Trial Outcomes & Findings for Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion (NCT NCT05236764)
NCT ID: NCT05236764
Last Updated: 2025-10-30
Results Overview
Cumulative incidence of neutrophil and platelet engraftment (composite measure) will be reported as rate and its associated 95% confidence interval. Competing risks methods will be utilized, with graft failure and death considered as competing risks. Neutrophil engraftment is defined as the first of 3 consecutive days with a peripheral blood absolute neutrophil count of ≥ 0.5x10\^9/L Platelet engraftment is defined as the first day with platelet count of ≥ 20 x10\^9/L without transfusion support for 7 consecutive days
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
NA
Target enrollment
3 participants
Primary outcome timeframe
42 days post-HCT
Results posted on
2025-10-30
Participant Flow
Participant milestones
| Measure |
Alpha beta+ T cell depleted CD34+ stem cells
The patient will be receiving a donor stem cell transplant with a preceding conditioning regimen (chemotherapy with, or without, radiation). The investigators will be specially treating the donor's blood cells used for the stem cell transplant.
CliniMACS: Peripheral blood stem cells from closely matched unrelated donors will be processed using the CliniMACS device to remove TCRalpha/beta (alpha beta+) T cells and B cells, in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique
|
|---|---|
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Overall Study
STARTED
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3
|
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Overall Study
COMPLETED
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0
|
|
Overall Study
NOT COMPLETED
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3
|
Reasons for withdrawal
| Measure |
Alpha beta+ T cell depleted CD34+ stem cells
The patient will be receiving a donor stem cell transplant with a preceding conditioning regimen (chemotherapy with, or without, radiation). The investigators will be specially treating the donor's blood cells used for the stem cell transplant.
CliniMACS: Peripheral blood stem cells from closely matched unrelated donors will be processed using the CliniMACS device to remove TCRalpha/beta (alpha beta+) T cells and B cells, in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique
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|---|---|
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Overall Study
In follow up (ongoing)
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1
|
|
Overall Study
Taken off study - secondary graft failure
|
1
|
|
Overall Study
Death
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1
|
Baseline Characteristics
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
Baseline characteristics by cohort
| Measure |
Alpha Beta+ T Cell Depleted CD34+ Stem Cells
n=3 Participants
The patient will be receiving a donor stem cell transplant with a preceding conditioning regimen (chemotherapy with, or without, radiation). The investigators will be specially treating the donor's blood cells used for the stem cell transplant.
CliniMACS: Peripheral blood stem cells from closely matched unrelated donors will be processed using the CliniMACS device to remove TCRalpha/beta (alpha beta+) T cells and B cells, in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique
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|---|---|
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Age, Continuous
|
10.3 years
STANDARD_DEVIATION 1.5 • n=5 Participants
|
|
Sex: Female, Male
Female
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1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 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
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
1 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
|
PRIMARY outcome
Timeframe: 42 days post-HCTPopulation: All patients who received transplant were included in analysis.
Cumulative incidence of neutrophil and platelet engraftment (composite measure) will be reported as rate and its associated 95% confidence interval. Competing risks methods will be utilized, with graft failure and death considered as competing risks. Neutrophil engraftment is defined as the first of 3 consecutive days with a peripheral blood absolute neutrophil count of ≥ 0.5x10\^9/L Platelet engraftment is defined as the first day with platelet count of ≥ 20 x10\^9/L without transfusion support for 7 consecutive days
Outcome measures
| Measure |
Alpha beta+ T cell depleted CD34+ stem cells
n=3 Participants
The patient will be receiving a donor stem cell transplant with a preceding conditioning regimen (chemotherapy with, or without, radiation). The investigators will be specially treating the donor's blood cells used for the stem cell transplant.
CliniMACS: Peripheral blood stem cells from closely matched unrelated donors will be processed using the CliniMACS device to remove TCRalpha/beta (alpha beta+) T cells and B cells, in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique
|
|---|---|
|
Cumulative Incidence of Neutrophil Engraftment and Platelet Engraftment
|
66.7 Percentage of participants
Interval 0.2 to 97.3
|
PRIMARY outcome
Timeframe: 100 days post-HCTPopulation: All patients who received transplant and did not encounter graft failure are included in analysis. Patients who had graft failure were not evaluable.
Cumulative incidence of grade III or higher acute GVHD among patients who achieve engraftment will be reported as rate and its associated 95% confidence interval. Competing risks methods will be utilized, with death considered the competing risk.
Outcome measures
| Measure |
Alpha beta+ T cell depleted CD34+ stem cells
n=2 Participants
The patient will be receiving a donor stem cell transplant with a preceding conditioning regimen (chemotherapy with, or without, radiation). The investigators will be specially treating the donor's blood cells used for the stem cell transplant.
CliniMACS: Peripheral blood stem cells from closely matched unrelated donors will be processed using the CliniMACS device to remove TCRalpha/beta (alpha beta+) T cells and B cells, in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique
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|---|---|
|
Cumulative Incidence of Grade III or Higher Acute GVHD
|
0.0 Percentage of participants
Interval 0.0 to 0.0
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SECONDARY outcome
Timeframe: 100 days and 365 days post-HCTCumulative incidence of transplant related mortality will be reported as rate and its associated 95% confidence interval. TRM is defined as death due to any transplantation-related cause, other than disease
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to one year post-HCTThe length of time from the day of transplant to death
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to two years post HCTCumulative incidence of chronic GVHD among patients who achieve engraftment will be reported as rate of chronic GvHD and its associated 95% confidence interval.
Outcome measures
Outcome data not reported
Adverse Events
Alpha beta+ T cell depleted CD34+ stem cells
Serious events: 1 serious events
Other events: 3 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Alpha beta+ T cell depleted CD34+ stem cells
n=3 participants at risk
The patient will be receiving a donor stem cell transplant with a preceding conditioning regimen (chemotherapy with, or without, radiation). The investigators will be specially treating the donor's blood cells used for the stem cell transplant.
CliniMACS: Peripheral blood stem cells from closely matched unrelated donors will be processed using the CliniMACS device to remove TCRalpha/beta (alpha beta+) T cells and B cells, in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique
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|---|---|
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General disorders and administration site conditions
Fever
|
33.3%
1/3 • Number of events 1 • Day 0 until 30-days post-transplant for adverse events, and until 100-days post-transplant for serious adverse events.
|
|
Infections and infestations
Encephalitis infection
|
33.3%
1/3 • Number of events 1 • Day 0 until 30-days post-transplant for adverse events, and until 100-days post-transplant for serious adverse events.
|
|
Infections and infestations
Viremia
|
33.3%
1/3 • Number of events 1 • Day 0 until 30-days post-transplant for adverse events, and until 100-days post-transplant for serious adverse events.
|
|
Psychiatric disorders
Confusion
|
33.3%
1/3 • Number of events 1 • Day 0 until 30-days post-transplant for adverse events, and until 100-days post-transplant for serious adverse events.
|
Other adverse events
| Measure |
Alpha beta+ T cell depleted CD34+ stem cells
n=3 participants at risk
The patient will be receiving a donor stem cell transplant with a preceding conditioning regimen (chemotherapy with, or without, radiation). The investigators will be specially treating the donor's blood cells used for the stem cell transplant.
CliniMACS: Peripheral blood stem cells from closely matched unrelated donors will be processed using the CliniMACS device to remove TCRalpha/beta (alpha beta+) T cells and B cells, in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
33.3%
1/3 • Number of events 1 • Day 0 until 30-days post-transplant for adverse events, and until 100-days post-transplant for serious adverse events.
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
66.7%
2/3 • Number of events 2 • Day 0 until 30-days post-transplant for adverse events, and until 100-days post-transplant for serious adverse events.
|
|
Gastrointestinal disorders
Abdominal pain
|
33.3%
1/3 • Number of events 1 • Day 0 until 30-days post-transplant for adverse events, and until 100-days post-transplant for serious adverse events.
|
|
Gastrointestinal disorders
Mucositis oral
|
66.7%
2/3 • Number of events 2 • Day 0 until 30-days post-transplant for adverse events, and until 100-days post-transplant for serious adverse events.
|
|
Infections and infestations
Cytomegalovirus infection reactivation
|
33.3%
1/3 • Number of events 1 • Day 0 until 30-days post-transplant for adverse events, and until 100-days post-transplant for serious adverse events.
|
|
Investigations
Aspartate aminotransferase increased
|
33.3%
1/3 • Number of events 1 • Day 0 until 30-days post-transplant for adverse events, and until 100-days post-transplant for serious adverse events.
|
|
Investigations
Creatinine increased
|
33.3%
1/3 • Number of events 1 • Day 0 until 30-days post-transplant for adverse events, and until 100-days post-transplant for serious adverse events.
|
|
Investigations
Neutrophil count decreased
|
33.3%
1/3 • Number of events 1 • Day 0 until 30-days post-transplant for adverse events, and until 100-days post-transplant for serious adverse events.
|
|
Metabolism and nutrition disorders
Anorexia
|
66.7%
2/3 • Number of events 2 • Day 0 until 30-days post-transplant for adverse events, and until 100-days post-transplant for serious adverse events.
|
|
Renal and urinary disorders
Acute kidney injury
|
33.3%
1/3 • Number of events 1 • Day 0 until 30-days post-transplant for adverse events, and until 100-days post-transplant for serious adverse events.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place