Trial Outcomes & Findings for VB-111 in Surgically Accessible Recurrent/Progressive GBM (NCT NCT04406272)

Last Updated: 2025-06-10

Results Overview

TIL Density Analysis was not - and will not be - performed on study samples, therefore no data was generated for this study objective. * Tumor infiltrating T cell (TIL) density is defined as the number of T lymphocytes per nucleated cell, and calculated by detailed sequencing of recombined T cell receptor sequences obtained from the tumor specimen gDNA. * Plan had been to use a two-sample t-test with tumor size stratification to test the difference of tumor infiltrating T cell density between the two groups (Group A vs combined Group B+C).

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

2 years

Results posted on

2025-06-10

Participant Flow

Participant milestones

Participant milestones
Measure	Group A: VB-111 Before and After Surgery VB-111 will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovering from surgery (within 28-35 days after surgery), participants will receive intravenous VB-111 every 6 weeks. Participants evidencing disease progression may initiate bevacizumab at a pre-determined dose as needed for supportive care and will continue with VB-111 infusions every 6 weeks until tumor growth is evidenced a two consecutive time points. VB11: Intravenously administered type of gene therapy that works by blocking the process of blood-vessel creation. Disrupting a cancer from growing blood vessels, might slow the growth of the cancer or shrink it. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Bevacizumab: A type of antibody, Bevacizumab is intravenously administered and works by binding to and disrupting the vascular endothelial growth factor (VEGF). VEGF is a signal protein produced by cells that stimulates the formation of blood vessels. By disrupting VEGF, Bevacizumab helps to prevent the growth and maintenance of tumor blood vessels.	Group B: Placebo Before Surgery; VB-111 After Surgery Placebo (IV solution with no medicine) will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovering from surgery (within 28-35 days after surgery), participants will receive intravenous VB-111 every 6 weeks. Participants evidencing disease progression may initiate bevacizumab at a pre-determined dose as needed for supportive care and will continue with VB-111 infusions every 6 weeks until tumor growth is evidenced a two consecutive time points. VB11: Intravenously administered type of gene therapy that works by blocking the process of blood-vessel creation. Disrupting a cancer from growing blood vessels, might slow the growth of the cancer or shrink it. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Placebo: Intravenous solution that has no therapeutic effect, used as a control in testing investigational drug. Bevacizumab: A type of antibody, Bevacizumab is intravenously administered and works by binding to and disrupting the vascular endothelial growth factor (VEGF). VEGF is a signal protein produced by cells that stimulates the formation of blood vessels. By disrupting VEGF, Bevacizumab helps to prevent the growth and maintenance of tumor blood vessels.	Group C: Placebo Before Surgery; Standard of Care After Surgery Placebo (IV solution with no medicine) will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovery from surgery, participants will receive standard of care treatment every 6 weeks until tumor growth is evidenced a two consecutive time points. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Placebo: Intravenous solution that has no therapeutic effect, used as a control in testing investigational drug.
NeoAdjuvant Treatment STARTED	6	4	5
NeoAdjuvant Treatment COMPLETED	6	4	5
NeoAdjuvant Treatment NOT COMPLETED	0	0	0
Adjuvant Therapy (Post-surgical) STARTED	5	4	5
Adjuvant Therapy (Post-surgical) COMPLETED	5	4	5
Adjuvant Therapy (Post-surgical) NOT COMPLETED	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

VB-111 in Surgically Accessible Recurrent/Progressive GBM

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Group A: VB-111 Before and After Surgery n=6 Participants VB-111 will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovering from surgery (within 28-35 days after surgery), participants will receive intravenous VB-111 every 6 weeks. Participants evidencing disease progression may initiate bevacizumab at a pre-determined dose as needed for supportive care and will continue with VB-111 infusions every 6 weeks until tumor growth is evidenced a two consecutive time points. VB11: Intravenously administered type of gene therapy that works by blocking the process of blood-vessel creation. Disrupting a cancer from growing blood vessels, might slow the growth of the cancer or shrink it. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Bevacizumab: A type of antibody, Bevacizumab is intravenously administered and works by binding to and disrupting the vascular endothelial growth factor (VEGF). VEGF is a signal protein produced by cells that stimulates the formation of blood vessels. By disrupting VEGF, Bevacizumab helps to prevent the growth and maintenance of tumor blood vessels.	Group B: Placebo Before Surgery; VB-111 After Surgery n=4 Participants Placebo (IV solution with no medicine) will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovering from surgery (within 28-35 days after surgery), participants will receive intravenous VB-111 every 6 weeks. Participants evidencing disease progression may initiate bevacizumab at a pre-determined dose as needed for supportive care and will continue with VB-111 infusions every 6 weeks until tumor growth is evidenced a two consecutive time points. VB11: Intravenously administered type of gene therapy that works by blocking the process of blood-vessel creation. Disrupting a cancer from growing blood vessels, might slow the growth of the cancer or shrink it. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Placebo: Intravenous solution that has no therapeutic effect, used as a control in testing investigational drug. Bevacizumab: A type of antibody, Bevacizumab is intravenously administered and works by binding to and disrupting the vascular endothelial growth factor (VEGF). VEGF is a signal protein produced by cells that stimulates the formation of blood vessels. By disrupting VEGF, Bevacizumab helps to prevent the growth and maintenance of tumor blood vessels.	Group C: Placebo Before Surgery; Standard of Care After Surgery n=5 Participants Placebo (IV solution with no medicine) will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovery from surgery, participants will receive standard of care treatment every 6 weeks until tumor growth is evidenced a two consecutive time points. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Placebo: Intravenous solution that has no therapeutic effect, used as a control in testing investigational drug.	Total n=15 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants	3 Participants n=7 Participants	4 Participants n=5 Participants	11 Participants n=4 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	4 Participants n=4 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	5 Participants n=4 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	10 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants	2 Participants n=7 Participants	1 Participants n=5 Participants	5 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants	8 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	2 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) White	6 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants	12 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	2 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants
Baseline Tumor Size </= 15 cc	4 Participants n=5 Participants	3 Participants n=7 Participants	2 Participants n=5 Participants	9 Participants n=4 Participants
Baseline Tumor Size > 15 cc	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants	6 Participants n=4 Participants

PRIMARY outcome

Timeframe: 2 years

Population: TIL Density Analysis was not - and will not be - performed on study samples, therefore no data was generated for this study objective. Because the trial was stopped after accrual of only 15 of 45 planned patients, it is believed that any results generated would not be of value, as there is no meaningful correlation that could be be drawn.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 2 years

AEs graded using CTCAE version 5.0 criteria. Safety will be assessed by quantifying the toxicities and grades experienced by participants who have received VB-111, including serious adverse events (SAEs) All participants who receive at least one dose of VB-111/placebo will be evaluable for toxicity from the time of their first treatment.

Outcome measures

Outcome measures
Measure	Group A: VB-111 Before and After Surgery n=6 Participants VB-111 will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovering from surgery (within 28-35 days after surgery), participants will receive intravenous VB-111 every 6 weeks. Participants evidencing disease progression may initiate bevacizumab at a pre-determined dose as needed for supportive care and will continue with VB-111 infusions every 6 weeks until tumor growth is evidenced a two consecutive time points. VB11: Intravenously administered type of gene therapy that works by blocking the process of blood-vessel creation. Disrupting a cancer from growing blood vessels, might slow the growth of the cancer or shrink it. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Bevacizumab: A type of antibody, Bevacizumab is intravenously administered and works by binding to and disrupting the vascular endothelial growth factor (VEGF). VEGF is a signal protein produced by cells that stimulates the formation of blood vessels. By disrupting VEGF, Bevacizumab helps to prevent the growth and maintenance of tumor blood vessels.	Group B: Placebo Before Surgery; VB-111 After Surgery n=4 Participants Placebo (IV solution with no medicine) will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovering from surgery (within 28-35 days after surgery), participants will receive intravenous VB-111 every 6 weeks. Participants evidencing disease progression may initiate bevacizumab at a pre-determined dose as needed for supportive care and will continue with VB-111 infusions every 6 weeks until tumor growth is evidenced a two consecutive time points. VB11: Intravenously administered type of gene therapy that works by blocking the process of blood-vessel creation. Disrupting a cancer from growing blood vessels, might slow the growth of the cancer or shrink it. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Placebo: Intravenous solution that has no therapeutic effect, used as a control in testing investigational drug. Bevacizumab: A type of antibody, Bevacizumab is intravenously administered and works by binding to and disrupting the vascular endothelial growth factor (VEGF). VEGF is a signal protein produced by cells that stimulates the formation of blood vessels. By disrupting VEGF, Bevacizumab helps to prevent the growth and maintenance of tumor blood vessels.	Group C: Placebo Before Surgery; Standard of Care After Surgery n=5 Participants Placebo (IV solution with no medicine) will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovery from surgery, participants will receive standard of care treatment every 6 weeks until tumor growth is evidenced a two consecutive time points. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Placebo: Intravenous solution that has no therapeutic effect, used as a control in testing investigational drug.
# of Participants to Experience a Reportable SAE on Study	3 Participants	1 Participants	2 Participants

SECONDARY outcome

Timeframe: 6 months

Defined as the %age of participants with progression-free survival at 6 months from surgery as defined by RANO. Recurrent/progressive GBM participants treated with VB-111 (Group A and Group B) compared to control (Group C), using RANO criteria. Progression is defined using Radiologic Assessment in Neuro-Oncology (RANO) criteria, as any of the following: * ≥ 25% increase in sum of the products of perpendicular diameters of enhancing lesions compared with the smallest tumor measurement, on stable or increasing doses of corticosteroids * Any new lesion * Significant increase in T2/FLAIR non-enhancing lesions on stable or increasing doses of corticosteroids not felt to be caused by co-morbid events * Clear clinical deterioration not attributable to other causes apart from the tumor or changes in corticosteroid dose * Clear progression of non-measurable disease * Or failure to return for evaluation due to death or deteriorating condition

Outcome measures

Outcome measures
Measure	Group A: VB-111 Before and After Surgery n=6 Participants VB-111 will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovering from surgery (within 28-35 days after surgery), participants will receive intravenous VB-111 every 6 weeks. Participants evidencing disease progression may initiate bevacizumab at a pre-determined dose as needed for supportive care and will continue with VB-111 infusions every 6 weeks until tumor growth is evidenced a two consecutive time points. VB11: Intravenously administered type of gene therapy that works by blocking the process of blood-vessel creation. Disrupting a cancer from growing blood vessels, might slow the growth of the cancer or shrink it. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Bevacizumab: A type of antibody, Bevacizumab is intravenously administered and works by binding to and disrupting the vascular endothelial growth factor (VEGF). VEGF is a signal protein produced by cells that stimulates the formation of blood vessels. By disrupting VEGF, Bevacizumab helps to prevent the growth and maintenance of tumor blood vessels.	Group B: Placebo Before Surgery; VB-111 After Surgery n=4 Participants Placebo (IV solution with no medicine) will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovering from surgery (within 28-35 days after surgery), participants will receive intravenous VB-111 every 6 weeks. Participants evidencing disease progression may initiate bevacizumab at a pre-determined dose as needed for supportive care and will continue with VB-111 infusions every 6 weeks until tumor growth is evidenced a two consecutive time points. VB11: Intravenously administered type of gene therapy that works by blocking the process of blood-vessel creation. Disrupting a cancer from growing blood vessels, might slow the growth of the cancer or shrink it. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Placebo: Intravenous solution that has no therapeutic effect, used as a control in testing investigational drug. Bevacizumab: A type of antibody, Bevacizumab is intravenously administered and works by binding to and disrupting the vascular endothelial growth factor (VEGF). VEGF is a signal protein produced by cells that stimulates the formation of blood vessels. By disrupting VEGF, Bevacizumab helps to prevent the growth and maintenance of tumor blood vessels.	Group C: Placebo Before Surgery; Standard of Care After Surgery n=5 Participants Placebo (IV solution with no medicine) will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovery from surgery, participants will receive standard of care treatment every 6 weeks until tumor growth is evidenced a two consecutive time points. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Placebo: Intravenous solution that has no therapeutic effect, used as a control in testing investigational drug.
6-month Progression-free Survival (PFS-6)	1 Participants	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Time from randomization until death from any cause; 37 months was the maximum time a participant remained in follow-up before passing away (10 pts), withdrawing consent (2 pts), being lost to follow-up (2 pts), or trial termination (1 pt).

OS, analyses will be conducted using historical comparison data which are available from a pooled analysis of Phase II experience in recurrent Grade IV gliomas who have undergone surgery either just prior to starting treatment or as part of. Kaplan-Meier (KM) curves and median estimates from the KM curves will be provided as appropriate. Participants without efficacy evaluation data or without survival data will be censored at Day 1. For evaluation of the expansion of TCR clones, a two-sample T-test with Bonferroni adjustment will be used to compare the increase number of expanded TCR clones after VB-111 in Group A+B vs Group C.

Outcome measures

Outcome measures
Measure	Group A: VB-111 Before and After Surgery n=6 Participants VB-111 will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovering from surgery (within 28-35 days after surgery), participants will receive intravenous VB-111 every 6 weeks. Participants evidencing disease progression may initiate bevacizumab at a pre-determined dose as needed for supportive care and will continue with VB-111 infusions every 6 weeks until tumor growth is evidenced a two consecutive time points. VB11: Intravenously administered type of gene therapy that works by blocking the process of blood-vessel creation. Disrupting a cancer from growing blood vessels, might slow the growth of the cancer or shrink it. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Bevacizumab: A type of antibody, Bevacizumab is intravenously administered and works by binding to and disrupting the vascular endothelial growth factor (VEGF). VEGF is a signal protein produced by cells that stimulates the formation of blood vessels. By disrupting VEGF, Bevacizumab helps to prevent the growth and maintenance of tumor blood vessels.	Group B: Placebo Before Surgery; VB-111 After Surgery n=4 Participants Placebo (IV solution with no medicine) will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovering from surgery (within 28-35 days after surgery), participants will receive intravenous VB-111 every 6 weeks. Participants evidencing disease progression may initiate bevacizumab at a pre-determined dose as needed for supportive care and will continue with VB-111 infusions every 6 weeks until tumor growth is evidenced a two consecutive time points. VB11: Intravenously administered type of gene therapy that works by blocking the process of blood-vessel creation. Disrupting a cancer from growing blood vessels, might slow the growth of the cancer or shrink it. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Placebo: Intravenous solution that has no therapeutic effect, used as a control in testing investigational drug. Bevacizumab: A type of antibody, Bevacizumab is intravenously administered and works by binding to and disrupting the vascular endothelial growth factor (VEGF). VEGF is a signal protein produced by cells that stimulates the formation of blood vessels. By disrupting VEGF, Bevacizumab helps to prevent the growth and maintenance of tumor blood vessels.	Group C: Placebo Before Surgery; Standard of Care After Surgery n=5 Participants Placebo (IV solution with no medicine) will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovery from surgery, participants will receive standard of care treatment every 6 weeks until tumor growth is evidenced a two consecutive time points. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Placebo: Intravenous solution that has no therapeutic effect, used as a control in testing investigational drug.
Overall Survival (OS) <5 months	2 Participants	1 Participants	0 Participants
Overall Survival (OS) 6-10 months	3 Participants	1 Participants	2 Participants
Overall Survival (OS) 11-15 months	0 Participants	1 Participants	1 Participants
Overall Survival (OS) 16-20 months	0 Participants	0 Participants	2 Participants
Overall Survival (OS) > 20 months	1 Participants	1 Participants	0 Participants

Adverse Events

Group A: VB-111 Before and After Surgery

Serious events: 3 serious events

Other events: 6 other events

Deaths: 3 deaths

Group B: Placebo Before Surgery; VB-111 After Surgery

Serious events: 1 serious events

Other events: 4 other events

Deaths: 2 deaths

Group C: Placebo Before Surgery; Standard of Care After Surgery

Serious events: 2 serious events

Other events: 5 other events

Deaths: 5 deaths

Serious adverse events

Serious adverse events
Measure	Group A: VB-111 Before and After Surgery n=6 participants at risk VB-111 will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovering from surgery (within 28-35 days after surgery), participants will receive intravenous VB-111 every 6 weeks. Participants evidencing disease progression may initiate bevacizumab at a pre-determined dose as needed for supportive care and will continue with VB-111 infusions every 6 weeks until tumor growth is evidenced a two consecutive time points. VB11: Intravenously administered type of gene therapy that works by blocking the process of blood-vessel creation. Disrupting a cancer from growing blood vessels, might slow the growth of the cancer or shrink it. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Bevacizumab: A type of antibody, Bevacizumab is intravenously administered and works by binding to and disrupting the vascular endothelial growth factor (VEGF). VEGF is a signal protein produced by cells that stimulates the formation of blood vessels. By disrupting VEGF, Bevacizumab helps to prevent the growth and maintenance of tumor blood vessels.	Group B: Placebo Before Surgery; VB-111 After Surgery n=4 participants at risk Placebo (IV solution with no medicine) will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovering from surgery (within 28-35 days after surgery), participants will receive intravenous VB-111 every 6 weeks. Participants evidencing disease progression may initiate bevacizumab at a pre-determined dose as needed for supportive care and will continue with VB-111 infusions every 6 weeks until tumor growth is evidenced a two consecutive time points. VB11: Intravenously administered type of gene therapy that works by blocking the process of blood-vessel creation. Disrupting a cancer from growing blood vessels, might slow the growth of the cancer or shrink it. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Placebo: Intravenous solution that has no therapeutic effect, used as a control in testing investigational drug. Bevacizumab: A type of antibody, Bevacizumab is intravenously administered and works by binding to and disrupting the vascular endothelial growth factor (VEGF). VEGF is a signal protein produced by cells that stimulates the formation of blood vessels. By disrupting VEGF, Bevacizumab helps to prevent the growth and maintenance of tumor blood vessels.	Group C: Placebo Before Surgery; Standard of Care After Surgery n=5 participants at risk Placebo (IV solution with no medicine) will be administered intravenously at a pre-determined dose at 14-28 days prior to surgery. Upon recovery from surgery, participants will receive standard of care treatment every 6 weeks until tumor growth is evidenced a two consecutive time points. Surgery: Treatment of disease or injury by cutting, abrading, suturing, or otherwise physically changing body tissues and organs. Placebo: Intravenous solution that has no therapeutic effect, used as a control in testing investigational drug.
Gastrointestinal disorders Nausea	16.7% 1/6 • Number of events 1 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	0.00% 0/4 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	0.00% 0/5 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.
Infections and infestations Memingitis	0.00% 0/6 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	0.00% 0/4 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	20.0% 1/5 • Number of events 1 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.
Injury, poisoning and procedural complications Injury, poisoning and procedural complications - Other, specify: Pseudomeningocele	0.00% 0/6 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	0.00% 0/4 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	20.0% 1/5 • Number of events 1 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.
Investigations Investigations - Other, specify: Elevated WBC in CSF	0.00% 0/6 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	0.00% 0/4 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	20.0% 1/5 • Number of events 1 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	0.00% 0/6 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	25.0% 1/4 • Number of events 1 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	0.00% 0/5 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.
Nervous system disorders Depressed level of consciousness	16.7% 1/6 • Number of events 2 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	0.00% 0/4 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	0.00% 0/5 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.
Nervous system disorders Edema cerebral	16.7% 1/6 • Number of events 1 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	0.00% 0/4 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	0.00% 0/5 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.
Nervous system disorders Hydrocephalus	16.7% 1/6 • Number of events 2 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	0.00% 0/4 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	0.00% 0/5 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.
Psychiatric disorders Confusion	0.00% 0/6 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	0.00% 0/4 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	20.0% 1/5 • Number of events 1 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.
Skin and subcutaneous tissue disorders Skin & subcutaneous tissue disorders - Other, specify: Preseptal cellulitis	0.00% 0/6 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	25.0% 1/4 • Number of events 1 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.	0.00% 0/5 • Adverse Events (AEs) monitored/assessed up to 2 years (as the maximum # of days a patient was on active study treatment was 665 days). AEs are collected from the time of screening through 30 days following end of study treatment and any AEs meeting serious criteria through 90 days following end of study treatment. An AE is any undesirable sign, symptom or medical condition that develops or worsens in severity after initiating study treatment or any protocol-specified procedure, regardless of attribution. An SAE is any AE at any dose \& regardless of causality that: results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, or is an important medical event.

Other adverse events

Additional Information

Patrick Y. Wen, MD

Dana-Farber Cancer Institute

Phone: 617-632-2166

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place