Trial Outcomes & Findings for T-DM1 and Palbociclib for Metastatic HER2 Breast Cancer (NCT NCT03530696)

Last Updated: 2024-08-13

Results Overview

Progression Free Survival (PFS) is defined as the time from date of first treatment to the date of investigator-determined objective disease progression as defined by Response Evaluation Criteria In Solid Tumors Criteria (RECIST) 1.1 or death from any cause. Per RECIST 1.1 for target lesions: Complete Response (CR) is the disappearance of all target lesions; Partial Response (PR) is at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters; Progressive Disease (PD) is at least a 20% increase in the sum of diameters of target lesions; Stable Disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. Patients who have neither progressed nor died will be censored at the day of their last radiographic tumor assessment (if available) or date of randomization if no post initiation (that is post baseline) radiographic assessment is available.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

55 participants

Primary outcome timeframe

Up to 4 years

Results posted on

2024-08-13

Participant Flow

Participant milestones

Participant milestones
Measure	T-DM1 With Palbociclib T-DM1 is given intravenously every 21 days (day 1 of each cycle) Palbociclib is administered orally on days 5-18 of each cycle Palbociclib: Palbociclib is to be taken orally on days 5-18 (14 days) of each cycle (each cycle length is 21 days). The starting dose will be 125mg. T-DM1: The recommended dose of T-DM1 is 3.6 mg/kg and is given as an intravenous infusion on Day 1 of every cycle (every 21 days).	Single Agent T-DM1 T-DM1 is given intravenously every 21 days (day 1 of each cycle) T-DM1: The recommended dose of T-DM1 is 3.6 mg/kg and is given as an intravenous infusion on Day 1 of every cycle (every 21 days).
Overall Study STARTED	39	16
Overall Study COMPLETED	38	14
Overall Study NOT COMPLETED	1	2

Reasons for withdrawal

Reasons for withdrawal
Measure	T-DM1 With Palbociclib T-DM1 is given intravenously every 21 days (day 1 of each cycle) Palbociclib is administered orally on days 5-18 of each cycle Palbociclib: Palbociclib is to be taken orally on days 5-18 (14 days) of each cycle (each cycle length is 21 days). The starting dose will be 125mg. T-DM1: The recommended dose of T-DM1 is 3.6 mg/kg and is given as an intravenous infusion on Day 1 of every cycle (every 21 days).	Single Agent T-DM1 T-DM1 is given intravenously every 21 days (day 1 of each cycle) T-DM1: The recommended dose of T-DM1 is 3.6 mg/kg and is given as an intravenous infusion on Day 1 of every cycle (every 21 days).
Overall Study Excluded for not meeting eligibility criteria upon further review	1	2

Baseline Characteristics

Age is missing for 1 participant in the Single Agent T-DM1 arm (n=13).

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	T-DM1 With Palbociclib n=38 Participants T-DM1 is given intravenously every 21 days (day 1 of each cycle) Palbociclib is administered orally on days 5-18 of each cycle Palbociclib: Palbociclib is to be taken orally on days 5-18 (14 days) of each cycle (each cycle length is 21 days). The starting dose will be 125mg. T-DM1: The recommended dose of T-DM1 is 3.6 mg/kg and is given as an intravenous infusion on Day 1 of every cycle (every 21 days).	Single Agent T-DM1 n=14 Participants T-DM1 is given intravenously every 21 days (day 1 of each cycle) T-DM1: The recommended dose of T-DM1 is 3.6 mg/kg and is given as an intravenous infusion on Day 1 of every cycle (every 21 days).	Total n=52 Participants Total of all reporting groups
Age, Continuous	56.5 Years STANDARD_DEVIATION 12.6 • n=38 Participants • Age is missing for 1 participant in the Single Agent T-DM1 arm (n=13).	57.8 Years STANDARD_DEVIATION 11.4 • n=13 Participants • Age is missing for 1 participant in the Single Agent T-DM1 arm (n=13).	56.8 Years STANDARD_DEVIATION 12.2 • n=51 Participants • Age is missing for 1 participant in the Single Agent T-DM1 arm (n=13).
Sex: Female, Male Female	38 Participants n=38 Participants	14 Participants n=14 Participants	52 Participants n=52 Participants
Sex: Female, Male Male	0 Participants n=38 Participants	0 Participants n=14 Participants	0 Participants n=52 Participants
Race/Ethnicity, Customized White	24 Participants n=36 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.	10 Participants n=13 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.	34 Participants n=49 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.
Race/Ethnicity, Customized Hispanic	4 Participants n=36 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.	2 Participants n=13 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.	6 Participants n=49 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.
Race/Ethnicity, Customized Black	4 Participants n=36 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.	1 Participants n=13 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.	5 Participants n=49 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.
Race/Ethnicity, Customized Asian or Pacific Islander	3 Participants n=36 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.	0 Participants n=13 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.	3 Participants n=49 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.
Race/Ethnicity, Customized American Indian or Alaskan Native	1 Participants n=36 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.	0 Participants n=13 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.	1 Participants n=49 Participants • Race/ethnicity is missing for 3 participants across both arms: n=36 for T-DM1 with palbociclib and n=13 for Single Agent T-DM1.
Region of Enrollment United States	38 participants n=38 Participants	14 participants n=14 Participants	52 participants n=52 Participants
Menopausal Status Surgical Menopause	3 Participants n=38 Participants	1 Participants n=14 Participants	4 Participants n=52 Participants
Menopausal Status Pre-Menopausal	10 Participants n=38 Participants	1 Participants n=14 Participants	11 Participants n=52 Participants
Menopausal Status Post-Menopausal	25 Participants n=38 Participants	12 Participants n=14 Participants	37 Participants n=52 Participants
Current HER2+ Breast Cancer Diagnosis Metastatic	37 Participants n=38 Participants	14 Participants n=14 Participants	51 Participants n=52 Participants
Current HER2+ Breast Cancer Diagnosis Recurrent	1 Participants n=38 Participants	0 Participants n=14 Participants	1 Participants n=52 Participants
Prior Treatment Lines in Metastatic Setting 0	13 Participants n=38 Participants	4 Participants n=14 Participants	17 Participants n=52 Participants
Prior Treatment Lines in Metastatic Setting 1	14 Participants n=38 Participants	3 Participants n=14 Participants	17 Participants n=52 Participants
Prior Treatment Lines in Metastatic Setting 2	8 Participants n=38 Participants	6 Participants n=14 Participants	14 Participants n=52 Participants
Prior Treatment Lines in Metastatic Setting 3	3 Participants n=38 Participants	1 Participants n=14 Participants	4 Participants n=52 Participants
Pertuzumab Prior to Study No	11 Participants n=38 Participants	4 Participants n=14 Participants	15 Participants n=52 Participants
Pertuzumab Prior to Study Yes	27 Participants n=38 Participants	10 Participants n=14 Participants	37 Participants n=52 Participants
Estrogen Receptor (ER) Status Negative	25 Participants n=38 Participants	9 Participants n=14 Participants	34 Participants n=52 Participants
Estrogen Receptor (ER) Status Positive	13 Participants n=38 Participants	5 Participants n=14 Participants	18 Participants n=52 Participants
Progesterone Receptor (PR) Status Negative	20 Participants n=38 Participants	5 Participants n=14 Participants	25 Participants n=52 Participants
Progesterone Receptor (PR) Status Positive	18 Participants n=38 Participants	9 Participants n=14 Participants	27 Participants n=52 Participants
Bone Metastasis No	22 Participants n=37 Participants • Bone metastasis data is missing for 1 participant in the T-DM1 with palbociclib arm (n=37).	9 Participants n=14 Participants • Bone metastasis data is missing for 1 participant in the T-DM1 with palbociclib arm (n=37).	31 Participants n=51 Participants • Bone metastasis data is missing for 1 participant in the T-DM1 with palbociclib arm (n=37).
Bone Metastasis Yes	15 Participants n=37 Participants • Bone metastasis data is missing for 1 participant in the T-DM1 with palbociclib arm (n=37).	5 Participants n=14 Participants • Bone metastasis data is missing for 1 participant in the T-DM1 with palbociclib arm (n=37).	20 Participants n=51 Participants • Bone metastasis data is missing for 1 participant in the T-DM1 with palbociclib arm (n=37).
Central Nervous System (CNS) Metastases No	7 Participants n=37 Participants • CNS metastasis data is missing for 1 participant in the T-DM1 with palbociclib arm (n=37).	0 Participants n=14 Participants • CNS metastasis data is missing for 1 participant in the T-DM1 with palbociclib arm (n=37).	7 Participants n=51 Participants • CNS metastasis data is missing for 1 participant in the T-DM1 with palbociclib arm (n=37).
Central Nervous System (CNS) Metastases Yes	30 Participants n=37 Participants • CNS metastasis data is missing for 1 participant in the T-DM1 with palbociclib arm (n=37).	14 Participants n=14 Participants • CNS metastasis data is missing for 1 participant in the T-DM1 with palbociclib arm (n=37).	44 Participants n=51 Participants • CNS metastasis data is missing for 1 participant in the T-DM1 with palbociclib arm (n=37).

PRIMARY outcome

Timeframe: Up to 4 years

Population: Number of patients included for PFS analysis: T-DM1 with Palbociclib Arm: n=36 T-DM1 Arm: n=11 5 patients lack data on response because they discontinued study participation (or withdrew consent) before the first scan. Thus, the final sample size for responses is n = 47.

Outcome measures

Outcome measures
Measure	T-DM1 With Palbociclib n=36 Participants T-DM1 is given intravenously every 21 days (day 1 of each cycle) Palbociclib is administered orally on days 5-18 of each cycle Palbociclib: Palbociclib is to be taken orally on days 5-18 (14 days) of each cycle (each cycle length is 21 days). The starting dose will be 125mg. T-DM1: The recommended dose of T-DM1 is 3.6 mg/kg and is given as an intravenous infusion on Day 1 of every cycle (every 21 days).	Single Agent T-DM1 n=11 Participants T-DM1 is given intravenously every 21 days (day 1 of each cycle) T-DM1: The recommended dose of T-DM1 is 3.6 mg/kg and is given as an intravenous infusion on Day 1 of every cycle (every 21 days).
Estimate Progression-free Survival	16.9 Months Interval 8.3 to The upper limit was not reached due to insufficient number of participants who progressed.	8.3 Months Interval 2.7 to 8.8

SECONDARY outcome

Timeframe: Up to 4 years

Population: 5 patients lack data on response because they discontinued study participation (or withdrew consent) before the first scan and 1 additional patient died before the first scan. Thus, the final sample size for responses is n = 46 (T-DM1 only: n = 11; T-DM1 + Palbociclib: n = 35).

Per RECIST 1.1 for target lesions: Complete Response (CR) is the disappearance of all target lesions; Partial Response (PR) is at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters; Progressive Disease (PD) is at least a 20% increase in the sum of diameters of target lesions; Stable Disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. Overall response rate (ORR) is defined as the proportion of patients with CR or PR per RECIST 1.1. Disease control rate (DCR) is defined as the proportion of patients with CR, PR or SD per RECIST 1.1. The data shown is the number of participants with ORR, DCR, CR, PR, PD, and SD.

Outcome measures

Outcome measures
Measure	T-DM1 With Palbociclib n=35 Participants T-DM1 is given intravenously every 21 days (day 1 of each cycle) Palbociclib is administered orally on days 5-18 of each cycle Palbociclib: Palbociclib is to be taken orally on days 5-18 (14 days) of each cycle (each cycle length is 21 days). The starting dose will be 125mg. T-DM1: The recommended dose of T-DM1 is 3.6 mg/kg and is given as an intravenous infusion on Day 1 of every cycle (every 21 days).	Single Agent T-DM1 n=11 Participants T-DM1 is given intravenously every 21 days (day 1 of each cycle) T-DM1: The recommended dose of T-DM1 is 3.6 mg/kg and is given as an intravenous infusion on Day 1 of every cycle (every 21 days).
Number of Participants With Response Overall Response Rate	15 Participants	2 Participants
Number of Participants With Response Disease Control Rate	30 Participants	7 Participants
Number of Participants With Response Complete Response	3 Participants	0 Participants
Number of Participants With Response Partial Response	12 Participants	2 Participants
Number of Participants With Response Stable Disease	15 Participants	5 Participants
Number of Participants With Response Progressive Disease	5 Participants	4 Participants

SECONDARY outcome

Timeframe: Up to 4 years

Population: We used date of study discontinuation as the censoring date for patients excluded from the survival follow-up file. There are not enough deaths recorded to calculate median OS in the T-DM1 only arm (nor the upper bound of the confidence interval for the T-DM1 + Palbociclib arm).

Estimate overall survival of T-DM1+Palbociclib treatment regimen. Overall survival (OS) is defined as the time from date of first treatment to date of death due to any cause. Patients last known to be alive are censored at their last contact date.

Outcome measures

Outcome measures
Measure	T-DM1 With Palbociclib n=38 Participants T-DM1 is given intravenously every 21 days (day 1 of each cycle) Palbociclib is administered orally on days 5-18 of each cycle Palbociclib: Palbociclib is to be taken orally on days 5-18 (14 days) of each cycle (each cycle length is 21 days). The starting dose will be 125mg. T-DM1: The recommended dose of T-DM1 is 3.6 mg/kg and is given as an intravenous infusion on Day 1 of every cycle (every 21 days).	Single Agent T-DM1 n=14 Participants T-DM1 is given intravenously every 21 days (day 1 of each cycle) T-DM1: The recommended dose of T-DM1 is 3.6 mg/kg and is given as an intravenous infusion on Day 1 of every cycle (every 21 days).
Estimate Overall Survival	35.1 Months Interval 24.9 to We used date of study discontinuation as the censoring date for patients excluded from the survival follow-up file. There are not enough deaths recorded to calculate the upper bound of the confidence interval for the T-DM1 + Palbociclib arm.	NA Months Interval 7.8 to The upper limit was not reached due to insufficient number of participants with events.

Adverse Events

T-DM1 With Palbociclib

Serious events: 8 serious events

Other events: 38 other events

Deaths: 5 deaths

Single Agent T-DM1

Serious events: 3 serious events

Other events: 14 other events

Deaths: 4 deaths

Serious adverse events

Serious adverse events
Measure	T-DM1 With Palbociclib n=38 participants at risk T-DM1 is given intravenously every 21 days (day 1 of each cycle) Palbociclib is administered orally on days 5-18 of each cycle Palbociclib: Palbociclib is to be taken orally on days 5-18 (14 days) of each cycle (each cycle length is 21 days). The starting dose will be 125mg. T-DM1: The recommended dose of T-DM1 is 3.6 mg/kg and is given as an intravenous infusion on Day 1 of every cycle (every 21 days).	Single Agent T-DM1 n=14 participants at risk T-DM1 is given intravenously every 21 days (day 1 of each cycle) T-DM1: The recommended dose of T-DM1 is 3.6 mg/kg and is given as an intravenous infusion on Day 1 of every cycle (every 21 days).
Blood and lymphatic system disorders Anaemia	2.6% 1/38 • Number of events 2 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).	0.00% 0/14 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).
Gastrointestinal disorders Appendicitis	2.6% 1/38 • Number of events 1 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).	0.00% 0/14 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).
Gastrointestinal disorders Colitis	2.6% 1/38 • Number of events 1 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).	0.00% 0/14 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).
Gastrointestinal disorders Enterocolitis	2.6% 1/38 • Number of events 1 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).	0.00% 0/14 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).
Gastrointestinal disorders Upper gastrointestinal haemorrhage	2.6% 1/38 • Number of events 1 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).	0.00% 0/14 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).
General disorders Non-cardiac chest pain	2.6% 1/38 • Number of events 1 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).	7.1% 1/14 • Number of events 1 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).
Infections and infestations Abdominal infection	0.00% 0/38 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).	7.1% 1/14 • Number of events 1 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).
Investigations Aspartate aminotransferase increased	0.00% 0/38 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).	7.1% 1/14 • Number of events 1 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).
Investigations Neutrophil count decreased	2.6% 1/38 • Number of events 1 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).	0.00% 0/14 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).
Investigations Platelet count decreased	2.6% 1/38 • Number of events 1 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).	7.1% 1/14 • Number of events 1 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).
Investigations White blood cell count decreased	2.6% 1/38 • Number of events 1 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).	0.00% 0/14 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).
Nervous system disorders Hydrocephalus	0.00% 0/38 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).	7.1% 1/14 • Number of events 1 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).
Respiratory, thoracic and mediastinal disorders Pleural effusion	2.6% 1/38 • Number of events 2 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).	0.00% 0/14 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).
Respiratory, thoracic and mediastinal disorders Respiratory failure	2.6% 1/38 • Number of events 1 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).	0.00% 0/14 • Adverse events (AEs) were assessed and collected starting from day 1 of study treatment and continued through the 30 day follow-up period after treatment discontinuation, up to 4 years. AEs were assessed using the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 for Toxicity and Adverse Event reporting. AEs meeting the below criteria were not collected: * Hospitalization for elective surgery or routine clinical procedures that are not the result of an AE (e.g., surgical insertion of central line). * Disease progression (unless it was attributable by the investigator to the study therapy).

Other adverse events

Additional Information

Dr. Pavani Chalasani

George Washington Cancer Center

Phone: (202) 741-2277

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place