Trial Outcomes & Findings for Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs (NCT NCT01033552)
NCT ID: NCT01033552
Last Updated: 2024-04-03
Results Overview
Event-free survival rate, with an event defined as death or failure to have a demonstrable increase in collagen, laminin, intergrin, keratin or plakin deposition. Assessed at follow up appointments through questionnaire and patient samples.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
32 participants
Primary outcome timeframe
1 year and 2 Years Post-transplant
Results posted on
2024-04-03
Participant Flow
Participant milestones
| Measure |
RDEB Mac
Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
RDEB RIC
Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB MAC
Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB RIC
Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
7
|
16
|
2
|
7
|
|
Overall Study
COMPLETED
|
7
|
16
|
2
|
7
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs
Baseline characteristics by cohort
| Measure |
RDEB Mac
n=7 Participants
Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
RDEB RIC
n=16 Participants
Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB MAC
n=2 Participants
Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB RIC
n=7 Participants
Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
Total
n=32 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
7 Participants
n=93 Participants
|
15 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
7 Participants
n=483 Participants
|
31 Participants
n=36 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
1 Participants
n=36 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=93 Participants
|
8 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
3 Participants
n=483 Participants
|
15 Participants
n=36 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=93 Participants
|
8 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
4 Participants
n=483 Participants
|
17 Participants
n=36 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
2 Participants
n=483 Participants
|
3 Participants
n=36 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=93 Participants
|
15 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
5 Participants
n=483 Participants
|
28 Participants
n=36 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
1 Participants
n=36 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
7 Participants
n=483 Participants
|
9 Participants
n=36 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
2 Participants
n=36 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=93 Participants
|
14 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
21 Participants
n=36 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
|
Region of Enrollment
United States
|
7 participants
n=93 Participants
|
16 participants
n=4 Participants
|
2 participants
n=27 Participants
|
7 participants
n=483 Participants
|
32 participants
n=36 Participants
|
PRIMARY outcome
Timeframe: 1 year and 2 Years Post-transplantEvent-free survival rate, with an event defined as death or failure to have a demonstrable increase in collagen, laminin, intergrin, keratin or plakin deposition. Assessed at follow up appointments through questionnaire and patient samples.
Outcome measures
| Measure |
RDEB Mac
n=7 Participants
Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
RDEB RIC
n=16 Participants
Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB MAC
n=2 Participants
Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB RIC
n=7 Participants
Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
|---|---|---|---|---|
|
Percentage of Participants With Event-free Survival
1 year
|
67 Percentage of participants
Interval 19.0 to 90.0
|
80 Percentage of participants
Interval 50.0 to 93.0
|
50 Percentage of participants
Interval 1.0 to 91.0
|
29 Percentage of participants
Interval 4.0 to 61.0
|
|
Percentage of Participants With Event-free Survival
2 year
|
44 Percentage of participants
Interval 7.0 to 78.0
|
80 Percentage of participants
Interval 50.0 to 93.0
|
50 Percentage of participants
Interval 1.0 to 91.0
|
14 Percentage of participants
Interval 1.0 to 46.0
|
SECONDARY outcome
Timeframe: 180 Days Post TransplantIncidence of transplant-related mortality (TRM)
Outcome measures
| Measure |
RDEB Mac
n=7 Participants
Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
RDEB RIC
n=16 Participants
Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB MAC
n=2 Participants
Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB RIC
n=7 Participants
Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
|---|---|---|---|---|
|
Percentage of Participants Transplant-related Mortality (TRM)
|
43 Percentage of participants
Interval 11.0 to 75.0
|
19 Percentage of participants
Interval 2.0 to 36.0
|
50 Percentage of participants
Interval 1.0 to 99.0
|
86 Percentage of participants
Interval 50.0 to 100.0
|
SECONDARY outcome
Timeframe: 1 Year Post-TransplantPattern of biochemical improvement measured by cumulative increase in protein expression and related structural and physical changes
Outcome measures
| Measure |
RDEB Mac
n=7 Participants
Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
RDEB RIC
n=16 Participants
Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB MAC
n=2 Participants
Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB RIC
n=7 Participants
Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
|---|---|---|---|---|
|
Average Biochemical Improvement
|
29 Percentage of participants
Interval 7.0 to 74.0
|
31 Percentage of participants
Interval 14.0 to 60.0
|
0 Percentage of participants
Interval 0.0 to 100.0
|
0 Percentage of participants
Interval 0.0 to 100.0
|
SECONDARY outcome
Timeframe: Pretreatment and 1 yearPopulation: Participants unnable to complete quality of life questionnaires at various time points.
Health quality of life questionnaire as compared to pretreatment results. Scores can range from 0 to 100. The QOLS scores are summed so that a higher score indicates higher quality of life.
Outcome measures
| Measure |
RDEB Mac
n=4 Participants
Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
RDEB RIC
n=6 Participants
Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB MAC
Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB RIC
n=2 Participants
Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
|---|---|---|---|---|
|
Measure Patients Quality of Life Using a Questionnaire
Pretreatment
|
43 Scores on a scale
Interval 1.0 to 85.0
|
17 Scores on a scale
Interval 1.0 to 79.0
|
—
|
1 Scores on a scale
Interval 1.0 to 1.0
|
|
Measure Patients Quality of Life Using a Questionnaire
1 year
|
37 Scores on a scale
Interval 1.0 to 79.0
|
68 Scores on a scale
Interval 52.0 to 84.0
|
—
|
—
|
SECONDARY outcome
Timeframe: 100 DaysIncidence of HSC donor engraftment in the skin
Outcome measures
| Measure |
RDEB Mac
n=6 Participants
Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
RDEB RIC
n=14 Participants
Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB MAC
n=1 Participants
Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB RIC
n=3 Participants
Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
|---|---|---|---|---|
|
Durability of HSC Donor Engraftment in the Skin
|
11 Percentage of Incidence
Interval 6.0 to 30.0
|
7 Percentage of Incidence
Interval 0.0 to 18.0
|
0 Percentage of Incidence
Interval 0.0 to 100.0
|
9 Percentage of Incidence
Interval 0.0 to 13.0
|
SECONDARY outcome
Timeframe: 1 YearSurviving patients one year after engraftment
Outcome measures
| Measure |
RDEB Mac
n=7 Participants
Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
RDEB RIC
n=16 Participants
Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB MAC
n=2 Participants
Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB RIC
n=7 Participants
Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
|---|---|---|---|---|
|
Probability of Survival
|
71 Percentage of participants
Interval 26.0 to 92.0
|
81 Percentage of participants
Interval 52.0 to 94.0
|
50 Percentage of participants
Interval 1.0 to 91.0
|
29 Percentage of participants
Interval 4.0 to 61.0
|
SECONDARY outcome
Timeframe: 100 DaysIncidence of acute GCHD
Outcome measures
| Measure |
RDEB Mac
n=7 Participants
Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
RDEB RIC
n=16 Participants
Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB MAC
n=2 Participants
Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB RIC
n=7 Participants
Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
|---|---|---|---|---|
|
Percentage of Participants Who Experienced Acute GVHD
|
14 Percentage of participants
Interval 0.0 to 38.0
|
19 Percentage of participants
Interval 0.0 to 37.0
|
0 Percentage of participants
Interval 0.0 to 100.0
|
29 Percentage of participants
Interval 0.0 to 60.0
|
Adverse Events
RDEB Mac
Serious events: 7 serious events
Other events: 7 other events
Deaths: 4 deaths
RDEB RIC
Serious events: 16 serious events
Other events: 16 other events
Deaths: 3 deaths
JEB MAC
Serious events: 2 serious events
Other events: 2 other events
Deaths: 1 deaths
JEB RIC
Serious events: 7 serious events
Other events: 7 other events
Deaths: 6 deaths
Serious adverse events
| Measure |
RDEB Mac
n=7 participants at risk
Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
RDEB RIC
n=16 participants at risk
Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB MAC
n=2 participants at risk
Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB RIC
n=7 participants at risk
Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
|---|---|---|---|---|
|
Blood and lymphatic system disorders
Bone marrow hypocellular
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders - Other, specify
|
0.00%
0/7 • 1 year through treatment
|
25.0%
4/16 • Number of events 5 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
0.00%
0/7 • 1 year through treatment
|
12.5%
2/16 • Number of events 2 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Cardiac disorders
Cardiac disorders - Other, specify
|
0.00%
0/7 • 1 year through treatment
|
6.2%
1/16 • Number of events 1 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Cardiac disorders
Heart failure
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/7 • 1 year through treatment
|
6.2%
1/16 • Number of events 1 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
6.2%
1/16 • Number of events 1 • 1 year through treatment
|
50.0%
1/2 • Number of events 1 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Gastrointestinal disorders
Mucositis oral
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
6.2%
1/16 • Number of events 2 • 1 year through treatment
|
50.0%
1/2 • Number of events 1 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Gastrointestinal disorders
Vomiting
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
6.2%
1/16 • Number of events 1 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Gastrointestinal disorders
Esophageal stenosis
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
General disorders
Fever
|
42.9%
3/7 • Number of events 8 • 1 year through treatment
|
50.0%
8/16 • Number of events 11 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
28.6%
2/7 • Number of events 4 • 1 year through treatment
|
|
General disorders
General disorders and administration site conditions - Other, specify
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
50.0%
1/2 • Number of events 1 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
General disorders
Multi-organ failure
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
6.2%
1/16 • Number of events 1 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Hepatobiliary disorders
Portal hypertension
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
50.0%
1/2 • Number of events 2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Hepatobiliary disorders
Hepatobiliary disorders - Other, specify
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
Infections and infestations
Catheter related infection
|
57.1%
4/7 • Number of events 4 • 1 year through treatment
|
37.5%
6/16 • Number of events 9 • 1 year through treatment
|
50.0%
1/2 • Number of events 2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
Infections and infestations
Sepsis
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
6.2%
1/16 • Number of events 1 • 1 year through treatment
|
50.0%
1/2 • Number of events 1 • 1 year through treatment
|
28.6%
2/7 • Number of events 2 • 1 year through treatment
|
|
Infections and infestations
Skin infection
|
0.00%
0/7 • 1 year through treatment
|
6.2%
1/16 • Number of events 1 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
Infections and infestations
Upper respiratory infection
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
6.2%
1/16 • Number of events 2 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
28.6%
2/7 • Number of events 2 • 1 year through treatment
|
|
Infections and infestations
Wound infection
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
28.6%
2/7 • Number of events 2 • 1 year through treatment
|
|
Infections and infestations
Infections and infestations - Other, specify
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 2 • 1 year through treatment
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders - Other, specify
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
50.0%
1/2 • Number of events 1 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Nervous system disorders
Intracranial hemorrhage
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
50.0%
1/2 • Number of events 2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Nervous system disorders
Seizure
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
Renal and urinary disorders
Acute kidney injury
|
28.6%
2/7 • Number of events 2 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
100.0%
2/2 • Number of events 2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 2 • 1 year through treatment
|
|
Respiratory, thoracic and mediastinal disorders
Laryngeal edema
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
Respiratory, thoracic and mediastinal disorders
Stridor
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
Respiratory, thoracic and mediastinal disorders
Bronchopulmonary hemorrhage
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
50.0%
1/2 • Number of events 1 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
0.00%
0/7 • 1 year through treatment
|
6.2%
1/16 • Number of events 1 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Skin and subcutaneous tissue disorders
Toxic epidermal necrolysis
|
0.00%
0/7 • 1 year through treatment
|
6.2%
1/16 • Number of events 1 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
Other adverse events
| Measure |
RDEB Mac
n=7 participants at risk
Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
RDEB RIC
n=16 participants at risk
Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB MAC
n=2 participants at risk
Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Myeloablative Busulfan: Targeting AUC 1000 umol/min
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
JEB RIC
n=7 participants at risk
Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.
Fludarabine: 40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.
Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2.
Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0
Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.
Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.
|
|---|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
28.6%
2/7 • Number of events 2 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
50.0%
1/2 • Number of events 1 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
General disorders
Hypoxia
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
85.7%
6/7 • Number of events 13 • 1 year through treatment
|
12.5%
2/16 • Number of events 2 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
28.6%
2/7 • Number of events 3 • 1 year through treatment
|
|
General disorders
Chills
|
42.9%
3/7 • Number of events 5 • 1 year through treatment
|
6.2%
1/16 • Number of events 1 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Vascular disorders
Hypertension
|
57.1%
4/7 • Number of events 5 • 1 year through treatment
|
75.0%
12/16 • Number of events 25 • 1 year through treatment
|
100.0%
2/2 • Number of events 4 • 1 year through treatment
|
85.7%
6/7 • Number of events 12 • 1 year through treatment
|
|
Vascular disorders
Hypotension
|
0.00%
0/7 • 1 year through treatment
|
18.8%
3/16 • Number of events 3 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
42.9%
3/7 • Number of events 6 • 1 year through treatment
|
|
Skin and subcutaneous tissue disorders
Rash
|
100.0%
7/7 • Number of events 17 • 1 year through treatment
|
6.2%
1/16 • Number of events 1 • 1 year through treatment
|
100.0%
2/2 • Number of events 6 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Infections and infestations
Infections and infestations - Other
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Gastrointestinal disorders
General disorders and administration site conditions - Other, specify
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
6.2%
1/16 • Number of events 1 • 1 year through treatment
|
50.0%
1/2 • Number of events 1 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Cardiac disorders
Sinus Tachycardia
|
85.7%
6/7 • Number of events 14 • 1 year through treatment
|
62.5%
10/16 • Number of events 22 • 1 year through treatment
|
50.0%
1/2 • Number of events 2 • 1 year through treatment
|
85.7%
6/7 • Number of events 11 • 1 year through treatment
|
|
General disorders
Pain
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
General disorders
General disorders - Other
|
0.00%
0/7 • 1 year through treatment
|
6.2%
1/16 • Number of events 1 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Respiratory, thoracic and mediastinal disorders
Stridor
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnea
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.00%
0/7 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
|
Gastrointestinal disorders
Vomiting
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
|
Psychiatric disorders
Agitation
|
14.3%
1/7 • Number of events 1 • 1 year through treatment
|
0.00%
0/16 • 1 year through treatment
|
0.00%
0/2 • 1 year through treatment
|
0.00%
0/7 • 1 year through treatment
|
Additional Information
Dr. Jakub Tolar
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