Trial Outcomes & Findings for SCID Bu/Flu/ATG Study With T Cell Depletion (NCT NCT02127892)
NCT ID: NCT02127892
Last Updated: 2017-09-18
Results Overview
Engraftment is defined as recovery of blood counts (neutrophil and platelet engraftment) with cells of donor origin, documented by either bone marrow or peripheral blood chimerism assays after hematopoietic stem cell transplant.
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
9 participants
Primary outcome timeframe
100 day
Results posted on
2017-09-18
Participant Flow
Participant milestones
| Measure |
Unrelated BM With T Cell Depletion
Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.
unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).
|
Unrelated Cord Blood
Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).
unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.
|
Haplo BM With T Cell Depletion
If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.
haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.
|
Unrelated PBSC With T Cell Depletion
The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.
unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
7
|
2
|
0
|
0
|
|
Overall Study
COMPLETED
|
6
|
1
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
SCID Bu/Flu/ATG Study With T Cell Depletion
Baseline characteristics by cohort
| Measure |
Unrelated BM With T Cell Depletion
n=7 Participants
Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.
unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).
|
Unrelated Cord Blood
n=2 Participants
Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).
unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.
|
Haplo BM With T Cell Depletion
If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.
haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.
|
Unrelated PBSC With T Cell Depletion
The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.
unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
|
Total
n=9 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
7 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
9 Participants
n=21 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
—
|
—
|
1 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
—
|
—
|
8 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
5 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
3 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
—
|
—
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
—
|
—
|
1 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
—
|
—
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
—
|
—
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
—
|
—
|
2 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
—
|
—
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
—
|
—
|
6 Participants
n=21 Participants
|
|
Region of Enrollment
United States
|
7 participants
n=5 Participants
|
2 participants
n=7 Participants
|
—
|
—
|
9 participants
n=21 Participants
|
PRIMARY outcome
Timeframe: 100 dayEngraftment is defined as recovery of blood counts (neutrophil and platelet engraftment) with cells of donor origin, documented by either bone marrow or peripheral blood chimerism assays after hematopoietic stem cell transplant.
Outcome measures
| Measure |
Unrelated BM With T Cell Depletion
n=7 Participants
Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.
unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).
|
Unrelated Cord Blood
n=2 Participants
Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).
unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.
|
Haplo BM With T Cell Depletion
If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.
haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.
|
Unrelated PBSC With T Cell Depletion
The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.
unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
|
|---|---|---|---|---|
|
Number of Participants With Engraftment
|
7 Participants
|
2 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: 1 yearAbsolute number of donor-derived CD3+ T lymphocytes \>/= 100/mm3 in participating subjects.
Outcome measures
| Measure |
Unrelated BM With T Cell Depletion
n=7 Participants
Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.
unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).
|
Unrelated Cord Blood
n=2 Participants
Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).
unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.
|
Haplo BM With T Cell Depletion
If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.
haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.
|
Unrelated PBSC With T Cell Depletion
The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.
unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
|
|---|---|---|---|---|
|
Number of Participants With Donor-derived CD3+ T Lymphocytes >/= 100/mm3
|
7 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 100 daysEvaluation of veno-occlusive disease determined by the presence of the following features; fluid retention, weight gain, leaky capillary syndrome, painful liver enlargement, refractoriness to platelet tranfusion and hyperbilirubinemia
Outcome measures
| Measure |
Unrelated BM With T Cell Depletion
n=7 Participants
Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.
unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).
|
Unrelated Cord Blood
n=2 Participants
Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).
unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.
|
Haplo BM With T Cell Depletion
If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.
haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.
|
Unrelated PBSC With T Cell Depletion
The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.
unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
|
|---|---|---|---|---|
|
Number of Participants With Veno-occlusive Disease (VOD) - Moderate and Severe
|
2 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 1 yearGVHD disease surveillance done by clinical evaluation, to include history, physical examination, specifically for rash, jaundice, liver dysfunction, nausea and vomiting, diarrhea and failure to thrive.
Outcome measures
| Measure |
Unrelated BM With T Cell Depletion
n=7 Participants
Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.
unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).
|
Unrelated Cord Blood
n=2 Participants
Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).
unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.
|
Haplo BM With T Cell Depletion
If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.
haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.
|
Unrelated PBSC With T Cell Depletion
The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.
unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
|
|---|---|---|---|---|
|
Number of Participants With Graft Versus Host Disease (GVHD) - Grade III or IV
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 1 yearOveralls survival of patient at 1 year post transplant
Outcome measures
| Measure |
Unrelated BM With T Cell Depletion
n=7 Participants
Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.
unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).
|
Unrelated Cord Blood
n=2 Participants
Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).
unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.
|
Haplo BM With T Cell Depletion
If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.
haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.
|
Unrelated PBSC With T Cell Depletion
The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.
unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
|
|---|---|---|---|---|
|
Overall Survival
|
6 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
Adverse Events
Unrelated BM With T Cell Depletion
Serious events: 1 serious events
Other events: 5 other events
Deaths: 1 deaths
Unrelated Cord Blood
Serious events: 1 serious events
Other events: 1 other events
Deaths: 1 deaths
Haplo BM With T Cell Depletion
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Unrelated PBSC With T Cell Depletion
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Unrelated BM With T Cell Depletion
n=7 participants at risk
Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.
unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).
|
Unrelated Cord Blood
n=2 participants at risk
Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).
unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.
|
Haplo BM With T Cell Depletion
If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.
haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.
|
Unrelated PBSC With T Cell Depletion
The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.
unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
|
|---|---|---|---|---|
|
Hepatobiliary disorders
Sinusoidal obstruction syndrome
|
14.3%
1/7 • Number of events 1 • Adverse event data was collected over a period of 1 year post transplant.
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected over a period of 1 year post transplant.
|
—
0/0 • Adverse event data was collected over a period of 1 year post transplant.
|
—
0/0 • Adverse event data was collected over a period of 1 year post transplant.
|
Other adverse events
| Measure |
Unrelated BM With T Cell Depletion
n=7 participants at risk
Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.
unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).
|
Unrelated Cord Blood
n=2 participants at risk
Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).
unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.
|
Haplo BM With T Cell Depletion
If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.
haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.
|
Unrelated PBSC With T Cell Depletion
The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.
unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
|
|---|---|---|---|---|
|
Skin and subcutaneous tissue disorders
Graft Versus Host Disease
|
71.4%
5/7 • Number of events 5 • Adverse event data was collected over a period of 1 year post transplant.
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected over a period of 1 year post transplant.
|
—
0/0 • Adverse event data was collected over a period of 1 year post transplant.
|
—
0/0 • Adverse event data was collected over a period of 1 year post transplant.
|
|
Infections and infestations
Infection - Aspergillis
|
14.3%
1/7 • Number of events 1 • Adverse event data was collected over a period of 1 year post transplant.
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected over a period of 1 year post transplant.
|
—
0/0 • Adverse event data was collected over a period of 1 year post transplant.
|
—
0/0 • Adverse event data was collected over a period of 1 year post transplant.
|
|
Infections and infestations
Infection - Fungal
|
14.3%
1/7 • Number of events 1 • Adverse event data was collected over a period of 1 year post transplant.
|
0.00%
0/2 • Adverse event data was collected over a period of 1 year post transplant.
|
—
0/0 • Adverse event data was collected over a period of 1 year post transplant.
|
—
0/0 • Adverse event data was collected over a period of 1 year post transplant.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Hemorrhage
|
0.00%
0/7 • Adverse event data was collected over a period of 1 year post transplant.
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected over a period of 1 year post transplant.
|
—
0/0 • Adverse event data was collected over a period of 1 year post transplant.
|
—
0/0 • Adverse event data was collected over a period of 1 year post transplant.
|
|
Nervous system disorders
Seizure
|
14.3%
1/7 • Number of events 1 • Adverse event data was collected over a period of 1 year post transplant.
|
0.00%
0/2 • Adverse event data was collected over a period of 1 year post transplant.
|
—
0/0 • Adverse event data was collected over a period of 1 year post transplant.
|
—
0/0 • Adverse event data was collected over a period of 1 year post transplant.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place