Trial Outcomes & Findings for Cord Blood With T-Cell Depleted Haplo-identical Peripheral Blood Stem Cell Transplantation for Hematological Malignancies (NCT NCT01682226)
NCT ID: NCT01682226
Last Updated: 2022-08-05
Results Overview
The primary endpoint is neutrophil recovery ie the speed \[median (range) days to recovery\] and success \[day 45 cumulative incidence percent\].
COMPLETED
PHASE2
84 participants
Day 45
2022-08-05
Participant Flow
Patients were accrued to this protocol in 2 arms based on the patients risk of transplant-related mortality. The division into 2 arms was for the purposes of the creation of clinically appropriate stopping rules to promote access to this therapy while ensuring safety. As the safety stopping rules were not reached in either arm all patients were combined for the analysis and analysis by individual arms was not indicated and not performed.
Participant milestones
| Measure |
Adult Haplo-dCBT Recipients
Participants included adult patients with high-risk hematologic malignancies without a suitable human leukocyte antigen (HLA)-matched related or unrelated donor, who had a suitable CB graft and a suitable haplo-identical donor.
Patients were accrued to this protocol in 2 arms based on the patients risk of transplant-related mortality. The division into 2 arms was for the purposes of the creation of clinically appropriate stopping rules to promote access to this therapy while ensuring safety. As the safety stopping rules were not reached in either arm all patients were combined for the analysis and analysis by individual arms was not indicated and not performed.
|
|---|---|
|
Overall Study
STARTED
|
84
|
|
Overall Study
COMPLETED
|
78
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
Adult Haplo-dCBT Recipients
Participants included adult patients with high-risk hematologic malignancies without a suitable human leukocyte antigen (HLA)-matched related or unrelated donor, who had a suitable CB graft and a suitable haplo-identical donor.
Patients were accrued to this protocol in 2 arms based on the patients risk of transplant-related mortality. The division into 2 arms was for the purposes of the creation of clinically appropriate stopping rules to promote access to this therapy while ensuring safety. As the safety stopping rules were not reached in either arm all patients were combined for the analysis and analysis by individual arms was not indicated and not performed.
|
|---|---|
|
Overall Study
Participants were pediatric subjects and not adults subjects
|
3
|
|
Overall Study
Participants did not receive treatment
|
2
|
|
Overall Study
Participants received a transplant under a different protocol
|
1
|
Baseline Characteristics
Cord Blood With T-Cell Depleted Haplo-identical Peripheral Blood Stem Cell Transplantation for Hematological Malignancies
Baseline characteristics by cohort
| Measure |
Adult Haplo-dCBT Recipients
n=78 Participants
Participants included adult patients with high-risk hematologic malignancies without a suitable human leukocyte antigen (HLA)-matched related or unrelated donor, who had a suitable CB graft and a suitable haplo-identical donor.
|
|---|---|
|
Age, Continuous
|
49.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
37 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
41 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
72 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
59 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
78 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 45The primary endpoint is neutrophil recovery ie the speed \[median (range) days to recovery\] and success \[day 45 cumulative incidence percent\].
Outcome measures
| Measure |
Hematopoietic Engraftment Participants
n=75 Participants
Adult participants who underwent haplodCBT
|
|---|---|
|
Percent of Engrafted Participants With Successful Neutrophil Recovery
|
96 % w/incidence of neutrophil recovery
Interval 87.0 to 99.0
|
SECONDARY outcome
Timeframe: up to 6 years78 adult patients were transplanted and received protocol therapy and that patient number was analyzed. Survival was analyzed in clinically meaningful patient groups according to having received protocol therapy, the diagnosis and patient comorbidity and have been published separately from the primary endpoint of engraftment.
Outcome measures
| Measure |
Hematopoietic Engraftment Participants
n=78 Participants
Adult participants who underwent haplodCBT
|
|---|---|
|
Overall Survival
|
3.75 years
Interval 1.0 to 6.0
|
Adverse Events
Adult Haplo-dCBT Recipients
Serious adverse events
| Measure |
Adult Haplo-dCBT Recipients
n=78 participants at risk
Participants included adult patients with high-risk hematologic malignancies without a suitable human leukocyte antigen (HLA)-matched related or unrelated donor, who had a suitable CB graft and a suitable haplo-identical donor.
|
|---|---|
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General disorders
Death- NOS
|
26.9%
21/78 • Up to 6 years
The research question of this clinical trial was whether the addition of CD34+ selected haploidentical cells to a standard double unit cord blood graft. There were no serious adverse events that were related to the protocol's treatment intervention, the addition of the haplo-identical cells.
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Other adverse events
Adverse event data not reported
Additional Information
Dr. Juliet Barker, MBBS
Memorial Sloan Kettering Cancer Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place