Trial Outcomes & Findings for Stem Cell Transplant in Treating Patients With Acute Myeloid Leukemia (NCT NCT00630565)
NCT ID: NCT00630565
Last Updated: 2024-01-29
Results Overview
Percentage of Participants with Engraftment measured by myeloid, platelet, and erythroid recovery
Recruitment status
TERMINATED
Study phase
PHASE2/PHASE3
Target enrollment
12 participants
Primary outcome timeframe
30 Days Post Transplant
Results posted on
2024-01-29
Participant Flow
Participant milestones
| Measure |
Bone Marrow Transplant (2-70 Years Old)
Patients over the age of two will receive a cytoreductive regimen of total-body irradiation and cyclophosphamide (TBI/CY) as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation).
sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed
cyclophosphamide: 4 gm/m\^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2.
dexamethasone: 20 mg/m\^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours
etoposide: 300 mg/m\^2/day x 2 days (day 0-1) over 3 hours intravenously (IV)
bone marrow transplantation: Day 0 infusion of bone marrow cells
peripheral blood stem cell transplantation: Day 0 infusion of peripheral blood stem cells
total-body irradiation: 165 cGy/dose given twice a day on days -7 through -4.
|
Bone Marrow Transplant (Less and 2 Years Old)
Patients under the age of two, and patients who cannot receive total body irradiation (TBI), will receive a cytoreductive regimen of Busulfan and cyclophosphamide (BU/CY) as per the Johns Hopkins University Hospital regimen as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation).
sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed
busulfan: 4 mg/kg po in 4 divided doses (.8 mg/kg/dose orally every 6 hours) on days -7 through -4.
cyclophosphamide: 4 gm/m\^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2.
dexamethasone: 20 mg/m\^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours
etoposide: 300 mg/m\^2/day x 2 days (day 0-1) over 3 hours intravenously (IV)
hematopoietic stem cell transplantation: Stem cell infusion (\>48 hours after the last dose of cyclophosphamide)
|
|---|---|---|
|
Overall Study
STARTED
|
12
|
0
|
|
Overall Study
COMPLETED
|
12
|
0
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Stem Cell Transplant in Treating Patients With Acute Myeloid Leukemia
Baseline characteristics by cohort
| Measure |
Bone Marrow Transplant (2-70 Years Old)
n=12 Participants
Patients over the age of two will receive a cytoreductive regimen of total-body irradiation and cyclophosphamide (TBI/CY) as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation).
sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed
cyclophosphamide: 4 gm/m\^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2.
dexamethasone: 20 mg/m\^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours
etoposide: 300 mg/m\^2/day x 2 days (day 0-1) over 3 hours intravenously (IV)
bone marrow transplantation: Day 0 infusion of bone marrow cells
peripheral blood stem cell transplantation: Day 0 infusion of peripheral blood stem cells
total-body irradiation: 165 cGy/dose given twice a day on days -7 through -4.
|
Bone Marrow Transplant (Less and 2 Years Old)
Patients under the age of two, and patients who cannot receive total body irradiation (TBI), will receive a cytoreductive regimen of Busulfan and cyclophosphamide (BU/CY) as per the Johns Hopkins University Hospital regimen as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation).
sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed
busulfan: 4 mg/kg po in 4 divided doses (.8 mg/kg/dose orally every 6 hours) on days -7 through -4.
cyclophosphamide: 4 gm/m\^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2.
dexamethasone: 20 mg/m\^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours
etoposide: 300 mg/m\^2/day x 2 days (day 0-1) over 3 hours intravenously (IV)
hematopoietic stem cell transplantation: Stem cell infusion (\>48 hours after the last dose of cyclophosphamide)
|
Total
n=12 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
12 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
11 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=5 Participants
|
—
|
12 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 30 Days Post TransplantPercentage of Participants with Engraftment measured by myeloid, platelet, and erythroid recovery
Outcome measures
| Measure |
Bone Marrow Transplant (2-70 Years Old)
n=12 Participants
Patients over the age of two will receive a cytoreductive regimen of total-body irradiation and cyclophosphamide (TBI/CY) as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation).
sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed
cyclophosphamide: 4 gm/m\^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2.
dexamethasone: 20 mg/m\^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours
etoposide: 300 mg/m\^2/day x 2 days (day 0-1) over 3 hours intravenously (IV)
bone marrow transplantation: Day 0 infusion of bone marrow cells
peripheral blood stem cell transplantation: Day 0 infusion of peripheral blood stem cells
total-body irradiation: 165 cGy/dose given twice a day on days -7 through -4.
|
|---|---|
|
Percentage of Participants With Engraftment
|
100 Participants of participants
Interval 76.0 to 100.0
|
SECONDARY outcome
Timeframe: 2 years Post TransplantDisease evaluation will be completed approximately 100 days after stem cell infusion and every 6 months, 1 year, and until 2 years after infusion.
Outcome measures
| Measure |
Bone Marrow Transplant (2-70 Years Old)
n=12 Participants
Patients over the age of two will receive a cytoreductive regimen of total-body irradiation and cyclophosphamide (TBI/CY) as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation).
sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed
cyclophosphamide: 4 gm/m\^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2.
dexamethasone: 20 mg/m\^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours
etoposide: 300 mg/m\^2/day x 2 days (day 0-1) over 3 hours intravenously (IV)
bone marrow transplantation: Day 0 infusion of bone marrow cells
peripheral blood stem cell transplantation: Day 0 infusion of peripheral blood stem cells
total-body irradiation: 165 cGy/dose given twice a day on days -7 through -4.
|
|---|---|
|
Percentage of Participants With Disease Response
|
83 Percentage of participants
Interval 46.0 to 95.0
|
SECONDARY outcome
Timeframe: 2 years Post TransplantPercentage of participants experiencing treatment failure.
Outcome measures
| Measure |
Bone Marrow Transplant (2-70 Years Old)
n=12 Participants
Patients over the age of two will receive a cytoreductive regimen of total-body irradiation and cyclophosphamide (TBI/CY) as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation).
sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed
cyclophosphamide: 4 gm/m\^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2.
dexamethasone: 20 mg/m\^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours
etoposide: 300 mg/m\^2/day x 2 days (day 0-1) over 3 hours intravenously (IV)
bone marrow transplantation: Day 0 infusion of bone marrow cells
peripheral blood stem cell transplantation: Day 0 infusion of peripheral blood stem cells
total-body irradiation: 165 cGy/dose given twice a day on days -7 through -4.
|
|---|---|
|
Treatment Failure
|
25 Percentage of participants
|
SECONDARY outcome
Timeframe: 2 years Post TransplantDescription: (e.g., thyroid function abnormalities - T4, TSH, gonadal abnormalities, cataracts, pulmonary dysfunctions, growth and development abnormalities, and second malignant neoplasms)
Outcome measures
| Measure |
Bone Marrow Transplant (2-70 Years Old)
n=12 Participants
Patients over the age of two will receive a cytoreductive regimen of total-body irradiation and cyclophosphamide (TBI/CY) as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation).
sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed
cyclophosphamide: 4 gm/m\^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2.
dexamethasone: 20 mg/m\^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours
etoposide: 300 mg/m\^2/day x 2 days (day 0-1) over 3 hours intravenously (IV)
bone marrow transplantation: Day 0 infusion of bone marrow cells
peripheral blood stem cell transplantation: Day 0 infusion of peripheral blood stem cells
total-body irradiation: 165 cGy/dose given twice a day on days -7 through -4.
|
|---|---|
|
Percent of Patients With Various Late Effects
|
0 Participants
|
SECONDARY outcome
Timeframe: 2 years Post TransplantDisease-free survival 2 years Post Transplant
Outcome measures
| Measure |
Bone Marrow Transplant (2-70 Years Old)
n=12 Participants
Patients over the age of two will receive a cytoreductive regimen of total-body irradiation and cyclophosphamide (TBI/CY) as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation).
sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed
cyclophosphamide: 4 gm/m\^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2.
dexamethasone: 20 mg/m\^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours
etoposide: 300 mg/m\^2/day x 2 days (day 0-1) over 3 hours intravenously (IV)
bone marrow transplantation: Day 0 infusion of bone marrow cells
peripheral blood stem cell transplantation: Day 0 infusion of peripheral blood stem cells
total-body irradiation: 165 cGy/dose given twice a day on days -7 through -4.
|
|---|---|
|
Disease-free Survival
|
73 Percentage of participants with DFS
Interval 38.0 to 91.0
|
SECONDARY outcome
Timeframe: Pre-TransplantThe proportion of primed patients with adequate number of cells collected will be calculated.
Outcome measures
| Measure |
Bone Marrow Transplant (2-70 Years Old)
n=12 Participants
Patients over the age of two will receive a cytoreductive regimen of total-body irradiation and cyclophosphamide (TBI/CY) as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation).
sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed
cyclophosphamide: 4 gm/m\^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2.
dexamethasone: 20 mg/m\^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours
etoposide: 300 mg/m\^2/day x 2 days (day 0-1) over 3 hours intravenously (IV)
bone marrow transplantation: Day 0 infusion of bone marrow cells
peripheral blood stem cell transplantation: Day 0 infusion of peripheral blood stem cells
total-body irradiation: 165 cGy/dose given twice a day on days -7 through -4.
|
|---|---|
|
Percentage of Patients With Adequate Cells Collected
|
75 Percentage of participants
Interval 47.0 to 91.0
|
Adverse Events
Bone Marrow Transplant (2-70 Years Old)
Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Bone Marrow Transplant (2-70 Years Old)
n=12 participants at risk
Patients over the age of two will receive a cytoreductive regimen of total-body irradiation and cyclophosphamide (TBI/CY) as well as sargramostim, dexamethasone, etoposide, transplantation (bone marrow transplantation/hematopoietic stem cell transplantation/peripheral blood stem cell transplantation).
sargramostim: Given subcutaneously (SC) 10 μg/kg/day from day +3 until apheresis is completed
cyclophosphamide: 4 gm/m\^2 x 1 (day 0) and 60 mg/kg intravenous (IV) over 2 hours on days -3 and -2.
dexamethasone: 20 mg/m\^2 x 4 doses every 12 hours given intravenously (IV) push before cytoxan on day 0 and then every 12 hours
etoposide: 300 mg/m\^2/day x 2 days (day 0-1) over 3 hours intravenously (IV)
bone marrow transplantation: Day 0 infusion of bone marrow cells
peripheral blood stem cell transplantation: Day 0 infusion of peripheral blood stem cells
total-body irradiation: 165 cGy/dose given twice a day on days -7 through -4.
|
|---|---|
|
Blood and lymphatic system disorders
Relapse
|
8.3%
1/12 • Number of events 1 • 2 years
|
Other adverse events
Adverse event data not reported
Additional Information
Dr. Daniel Weisdorf, MD
University of Minnesota, Masonic Cancer Center
Phone: +1 612-624-3101
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place