| ID | Type | Description | Link |
|---|---|---|---|
| NCI-2012-02987 | Registry Identifier | CTRP (Clinical Trial Reporting Program) | |
| J1002 | |||
| NA_00034732 | Other Identifier | Johns Hopkins University/Sidney Kimmel Cancer Center | |
| 8248 | Other Identifier | CTEP | |
| P30CA006973 | U.S. NIH Grant/Contract | View source |
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People who have neurofibromatosis type 2 (NF2) can have tumors that grow on the auditory nerves and cause hearing loss. These tumors are called vestibular schwannomas (VSs), or acoustic neuromas. People with NF2 can also get schwannomas in other parts of their body, as well as tumors called meningiomas and ependymomas. Because VSs can cause hearing loss, many people with NF2 will have treatment to preserve their hearing. This treatment usually involves surgery. Because surgery has risks and is not able to help everyone with VSs, other methods of treatment are being explored. One area of exploration is looking to see if there is a drug that can be taken that might prevent the VSs from growing larger and causing hearing loss or brainstem compression. This study is exploring whether a drug that is approved by the FDA and is currently used to treat other tumors might also work to treat VSs. Based on people who have taken this drug to treat VSs already, there is some reason to think that it might be helpful to certain people with NF2. People enrolled in this study will receive the drug one time every three weeks for one year by infusion. This study will follow subjects over the course of the year that the person is taking the drug and for six months after the drug is stopped. This study is recruiting people who have NF2 and are currently having symptoms of tinnitus, dizziness, and/or hearing loss from their VSs. If you have NF2 and are currently having symptoms caused by your VSs, you may be eligible to participate.
PRIMARY OBJECTIVES:
I. The primary objective of this study is to determine the activity of bevacizumab for treatment of symptomatic vestibular schwannomas (VS) defined as progressive hearing loss in patients with neurofibromatosis type 2 (NF2) based on objective hearing response.
SECONDARY OJBECTIVES:
I. Determine the safety and tolerability of bevacizumab in this patient population on an every three week dosing schedule of 7.5mg/kg for 12 months of therapy.
II. Assess the rate of radiographic response (>= 20% reduction in volume). III. Determine the growth rate of VS using volumetric MRI analysis in comparison to 1-dimensional and 2-dimensional measurements.
IV. Assess changes in function of the auditory system during bevacizumab treatment.
V. Assess the vascular permeability (Ktrans), relative cerebral blood volume/flow, mean transit time, and mean vessel diameter from perfusion-weighted MRI.
VI. Assess the change in circulating endothelial cells, circulating progenitor cells, and plasma angiogenic proteins in subjects receiving bevacizumab treatment.
VII. Observe the impact of bevacizumab on non-VS tumors in patients with NF2 via whole body MRI.
VIII. Explore hearing related QOL measures throughout treatment. IX. Explore the effect of treatment with bevacizumab on auditory function using distortion product optoacoustic emissions (DPOAE) (to be evaluated at NCI only).
OUTLINE:
Patients receive bevacizumab intravenously (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.
After completion of study treatment, patients are followed up at 3 and 6 months.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment (bevacizumab) | Experimental | Patients receive 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| bevacizumab | Biological | Given IV |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Patients With Hearing Response | 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. | Baseline to 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Serious or Life Threatening Toxicities | The number of patients with serious or life threatening toxicities (CTCAE grade 3 or above) | Up to 6 months post-treatment |
| Radiographic Response |
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Inclusion Criteria:
Patients must have a confirmed diagnosis of neurofibromatosis 2 by fulfilling National Institute of Health (NIH) criteria or Manchester criteria, or by detection of a causative mutation in the NF2 gene
The NIH criteria (82) includes presence of:
The Manchester criteria (101) includes presence of:
Patients must have measurable disease, defined as at least one VS >= 1.5 cm (on longest diameter) as measured by contrast-enhanced cranial MRI scan with fine cuts through the internal auditory canal (3 mm slices, no skip)
Life expectancy of greater than 6 months
ECOG performance status (Karnofsky >= 60% or Lansky Score >= 60)
Patients must have normal organ and marrow function as defined below:
Leukocytes >= 3,000/mcL
Absolute neutrophil count >= 1,500/mcL
Platelets >= 150,000/mcL or lower limit of institutional normal
Total bilirubin =< 2 X institutional upper limit of normal
AST(SGOT)/ALT(SGPT) =< 2.5 X institutional upper limit of normal
Patients must have recovered from acute toxicity of prior treatment to grade 1 or less unless otherwise specified
Patients must have a creatinine clearance or radioisotope GFR >= 60ml/min/1.73 m^2 or a normal serum creatinine based on age described in the table below:
Subjects must have a VS not amenable to surgery or have refused surgery due to high risk for permanent complications related to surgery (e.g. damage to lower cranial nerve function, facial palsy, risk for cerebrospinal fluid leak, etc.) as determined by a surgeon with experience in management of NF2 associated VS
Subjects must have had a discussion of all available treatment options and their risks and benefits of these options including surgery, radiation therapy, observation, other clinical trials and expressed their preference for participation in this trial in the informed consent process
The effects of bevacizumab on the developing human fetus at the recommended therapeutic dose are unknown; for this reason and because anti-angiogenic agents are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation; should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately
Ability to understand and the willingness give written informed consent or assent
Evidence of active disease, defined as progressive hearing loss (with decrease in word recognition score) related to VS (i.e., not due to prior interventions such as surgery or radiation) documented in the preceding 24 months with a word recognition score of < 90% in the target ear
Proteinuria (including albuminuria) should be screened for by either urine analysis for urine protein creatinine (UPC) ratio or by urine dipstick; if the UPC ratio is greater than or equal to 0.5 or if urine dipstick shows 2+ proteinuria, 24-hour urine protein should be obtained and the level should be < 1000 mg for patient enrollment
Exclusion Criteria:
Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier
Patients may not be receiving any other investigational agents
Patients with nervous system tumors associated with NF2 (e.g., schwannomas, meningiomas, ependymomas, or gliomas) will not be excluded from this clinical trial as long as these tumors do not require treatment with radiation, surgery, or medical treatment at the time of enrollment on trial
Patients with known hypersensitivity of Chinese hamster ovary cell products, other recombinant human antibodies, or compounds of similar chemical or biologic composition to bevacizumab
Inability to tolerate periodic MRI scans or gadolinium contrast without general anesthesia
Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, or psychiatric illness/social situations that would limit compliance with study requirements
Clinically significant cardiovascular disease, such as:
Pregnant women (positive pregnancy test) are excluded from this study because bevacizumab is an anti-angiogenic agent with the potential for teratogenic or abortifacient effects; because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with bevacizumab, breastfeeding should be discontinued if the mother is treated with bevacizumab; both fertile men and women must agree to use adequate contraceptive measures during study therapy and for at least 6 months after the completion of bevacizumab therapy; abstinence is considered an adequate contraceptive measure
HIV-positive patients or cancer survivors are eligible for this study if they fulfill all other eligibility criteria
Inability to perform volumetric measurement of target VS (e.g., due to the MRI artifact from auditory brainstem implant or due to presence of collision tumor (two or more tumors abutting each other) in the cerebellopontine angle); Note: questions about the ability to perform volumetric analysis on a baseline MRI scan should be directed to the study radiologist, Dr. Gregory Sorensen
Concurrent use of anti-coagulant drugs (not including prophylactic doses), history of coagulopathy, or evidence of bleeding diathesis or coagulopathy
Imaging (CT or MRI) evidence of newly identified hemorrhage (new within the last in the 6 months prior to enrollment), any history of symptomatic intracranial hemorrhage, or any history of spontaneous intracranial hemorrhage
Serious or non-healing wound, ulcer or bone fracture
History of abdominal fistula, gastrointestinal perforation or intra-abdominal abscess within 6 months prior to day 1
Invasive procedures defined as follows:
Prior treatment with bevacizumab or other VEGF targeting therapies
Personal history of autoimmune coagulopathy, including idiopathic thrombocytopenia purpura (ITP)
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| Name | Affiliation | Role |
|---|---|---|
| Jaishri Blakeley | Johns Hopkins University/Sidney Kimmel Cancer Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Johns Hopkins University/Sidney Kimmel Cancer Center | Baltimore | Maryland | 21287 | United States | ||
| National Cancer Institute |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 26976425 | Result | Blakeley JO, Ye X, Duda DG, Halpin CF, Bergner AL, Muzikansky A, Merker VL, Gerstner ER, Fayad LM, Ahlawat S, Jacobs MA, Jain RK, Zalewski C, Dombi E, Widemann BC, Plotkin SR. Efficacy and Biomarker Study of Bevacizumab for Hearing Loss Resulting From Neurofibromatosis Type 2-Associated Vestibular Schwannomas. J Clin Oncol. 2016 May 10;34(14):1669-75. doi: 10.1200/JCO.2015.64.3817. Epub 2016 Mar 14. |
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No individual patient data will be shared
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Treatment (Bevacizumab) | People with NF2, not eligible for surgery with progressive vestibular schwannoma |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Proportion of Patients With Hearing Response | 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. | 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. | Posted | Number | 95% Confidence Interval | Proportion with hearing response | Baseline to 12 months |
|
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
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Treatment (Bevacizumab) - All Participants | All enrolled participants received bevacizumab 7.5mg/kg IV once every 3 weeks for up to 12 months. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypertension | Vascular disorders | CTCAE (4.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Jaishri Blakeley | Johns Hopkins Comprehensive Neurofibromatosis Center | 410-614-3853 | jblakel3@jhmi.edu |
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| ID | Term |
|---|---|
| D009464 | Neuroma, Acoustic |
| D016518 | Neurofibromatosis 2 |
| ID | Term |
|---|---|
| D009442 | Neurilemmoma |
| D018358 | Neuroendocrine Tumors |
| D017599 | Neuroectodermal Tumors |
| D009373 | Neoplasms, Germ Cell and Embryonal |
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| ID | Term |
|---|---|
| D000068258 | Bevacizumab |
| ID | Term |
|---|---|
| D061067 | Antibodies, Monoclonal, Humanized |
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
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| laboratory biomarker analysis | Other | Correlative studies |
|
| quality-of-life assessment | Procedure | Ancillary studies |
|
|
The 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.
| Baseline to 6 months post-treatment |
| Median Percent Change in Target Vestibular Schwannoma Volume Using Volumetric MRI | The median percent change in the volume of the target vestibular schwannoma using volumetric MRI | Baseline to 12 months |
| Number of Participants With Changes in Function of the Auditory System | 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. | Baseline to 6 months post-treatment |
| Percent Change in Median Vascular Permeability (Ktrans) | Correlation 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. | Baseline to week 72 |
| Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Total Score | 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. | Baseline |
| Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores | 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. | Baseline |
| Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Total Score | 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. | 6 months |
| Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores | 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. | 6 months |
| Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Total Score | 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. | 12 months |
| Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores | 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. | 12 months |
| Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Total Score | 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. | 18 months |
| Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores | 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. | 18 months |
| Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ) | 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. 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. | baseline |
| Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ) | 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. | 6 months |
| Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ) | 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. | 12 months |
| Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ) | 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. | 18 months |
| Quality of Life Assessed by the Tinnitus Reaction Questionnaire (TRQ) | 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. | baseline |
| Quality of Life Assessed by the Tinnitus Reaction Questionnaire (TRQ) | 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. | 6 months |
| Quality of Life Assessed by the Tinnitus Reaction Questionnaire (TRQ) | 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. | 12 months |
| Quality of Life Assessed by the Tinnitus Reaction Questionnaire (TRQ) | 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. | 18 months |
| Rockville |
| Maryland |
| 20850 |
| United States |
| Massachusetts General Hospital | Charlestown | Massachusetts | 02129 | United States |
| Participants |
|
| Age, Continuous | Median | Full Range | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| Karnofsky performance status scale | The Karnofsky performance status (KPS) scale is a provider reported measure of functional status. Decile scores are assigned, ranging from 0 to 100. A lower score (minimum 0) represents greater functional impairment. A higher score (maximum 100) represents less functional impairment. KPS can be used measure changes in a patient's functional status. | Median | Full Range | units on a scale |
|
| word recognition score of target ear | 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 | Median | Full Range | units on a scale |
|
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.
| OG001 | 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. |
|
|
| Secondary | Incidence of Serious or Life Threatening Toxicities | The number of patients with serious or life threatening toxicities (CTCAE grade 3 or above) | Participants evaluated for serious or life threatening toxicities | Posted | Count of Participants | Participants | Up to 6 months post-treatment |
|
|
|
| Secondary | Radiographic Response | The 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. | Posted | Number | 95% Confidence Interval | participants | Baseline to 6 months post-treatment |
|
|
|
| Secondary | Median Percent Change in Target Vestibular Schwannoma Volume Using Volumetric MRI | The median percent change in the volume of the target vestibular schwannoma using volumetric MRI | Posted | Median | Full Range | percentage of change in tumor volume | Baseline to 12 months |
|
|
|
| Secondary | Number of Participants With Changes in Function of the Auditory System | 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. | Distortion product optoacoustic emissions (DPOEs) were only obtained for participants at the NCI site; thus, data from 5 participants were analyzed. | Posted | Count of Participants | Participants | Baseline to 6 months post-treatment |
|
|
|
| Secondary | Percent Change in Median Vascular Permeability (Ktrans) | Correlation 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. | Posted | Number | percent change in median Ktrans | Baseline to week 72 |
|
|
|
| Secondary | Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Total Score | 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. | Posted | Median | Full Range | units on a scale | Baseline |
|
|
|
| Secondary | Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores | 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. | Posted | Median | Full Range | T-score | Baseline |
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|
|
| Secondary | Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Total Score | 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. | 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. | Posted | Median | Full Range | units on a scale | 6 months |
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| Secondary | Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores | 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. | 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. | Posted | Median | Full Range | T-score | 6 months |
|
|
|
| Secondary | Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Total Score | 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. | 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. | Posted | Median | Full Range | units on a scale | 12 months |
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| Secondary | Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores | 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. | 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. | Posted | Median | Full Range | T-score | 12 months |
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| Secondary | Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Total Score | 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. | 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. | Posted | Median | Full Range | units on a scale | 18 months |
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| Secondary | Quality of Life Assessed Using Health Survey Short Form-36 (SF-36) - Component Scores | 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. | 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. | Posted | Median | Full Range | T-score | 18 months |
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| Secondary | Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ) | 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. 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. | Posted | Median | Full Range | units on a scale | baseline |
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| Secondary | Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ) | 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. | 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. | Posted | Median | Full Range | units on a scale | 6 months |
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| Secondary | Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ) | 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. | 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. | Posted | Median | Full Range | units on a scale | 12 months |
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| Secondary | Quality of Life as Assessed by the Speech and Spatial Qualities Questionnaire (SSQ) | 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. | 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. | Posted | Median | Full Range | units on a scale | 18 months |
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| Secondary | Quality of Life Assessed by the Tinnitus Reaction Questionnaire (TRQ) | 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. | Posted | Median | Full Range | units on a scale | baseline |
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| Secondary | Quality of Life Assessed by the Tinnitus Reaction Questionnaire (TRQ) | 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. | 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. | Posted | Median | Full Range | units on a scale | 6 months |
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| Secondary | Quality of Life Assessed by the Tinnitus Reaction Questionnaire (TRQ) | 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. | 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. | Posted | Median | Full Range | units on a scale | 12 months |
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| Secondary | Quality of Life Assessed by the Tinnitus Reaction Questionnaire (TRQ) | 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. | 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. | Posted | Median | Full Range | units on a scale | 18 months |
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| 0 |
| 14 |
| 2 |
| 14 |
| 13 |
| 14 |
| EG001 | Treatment (Bevacizumab) - Pediatric Participants | All enrolled patients < 18 years of age received bevacizumab 7.5mg/kg IV once every 3 weeks for 12 months. | 0 | 2 | 0 | 2 | 1 | 2 |
| Idiopathic thrombocytopenia purpura | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
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| ALT increased | Investigations | CTCAE (4.0) | Systematic Assessment |
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| Allergic rhinitis | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Anemia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
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| Anorexia | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
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| AST increase | Investigations | CTCAE (4.0) | Systematic Assessment |
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| Bruising | Injury, poisoning and procedural complications | CTCAE (4.0) | Systematic Assessment |
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| CPK increase | Investigations | CTCAE (4.0) | Systematic Assessment |
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| Diarrhea | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Dizziness | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
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| Dry skin | Skin and subcutaneous tissue disorders | CTCAE (4.0) | Systematic Assessment |
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| Dyspepsia | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Dyspnea | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Electrocardiogram with prolonged QTc | Investigations | CTCAE (4.0) | Systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Fatigue | General disorders | CTCAE (4.0) | Systematic Assessment |
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| Headache | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
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| Hemoglobinuria | Renal and urinary disorders | CTCAE (4.0) | Systematic Assessment |
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| Hemolysis | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
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| Hemorrhoidal hemorrhage | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Hoarseness | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Hyperglycemia | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
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| Hypermagnesemia | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
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| Hypomagnesemia | Vascular disorders | CTCAE (4.0) | Systematic Assessment |
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| Increased blood bicarbonate | Investigations | CTCAE (4.0) | Systematic Assessment |
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| Irregular menstruation | Reproductive system and breast disorders | CTCAE (4.0) | Systematic Assessment |
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| Menorrhagia | Reproductive system and breast disorders | CTCAE (4.0) | Systematic Assessment |
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| Mucositis oral | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Oral hemorrhage | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Oral pain | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Palpitations | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
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| Peripheral sensory neuropathy | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
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| Platelet count decreased | Investigations | CTCAE (4.0) | Systematic Assessment |
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| Proteinuria | Renal and urinary disorders | CTCAE (4.0) | Systematic Assessment |
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| Rectal hemorrhage | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Nasal septum perforation | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Sore throat | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Vertigo | Ear and labyrinth disorders | CTCAE (4.0) | Systematic Assessment |
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| Voice alteration | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Weight gain | Investigations | CTCAE (4.0) | Systematic Assessment |
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| Weight loss | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Wound complication | Injury, poisoning and procedural complications | CTCAE (4.0) | Systematic Assessment |
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Not provided
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009463 | Neuroma |
| D018317 | Nerve Sheath Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
| D003390 | Cranial Nerve Neoplasms |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D010524 | Peripheral Nervous System Neoplasms |
| D000160 | Vestibulocochlear Nerve Diseases |
| D012181 | Retrocochlear Diseases |
| D004427 | Ear Diseases |
| D010038 | Otorhinolaryngologic Diseases |
| D010039 | Otorhinolaryngologic Neoplasms |
| D003389 | Cranial Nerve Diseases |
| D009422 | Nervous System Diseases |
| D017253 | Neurofibromatoses |
| D009455 | Neurofibroma |
| D009386 | Neoplastic Syndromes, Hereditary |
| D020752 | Neurocutaneous Syndromes |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007162 |
| Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
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