Trial Outcomes & Findings for TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PID (NCT NCT02327351)
NCT ID: NCT02327351
Last Updated: 2021-02-26
Results Overview
The probability of overall survival estimated by the Kaplan-Meier method at 1 year after HSCT
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
98 participants
Primary outcome timeframe
1 year after HSCT
Results posted on
2021-02-26
Participant Flow
Participant milestones
| Measure |
TCR Alfa Beta Depletion
TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.
Biological: TCR alfa beta T cell depletion
|
|---|---|
|
Overall Study
STARTED
|
98
|
|
Overall Study
COMPLETED
|
98
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
TCR Alfa Beta Depletion
n=98 Participants
TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.
Biological: TCR alfa beta T cell depletion
|
|---|---|
|
Age, Categorical
<=18 years
|
98 Participants
n=98 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=98 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=98 Participants
|
|
Age, Continuous
|
4.2 years
STANDARD_DEVIATION 2 • n=98 Participants
|
|
Sex: Female, Male
Female
|
23 Participants
n=98 Participants
|
|
Sex: Female, Male
Male
|
75 Participants
n=98 Participants
|
|
Region of Enrollment
Russia
|
98 participants
n=98 Participants
|
PRIMARY outcome
Timeframe: 1 year after HSCTThe probability of overall survival estimated by the Kaplan-Meier method at 1 year after HSCT
Outcome measures
| Measure |
TCR Alfa Beta Depletion
n=98 Participants
TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.
Biological: TCR alfa beta T cell depletion
|
Matched Unrelated Donor
n=75 Participants
type of HSCT donor - HLA-matched unrelated donor
|
HLA-mismatched Related Donor
n=23 Participants
type of HSCT donor - HLA-mismatched related
|
|---|---|---|---|
|
Overall Survival
|
86 percentage of survival probability
Interval 79.0 to 94.0
|
86 percentage of survival probability
Interval 79.0 to 94.0
|
87 percentage of survival probability
Interval 73.0 to 100.0
|
SECONDARY outcome
Timeframe: 24 months after transplantationtransplant-related mortality estimated with cumulative incidence curve, considering relapse as a competitive risk
Outcome measures
| Measure |
TCR Alfa Beta Depletion
n=98 Participants
TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.
Biological: TCR alfa beta T cell depletion
|
Matched Unrelated Donor
type of HSCT donor - HLA-matched unrelated donor
|
HLA-mismatched Related Donor
type of HSCT donor - HLA-mismatched related
|
|---|---|---|---|
|
Transplant Related Mortality (TRM)
|
11 percentage of cumulative incidence
Interval 6.0 to 21.0
|
—
|
—
|
SECONDARY outcome
Timeframe: 12 months after transplantationincidence of aGVHD II-IV stage estimated with cumulative incidence curve, considering graft rejection and death as competitive risks
Outcome measures
| Measure |
TCR Alfa Beta Depletion
n=98 Participants
TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.
Biological: TCR alfa beta T cell depletion
|
Matched Unrelated Donor
type of HSCT donor - HLA-matched unrelated donor
|
HLA-mismatched Related Donor
type of HSCT donor - HLA-mismatched related
|
|---|---|---|---|
|
Acute Graft Versus Host Diseases (аGVHD)
|
17 percentage of cumulative incidence
Interval 1.0 to 28.0
|
—
|
—
|
SECONDARY outcome
Timeframe: 1 year after HSCTincidence of cGVHD estimated with cumulative incidence curve, considering graft rejection and death as competitive risks
Outcome measures
| Measure |
TCR Alfa Beta Depletion
n=98 Participants
TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.
Biological: TCR alfa beta T cell depletion
|
Matched Unrelated Donor
type of HSCT donor - HLA-matched unrelated donor
|
HLA-mismatched Related Donor
type of HSCT donor - HLA-mismatched related
|
|---|---|---|---|
|
Chronic Graft Versus Host Diseases (cGVHD)
|
9 percentage of cumulative incidence
Interval 4.0 to 19.0
|
—
|
—
|
SECONDARY outcome
Timeframe: 2 years after HSCTPopulation: alive at a time-point 2 years after HSCT
Number of participants, who reached immune recovery - CD19+ lymphocytes subsets
Outcome measures
| Measure |
TCR Alfa Beta Depletion
n=70 Participants
TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.
Biological: TCR alfa beta T cell depletion
|
Matched Unrelated Donor
type of HSCT donor - HLA-matched unrelated donor
|
HLA-mismatched Related Donor
type of HSCT donor - HLA-mismatched related
|
|---|---|---|---|
|
Cellular Immunological Reconstitution
|
67 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: last follow-upPopulation: survivals at the last folow-up
Percentage of patients with full (more than 90%) donor chimerism among survivals
Outcome measures
| Measure |
TCR Alfa Beta Depletion
n=72 Participants
TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.
Biological: TCR alfa beta T cell depletion
|
Matched Unrelated Donor
type of HSCT donor - HLA-matched unrelated donor
|
HLA-mismatched Related Donor
type of HSCT donor - HLA-mismatched related
|
|---|---|---|---|
|
Percentage of Patients With Full Donor Chimerism
|
48 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: 12 months after transplantationnumber of patients with CMV reactivation (detection of any grade of CMV viremia after HSCT)
Outcome measures
| Measure |
TCR Alfa Beta Depletion
n=74 Participants
TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.
Biological: TCR alfa beta T cell depletion
|
Matched Unrelated Donor
n=20 Participants
type of HSCT donor - HLA-matched unrelated donor
|
HLA-mismatched Related Donor
type of HSCT donor - HLA-mismatched related
|
|---|---|---|---|
|
Viral Infections After Transplant
|
50 percentage of cumulative incidence
Interval 38.0 to 65.0
|
60 percentage of cumulative incidence
Interval 42.0 to 86.0
|
—
|
Adverse Events
TCR Alfa Beta Depletion
Serious events: 17 serious events
Other events: 0 other events
Deaths: 13 deaths
Serious adverse events
| Measure |
TCR Alfa Beta Depletion
n=98 participants at risk
TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.
Biological: TCR alfa beta T cell depletion
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
CMV pneumonia
|
6.1%
6/98 • 1 year
|
|
Hepatobiliary disorders
ADV hepatitis
|
2.0%
2/98 • 1 year
|
|
Blood and lymphatic system disorders
Bacterial sepsis
|
7.1%
7/98 • 1 year
|
|
Renal and urinary disorders
TMA
|
2.0%
2/98 • 1 year
|
Other adverse events
Adverse event data not reported
Additional Information
Balashov Dmitriy, head of HSCT department
National Research Center for Pediatric Hematology , Moscow, Russian Federation
Phone: 84956647078
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place