Trial Outcomes & Findings for Graft-Versus-Host Disease Prophylaxis in Treating Patients With Hematologic Malignancies Undergoing Unrelated Donor Peripheral Blood Stem Cell Transplant (NCT NCT01231412)
NCT ID: NCT01231412
Last Updated: 2019-10-24
Results Overview
Number of patients with grades II-IV acute GVHD aGVHD Stages Skin: 1. a maculopapular eruption involving \< 25% BSA 2. a maculopapular eruption involving 25 - 50% BSA 3. generalized erythroderma 4. generalized erythroderma w/ bullous formation and often w/ desquamation Liver: 1. bilirubin 2.0 - 3.0 mg/100 mL 2. bilirubin 3 - 5.9 mg/100 mL 3. bilirubin 6 - 14.9 mg/100 mL 4. bilirubin \> 15 mg/100 mL Gut: Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall. aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
174 participants
Primary outcome timeframe
At day 100 post-transplant
Results posted on
2019-10-24
Participant Flow
Participant milestones
| Measure |
Arm I (MMF and CSP)
Patients receive FLU IV over 30 minutes on days -4 to -2. Patients also receive CSP PO BID on days -3 to 96 with taper to day 150 and MMF PO TID daily on days 0-29 and then BID on days 30-150 with taper to day 180. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Total-Body Irradiation: Undergo TBI
|
Arm II (MMF, CSP, and Sirolimus)
Patients receive FLU and CSP as in Arm I and sirolimus PO QD on days -3 to 150 with taper to day 180. Patients also receive MMF PO TID on days 0-29 and then BID on days 30-40. MMF will then be discontinued without taper unless GVHD or disease relapse/progression occurs. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Sirolimus: Given PO
Total-Body Irradiation: Undergo TBI
|
Arm 0 (CSP and Sirolimus)
Patients receive CSP orally (PO) twice daily (BID) on days -3 to 96 with taper to day 150 and and sirolimus PO once daily (QD) on days -3 to 150 with taper to day 180. Arm removed as of 14-Sep-2011
|
|---|---|---|---|
|
Overall Study
STARTED
|
77
|
91
|
6
|
|
Overall Study
COMPLETED
|
77
|
90
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Graft-Versus-Host Disease Prophylaxis in Treating Patients With Hematologic Malignancies Undergoing Unrelated Donor Peripheral Blood Stem Cell Transplant
Baseline characteristics by cohort
| Measure |
Arm I (MMF and CSP)
n=77 Participants
Patients receive FLU IV over 30 minutes on days -4 to -2. Patients also receive CSP PO BID on days -3 to 96 with taper to day 150 and MMF PO TID daily on days 0-29 and then BID on days 30-150 with taper to day 180. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Total-Body Irradiation: Undergo TBI
|
Arm II (MMF, CSP, and Sirolimus)
n=91 Participants
Patients receive FLU and CSP as in Arm I and sirolimus PO QD on days -3 to 150 with taper to day 180. Patients also receive MMF PO TID on days 0-29 and then BID on days 30-40. MMF will then be discontinued without taper unless GVHD or disease relapse/progression occurs. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Sirolimus: Given PO
Total-Body Irradiation: Undergo TBI
|
Arm 0 (CSP and Sirolimus)
n=6 Participants
Patients receive CSP orally (PO) twice daily (BID) on days -3 to 96 with taper to day 150 and and sirolimus PO once daily (QD) on days -3 to 150 with taper to day 180. Arm removed as of 14-Sep-2011
|
Total
n=174 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
52 Participants
n=93 Participants
|
50 Participants
n=4 Participants
|
4 Participants
n=27 Participants
|
106 Participants
n=483 Participants
|
|
Age, Categorical
>=65 years
|
25 Participants
n=93 Participants
|
41 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
68 Participants
n=483 Participants
|
|
Age, Continuous
|
61.94 years
n=93 Participants
|
63.75 years
n=4 Participants
|
59.515 years
n=27 Participants
|
62.655 years
n=483 Participants
|
|
Sex: Female, Male
Female
|
27 Participants
n=93 Participants
|
28 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
57 Participants
n=483 Participants
|
|
Sex: Female, Male
Male
|
50 Participants
n=93 Participants
|
63 Participants
n=4 Participants
|
4 Participants
n=27 Participants
|
117 Participants
n=483 Participants
|
|
Region of Enrollment
United States
|
65 participants
n=93 Participants
|
78 participants
n=4 Participants
|
6 participants
n=27 Participants
|
149 participants
n=483 Participants
|
|
Region of Enrollment
Denmark
|
10 participants
n=93 Participants
|
12 participants
n=4 Participants
|
0 participants
n=27 Participants
|
22 participants
n=483 Participants
|
|
Region of Enrollment
Germany
|
2 participants
n=93 Participants
|
1 participants
n=4 Participants
|
0 participants
n=27 Participants
|
3 participants
n=483 Participants
|
PRIMARY outcome
Timeframe: At day 100 post-transplantPopulation: One subject on Arm II aborted transplant during conditioning and subsequently went on to transplant on a different study. This subject was counted towards accrual but not evaluated with respect to outcome measures.
Number of patients with grades II-IV acute GVHD aGVHD Stages Skin: 1. a maculopapular eruption involving \< 25% BSA 2. a maculopapular eruption involving 25 - 50% BSA 3. generalized erythroderma 4. generalized erythroderma w/ bullous formation and often w/ desquamation Liver: 1. bilirubin 2.0 - 3.0 mg/100 mL 2. bilirubin 3 - 5.9 mg/100 mL 3. bilirubin 6 - 14.9 mg/100 mL 4. bilirubin \> 15 mg/100 mL Gut: Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall. aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death
Outcome measures
| Measure |
Arm I (MMF and CSP)
n=77 Participants
Patients receive FLU IV over 30 minutes on days -4 to -2. Patients also receive CSP PO BID on days -3 to 96 with taper to day 150 and MMF PO TID daily on days 0-29 and then BID on days 30-150 with taper to day 180. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Total-Body Irradiation: Undergo TBI
|
Arm II (MMF, CSP, and Sirolimus)
n=90 Participants
Patients receive FLU and CSP as in Arm I and sirolimus PO QD on days -3 to 150 with taper to day 180. Patients also receive MMF PO TID on days 0-29 and then BID on days 30-40. MMF will then be discontinued without taper unless GVHD or disease relapse/progression occurs. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Sirolimus: Given PO
Total-Body Irradiation: Undergo TBI
|
Arm 0 (CSP and Sirolimus)
n=6 Participants
Patients receive CSP orally (PO) twice daily (BID) on days -3 to 96 with taper to day 150 and and sirolimus PO once daily (QD) on days -3 to 150 with taper to day 180. Arm removed as of 14-Sep-2011
|
|---|---|---|---|
|
Number of Patients With Grades II-IV Acute GVHD
|
39 Participants
|
22 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: Up to 1 yearPopulation: One subject on Arm II aborted transplant during conditioning and subsequently went on to transplant on a different study. This subject was counted towards accrual but not evaluated with respect to outcome measures.
Number of patients who developed chronic extensive GVHD post-transplant. The diagnosis of chronic GVHD requires at least one manifestation that is distinctive for chronic GVHD as opposed to acute GVHD. In all cases, infection and others causes must be ruled out in the differential diagnosis of chronic GVHD.
Outcome measures
| Measure |
Arm I (MMF and CSP)
n=77 Participants
Patients receive FLU IV over 30 minutes on days -4 to -2. Patients also receive CSP PO BID on days -3 to 96 with taper to day 150 and MMF PO TID daily on days 0-29 and then BID on days 30-150 with taper to day 180. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Total-Body Irradiation: Undergo TBI
|
Arm II (MMF, CSP, and Sirolimus)
n=90 Participants
Patients receive FLU and CSP as in Arm I and sirolimus PO QD on days -3 to 150 with taper to day 180. Patients also receive MMF PO TID on days 0-29 and then BID on days 30-40. MMF will then be discontinued without taper unless GVHD or disease relapse/progression occurs. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Sirolimus: Given PO
Total-Body Irradiation: Undergo TBI
|
Arm 0 (CSP and Sirolimus)
n=6 Participants
Patients receive CSP orally (PO) twice daily (BID) on days -3 to 96 with taper to day 150 and and sirolimus PO once daily (QD) on days -3 to 150 with taper to day 180. Arm removed as of 14-Sep-2011
|
|---|---|---|---|
|
Number of Patients With Chronic Extensive GVHD
|
38 Participants
|
43 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: Up to 100 daysPopulation: One subject on Arm II aborted transplant during conditioning and subsequently went on to transplant on a different study. This subject was counted towards accrual but not evaluated with respect to outcome measures.
Number of patients with grades III-IV acute GVHD aGVHD Stages Skin: 1. a maculopapular eruption involving \< 25% BSA 2. a maculopapular eruption involving 25 - 50% BSA 3. generalized erythroderma 4. generalized erythroderma w/ bullous formation and often w/ desquamation Liver: 1. bilirubin 2.0 - 3.0 mg/100 mL 2. bilirubin 3 - 5.9 mg/100 mL 3. bilirubin 6 - 14.9 mg/100 mL 4. bilirubin \> 15 mg/100 mL Gut: Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall. aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death
Outcome measures
| Measure |
Arm I (MMF and CSP)
n=77 Participants
Patients receive FLU IV over 30 minutes on days -4 to -2. Patients also receive CSP PO BID on days -3 to 96 with taper to day 150 and MMF PO TID daily on days 0-29 and then BID on days 30-150 with taper to day 180. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Total-Body Irradiation: Undergo TBI
|
Arm II (MMF, CSP, and Sirolimus)
n=90 Participants
Patients receive FLU and CSP as in Arm I and sirolimus PO QD on days -3 to 150 with taper to day 180. Patients also receive MMF PO TID on days 0-29 and then BID on days 30-40. MMF will then be discontinued without taper unless GVHD or disease relapse/progression occurs. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Sirolimus: Given PO
Total-Body Irradiation: Undergo TBI
|
Arm 0 (CSP and Sirolimus)
n=6 Participants
Patients receive CSP orally (PO) twice daily (BID) on days -3 to 96 with taper to day 150 and and sirolimus PO once daily (QD) on days -3 to 150 with taper to day 180. Arm removed as of 14-Sep-2011
|
|---|---|---|---|
|
Number of Patients With Grades III-IV Acute GVHD
|
8 Participants
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 1 yearPopulation: One subject on Arm II aborted transplant during conditioning and subsequently went on to transplant on a different study. This subject was counted towards accrual but not evaluated with respect to outcome measures.
Number of subjects expired without disease progression/relapse.
Outcome measures
| Measure |
Arm I (MMF and CSP)
n=77 Participants
Patients receive FLU IV over 30 minutes on days -4 to -2. Patients also receive CSP PO BID on days -3 to 96 with taper to day 150 and MMF PO TID daily on days 0-29 and then BID on days 30-150 with taper to day 180. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Total-Body Irradiation: Undergo TBI
|
Arm II (MMF, CSP, and Sirolimus)
n=90 Participants
Patients receive FLU and CSP as in Arm I and sirolimus PO QD on days -3 to 150 with taper to day 180. Patients also receive MMF PO TID on days 0-29 and then BID on days 30-40. MMF will then be discontinued without taper unless GVHD or disease relapse/progression occurs. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Sirolimus: Given PO
Total-Body Irradiation: Undergo TBI
|
Arm 0 (CSP and Sirolimus)
n=6 Participants
Patients receive CSP orally (PO) twice daily (BID) on days -3 to 96 with taper to day 150 and and sirolimus PO once daily (QD) on days -3 to 150 with taper to day 180. Arm removed as of 14-Sep-2011
|
|---|---|---|---|
|
Number of Non-Relapse Mortalities
|
12 Participants
|
4 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 1 yearPopulation: One subject on Arm II aborted transplant during conditioning and subsequently went on to transplant on a different study. This subject was counted towards accrual but not evaluated with respect to outcome measures.
Number of subjects surviving overall post-transplant.
Outcome measures
| Measure |
Arm I (MMF and CSP)
n=77 Participants
Patients receive FLU IV over 30 minutes on days -4 to -2. Patients also receive CSP PO BID on days -3 to 96 with taper to day 150 and MMF PO TID daily on days 0-29 and then BID on days 30-150 with taper to day 180. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Total-Body Irradiation: Undergo TBI
|
Arm II (MMF, CSP, and Sirolimus)
n=90 Participants
Patients receive FLU and CSP as in Arm I and sirolimus PO QD on days -3 to 150 with taper to day 180. Patients also receive MMF PO TID on days 0-29 and then BID on days 30-40. MMF will then be discontinued without taper unless GVHD or disease relapse/progression occurs. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Sirolimus: Given PO
Total-Body Irradiation: Undergo TBI
|
Arm 0 (CSP and Sirolimus)
n=6 Participants
Patients receive CSP orally (PO) twice daily (BID) on days -3 to 96 with taper to day 150 and and sirolimus PO once daily (QD) on days -3 to 150 with taper to day 180. Arm removed as of 14-Sep-2011
|
|---|---|---|---|
|
Number of of Participants Surviving Overall
|
53 Participants
|
75 Participants
|
6 Participants
|
SECONDARY outcome
Timeframe: Up to 1 yearPopulation: One subject on Arm II aborted transplant during conditioning and subsequently went on to transplant on a different study. This subject was counted towards accrual but not evaluated with respect to outcome measures.
Relapse/Progression criteria: CML New cytogenetic abnormality and/or development of accelerated phase or blast crisis. The criteria for accelerated phase will be defined as unexplained fever \>38.3°C, new clonal cytogenetic abnormalities in addition to a single Ph-positive chromosome, marrow blasts and promyelocytes \>20%. AML, ALL, MDS \>5% blasts by morphologic or flow cytometric evaluation of the BMA or appearance of extramedullary disease CLL ≥1 of: Physical exam/imaging studies ≥50% increase or new, circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, and lymph node biopsy w/ Richter's transformation. NHL \>25% increase in the sum of the products of the perpendicular diameters of marker lesions, or the appearance of new lesions. MM ≥100% increase of the serum myeloma protein from its lowest level, or reappearance of myeloma peaks that had disappeared w/ treatment; or definite increase in the size or numb
Outcome measures
| Measure |
Arm I (MMF and CSP)
n=77 Participants
Patients receive FLU IV over 30 minutes on days -4 to -2. Patients also receive CSP PO BID on days -3 to 96 with taper to day 150 and MMF PO TID daily on days 0-29 and then BID on days 30-150 with taper to day 180. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Total-Body Irradiation: Undergo TBI
|
Arm II (MMF, CSP, and Sirolimus)
n=90 Participants
Patients receive FLU and CSP as in Arm I and sirolimus PO QD on days -3 to 150 with taper to day 180. Patients also receive MMF PO TID on days 0-29 and then BID on days 30-40. MMF will then be discontinued without taper unless GVHD or disease relapse/progression occurs. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Sirolimus: Given PO
Total-Body Irradiation: Undergo TBI
|
Arm 0 (CSP and Sirolimus)
n=6 Participants
Patients receive CSP orally (PO) twice daily (BID) on days -3 to 96 with taper to day 150 and and sirolimus PO once daily (QD) on days -3 to 150 with taper to day 180. Arm removed as of 14-Sep-2011
|
|---|---|---|---|
|
Number of Participants With Relapse/Progression
|
16 Participants
|
16 Participants
|
1 Participants
|
Adverse Events
Arm I (MMF and CSP)
Serious events: 0 serious events
Other events: 27 other events
Deaths: 0 deaths
Arm II (MMF, CSP, and Sirolimus)
Serious events: 0 serious events
Other events: 26 other events
Deaths: 0 deaths
Arm 0 (CSP and Sirolimus)
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Arm I (MMF and CSP)
n=77 participants at risk
Patients receive FLU IV over 30 minutes on days -4 to -2. Patients also receive CSP PO BID on days -3 to 96 with taper to day 150 and MMF PO TID daily on days 0-29 and then BID on days 30-150 with taper to day 180. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Total-Body Irradiation: Undergo TBI
|
Arm II (MMF, CSP, and Sirolimus)
n=90 participants at risk
Patients receive FLU and CSP as in Arm I and sirolimus PO QD on days -3 to 150 with taper to day 180. Patients also receive MMF PO TID on days 0-29 and then BID on days 30-40. MMF will then be discontinued without taper unless GVHD or disease relapse/progression occurs. Patients undergo allogeneic PBSCT on day 0 following the TBI.
Allogeneic Hematopoietic Stem Cell Transplantation: Undergo allogeneic PBSCT
Cyclosporine: Given PO or IV
Fludarabine Phosphate: Given IV
Mycophenolate Mofetil: Given PO
Peripheral Blood Stem Cell Transplantation: Undergo allogeneic PBSCT
Sirolimus: Given PO
Total-Body Irradiation: Undergo TBI
|
Arm 0 (CSP and Sirolimus)
n=6 participants at risk
Patients receive CSP orally (PO) twice daily (BID) on days -3 to 96 with taper to day 150 and and sirolimus PO once daily (QD) on days -3 to 150 with taper to day 180. Arm removed as of 14-Sep-2011
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/77 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
3.3%
3/90 • Number of events 3 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
"Adult respiratory distress syndrome"
|
3.9%
3/77 • Number of events 3 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
16.7%
1/6 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Investigations
Alanine aminotransferase increased
|
2.6%
2/77 • Number of events 2 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Immune system disorders
Allergic reaction
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Investigations
Aspartate aminotransferase increased
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
1.1%
1/90 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Cardiac disorders
Atrial fibrillation
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
1.1%
1/90 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Investigations
Blood bilirubin increased
|
9.1%
7/77 • Number of events 7 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
1.1%
1/90 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
16.7%
1/6 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
Bronchopulmonary hemorrhage
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
2.2%
2/90 • Number of events 2 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Renal and urinary disorders
Chronic kidney disease
|
0.00%
0/77 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
1.1%
1/90 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Gastrointestinal disorders
Colonic hemorrhage
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Investigations
Creatinine increased
|
5.2%
4/77 • Number of events 4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
4.4%
4/90 • Number of events 4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Gastrointestinal disorders
Diarrhea
|
2.6%
2/77 • Number of events 2 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
1.1%
1/90 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Infections and infestations
Encephalitis infection
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Infections and infestations
Enterocolitis infectious
|
0.00%
0/77 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
1.1%
1/90 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
7.8%
6/77 • Number of events 7 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
3.3%
3/90 • Number of events 3 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Gastrointestinal disorders
Fecal incontinence
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
General disorders
Fever
|
0.00%
0/77 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
1.1%
1/90 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Gastrointestinal disorders
Gastric ulcer
|
0.00%
0/77 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
1.1%
1/90 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Gastrointestinal disorders
Gastritis
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Blood and lymphatic system disorders
Hemolysis
|
3.9%
3/77 • Number of events 3 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
0.00%
0/77 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
4.4%
4/90 • Number of events 4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Vascular disorders
Hypotension
|
2.6%
2/77 • Number of events 3 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
7.8%
6/77 • Number of events 6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
3.3%
3/90 • Number of events 4 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
Laryngeal inflammation
|
0.00%
0/77 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
16.7%
1/6 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
Lung infection
|
3.9%
3/77 • Number of events 3 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Gastrointestinal disorders
Mucositis oral
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
2.2%
2/90 • Number of events 2 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/77 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
1.1%
1/90 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Infections and infestations
Pleural infection
|
0.00%
0/77 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
1.1%
1/90 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Renal and urinary disorders
Renal and urinary disorders - Other (Hemorrhagic cystitis)
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
2.6%
2/77 • Number of events 2 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
1.1%
1/90 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Infections and infestations
Sepsis
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Infections and infestations
Skin infection
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Nervous system disorders
Syncope
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
1.1%
1/90 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
16.7%
1/6 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Vascular disorders
Thromboembolic event
|
2.6%
2/77 • Number of events 3 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
1.1%
1/90 • Number of events 2 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Treatment related secondary malignancy
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Renal and urinary disorders
Urinary retention
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Cardiac disorders
Ventricular arrhythmia
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
|
Investigations
Weight loss
|
1.3%
1/77 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/90 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
0.00%
0/6 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
|
Additional Information
Dr. Brenda M. Sandmaier
Fred Hutchinson Cancer Research Center
Phone: (206) 667-4961
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place