Trial Outcomes & Findings for MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs (NCT NCT02582775)
NCT ID: NCT02582775
Last Updated: 2024-09-19
Results Overview
An event defined as death or a 50% increase in a patient's IScoreEB from baseline
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
17 participants
Primary outcome timeframe
1 year post-transplant
Results posted on
2024-09-19
Participant Flow
The JEB subject (n=1) was enrolled on Arm B, receiving the same therapy as the RDEB subjects, but pulled out and reported separately due to the very different baseline disease course.
Participant milestones
| Measure |
RDEB: HCT Plus MSC Arm B
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
RDEB: HCT Plus MSC Arm E
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
JEB: HCT Plus MSC Arm B
Junctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
HCT With 300 cGy of TBI Arm A
Epidermolysis bullosa patients treated per study regimen with chemotherapy and stem cell transplant without mesenchymal stem cell infusions.
|
Re-Transplant Arm C
Epidermolysis bullosa patients treated regardless of original transplant arm with re-transplant using 300 cGy of TBI.
|
HCT Arm D
HLA-matched epidermolysis bullosa patients treated with hematopoietic cell transplant alone using 200 cGY BID of TBI (400 cGy total).
|
HCT Alone Arm F
HLA-mismatched epidermolysis bullosa patients treated with hematopoietic cell transplant alone using 200 cGy BID of TBI (400 cGy total) + addition of low dose busulfan for recipients of HLA-mismatched bone marrow
|
HCT Plus MSC Arm G
HLA-mismatched epidermolysis bullosa patients treated with hematopoietic cell transplant plus serial MSC infusions using 200 cGy BID of TBI (400 cGy total) + addition of low dose busulfan for recipients of HLA-mismatched bone marrow
|
|---|---|---|---|---|---|---|---|---|
|
Overall Study
STARTED
|
7
|
9
|
1
|
0
|
0
|
0
|
0
|
0
|
|
Overall Study
COMPLETED
|
7
|
9
|
1
|
0
|
0
|
0
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs
Baseline characteristics by cohort
| Measure |
RDEB: HCT Plus MSC B
n=7 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
RDEB: HCT Plus MSC E
n=9 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
JEB: HCT Plus MSC Arm B
n=1 Participants
Junctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
Total
n=17 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
7 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
16 Participants
n=7 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
8 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
9 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
7 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
9 Participants
n=7 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
15 Participants
n=7 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Region of Enrollment
United States
|
7 participants
n=99 Participants
|
9 participants
n=107 Participants
|
1 participants
n=206 Participants
|
17 participants
n=7 Participants
|
PRIMARY outcome
Timeframe: 1 year post-transplantAn event defined as death or a 50% increase in a patient's IScoreEB from baseline
Outcome measures
| Measure |
RDEB: HCT Plus MSC Arm B
n=7 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
RDEB: HCT Plus MSC Arm E
n=9 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
JEB: HCT Plus MSC Arm B
n=1 Participants
Junctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
|---|---|---|---|
|
Event-free Survival
|
4 Count of Participants
|
2 Count of Participants
|
1 Count of Participants
|
SECONDARY outcome
Timeframe: 1 year post-transplantiscorEB surveys are a validated, standard of care tool used to assess disease status in patients with Epidermolysis Bullosa. Measure changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire. Scores can range from 16 to 112. The QOLS scores are summed so that a higher score indicates higher quality of life.
Outcome measures
| Measure |
RDEB: HCT Plus MSC Arm B
n=4 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
RDEB: HCT Plus MSC Arm E
n=6 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
JEB: HCT Plus MSC Arm B
n=1 Participants
Junctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
|---|---|---|---|
|
Change of a Patient's iscorEB
|
-10 units on a scale
Interval -10.0 to 112.0
|
-24 units on a scale
Interval -24.0 to 112.0
|
110 units on a scale
Interval 16.0 to 112.0
|
SECONDARY outcome
Timeframe: 180 days post-transplantCumulative incidence will be used to estimate the probability of relapse treating non-relapse death as a competing risk and transplant-related mortality conversely treating relapse as a competing risk.
Outcome measures
| Measure |
RDEB: HCT Plus MSC Arm B
n=7 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
RDEB: HCT Plus MSC Arm E
n=9 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
JEB: HCT Plus MSC Arm B
n=1 Participants
Junctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
|---|---|---|---|
|
Transplant-related Mortality
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 1 year post-transplantMeasured by the Lansky or Karnofsky score (10-100). Higher scores indicate a better outcome. Midpoint of change in scores used to indicate median.
Outcome measures
| Measure |
RDEB: HCT Plus MSC Arm B
n=3 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
RDEB: HCT Plus MSC Arm E
n=5 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
JEB: HCT Plus MSC Arm B
n=1 Participants
Junctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
|---|---|---|---|
|
Average Change in Quality of Life
|
10 Change in score
Interval 10.0 to 100.0
|
0 Change in score
Interval 10.0 to 100.0
|
-30 Change in score
Interval 10.0 to 100.0
|
SECONDARY outcome
Timeframe: 2 years post-transplantPopulation: Participant in "JEB: HCT plus MSC Arm B" deceased at 2 year post-transplant timepoint.
Measured by the Lansky or Karnofsky score (10-100). The Karnofsky Performance Scale is an assessment tool intended to gauge a patient's functional status and ability to carry out activities of daily living. Higher scores indicate a better outcome. Midpoint of change in scores used to indicate median.
Outcome measures
| Measure |
RDEB: HCT Plus MSC Arm B
n=3 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
RDEB: HCT Plus MSC Arm E
n=4 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
JEB: HCT Plus MSC Arm B
Junctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
|---|---|---|---|
|
Average Change in Quality of Life
|
10 Change in score
Interval 10.0 to 100.0
|
0 Change in score
Interval 10.0 to 100.0
|
—
|
SECONDARY outcome
Timeframe: Day 28, 60, 100, 180, and year 1 and 2 post-transplantPopulation: Participants in "RDEB: HCT plus MSC" arm unable to be analyzed due to missed visits.
Median hematopoietic cells that are of donor origin per participant.
Outcome measures
| Measure |
RDEB: HCT Plus MSC Arm B
n=7 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
RDEB: HCT Plus MSC Arm E
n=9 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
JEB: HCT Plus MSC Arm B
n=1 Participants
Junctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
|---|---|---|---|
|
Lymphoid Chimerism
Day 28
|
100 Percentage of donor derived cells
Interval 0.0 to 100.0
|
60 Percentage of donor derived cells
Interval 0.0 to 100.0
|
NA Percentage of donor derived cells
Participant unable to be analyzed.
|
|
Lymphoid Chimerism
Day 60
|
99 Percentage of donor derived cells
Interval 0.0 to 100.0
|
78 Percentage of donor derived cells
Interval 0.0 to 100.0
|
NA Percentage of donor derived cells
Participant unable to be analyzed.
|
|
Lymphoid Chimerism
Day 100
|
88 Percentage of donor derived cells
Interval 0.0 to 100.0
|
94 Percentage of donor derived cells
Interval 0.0 to 100.0
|
NA Percentage of donor derived cells
Participant unable to be analyzed.
|
|
Lymphoid Chimerism
Day 180
|
100 Percentage of donor derived cells
Interval 0.0 to 100.0
|
98.5 Percentage of donor derived cells
Interval 0.0 to 100.0
|
NA Percentage of donor derived cells
Participant unable to be analyzed.
|
|
Lymphoid Chimerism
1 year
|
100 Percentage of donor derived cells
Interval 0.0 to 100.0
|
100 Percentage of donor derived cells
Interval 0.0 to 100.0
|
—
|
|
Lymphoid Chimerism
2 years
|
100 Percentage of donor derived cells
Interval 0.0 to 100.0
|
100 Percentage of donor derived cells
Interval 0.0 to 100.0
|
—
|
SECONDARY outcome
Timeframe: Day 28, 60, 100, 180, and year 1 and 2 post-transplantPopulation: Participants in "RDEB: HCT plus MSC" arm unable to be analyzed due to missed visits.
Median hematopoietic cells that are of donor origin per participant.
Outcome measures
| Measure |
RDEB: HCT Plus MSC Arm B
n=7 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
RDEB: HCT Plus MSC Arm E
n=9 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
JEB: HCT Plus MSC Arm B
n=1 Participants
Junctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
|---|---|---|---|
|
Myeloid Chimerism
Day 100
|
46 Percentage of donor derived cells
Interval 0.0 to 100.0
|
96 Percentage of donor derived cells
Interval 0.0 to 100.0
|
0 Percentage of donor derived cells
Interval 0.0 to 100.0
|
|
Myeloid Chimerism
Day 28
|
76 Percentage of donor derived cells
Interval 0.0 to 100.0
|
95 Percentage of donor derived cells
Interval 0.0 to 100.0
|
23 Percentage of donor derived cells
Interval 0.0 to 100.0
|
|
Myeloid Chimerism
Day 60
|
32 Percentage of donor derived cells
Interval 0.0 to 100.0
|
96 Percentage of donor derived cells
Interval 0.0 to 100.0
|
0 Percentage of donor derived cells
Interval 0.0 to 100.0
|
|
Myeloid Chimerism
Day 180
|
100 Percentage of donor derived cells
Interval 0.0 to 100.0
|
97.5 Percentage of donor derived cells
Interval 0.0 to 100.0
|
0 Percentage of donor derived cells
Interval 0.0 to 100.0
|
|
Myeloid Chimerism
1 year
|
100 Percentage of donor derived cells
Interval 0.0 to 100.0
|
100 Percentage of donor derived cells
Interval 0.0 to 100.0
|
—
|
|
Myeloid Chimerism
2 years
|
100 Percentage of donor derived cells
Interval 0.0 to 100.0
|
100 Percentage of donor derived cells
Interval 0.0 to 100.0
|
—
|
SECONDARY outcome
Timeframe: 2 year post-transplantPopulation: Participant in "JEB: HCT plus MSC Arm B" deceased at 2 year post-transplant timepoint.
iscorEB surveys are a validated, standard of care tool used to assess disease status in patients with Epidermolysis Bullosa. Measure percent of changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire. Scores can range from 16 to 112. The QOLS scores are summed so that a higher score indicates higher quality of life.
Outcome measures
| Measure |
RDEB: HCT Plus MSC Arm B
n=2 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
RDEB: HCT Plus MSC Arm E
n=4 Participants
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
JEB: HCT Plus MSC Arm B
Junctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
|---|---|---|---|
|
Average Change of a Patient's iscorEB
|
9 units on a scale
Interval 6.0 to 112.0
|
-2 units on a scale
Interval 16.0 to 112.0
|
—
|
Adverse Events
RDEB: HCT Plus MSC Arm B
Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths
RDEB: HCT Plus MSC Arm E
Serious events: 1 serious events
Other events: 1 other events
Deaths: 1 deaths
JEB: HCT Plus MSC Arm B
Serious events: 0 serious events
Other events: 1 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
RDEB: HCT Plus MSC Arm B
n=7 participants at risk
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
RDEB: HCT Plus MSC Arm E
n=9 participants at risk
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
JEB: HCT Plus MSC Arm B
n=1 participants at risk
Junctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
|---|---|---|---|
|
Hepatobiliary disorders
Other, specify - Veno-occlusive disease
|
28.6%
2/7 • Number of events 2 • 2 years
|
11.1%
1/9 • Number of events 1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Investigations
Reversible posterior leukoencephalopathy syndrome
|
14.3%
1/7 • Number of events 1 • 2 years
|
0.00%
0/9 • 2 years
|
0.00%
0/1 • 2 years
|
Other adverse events
| Measure |
RDEB: HCT Plus MSC Arm B
n=7 participants at risk
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
RDEB: HCT Plus MSC Arm E
n=9 participants at risk
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
JEB: HCT Plus MSC Arm B
n=1 participants at risk
Junctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
|
|---|---|---|---|
|
Hepatobiliary disorders
Hepatobiliary disorders - Other, specify
|
0.00%
0/7 • 2 years
|
11.1%
1/9 • Number of events 1 • 2 years
|
100.0%
1/1 • Number of events 1 • 2 years
|
|
Infections and infestations
Sepsis
|
14.3%
1/7 • Number of events 1 • 2 years
|
0.00%
0/9 • 2 years
|
0.00%
0/1 • 2 years
|
|
Nervous system disorders
Nervous system disorders - Other, specify
|
14.3%
1/7 • Number of events 1 • 2 years
|
0.00%
0/9 • 2 years
|
0.00%
0/1 • 2 years
|
Additional Information
Dr. Christen Ebens
University of Minnesota, Masonic Cancer Center
Phone: (612) 626-5654
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place