Trial Outcomes & Findings for Safety and Efficacy of ATIR101 as Adjunctive Treatment to Blood Stem Cell Transplantation From a Haploidentical Family Donor Compared to Post-transplant Cyclophosphamide in Patients With Blood Cancer (NCT NCT02999854)
NCT ID: NCT02999854
Last Updated: 2022-05-24
Results Overview
Defined as the time until acute GVHD grade III/IV, chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of GRFS were calculated at 24 months post HSCT.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
63 participants
Primary outcome timeframe
24 months post-HSCT
Results posted on
2022-05-24
Participant Flow
Participant milestones
| Measure |
ATIR101
T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
PTCy
T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
|---|---|---|
|
Active Study Phase (up to 18 Months)
STARTED
|
32
|
31
|
|
Active Study Phase (up to 18 Months)
COMPLETED
|
8
|
22
|
|
Active Study Phase (up to 18 Months)
NOT COMPLETED
|
24
|
9
|
|
Long Term Safety FU (up to 24 Months)
STARTED
|
8
|
22
|
|
Long Term Safety FU (up to 24 Months)
COMPLETED
|
5
|
19
|
|
Long Term Safety FU (up to 24 Months)
NOT COMPLETED
|
3
|
3
|
Reasons for withdrawal
| Measure |
ATIR101
T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
PTCy
T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
|---|---|---|
|
Active Study Phase (up to 18 Months)
Screen failures
|
7
|
2
|
|
Active Study Phase (up to 18 Months)
Discontinued before HSCT
|
8
|
2
|
|
Active Study Phase (up to 18 Months)
Death
|
7
|
5
|
|
Active Study Phase (up to 18 Months)
Completed 2 year follow-up
|
1
|
0
|
|
Active Study Phase (up to 18 Months)
End of study after HSCT but before treatment
|
1
|
0
|
|
Long Term Safety FU (up to 24 Months)
Death
|
1
|
1
|
|
Long Term Safety FU (up to 24 Months)
Withdrawal by Subject
|
1
|
1
|
|
Long Term Safety FU (up to 24 Months)
Lost to Follow-up
|
1
|
1
|
Baseline Characteristics
Safety and Efficacy of ATIR101 as Adjunctive Treatment to Blood Stem Cell Transplantation From a Haploidentical Family Donor Compared to Post-transplant Cyclophosphamide in Patients With Blood Cancer
Baseline characteristics by cohort
| Measure |
ATIR101
n=16 Participants
T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
PTCy
n=27 Participants
T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
Total
n=43 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
36.2 years
STANDARD_DEVIATION 9.63 • n=5 Participants
|
32.8 years
STANDARD_DEVIATION 13.20 • n=7 Participants
|
34.5 years
STANDARD_DEVIATION 11.42 • n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
20 Participants
n=7 Participants
|
30 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
12 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
34 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
14 Participants
n=5 Participants
|
23 Participants
n=7 Participants
|
37 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Hematologic malignancy
Acute lymphatic leukemia (ALL)
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Hematologic malignancy
Acute myeloid leukemia (AML)
|
9 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
|
Hematologic malignancy
Myelodysplastic syndrome (MDS)
|
3 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 months post-HSCTDefined as the time until acute GVHD grade III/IV, chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of GRFS were calculated at 24 months post HSCT.
Outcome measures
| Measure |
ATIR101
n=16 Participants
T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
PTCy
n=27 Participants
T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
|---|---|---|
|
Graft-versus-host Disease-free, Relapse-free Survival (GRFS)
|
25 Percentage of participants
Interval 8.0 to 47.0
|
62 Percentage of participants
Interval 41.0 to 78.0
|
SECONDARY outcome
Timeframe: 24 months post-HSCTOS is defined as the time from HSCT until death from any cause. Kaplan-Meier estimates (percentage of participants) of OS were calculated at 24 months post HSCT.
Outcome measures
| Measure |
ATIR101
n=16 Participants
T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
PTCy
n=27 Participants
T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
|---|---|---|
|
Overall Survival (OS)
|
49 percentage of participants
Interval 24.0 to 71.0
|
77 percentage of participants
Interval 55.0 to 89.0
|
SECONDARY outcome
Timeframe: 24 months post-HSCTDefined as the time from HSCT until relapse, disease progression, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.
Outcome measures
| Measure |
ATIR101
n=16 Participants
T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
PTCy
n=27 Participants
T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
|---|---|---|
|
Progression-free Survival (PFS)
|
44 percentage of participants
Interval 20.0 to 66.0
|
73 percentage of participants
Interval 52.0 to 86.0
|
SECONDARY outcome
Timeframe: Through study completion, at least two years post HSCTPopulation: Study was pre-maturely terminated. Data not collected.
Time from randomization to death due to disease relapse or disease progression
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 months post-HSCTDefined as death due to causes other than disease relapse or progression, or other causes which are unrelated to the transplantation procedure (e.g. accident, suicide). Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.
Outcome measures
| Measure |
ATIR101
n=16 Participants
T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
PTCy
n=27 Participants
T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
|---|---|---|
|
Transplant-related Mortality (TRM)
|
44 percentage of participants
Interval 19.0 to 66.0
|
15 percentage of participants
Interval 5.0 to 31.0
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Through study completion, at least two years post HSCTPopulation: Study was pre-maturely terminated. Data not collected.
Time to CD3+ \> 0.2×10E9/l in peripheral blood (at two consecutive measurements; time to first measurement)
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Through study completion, at least two years post HSCTPopulation: Study was pre-maturely terminated. Data not collected.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Through study completion, at least two years post HSCTPopulation: Study was pre-maturely terminated. Data not collected.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Through study completion, at least two years post HSCTPopulation: Study was pre-maturely terminated. Data not collected.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Through study completion, at least two years post HSCTPopulation: Study was pre-maturely terminated. Data not collected.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Until 2 years after the HSCTPopulation: Study was pre-maturely terminated. Data not collected.
Viral, fungal, and bacterial infections
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Until 2 years after the HSCTPopulation: Study was pre-maturely terminated. Data not collected.
Viral, fungal, and bacterial infections
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)Population: Study was pre-maturely terminated. Data not collected.
Quality of life: Foundation for the Accreditation of Cellular Therapy - Bone Marrow Transplantation questionnaire (FACT-BMT)
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)Population: Study was pre-maturely terminated. Data not collected.
Quality of life: Short Form 36-item health survey (SF-36)
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)Population: Study was pre-maturely terminated. Data not collected.
Quality of life: MD Anderson Symptom Inventory (MDASI)
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)Population: Study was pre-maturely terminated. Data not collected.
Quality of life: EQ-5D-5L
Outcome measures
Outcome data not reported
Adverse Events
ATIR101
Serious events: 13 serious events
Other events: 0 other events
Deaths: 8 deaths
PTCy
Serious events: 19 serious events
Other events: 0 other events
Deaths: 6 deaths
Serious adverse events
| Measure |
ATIR101
n=16 participants at risk
T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
PTCy
n=27 participants at risk
T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
|
|---|---|---|
|
Infections and infestations
Infections and infestations
|
62.5%
10/16 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
44.4%
12/27 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders
|
31.2%
5/16 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
18.5%
5/27 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
|
General disorders
General disorders and administration site conditions
|
25.0%
4/16 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
22.2%
6/27 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
|
Cardiac disorders
Cardiac disorders
|
12.5%
2/16 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
18.5%
5/27 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
|
Gastrointestinal disorders
Gastrointestinal disorders
|
0.00%
0/16 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
3.7%
1/27 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders
|
0.00%
0/16 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
11.1%
3/27 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
|
Nervous system disorders
Nervous system disorders
|
18.8%
3/16 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
0.00%
0/27 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders
|
12.5%
2/16 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
3.7%
1/27 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications
|
12.5%
2/16 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
0.00%
0/27 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
|
Investigations
Investigations
|
0.00%
0/16 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
7.4%
2/27 • Long term safety follow-up, 24 months.
Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat \[ITT\] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
|
Other adverse events
Adverse event data not reported
Additional Information
Klaudia Traudtner, MD. Head of Safety and Pharmacovigilance.
Kiadis Pharma Netherlands B.V
Phone: +31 (0)20 240 52 50
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place