scores indicating more distress related to tinnitus.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=13 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life Assessed by the Tinnitus Reaction Questionnaire (TRQ)
|
10 units on a scale
Interval 0.0 to 56.0
|
—
|
SECONDARY outcome
Timeframe: 18 monthsPopulation: At this time point, one participant had withdrawn consent from the study and did not complete the questionnaire. Hence, data from 13 of the 14 participants were available for analysis at this time point.
The TRQ is a 26 item patient reported outcome. It is scored using a 5 point Likert scale (0-4). The responses are summed, resulting in a range of 0 - 104 with higher scores indicating more distress related to tinnitus.
Outcome measures
| Measure |
Treatment (Bevacizumab) - All Participants
n=13 Participants
Patients who met all eligibility criteria underwent baseline evaluation and then received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 48 weeks in the absence of disease progression or unacceptable toxicity. Hearing evaluation for word recognition score was evaluated every 12 weeks. Hearing response was defined as improvement beyond the 95% CI maintained across 2 timepoints.
|
Treatment (Bevacizumab) - Pediatric Participants Only
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Quality of Life Assessed by the Tinnitus Reaction Questionnaire (TRQ)
|
23 units on a scale
Interval 0.0 to 40.0
|
—
|
Adverse Events
Treatment (Bevacizumab) - All Participants
Treatment (Bevacizumab) - Pediatric Participants
Serious adverse events
| Measure |
Treatment (Bevacizumab) - All Participants
n=14 participants at risk
All enrolled participants received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 12 months.
|
Treatment (Bevacizumab) - Pediatric Participants
n=2 participants at risk
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Vascular disorders
Hypertension
|
7.1%
1/14 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Blood and lymphatic system disorders
Idiopathic thrombocytopenia purpura
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
Other adverse events
| Measure |
Treatment (Bevacizumab) - All Participants
n=14 participants at risk
All enrolled participants received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 12 months.
|
Treatment (Bevacizumab) - Pediatric Participants
n=2 participants at risk
All enrolled patients \< 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
14.3%
2/14 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Investigations
ALT increased
|
35.7%
5/14 • Number of events 8 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Blood and lymphatic system disorders
Anemia
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Metabolism and nutrition disorders
Anorexia
|
7.1%
1/14 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Investigations
AST increase
|
28.6%
4/14 • Number of events 8 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Injury, poisoning and procedural complications
Bruising
|
14.3%
2/14 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Investigations
CPK increase
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Gastrointestinal disorders
Diarrhea
|
21.4%
3/14 • Number of events 3 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Nervous system disorders
Dizziness
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
14.3%
2/14 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Gastrointestinal disorders
Dyspepsia
|
14.3%
2/14 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
14.3%
2/14 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Investigations
Electrocardiogram with prolonged QTc
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
50.0%
7/14 • Number of events 10 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
General disorders
Fatigue
|
64.3%
9/14 • Number of events 13 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Nervous system disorders
Headache
|
7.1%
1/14 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Renal and urinary disorders
Hemoglobinuria
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Blood and lymphatic system disorders
Hemolysis
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Gastrointestinal disorders
Hemorrhoidal hemorrhage
|
14.3%
2/14 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
14.3%
2/14 • Number of events 4 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
14.3%
2/14 • Number of events 3 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Vascular disorders
Hypomagnesemia
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Investigations
Increased blood bicarbonate
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Reproductive system and breast disorders
Irregular menstruation
|
28.6%
2/7 • Number of events 6 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 4 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Reproductive system and breast disorders
Menorrhagia
|
14.3%
1/7 • Number of events 3 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Gastrointestinal disorders
Mucositis oral
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Gastrointestinal disorders
Nausea
|
28.6%
4/14 • Number of events 7 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Gastrointestinal disorders
Oral hemorrhage
|
14.3%
2/14 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Gastrointestinal disorders
Oral pain
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Cardiac disorders
Palpitations
|
14.3%
2/14 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Investigations
Platelet count decreased
|
7.1%
1/14 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Renal and urinary disorders
Proteinuria
|
14.3%
2/14 • Number of events 10 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 6 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Gastrointestinal disorders
Rectal hemorrhage
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Respiratory, thoracic and mediastinal disorders
Nasal septum perforation
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
14.3%
2/14 • Number of events 4 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Ear and labyrinth disorders
Vertigo
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
50.0%
1/2 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Respiratory, thoracic and mediastinal disorders
Voice alteration
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Gastrointestinal disorders
Vomiting
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Investigations
Weight gain
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Gastrointestinal disorders
Weight loss
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
|
Injury, poisoning and procedural complications
Wound complication
|
7.1%
1/14 • Number of events 1 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
0.00%
0/2 • From first dose until 6 months off of drug or until resolution of all SAE to baseline, assessed up to 12 months
definition is that used in clinicaltrials.gov
|
Additional Information
Dr. Jaishri Blakeley
Johns Hopkins Comprehensive Neurofibromatosis Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60