Trial Outcomes & Findings for Therapeutic Effects of Epstein-Barr Virus Immune T-Lymphocytes Derived From a Normal HLA-Compatible Or Partially-Matched Third-Party Donor in the Treatment of EBV Lymphoproliferative Disorders and EBV-Associated Malignancies (NCT NCT01498484)
NCT ID: NCT01498484
Last Updated: 2022-10-21
Results Overview
The ORR is defined as percentage of participants with best overall response of complete remission/response (CR) or partial remission/response (PR) based on investigator's assessment. For participants with clinically and/or radiologically evident EBV LPD or malignancies, CR is complete resolution of all clinical and radiologic evidence of lymphoma, confirmed by biopsy of the affected tissues when indicated, lasting for at least 3 weeks following completion of a cycle of tabelecleucel; and PR is a 50 % or greater reduction in the size of all lymphomatous lesions as determined by computed tomography (CT) or magnetic resonance imaging (MRI) measurements of tumor volume, which was maintained for at least 3 weeks following completion of a cycle of tabelecleucel. For participants without clinically and/or radiologically evident tumors with increasing levels of EBV DNA, CR is clearance of EBV without subsequent development of EBV+ LPD; and PR is at least a 10-fold decrease in EBV DNA levels.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
87 participants
Primary outcome timeframe
From Day 1 through 65.3 months after Day 1 dose
Results posted on
2022-10-21
Participant Flow
Participant milestones
| Measure |
HCT EBV+ PTLD R/R Rituximab
Participants with Epstein-Barr virus positive (EBV+) posttransplant lymphoproliferative disorders (PTLD) hematopoietic cell transplant (HCT) who were relapse/refractory (R/R) to rituximab received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
SOT EBV+ PTLD R/R Rituximab
Participants with EBV+PTLD solid organ transplant (SOT) who were R/R to rituximab or R/R to rituximab and chemotherapy received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After 3 week observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ AID-LPD
Participants with EBV+ acquired immunodeficiency (AID) lymphoproliferative disorder (LPD) received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity. Results from participants in this arm are being excluded due to the low single digit numbers of participants; these low numbers pose a risk for participant re-identification in these rare indications in this single center study.
|
EBV+ PID-LPD
Participants with EBV+ primary immunodeficiency (PID) LPD received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity. Results from participants in this arm are being excluded due to the low single digit numbers of participants; these low numbers pose a risk for participant re-identification in these rare indications in this single center study.
|
EBV+ Viremia
Participants with EBV+ viremia received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity. Results from participants in this arm are being excluded due to the low single digit numbers of participants; these low numbers pose a risk for participant re-identification in these rare indications in this single center study.
|
EBV+ Leiomyosarcoma
Participants with EBV+ leiomyosarcoma (LMS) received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity. Results from participants in this arm are being excluded due to the low single digit numbers of participants; these low numbers pose a risk for participant re-identification in these rare indications in this single center study.
|
EBV+ Lymphoma
Participants with EBV+ lymphoma received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ NPC
Participants with EBV+ nasopharyngeal carcinoma (NPC) received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ Other Solid Tumor
Participants with EBV+ other solid tumors received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity. Results from participants in this arm are being excluded due to the low single digit numbers of participants; these low numbers pose a risk for participant re-identification in these rare indications in this single center study.
|
|---|---|---|---|---|---|---|---|---|---|
|
Overall Study
STARTED
|
25
|
10
|
3
|
3
|
2
|
3
|
25
|
14
|
2
|
|
Overall Study
COMPLETED
|
11
|
3
|
1
|
0
|
1
|
2
|
3
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
14
|
7
|
2
|
3
|
1
|
1
|
22
|
14
|
2
|
Reasons for withdrawal
| Measure |
HCT EBV+ PTLD R/R Rituximab
Participants with Epstein-Barr virus positive (EBV+) posttransplant lymphoproliferative disorders (PTLD) hematopoietic cell transplant (HCT) who were relapse/refractory (R/R) to rituximab received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
SOT EBV+ PTLD R/R Rituximab
Participants with EBV+PTLD solid organ transplant (SOT) who were R/R to rituximab or R/R to rituximab and chemotherapy received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After 3 week observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ AID-LPD
Participants with EBV+ acquired immunodeficiency (AID) lymphoproliferative disorder (LPD) received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity. Results from participants in this arm are being excluded due to the low single digit numbers of participants; these low numbers pose a risk for participant re-identification in these rare indications in this single center study.
|
EBV+ PID-LPD
Participants with EBV+ primary immunodeficiency (PID) LPD received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity. Results from participants in this arm are being excluded due to the low single digit numbers of participants; these low numbers pose a risk for participant re-identification in these rare indications in this single center study.
|
EBV+ Viremia
Participants with EBV+ viremia received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity. Results from participants in this arm are being excluded due to the low single digit numbers of participants; these low numbers pose a risk for participant re-identification in these rare indications in this single center study.
|
EBV+ Leiomyosarcoma
Participants with EBV+ leiomyosarcoma (LMS) received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity. Results from participants in this arm are being excluded due to the low single digit numbers of participants; these low numbers pose a risk for participant re-identification in these rare indications in this single center study.
|
EBV+ Lymphoma
Participants with EBV+ lymphoma received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ NPC
Participants with EBV+ nasopharyngeal carcinoma (NPC) received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ Other Solid Tumor
Participants with EBV+ other solid tumors received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity. Results from participants in this arm are being excluded due to the low single digit numbers of participants; these low numbers pose a risk for participant re-identification in these rare indications in this single center study.
|
|---|---|---|---|---|---|---|---|---|---|
|
Overall Study
Death
|
6
|
3
|
1
|
1
|
0
|
0
|
10
|
1
|
0
|
|
Overall Study
Physician Decision
|
3
|
2
|
1
|
1
|
0
|
0
|
8
|
13
|
1
|
|
Overall Study
Adverse Event
|
0
|
0
|
0
|
0
|
0
|
1
|
0
|
0
|
0
|
|
Overall Study
Withdrawal by Subject
|
0
|
0
|
0
|
1
|
0
|
0
|
1
|
0
|
0
|
|
Overall Study
Other
|
5
|
2
|
0
|
0
|
1
|
0
|
3
|
0
|
1
|
Baseline Characteristics
Therapeutic Effects of Epstein-Barr Virus Immune T-Lymphocytes Derived From a Normal HLA-Compatible Or Partially-Matched Third-Party Donor in the Treatment of EBV Lymphoproliferative Disorders and EBV-Associated Malignancies
Baseline characteristics by cohort
| Measure |
HCT EBV+ PTLD R/R Rituximab
n=25 Participants
Participants with EBV+ PTLD HCT who were R/R to rituximab received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
SOT EBV+ PTLD R/R Rituximab
n=10 Participants
Participants with EBV+PTLD SOT who were R/R to rituximab or R/R to rituximab and chemotherapy received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After 3 week observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ Lymphoma
n=25 Participants
Participants with EBV+ lymphoma received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ NPC
n=14 Participants
Participants with EBV+ NPC received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
Total
n=74 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Customized
<18 years
|
5 Participants
n=39 Participants
|
4 Participants
n=41 Participants
|
2 Participants
n=35 Participants
|
2 Participants
n=31 Participants
|
13 Participants
n=146 Participants
|
|
Age, Customized
>=18 years
|
20 Participants
n=39 Participants
|
6 Participants
n=41 Participants
|
23 Participants
n=35 Participants
|
12 Participants
n=31 Participants
|
61 Participants
n=146 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=39 Participants
|
7 Participants
n=41 Participants
|
15 Participants
n=35 Participants
|
4 Participants
n=31 Participants
|
37 Participants
n=146 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=39 Participants
|
3 Participants
n=41 Participants
|
10 Participants
n=35 Participants
|
10 Participants
n=31 Participants
|
37 Participants
n=146 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
3 Participants
n=146 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
3 Participants
n=35 Participants
|
3 Participants
n=31 Participants
|
10 Participants
n=146 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
19 Participants
n=39 Participants
|
10 Participants
n=41 Participants
|
21 Participants
n=35 Participants
|
11 Participants
n=31 Participants
|
61 Participants
n=146 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=146 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
5 Participants
n=35 Participants
|
11 Participants
n=31 Participants
|
17 Participants
n=146 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=146 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
2 Participants
n=31 Participants
|
7 Participants
n=146 Participants
|
|
Race (NIH/OMB)
White
|
17 Participants
n=39 Participants
|
9 Participants
n=41 Participants
|
15 Participants
n=35 Participants
|
1 Participants
n=31 Participants
|
42 Participants
n=146 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=146 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
4 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
8 Participants
n=146 Participants
|
PRIMARY outcome
Timeframe: From Day 1 through 65.3 months after Day 1 dosePopulation: Full analysis set included all participants who received at least one dose of tabelecleucel.
The ORR is defined as percentage of participants with best overall response of complete remission/response (CR) or partial remission/response (PR) based on investigator's assessment. For participants with clinically and/or radiologically evident EBV LPD or malignancies, CR is complete resolution of all clinical and radiologic evidence of lymphoma, confirmed by biopsy of the affected tissues when indicated, lasting for at least 3 weeks following completion of a cycle of tabelecleucel; and PR is a 50 % or greater reduction in the size of all lymphomatous lesions as determined by computed tomography (CT) or magnetic resonance imaging (MRI) measurements of tumor volume, which was maintained for at least 3 weeks following completion of a cycle of tabelecleucel. For participants without clinically and/or radiologically evident tumors with increasing levels of EBV DNA, CR is clearance of EBV without subsequent development of EBV+ LPD; and PR is at least a 10-fold decrease in EBV DNA levels.
Outcome measures
| Measure |
HCT EBV+ PTLD R/R Rituximab
n=25 Participants
Participants with EBV+ PTLD HCT who were R/R to rituximab received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
SOT EBV+ PTLD R/R Rituximab
n=10 Participants
Participants with EBV+PTLD SOT who were R/R to rituximab or R/R to rituximab and chemotherapy received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After 3 week observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ Lymphoma
n=25 Participants
Participants with EBV+ lymphoma received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ NPC
n=14 Participants
Participants with EBV+ NPC received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
|---|---|---|---|---|
|
Objective Response Rate (ORR)
|
68.0 Percentage of participants
Interval 46.5 to 85.1
|
50.0 Percentage of participants
Interval 18.7 to 81.3
|
16.0 Percentage of participants
Interval 4.5 to 36.1
|
14.3 Percentage of participants
Interval 1.8 to 42.8
|
SECONDARY outcome
Timeframe: From Day 1 through 65.3 months after Day 1 dosePopulation: Full analysis set included all participants who received at least one dose of tabelecleucel. Participants who were lost to follow-up or still alive were censored on the last known-to-be-alive date.
The OS is defined as the time from the first dose of tabelecleucel to the date of death due to any cause. The OS was estimated using Kaplan-Meier method. Participants who were lost to follow-up or still alive were censored on the last known-to-be-alive date.
Outcome measures
| Measure |
HCT EBV+ PTLD R/R Rituximab
n=25 Participants
Participants with EBV+ PTLD HCT who were R/R to rituximab received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
SOT EBV+ PTLD R/R Rituximab
n=10 Participants
Participants with EBV+PTLD SOT who were R/R to rituximab or R/R to rituximab and chemotherapy received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After 3 week observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ Lymphoma
n=25 Participants
Participants with EBV+ lymphoma received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ NPC
n=14 Participants
Participants with EBV+ NPC received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
|---|---|---|---|---|
|
Overall Survival (OS)
|
NA Months
Interval 7.1 to
Median and upper limit of confidence interval are not estimable due to insufficient events being observed at the time of the analysis.
|
14.9 Months
Interval 0.4 to
Upper limit of confidence interval is not estimable due to insufficient events being observed at the time of the analysis.
|
12.3 Months
Interval 1.5 to 21.6
|
NA Months
Interval 4.9 to
Median and upper limit of confidence interval are not estimable due to insufficient events being observed at the time of the analysis.
|
SECONDARY outcome
Timeframe: From Day 1 through 12 months after Day 1 dosePopulation: Full analysis set included all participants who received at least one dose of tabelecleucel. Participants who were lost to follow-up or still alive were censored on the last known-to-be-alive date.
Percentage of participants with OS at 12 months are reported. The OS is defined as the time from the first dose of tabelecleucel to the date of death due to any cause. The OS was estimated using Kaplan-Meier method. Participants who were lost to follow-up or still alive were censored on the last known-to-be-alive date.
Outcome measures
| Measure |
HCT EBV+ PTLD R/R Rituximab
n=25 Participants
Participants with EBV+ PTLD HCT who were R/R to rituximab received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
SOT EBV+ PTLD R/R Rituximab
n=10 Participants
Participants with EBV+PTLD SOT who were R/R to rituximab or R/R to rituximab and chemotherapy received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After 3 week observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ Lymphoma
n=25 Participants
Participants with EBV+ lymphoma received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ NPC
n=14 Participants
Participants with EBV+ NPC received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
|---|---|---|---|---|
|
OS Rate at 12 Months
|
68.0 Percentage of participants
Interval 46.1 to 82.5
|
60.0 Percentage of participants
Interval 25.3 to 82.7
|
50.3 Percentage of participants
Interval 29.1 to 68.2
|
77.9 Percentage of participants
Interval 45.9 to 92.3
|
SECONDARY outcome
Timeframe: From Day 1 through 65.3 months after Day 1 dosePopulation: Full analysis set included all participants who received at least one dose of tabelecleucel.
The OS follow-up time are reported. The OS is defined as the time from the first dose of tabelecleucel to the date of death due to any cause. The OS was estimated using Kaplan-Meier method. Participants who were lost to follow-up or still alive were censored on the last known-to-be-alive date.
Outcome measures
| Measure |
HCT EBV+ PTLD R/R Rituximab
n=25 Participants
Participants with EBV+ PTLD HCT who were R/R to rituximab received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
SOT EBV+ PTLD R/R Rituximab
n=10 Participants
Participants with EBV+PTLD SOT who were R/R to rituximab or R/R to rituximab and chemotherapy received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After 3 week observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ Lymphoma
n=25 Participants
Participants with EBV+ lymphoma received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ NPC
n=14 Participants
Participants with EBV+ NPC received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
|---|---|---|---|---|
|
OS Follow-up Time
|
23.33 Months
Interval 0.6 to 60.6
|
14.88 Months
Interval 0.4 to 55.4
|
7.20 Months
Interval 0.3 to 65.3
|
16.0 Months
Interval 3.2 to 46.5
|
SECONDARY outcome
Timeframe: From Day 1 through 65.3 months after Day 1 dosePopulation: Full analysis set included all participants who received at least one dose of tabelecleucel. Participants who achieved CR or PR were analyzed for this outcome measure.
The TTR is defined as the time from the date of the first dose of tabelecleucel to the date of a PR or CR, whichever occurred first. For participants with clinically and/or radiologically evident EBV LPD or malignancies, CR is defined as complete resolution of all clinical and radiologic evidence of lymphoma, confirmed by biopsy of the affected tissues when indicated, lasting for at least 3 weeks following completion of a cycle of tabelecleucel; and a PR is defined as a 50 % or greater reduction in the size of all lymphomatous lesions as determined by CT or MRI scan measurements of tumor volume, which was maintained for at least 3 weeks following completion of a cycle of tabelecleucel. For participants without clinically and/or radiologically evident tumors with increasing levels of EBV DNA, CR is defined as clearance of EBV without subsequent development of EBV+ LPD; and PR is defined as at least a 10-fold decrease in EBV DNA levels.
Outcome measures
| Measure |
HCT EBV+ PTLD R/R Rituximab
n=17 Participants
Participants with EBV+ PTLD HCT who were R/R to rituximab received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
SOT EBV+ PTLD R/R Rituximab
n=5 Participants
Participants with EBV+PTLD SOT who were R/R to rituximab or R/R to rituximab and chemotherapy received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After 3 week observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ Lymphoma
n=4 Participants
Participants with EBV+ lymphoma received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
EBV+ NPC
n=2 Participants
Participants with EBV+ NPC received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
|---|---|---|---|---|
|
Time to Response (TTR)
|
1.77 Months
Interval 0.7 to 6.4
|
3.32 Months
Interval 1.6 to 5.4
|
2.28 Months
Interval 1.2 to 8.4
|
1.76 Months
Interval 1.2 to 2.3
|
Adverse Events
Overall Total
Serious events: 46 serious events
Other events: 0 other events
Deaths: 46 deaths
Serious adverse events
| Measure |
Overall Total
n=87 participants at risk
All eligible participants with EBV+ received IV infusion of tabelecleucel 2 × 10\^6 T-cells/kg on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
5.7%
5/87 • Number of events 6 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Cardiac disorders
Atrial fibrillation
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Cardiac disorders
Pericardial effusion
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Cardiac disorders
Pericarditis constrictive
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Endocrine disorders
Adrenal insufficiency
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Endocrine disorders
Diabetes insipidus
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Eye disorders
Periorbital oedema
|
1.1%
1/87 • Number of events 2 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Gastrointestinal disorders
Abdominal distension
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Gastrointestinal disorders
Ascites
|
1.1%
1/87 • Number of events 2 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Gastrointestinal disorders
Colitis
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Gastrointestinal disorders
Diarrhoea
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Gastrointestinal disorders
Dysphagia
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Gastrointestinal disorders
Enterocolitis
|
2.3%
2/87 • Number of events 2 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Gastrointestinal disorders
Nausea
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Gastrointestinal disorders
Small intestinal haemorrhage
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Gastrointestinal disorders
Vomiting
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
General disorders
Death
|
14.9%
13/87 • Number of events 13 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
General disorders
Non-cardiac chest pain
|
2.3%
2/87 • Number of events 2 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
General disorders
Pyrexia
|
9.2%
8/87 • Number of events 8 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Hepatobiliary disorders
Cholecystitis
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Hepatobiliary disorders
Hepatic failure
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Hepatobiliary disorders
Hepatobiliary disease
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Clostridium difficile infection
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Device related infection
|
3.4%
3/87 • Number of events 3 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Gastroenteritis
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Influenza
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Lower respiratory tract infection
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Lung infection
|
11.5%
10/87 • Number of events 13 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Otitis media
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Periorbital infection
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Pharyngitis
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Pneumonia
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Sepsis
|
3.4%
3/87 • Number of events 3 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Sinusitis
|
3.4%
3/87 • Number of events 3 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Skin infection
|
2.3%
2/87 • Number of events 3 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Upper respiratory tract infection
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Infections and infestations
Urinary tract infection
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Injury, poisoning and procedural complications
Vascular access complication
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Investigations
Lymphocyte count decreased
|
2.3%
2/87 • Number of events 2 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Investigations
Neutrophil count decreased
|
2.3%
2/87 • Number of events 2 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Metabolism and nutrition disorders
Acidosis
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Metabolism and nutrition disorders
Dehydration
|
3.4%
3/87 • Number of events 3 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Metabolism and nutrition disorders
Tumour lysis syndrome
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Musculoskeletal and connective tissue disorders
Joint effusion
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Musculoskeletal and connective tissue disorders
Osteonecrosis
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Second primary malignancy
|
1.1%
1/87 • Number of events 2 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Nervous system disorders
Cerebrovascular accident
|
2.3%
2/87 • Number of events 2 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Nervous system disorders
Depressed level of consciousness
|
2.3%
2/87 • Number of events 3 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Nervous system disorders
Encephalopathy
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Nervous system disorders
Seizure
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Psychiatric disorders
Confusional state
|
3.4%
3/87 • Number of events 4 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Psychiatric disorders
Mental status changes
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Psychiatric disorders
Suicide attempt
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Renal and urinary disorders
Acute kidney injury
|
4.6%
4/87 • Number of events 4 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Renal and urinary disorders
Chronic kidney disease
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Renal and urinary disorders
Renal disorder
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
2.3%
2/87 • Number of events 2 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.3%
2/87 • Number of events 2 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
1.1%
1/87 • Number of events 2 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
|
2.3%
2/87 • Number of events 2 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
2.3%
2/87 • Number of events 2 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Respiratory, thoracic and mediastinal disorders
Stridor
|
1.1%
1/87 • Number of events 1 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Vascular disorders
Embolism
|
2.3%
2/87 • Number of events 2 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
|
Vascular disorders
Hypotension
|
2.3%
2/87 • Number of events 3 • From Day 1 through 65.3 months after Day 1 dose
Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place