Trial Outcomes & Findings for Novel Approaches for Graft-versus-Host Disease Prevention Compared to Contemporary Controls (BMT CTN 1203) (NCT NCT02208037)
NCT ID: NCT02208037
Last Updated: 2019-01-23
Results Overview
GRFS is defined as being free of grade III-IV acute GVHD onset, chronic GVHD onset requiring systemic immunosuppressive therapy, disease relapse or progression, and death from any cause.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
279 participants
Primary outcome timeframe
1 Year Post-transplant
Results posted on
2019-01-23
Participant Flow
Participant milestones
| Measure |
Tacrolimus/Methotrexate/Bortezomib
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Overall Study
STARTED
|
93
|
93
|
93
|
|
Overall Study
COMPLETED
|
89
|
92
|
92
|
|
Overall Study
NOT COMPLETED
|
4
|
1
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Novel Approaches for Graft-versus-Host Disease Prevention Compared to Contemporary Controls (BMT CTN 1203)
Baseline characteristics by cohort
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
Total
n=273 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
64 years
n=5 Participants
|
64 years
n=7 Participants
|
64 years
n=5 Participants
|
64 years
n=4 Participants
|
|
Sex: Female, Male
Female
|
31 Participants
n=5 Participants
|
37 Participants
n=7 Participants
|
30 Participants
n=5 Participants
|
98 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
58 Participants
n=5 Participants
|
55 Participants
n=7 Participants
|
62 Participants
n=5 Participants
|
175 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
11 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
85 Participants
n=5 Participants
|
86 Participants
n=7 Participants
|
81 Participants
n=5 Participants
|
252 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
10 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
6 Participants
n=4 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
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
79 Participants
n=5 Participants
|
81 Participants
n=7 Participants
|
82 Participants
n=5 Participants
|
242 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
16 Participants
n=4 Participants
|
|
HCT-CI Comorbidity Index
0
|
24 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
68 Participants
n=4 Participants
|
|
HCT-CI Comorbidity Index
1-2
|
31 Participants
n=5 Participants
|
37 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
95 Participants
n=4 Participants
|
|
HCT-CI Comorbidity Index
3 or greater
|
34 Participants
n=5 Participants
|
37 Participants
n=7 Participants
|
39 Participants
n=5 Participants
|
110 Participants
n=4 Participants
|
|
Karnofsky Performance Score
100
|
13 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
34 Participants
n=4 Participants
|
|
Karnofsky Performance Score
90
|
39 Participants
n=5 Participants
|
39 Participants
n=7 Participants
|
42 Participants
n=5 Participants
|
120 Participants
n=4 Participants
|
|
Karnofsky Performance Score
80
|
29 Participants
n=5 Participants
|
27 Participants
n=7 Participants
|
28 Participants
n=5 Participants
|
84 Participants
n=4 Participants
|
|
Karnofsky Performance Score
70
|
8 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
35 Participants
n=4 Participants
|
|
HLA Matching and Donor Type
Matched Sibling (6/6)
|
29 Participants
n=5 Participants
|
33 Participants
n=7 Participants
|
29 Participants
n=5 Participants
|
91 Participants
n=4 Participants
|
|
HLA Matching and Donor Type
Matched Other Relative (6/6)
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
|
HLA Matching and Donor Type
Matched Unrelated (8/8)
|
53 Participants
n=5 Participants
|
48 Participants
n=7 Participants
|
50 Participants
n=5 Participants
|
151 Participants
n=4 Participants
|
|
HLA Matching and Donor Type
Mismatched Unrelated (7/8)
|
6 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
25 Participants
n=4 Participants
|
|
Primary Diagnosis
Acute Myeloid Leukemia (AML)
|
46 Participants
n=5 Participants
|
49 Participants
n=7 Participants
|
49 Participants
n=5 Participants
|
144 Participants
n=4 Participants
|
|
Primary Diagnosis
Acute Lymphoblastic Leukemia (ALL)
|
12 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
31 Participants
n=4 Participants
|
|
Primary Diagnosis
Chronic Myelogeneous Leukemia (CML)
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
|
Primary Diagnosis
Chronic Lymphocytic Leukemia (CLL)
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
Primary Diagnosis
Myelodysplastic Syndrome (MDS)
|
16 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
48 Participants
n=4 Participants
|
|
Primary Diagnosis
Follicular Lymphoma
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
14 Participants
n=4 Participants
|
|
Primary Diagnosis
Diffuse Large B-Cell Lymphoma
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
12 Participants
n=4 Participants
|
|
Primary Diagnosis
Mantle Cell Lymphoma
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
9 Participants
n=4 Participants
|
|
Primary Diagnosis
Hodgkin's Lymphoma
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Time from Diagnosis to Transplant
|
7.1 months
n=5 Participants
|
6.3 months
n=7 Participants
|
7.4 months
n=5 Participants
|
7.1 months
n=4 Participants
|
|
Donor/Recipient Sex Matching
Male donor / Male Recipient
|
44 Participants
n=5 Participants
|
39 Participants
n=7 Participants
|
43 Participants
n=5 Participants
|
126 Participants
n=4 Participants
|
|
Donor/Recipient Sex Matching
Male donor / Female Recipient
|
19 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
57 Participants
n=4 Participants
|
|
Donor/Recipient Sex Matching
Female donor / Male Recipient
|
14 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
49 Participants
n=4 Participants
|
|
Donor/Recipient Sex Matching
Female donor / Female Recipient
|
12 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
41 Participants
n=4 Participants
|
|
Donor/Recipient Cytomegalovirus (CMV) Status
Donor Positive / Recipient Positive
|
29 Participants
n=5 Participants
|
28 Participants
n=7 Participants
|
25 Participants
n=5 Participants
|
82 Participants
n=4 Participants
|
|
Donor/Recipient Cytomegalovirus (CMV) Status
Donor Positive / Recipient Negative
|
4 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
30 Participants
n=4 Participants
|
|
Donor/Recipient Cytomegalovirus (CMV) Status
Donor Negative / Recipient Positive
|
25 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
70 Participants
n=4 Participants
|
|
Donor/Recipient Cytomegalovirus (CMV) Status
Donor Negative / Recipient Negative
|
31 Participants
n=5 Participants
|
26 Participants
n=7 Participants
|
34 Participants
n=5 Participants
|
91 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 1 Year Post-transplantGRFS is defined as being free of grade III-IV acute GVHD onset, chronic GVHD onset requiring systemic immunosuppressive therapy, disease relapse or progression, and death from any cause.
Outcome measures
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Percentage of Participants With GVHD/Relapse or Progression-free Survival (GRFS)
|
35.5 percentage of participants
Interval 27.2 to 43.9
|
27.2 percentage of participants
Interval 19.9 to 35.0
|
44.1 percentage of participants
Interval 35.3 to 52.4
|
SECONDARY outcome
Timeframe: Day 180 Post-transplantAcute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995: Skin stage: 0: No rash 1. Rash \<25% of body surface area 2. Rash on 25-50% of body surface area 3. Rash on \> 50% of body surface area 4. Generalized erythroderma with bullous formation Liver stage (based on bilirubin level)\*: 0: \<2 mg/dL 1. 2-3 mg/dL 2. 3.01-6 mg/dL 3. 6.01-15.0 mg/dL 4. \>15 mg/dL GI stage\*: 0: No diarrhea or diarrhea \<500 mL/day 1. Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD 2. Diarrhea 1000-1499 mL/day 3. Diarrhea \>1500 mL/day 4. Severe abdominal pain with or without ileus \* If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1. GVHD grade: 0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4
Outcome measures
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Percentage of Participants With Grade II-IV Acute GVHD
|
26 percentage of participants
Interval 19.0 to 34.0
|
32 percentage of participants
Interval 24.0 to 40.0
|
27 percentage of participants
Interval 20.0 to 35.0
|
SECONDARY outcome
Timeframe: Day 180 Post-transplantAcute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995: Skin stage: 0: No rash 1. Rash \<25% of body surface area 2. Rash on 25-50% of body surface area 3. Rash on \> 50% of body surface area 4. Generalized erythroderma with bullous formation Liver stage (based on bilirubin level)\*: 0: \<2 mg/dL 1.2-3 mg/dL 2.3.01-6 mg/dL 3.6.01-15.0 mg/dL 4.\>15 mg/dL GI stage\*: 0: No diarrhea or diarrhea \<500 mL/day 1. Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD 2. Diarrhea 1000-1499 mL/day 3. Diarrhea \>1500 mL/day 4. Severe abdominal pain with or without ileus \* If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1. GVHD grade: 0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4
Outcome measures
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Percentage of Participants With Grade III-IV Acute GVHD
|
8 percentage of participants
Interval 4.0 to 13.0
|
9 percentage of participants
Interval 4.0 to 14.0
|
2 percentage of participants
Interval 0.0 to 5.0
|
SECONDARY outcome
Timeframe: 1 Year Post-transplantChronic GVHD is classified per 2005 NIH Consensus Criteria (Filipovich et al. 2005) into categories of severity: none, mild, moderate, and severe. Occurrence of chronic GVHD is defined as the occurrence of mild, moderate, or severe chronic GVHD per this classification.
Outcome measures
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Percentage of Participants With Chronic GVHD
|
39 percentage of participants
Interval 30.0 to 48.0
|
43 percentage of participants
Interval 35.0 to 52.0
|
28 percentage of participants
Interval 20.0 to 36.0
|
SECONDARY outcome
Timeframe: 1 Year Post-transplantChronic GVHD is classified per 2005 NIH Consensus Criteria (Filipovich et al. 2005) into categories of severity: none, mild, moderate, and severe. Occurrence of chronic GVHD is defined as the occurrence of mild, moderate, or severe chronic GVHD per this classification. This endpoint considers the occurrence of chronic GVHD that necessitated initiation of immunosuppressive therapy for treatment.
Outcome measures
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Percentage of Participants With Chronic GVHD Requiring Immunosupressive Therapy
|
29 percentage of participants
Interval 22.0 to 38.0
|
33 percentage of participants
Interval 25.0 to 41.0
|
22 percentage of participants
Interval 15.0 to 30.0
|
SECONDARY outcome
Timeframe: 1 Year Post-transplantRelapse is defined by either morphological or cytogenetic evidence of acute leukemia or MDS consistent with pretransplant features, or radiologic evidence of lymphoma. Progression of disease applies to patients with lymphoproliferative diseases (lymphoma or chronic lymphocytic leukemia) not in remission prior to transplantation and is defined as increase in size of prior sites of disease or evidence of new sites of disease.
Outcome measures
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Percentage of Participants With Disease Relapse or Progression
|
24 percentage of participants
Interval 17.0 to 32.0
|
31 percentage of participants
Interval 23.0 to 39.0
|
28 percentage of participants
Interval 21.0 to 37.0
|
SECONDARY outcome
Timeframe: 1 Year Post-transplantTRM is defined as death without prior disease relapse or progression.
Outcome measures
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Percentage of Participants With Transplant-Related Mortality (TRM)
|
17 percentage of participants
Interval 11.0 to 24.0
|
16 percentage of participants
Interval 10.0 to 23.0
|
11 percentage of participants
Interval 6.0 to 17.0
|
SECONDARY outcome
Timeframe: 1 Year Post-transplantDisease-free survival is defined as being alive and free of disease relapse or progression.
Outcome measures
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Percentage of Participants With Disease-free Survival
|
58 percentage of participants
Interval 49.0 to 66.0
|
56 percentage of participants
Interval 47.0 to 64.0
|
60 percentage of participants
Interval 51.0 to 68.0
|
SECONDARY outcome
Timeframe: 1 Year Post-transplantGVHD-free survival is defined as being alive without previous onset of Grade III-IV acute GVHD or chronic GVHD requiring immunosuppressive therapy.
Outcome measures
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Percentage of Participants With GVHD-free Survival
|
43 percentage of participants
Interval 34.0 to 52.0
|
34 percentage of participants
Interval 26.0 to 42.0
|
53 percentage of participants
Interval 44.0 to 61.0
|
SECONDARY outcome
Timeframe: 1 Year Post-transplantOutcome measures
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Percentage of Participants With Overall Survival
|
68 percentage of participants
Interval 59.0 to 76.0
|
66 percentage of participants
Interval 57.0 to 74.0
|
71 percentage of participants
Interval 63.0 to 78.0
|
SECONDARY outcome
Timeframe: Days 28 and 100 Post-transplantNeutrophil recovery is defined as achieving an absolute neutrophil count (ANC) ≥ 500/mm\^3 for three consecutive measurements on three different days.
Outcome measures
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Percentage of Participants With Neutrophil Recovery
Day 28
|
94 percentage of participants
Interval 89.0 to 98.0
|
93 percentage of participants
Interval 89.0 to 97.0
|
95 percentage of participants
Interval 90.0 to 98.0
|
|
Percentage of Participants With Neutrophil Recovery
Day 100
|
96 percentage of participants
Interval 91.0 to 99.0
|
95 percentage of participants
Interval 90.0 to 98.0
|
98 percentage of participants
Interval 94.0 to 100.0
|
SECONDARY outcome
Timeframe: Days 60 and 100 Post-transplantPlatelet recovery is defined as the first day of a sustained platelet count \>20,000/mm\^3 with no platelet transfusion in the preceding seven days.
Outcome measures
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Percentage of Participants With Platelet Recovery
Day 60
|
91 percentage of participants
Interval 85.0 to 96.0
|
92 percentage of participants
Interval 87.0 to 96.0
|
90 percentage of participants
Interval 84.0 to 95.0
|
|
Percentage of Participants With Platelet Recovery
Day 100
|
91 percentage of participants
Interval 85.0 to 96.0
|
92 percentage of participants
Interval 87.0 to 96.0
|
96 percentage of participants
Interval 91.0 to 99.0
|
SECONDARY outcome
Timeframe: Days 28 and 100 Post-transplantDonor cell engraftment will be assessed with donor/recipient chimerism. Chimerism may be evaluated in bone marrow, whole blood, or CD3 fractions. Full donor chimerism is defined as the presence of ≥ 95% of donor cells as a proportion of total cells. Mixed chimerism is defined as the presence of donor cells, as a proportion of total cells, of \< 95% but \> 5% in the bone marrow or peripheral blood. Full and mixed chimerism will be evidence of donor cell engraftment. Donor cells of ≤ 5% will be considered as graft rejection.
Outcome measures
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Donor Cell Engraftment
Day 100 · Mixed Chimerism
|
14 Participants
|
17 Participants
|
13 Participants
|
|
Donor Cell Engraftment
Day 28 · Full Chimerism
|
58 Participants
|
56 Participants
|
64 Participants
|
|
Donor Cell Engraftment
Day 28 · Mixed Chimerism
|
11 Participants
|
10 Participants
|
8 Participants
|
|
Donor Cell Engraftment
Day 28 · Graft Rejection
|
1 Participants
|
1 Participants
|
2 Participants
|
|
Donor Cell Engraftment
Day 28 · Dead
|
2 Participants
|
4 Participants
|
0 Participants
|
|
Donor Cell Engraftment
Day 28 · No Assay Performed
|
17 Participants
|
21 Participants
|
18 Participants
|
|
Donor Cell Engraftment
Day 100 · Full Chimerism
|
63 Participants
|
56 Participants
|
62 Participants
|
|
Donor Cell Engraftment
Day 100 · Graft Rejection
|
5 Participants
|
3 Participants
|
3 Participants
|
|
Donor Cell Engraftment
Day 100 · Dead
|
4 Participants
|
10 Participants
|
5 Participants
|
|
Donor Cell Engraftment
Day 100 · No Assay Performed
|
3 Participants
|
6 Participants
|
9 Participants
|
SECONDARY outcome
Timeframe: 1 Year Post-transplantOutcome measures
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=89 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=92 Participants
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Primary Cause of Death
Hemorrhage
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Primary Cause of Death
Interstitial Pneumonia
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Primary Cause of Death
Recurrence/Persistence
|
11 Participants
|
12 Participants
|
15 Participants
|
|
Primary Cause of Death
Acute GVHD
|
3 Participants
|
7 Participants
|
2 Participants
|
|
Primary Cause of Death
Chronic GVHD
|
2 Participants
|
1 Participants
|
1 Participants
|
|
Primary Cause of Death
Infection
|
4 Participants
|
7 Participants
|
4 Participants
|
|
Primary Cause of Death
Organ Failure
|
2 Participants
|
2 Participants
|
2 Participants
|
|
Primary Cause of Death
Adult Respiratory Distress Syndrome
|
2 Participants
|
1 Participants
|
0 Participants
|
|
Primary Cause of Death
Metastatis Breast Cancer
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Primary Cause of Death
Squamous Cell Carcinoma
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Primary Cause of Death
Toxicity - Not Specified
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Primary Cause of Death
Still Alive
|
61 Participants
|
61 Participants
|
66 Participants
|
Adverse Events
Tacrolimus/Methotrexate/Bortezomib
Serious events: 21 serious events
Other events: 0 other events
Deaths: 30 deaths
Tacrolimus/Methotrexate/Maraviroc
Serious events: 14 serious events
Other events: 0 other events
Deaths: 31 deaths
Tacrolimus/MMF/Cyclophosphamide
Serious events: 12 serious events
Other events: 0 other events
Deaths: 27 deaths
Serious adverse events
| Measure |
Tacrolimus/Methotrexate/Bortezomib
n=93 participants at risk
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Bortezomib.
|
Tacrolimus/Methotrexate/Maraviroc
n=93 participants at risk
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Methotrexate, and Maraviroc.
|
Tacrolimus/MMF/Cyclophosphamide
n=93 participants at risk
Participants will receive a GVHD prophylactic regimen of three agents: Tacrolimus, Mycophenolate Mofetil (MMF), and Cyclophosphamide.
|
|---|---|---|---|
|
Cardiac disorders
Atrial fibrillation
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Cardiac disorders
Cardiac Arrest
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
|
Cardiac disorders
Cardiac failure
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
|
Cardiac disorders
Cardiogenic shock
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Cardiac disorders
Cardiomyopathy
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Cardiac disorders
Myocardial ischaemia
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Cardiac disorders
Pericardial effusion
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
|
Gastrointestinal disorders
Abdominal pain
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Gastrointestinal disorders
Ascites
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Gastrointestinal disorders
Colonic pseudo-obstruction
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
|
Gastrointestinal disorders
Gastrointestinal perforation
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Gastrointestinal disorders
Ileus
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Gastrointestinal disorders
Intestinal perforation
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
General disorders
Death
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
|
Hepatobiliary disorders
Cholecystitis
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
2.2%
2/93 • Number of events 2 • Up to 1 Year Post-transplant
|
|
Infections and infestations
Anorectal cellulitis
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
|
Infections and infestations
Appendicitis
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
|
Infections and infestations
Pneumonia
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
|
Infections and infestations
Septic shock
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Injury, poisoning and procedural complications
Fracture
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
2.2%
2/93 • Number of events 2 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Musculoskeletal and connective tissue disorders
Vertebral foraminal stenosis
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Nervous system disorders
Encephalopathy
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Nervous system disorders
Guillain-Barre syndrome
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Nervous system disorders
Haemorrhage intracranial
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
|
Nervous system disorders
Syncope
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Psychiatric disorders
Delirium
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Psychiatric disorders
Mental status changes
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Renal and urinary disorders
Acute kidney injury
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory distress syndrome
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
2.2%
2/93 • Number of events 2 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Vascular disorders
Embolism
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
|
Vascular disorders
Hypotension
|
1.1%
1/93 • Number of events 1 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
0.00%
0/93 • Up to 1 Year Post-transplant
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place