Trial Outcomes & Findings for Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA (NCT NCT00455312)
NCT ID: NCT00455312
Last Updated: 2017-12-05
Results Overview
Defined as an absolute neutrophil count (ANC) \>5 x 10\^8/L (first of three consecutive laboratory measurements on different days) with at least 10% donor cells by day 100. Demonstrate sustained engraftment after a fludarabine based preparative regimen in patients with dyskeratosis congenita followed by hematopoietic cell transplantation.
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
36 participants
Primary outcome timeframe
Day 100
Results posted on
2017-12-05
Participant Flow
Participant milestones
| Measure |
Patients With DC
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
Patients With SAA
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin (ATG), total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
|---|---|---|
|
Overall Study
STARTED
|
15
|
21
|
|
Overall Study
COMPLETED
|
15
|
20
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
Patients With DC
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
Patients With SAA
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin (ATG), total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
|---|---|---|
|
Overall Study
Death
|
0
|
1
|
Baseline Characteristics
Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Baseline characteristics by cohort
| Measure |
Patients With SAA
n=21 Participants
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
Total
n=36 Participants
Total of all reporting groups
|
Patients With DC
n=15 Participants
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
15 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
6 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
9 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
10 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 100Defined as an absolute neutrophil count (ANC) \>5 x 10\^8/L (first of three consecutive laboratory measurements on different days) with at least 10% donor cells by day 100. Demonstrate sustained engraftment after a fludarabine based preparative regimen in patients with dyskeratosis congenita followed by hematopoietic cell transplantation.
Outcome measures
| Measure |
Patients With DC
n=15 Participants
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
Patients With SAA
n=20 Participants
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
|---|---|---|
|
Neutrophil Engraftment
|
15 Participants
|
20 Participants
|
SECONDARY outcome
Timeframe: 100 daysall deaths without previous relapse or progression
Outcome measures
| Measure |
Patients With DC
n=15 Participants
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
Patients With SAA
n=21 Participants
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
|---|---|---|
|
Incidence of Regimen Related Mortality at 100 Days
|
1 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: 6 monthsChronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host.
Outcome measures
| Measure |
Patients With DC
n=15 Participants
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
Patients With SAA
n=21 Participants
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
|---|---|---|
|
Incidence of Chronic GVHD
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 1 yearChronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host.
Outcome measures
| Measure |
Patients With DC
n=15 Participants
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
Patients With SAA
n=21 Participants
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
|---|---|---|
|
Incidence of Chronic GVHD
|
1 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: 1 YearDefined as patients who have a secondary malignancy (cancer) occurring.
Outcome measures
| Measure |
Patients With DC
n=15 Participants
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
Patients With SAA
n=21 Participants
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
|---|---|---|
|
Incidence of Late Secondary Malignancies
|
0 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: Day 100Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.
Outcome measures
| Measure |
Patients With DC
n=15 Participants
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
Patients With SAA
n=21 Participants
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
|---|---|---|
|
Incidence of Grade 2-4 Acute Graft Versus Host Disease (GVHD)
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 100Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.
Outcome measures
| Measure |
Patients With DC
n=15 Participants
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
Patients With SAA
n=21 Participants
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
|---|---|---|
|
Incidence of Grade 3-4 Acute Graft Versus Host Disease (GVHD)
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 100Overall survival is defined as time from date of transplant to date of death or censored at the date of last documented contact for patients still alive.
Outcome measures
| Measure |
Patients With DC
n=15 Participants
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
Patients With SAA
n=21 Participants
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
|---|---|---|
|
Overall Survival
|
14 Participants
|
19 Participants
|
SECONDARY outcome
Timeframe: 1 YearOverall survival is defined as time from date of transplant to date of death or censored at the date of last documented contact for patients still alive.
Outcome measures
| Measure |
Patients With DC
n=15 Participants
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
Patients With SAA
n=21 Participants
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
|---|---|---|
|
Overall Survival
|
12 Participants
|
19 Participants
|
SECONDARY outcome
Timeframe: 6 MonthsDefined as patients who exhibit a pulmonary (lung) adverse event.
Outcome measures
| Measure |
Patients With DC
n=15 Participants
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
Patients With SAA
n=21 Participants
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
|---|---|---|
|
Incidence of Pulmonary Complications
|
3 Participants
|
3 Participants
|
Adverse Events
Patients With DC
Serious events: 2 serious events
Other events: 15 other events
Deaths: 0 deaths
Patients With SAA
Serious events: 1 serious events
Other events: 20 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Patients With DC
n=15 participants at risk
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
Patients With SAA
n=21 participants at risk
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Adult Respiratory Distress Syndrome
|
0.00%
0/15
|
4.8%
1/21
|
|
Nervous system disorders
Intracranial Hemorrhage
|
6.7%
1/15
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Failure
|
6.7%
1/15
|
0.00%
0/21
|
|
Renal and urinary disorders
Renal Failure
|
6.7%
1/15
|
0.00%
0/21
|
Other adverse events
| Measure |
Patients With DC
n=15 participants at risk
Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.
Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
|
Patients With SAA
n=21 participants at risk
Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.
Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell Transplantation: Infusion of stem cells on Day 0.
antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.
Methylprednisolone: 2mg/kg IV is given before each dose of ATG.
|
|---|---|---|
|
Nervous system disorders
Acute Brain Infarction
|
0.00%
0/15
|
4.8%
1/21
|
|
Renal and urinary disorders
Acute Kidney Injury
|
0.00%
0/15
|
4.8%
1/21
|
|
Gastrointestinal disorders
Acute Pancreatitis
|
0.00%
0/15
|
4.8%
1/21
|
|
Respiratory, thoracic and mediastinal disorders
Acute Respiratory Distress Syndrome
|
20.0%
3/15
|
9.5%
2/21
|
|
Musculoskeletal and connective tissue disorders
Aseptic/Avascular Necrosis
|
13.3%
2/15
|
4.8%
1/21
|
|
Cardiac disorders
Atrial Fibrillation
|
0.00%
0/15
|
4.8%
1/21
|
|
Infections and infestations
Blood Infection
|
66.7%
10/15
|
66.7%
14/21
|
|
Infections and infestations
Bone Marrow Biopsy Site Infection
|
6.7%
1/15
|
0.00%
0/21
|
|
Infections and infestations
Bone Marrow Infection
|
0.00%
0/15
|
4.8%
1/21
|
|
Vascular disorders
Capillary Leak Syndrome
|
0.00%
0/15
|
4.8%
1/21
|
|
Infections and infestations
Cellulitis
|
13.3%
2/15
|
9.5%
2/21
|
|
Investigations
Coagulopathy
|
0.00%
0/15
|
4.8%
1/21
|
|
Infections and infestations
Colitis
|
0.00%
0/15
|
4.8%
1/21
|
|
Cardiac disorders
Congestive Heart Failure
|
6.7%
1/15
|
0.00%
0/21
|
|
Renal and urinary disorders
Cystitis
|
13.3%
2/15
|
14.3%
3/21
|
|
Blood and lymphatic system disorders
Cytopenias
|
0.00%
0/15
|
4.8%
1/21
|
|
Vascular disorders
Deep Vein Thrombosis
|
6.7%
1/15
|
4.8%
1/21
|
|
Infections and infestations
Ear Infection
|
6.7%
1/15
|
0.00%
0/21
|
|
General disorders
Early Small Bowel Obstruction vs. Ileus
|
6.7%
1/15
|
0.00%
0/21
|
|
Infections and infestations
Epstein Barr Virus Infection
|
0.00%
0/15
|
4.8%
1/21
|
|
Skin and subcutaneous tissue disorders
Erythema at Central Line Site
|
0.00%
0/15
|
4.8%
1/21
|
|
Blood and lymphatic system disorders
Erythroid Aplasia
|
0.00%
0/15
|
4.8%
1/21
|
|
Infections and infestations
Eye Infection
|
6.7%
1/15
|
0.00%
0/21
|
|
General disorders
Fever
|
0.00%
0/15
|
4.8%
1/21
|
|
General disorders
Fever with Chills
|
0.00%
0/15
|
4.8%
1/21
|
|
Skin and subcutaneous tissue disorders
Folliculitis
|
6.7%
1/15
|
0.00%
0/21
|
|
Hepatobiliary disorders
Gallstones
|
0.00%
0/15
|
4.8%
1/21
|
|
Gastrointestinal disorders
GI Bleed
|
20.0%
3/15
|
9.5%
2/21
|
|
Infections and infestations
GI Infection
|
6.7%
1/15
|
19.0%
4/21
|
|
Gastrointestinal disorders
Hemorrhagic Pancreatitis
|
0.00%
0/15
|
4.8%
1/21
|
|
Infections and infestations
Herpes Zoster
|
6.7%
1/15
|
4.8%
1/21
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
0.00%
0/15
|
4.8%
1/21
|
|
Vascular disorders
Hypertension
|
26.7%
4/15
|
38.1%
8/21
|
|
Endocrine disorders
Hypogonadism
|
6.7%
1/15
|
0.00%
0/21
|
|
Vascular disorders
Hypotension
|
0.00%
0/15
|
4.8%
1/21
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/15
|
4.8%
1/21
|
|
Infections and infestations
Infection at Central Line Site
|
0.00%
0/15
|
4.8%
1/21
|
|
Infections and infestations
Labia Infection
|
6.7%
1/15
|
0.00%
0/21
|
|
Blood and lymphatic system disorders
Low Reticulocyte Count
|
0.00%
0/15
|
4.8%
1/21
|
|
Respiratory, thoracic and mediastinal disorders
Lung Collapse
|
0.00%
0/15
|
4.8%
1/21
|
|
Nervous system disorders
Meningitis
|
6.7%
1/15
|
0.00%
0/21
|
|
General disorders
Multi Organ Failure
|
6.7%
1/15
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Infection
|
6.7%
1/15
|
0.00%
0/21
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/15
|
4.8%
1/21
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/15
|
4.8%
1/21
|
|
Gastrointestinal disorders
Anorexia
|
0.00%
0/15
|
4.8%
1/21
|
|
Nervous system disorders
Neuropathy
|
6.7%
1/15
|
4.8%
1/21
|
|
Nervous system disorders
Brain Lesion, Nonspecific
|
0.00%
0/15
|
4.8%
1/21
|
|
Respiratory, thoracic and mediastinal disorders
Nose Bleeds
|
0.00%
0/15
|
4.8%
1/21
|
|
Ear and labyrinth disorders
Otomastoiditis
|
0.00%
0/15
|
4.8%
1/21
|
|
Cardiac disorders
Pericardial Effusion
|
6.7%
1/15
|
4.8%
1/21
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
26.7%
4/15
|
47.6%
10/21
|
|
Infections and infestations
Intra-Abdominal Infection, Unconfirmed
|
0.00%
0/15
|
4.8%
1/21
|
|
Cardiac disorders
Pulmonary Hypertension
|
0.00%
0/15
|
4.8%
1/21
|
|
Blood and lymphatic system disorders
Post Transplant Lymphoproliferative Disease
|
0.00%
0/15
|
4.8%
1/21
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Hemorrhage
|
6.7%
1/15
|
4.8%
1/21
|
|
Skin and subcutaneous tissue disorders
Pustular Rash
|
6.7%
1/15
|
0.00%
0/21
|
|
Renal and urinary disorders
Pyelocaliectasis
|
0.00%
0/15
|
4.8%
1/21
|
|
Renal and urinary disorders
Renal Injury Requiring Dialysis
|
0.00%
0/15
|
4.8%
1/21
|
|
Infections and infestations
Respiratory Infection
|
6.7%
1/15
|
14.3%
3/21
|
|
Eye disorders
Retinal Hemorrhage
|
6.7%
1/15
|
0.00%
0/21
|
|
Nervous system disorders
Seizure
|
0.00%
0/15
|
4.8%
1/21
|
|
Infections and infestations
Sepsis
|
6.7%
1/15
|
4.8%
1/21
|
|
Respiratory, thoracic and mediastinal disorders
Sinusitis
|
6.7%
1/15
|
4.8%
1/21
|
|
Infections and infestations
Skin Infection
|
13.3%
2/15
|
14.3%
3/21
|
|
Skin and subcutaneous tissue disorders
Spongiotic Dermatitis
|
6.7%
1/15
|
0.00%
0/21
|
|
Infections and infestations
Tonsilar Infection
|
0.00%
0/15
|
4.8%
1/21
|
|
Investigations
Transaminitis
|
0.00%
0/15
|
9.5%
2/21
|
|
Infections and infestations
Urinary Tract Infection
|
13.3%
2/15
|
0.00%
0/21
|
|
Infections and infestations
Vaginal Infection
|
0.00%
0/15
|
4.8%
1/21
|
|
Hepatobiliary disorders
Veno-Occlusive Disease
|
0.00%
0/15
|
4.8%
1/21
|
|
Respiratory, thoracic and mediastinal disorders
Vocal Fold Erythema and Leukoplakia
|
6.7%
1/15
|
0.00%
0/21
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place