Trial Outcomes & Findings for Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer (NCT NCT00003816)
NCT ID: NCT00003816
Last Updated: 2021-08-13
Results Overview
Rate of Complete Remission by Day +100
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
361 participants
Primary outcome timeframe
day 100
Results posted on
2021-08-13
Participant Flow
Participant milestones
| Measure |
BuCy
Patients receive busulfan IV over 2 hours every 6 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
busulfan: Given IV
cyclophosphamide: Given IV
|
CyTBI
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4 and total-body irradiation (TBI) twice daily on days -3 to -1.
cyclophosphamide: Given IV
total-body irradiation: Given twice daily for 3 days
|
FluMel
Patients receive fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 1 hour on days -3 and -2.
fludarabine phosphate: Given IV
melphalan: Given IV
|
VpCyTBI
Patients receive etoposide IV over 26 hours beginning on day -5, cyclophosphamide IV over 2 hours on day -4, and TBI twice daily on days -3 to -1.
cyclophosphamide: Given IV
etoposide: Given IV
total-body irradiation: Given twice daily for 3 days
|
Other
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4, TBI twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4-8 hours on days -3 to -1. OR Patients receive cyclophosphamide IV over 2 hours on days -5 to -2 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -3. OR Patients receive cyclophosphamide IV over 24 hours, carboplatin IV over 24 hours, and thiotepa IV over 24 hours on days -7 to -4. OR Patients receive fludarabine IV over 30 minutes on days -5 to -1 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -2. OR Patients receive busulfan IV over 2 hours every 6 hours and anti-thymocyte globulin IV over 4-8 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
|
|---|---|---|---|---|---|
|
Overall Study
STARTED
|
55
|
69
|
199
|
23
|
15
|
|
Overall Study
COMPLETED
|
55
|
69
|
199
|
23
|
15
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer
Baseline characteristics by cohort
| Measure |
BuCy
n=55 Participants
Patients receive busulfan IV over 2 hours every 6 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
busulfan: Given IV
cyclophosphamide: Given IV
|
CyTBI
n=69 Participants
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4 and total-body irradiation (TBI) twice daily on days -3 to -1.
cyclophosphamide: Given IV
total-body irradiation: Given twice daily for 3 days
|
FluMel
n=199 Participants
Patients receive fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 1 hour on days -3 and -2.
fludarabine phosphate: Given IV
melphalan: Given IV
|
VpCyTBI
n=23 Participants
Patients receive etoposide IV over 26 hours beginning on day -5, cyclophosphamide IV over 2 hours on day -4, and TBI twice daily on days -3 to -1.
cyclophosphamide: Given IV
etoposide: Given IV
total-body irradiation: Given twice daily for 3 days
|
Other
n=15 Participants
Patients receive cyclophosphamide IV over 2 hours on days -5 to -2 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -3. OR Patients receive cyclophosphamide IV over 24 hours, carboplatin IV over 24 hours, and thiotepa IV over 24 hours on days -7 to -4. OR Patients receive fludarabine IV over 30 minutes on days -5 to -1 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -2. OR Patients receive cyclophosphamide IV over 2 hours on days -5 and -4, TBI twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4-8 hours on days -3 to -1.
OR Patients receive busulfan IV over 2 hours every 6 hours and anti-thymocyte globulin IV over 4-8 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
|
Total
n=361 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Age, Continuous
|
46 years
n=99 Participants
|
28 years
n=107 Participants
|
48 years
n=206 Participants
|
37 years
n=7 Participants
|
41 years
n=31 Participants
|
44 years
n=30 Participants
|
|
Sex: Female, Male
Female
|
24 Participants
n=99 Participants
|
24 Participants
n=107 Participants
|
80 Participants
n=206 Participants
|
7 Participants
n=7 Participants
|
4 Participants
n=31 Participants
|
139 Participants
n=30 Participants
|
|
Sex: Female, Male
Male
|
31 Participants
n=99 Participants
|
45 Participants
n=107 Participants
|
119 Participants
n=206 Participants
|
16 Participants
n=7 Participants
|
11 Participants
n=31 Participants
|
222 Participants
n=30 Participants
|
|
Race/Ethnicity, Customized
Non-Hispanic White
|
53 Participants
n=99 Participants
|
68 Participants
n=107 Participants
|
188 Participants
n=206 Participants
|
20 Participants
n=7 Participants
|
12 Participants
n=31 Participants
|
341 Participants
n=30 Participants
|
|
Race/Ethnicity, Customized
Non-Hispanic African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=31 Participants
|
12 Participants
n=30 Participants
|
|
Race/Ethnicity, Customized
Non-Hispanic Asian
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
3 Participants
n=30 Participants
|
|
Race/Ethnicity, Customized
Hispanic, White
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
3 Participants
n=30 Participants
|
|
Race/Ethnicity, Customized
Non-Hispanic Native Am
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
2 Participants
n=30 Participants
|
|
Region of Enrollment
United States
|
55 participants
n=99 Participants
|
69 participants
n=107 Participants
|
199 participants
n=206 Participants
|
23 participants
n=7 Participants
|
15 participants
n=31 Participants
|
361 participants
n=30 Participants
|
|
Disease
Acute myeloid leukemia
|
17 Participants
n=99 Participants
|
25 Participants
n=107 Participants
|
89 Participants
n=206 Participants
|
13 Participants
n=7 Participants
|
3 Participants
n=31 Participants
|
147 Participants
n=30 Participants
|
|
Disease
Acute lymphoblastic leukemia
|
2 Participants
n=99 Participants
|
30 Participants
n=107 Participants
|
16 Participants
n=206 Participants
|
5 Participants
n=7 Participants
|
1 Participants
n=31 Participants
|
54 Participants
n=30 Participants
|
|
Disease
Chronic myeloid or lymphocytic leukemia
|
14 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
14 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=31 Participants
|
37 Participants
n=30 Participants
|
|
Disease
Hodgkin or Non-Hodgkin lymphoma or multiple myeloma
|
6 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
56 Participants
n=206 Participants
|
4 Participants
n=7 Participants
|
4 Participants
n=31 Participants
|
72 Participants
n=30 Participants
|
|
Disease
Myelodysplastic syndrome or myeloproliferative disorder
|
15 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
23 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=31 Participants
|
43 Participants
n=30 Participants
|
|
Disease
Severe Aplastic anemia
|
1 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
3 Participants
n=31 Participants
|
7 Participants
n=30 Participants
|
|
Disease
Solid Tumor
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=31 Participants
|
1 Participants
n=30 Participants
|
|
Risk group
Standard
|
32 Participants
n=99 Participants
|
42 Participants
n=107 Participants
|
73 Participants
n=206 Participants
|
10 Participants
n=7 Participants
|
4 Participants
n=31 Participants
|
161 Participants
n=30 Participants
|
|
Risk group
High
|
23 Participants
n=99 Participants
|
27 Participants
n=107 Participants
|
126 Participants
n=206 Participants
|
13 Participants
n=7 Participants
|
11 Participants
n=31 Participants
|
200 Participants
n=30 Participants
|
PRIMARY outcome
Timeframe: day 100Rate of Complete Remission by Day +100
Outcome measures
| Measure |
BuCy
n=55 Participants
Patients receive busulfan IV over 2 hours every 6 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
busulfan: Given IV
cyclophosphamide: Given IV
|
CyTBI
n=69 Participants
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4 and total-body irradiation (TBI) twice daily on days -3 to -1.
cyclophosphamide: Given IV
total-body irradiation: Given twice daily for 3 days
|
FluMel
n=199 Participants
Patients receive fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 1 hour on days -3 and -2.
fludarabine phosphate: Given IV
melphalan: Given IV
|
VpCyTBI
n=23 Participants
Patients receive etoposide IV over 26 hours beginning on day -5, cyclophosphamide IV over 2 hours on day -4, and TBI twice daily on days -3 to -1.
cyclophosphamide: Given IV
etoposide: Given IV
total-body irradiation: Given twice daily for 3 days
|
Other
n=15 Participants
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4, TBI twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4-8 hours on days -3 to -1. OR Patients receive cyclophosphamide IV over 2 hours on days -5 to -2 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -3. OR Patients receive cyclophosphamide IV over 24 hours, carboplatin IV over 24 hours, and thiotepa IV over 24 hours on days -7 to -4. OR Patients receive fludarabine IV over 30 minutes on days -5 to -1 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -2. OR Patients receive busulfan IV over 2 hours every 6 hours and anti-thymocyte globulin IV over 4-8 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
|
|---|---|---|---|---|---|
|
CR Rate
|
42 Participants
|
55 Participants
|
134 Participants
|
18 Participants
|
7 Participants
|
SECONDARY outcome
Timeframe: Day +100Death due to treatment related causes before day +100 after BMT
Outcome measures
| Measure |
BuCy
n=55 Participants
Patients receive busulfan IV over 2 hours every 6 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
busulfan: Given IV
cyclophosphamide: Given IV
|
CyTBI
n=69 Participants
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4 and total-body irradiation (TBI) twice daily on days -3 to -1.
cyclophosphamide: Given IV
total-body irradiation: Given twice daily for 3 days
|
FluMel
n=199 Participants
Patients receive fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 1 hour on days -3 and -2.
fludarabine phosphate: Given IV
melphalan: Given IV
|
VpCyTBI
n=23 Participants
Patients receive etoposide IV over 26 hours beginning on day -5, cyclophosphamide IV over 2 hours on day -4, and TBI twice daily on days -3 to -1.
cyclophosphamide: Given IV
etoposide: Given IV
total-body irradiation: Given twice daily for 3 days
|
Other
n=15 Participants
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4, TBI twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4-8 hours on days -3 to -1. OR Patients receive cyclophosphamide IV over 2 hours on days -5 to -2 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -3. OR Patients receive cyclophosphamide IV over 24 hours, carboplatin IV over 24 hours, and thiotepa IV over 24 hours on days -7 to -4. OR Patients receive fludarabine IV over 30 minutes on days -5 to -1 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -2. OR Patients receive busulfan IV over 2 hours every 6 hours and anti-thymocyte globulin IV over 4-8 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
|
|---|---|---|---|---|---|
|
Toxicity/TRM at Day 100
|
3 Participants
|
4 Participants
|
31 Participants
|
4 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: 4 yearsprogression free survival estimate at 4 years post BMT (events are disease progression/relapse and death due to any cause)
Outcome measures
| Measure |
BuCy
n=55 Participants
Patients receive busulfan IV over 2 hours every 6 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
busulfan: Given IV
cyclophosphamide: Given IV
|
CyTBI
n=69 Participants
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4 and total-body irradiation (TBI) twice daily on days -3 to -1.
cyclophosphamide: Given IV
total-body irradiation: Given twice daily for 3 days
|
FluMel
n=199 Participants
Patients receive fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 1 hour on days -3 and -2.
fludarabine phosphate: Given IV
melphalan: Given IV
|
VpCyTBI
n=23 Participants
Patients receive etoposide IV over 26 hours beginning on day -5, cyclophosphamide IV over 2 hours on day -4, and TBI twice daily on days -3 to -1.
cyclophosphamide: Given IV
etoposide: Given IV
total-body irradiation: Given twice daily for 3 days
|
Other
n=15 Participants
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4, TBI twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4-8 hours on days -3 to -1. OR Patients receive cyclophosphamide IV over 2 hours on days -5 to -2 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -3. OR Patients receive cyclophosphamide IV over 24 hours, carboplatin IV over 24 hours, and thiotepa IV over 24 hours on days -7 to -4. OR Patients receive fludarabine IV over 30 minutes on days -5 to -1 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -2. OR Patients receive busulfan IV over 2 hours every 6 hours and anti-thymocyte globulin IV over 4-8 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
|
|---|---|---|---|---|---|
|
4 Year PFS
|
49.1 percentage of participants
Interval 36.0 to 62.2
|
52.2 percentage of participants
Interval 40.4 to 64.0
|
33.2 percentage of participants
Interval 26.7 to 39.7
|
26.1 percentage of participants
Interval 8.1 to 44.1
|
40 percentage of participants
Interval 15.3 to 64.7
|
SECONDARY outcome
Timeframe: 4-yearOverall survival estimate at 4 years post BMT
Outcome measures
| Measure |
BuCy
n=55 Participants
Patients receive busulfan IV over 2 hours every 6 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
busulfan: Given IV
cyclophosphamide: Given IV
|
CyTBI
n=69 Participants
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4 and total-body irradiation (TBI) twice daily on days -3 to -1.
cyclophosphamide: Given IV
total-body irradiation: Given twice daily for 3 days
|
FluMel
n=199 Participants
Patients receive fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 1 hour on days -3 and -2.
fludarabine phosphate: Given IV
melphalan: Given IV
|
VpCyTBI
n=23 Participants
Patients receive etoposide IV over 26 hours beginning on day -5, cyclophosphamide IV over 2 hours on day -4, and TBI twice daily on days -3 to -1.
cyclophosphamide: Given IV
etoposide: Given IV
total-body irradiation: Given twice daily for 3 days
|
Other
n=15 Participants
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4, TBI twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4-8 hours on days -3 to -1. OR Patients receive cyclophosphamide IV over 2 hours on days -5 to -2 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -3. OR Patients receive cyclophosphamide IV over 24 hours, carboplatin IV over 24 hours, and thiotepa IV over 24 hours on days -7 to -4. OR Patients receive fludarabine IV over 30 minutes on days -5 to -1 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -2. OR Patients receive busulfan IV over 2 hours every 6 hours and anti-thymocyte globulin IV over 4-8 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
|
|---|---|---|---|---|---|
|
4 yr OS
|
56.4 percentage of participants
Interval 43.3 to 69.5
|
56.5 percentage of participants
Interval 44.7 to 68.3
|
38.2 percentage of participants
Interval 31.5 to 44.9
|
39.1 percentage of participants
Interval 19.1 to 59.1
|
53.3 percentage of participants
Interval 28.0 to 78.6
|
Adverse Events
BuCy
Serious events: 11 serious events
Other events: 25 other events
Deaths: 30 deaths
CyTBI
Serious events: 10 serious events
Other events: 35 other events
Deaths: 36 deaths
FluMel
Serious events: 62 serious events
Other events: 100 other events
Deaths: 150 deaths
VpCyTBI
Serious events: 6 serious events
Other events: 6 other events
Deaths: 19 deaths
Other
Serious events: 4 serious events
Other events: 9 other events
Deaths: 9 deaths
Serious adverse events
| Measure |
BuCy
n=55 participants at risk
Patients receive busulfan IV over 2 hours every 6 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
busulfan: Given IV
cyclophosphamide: Given IV
|
CyTBI
n=69 participants at risk
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4 and total-body irradiation (TBI) twice daily on days -3 to -1.
cyclophosphamide: Given IV
total-body irradiation: Given twice daily for 3 days
|
FluMel
n=199 participants at risk
Patients receive fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 1 hour on days -3 and -2.
fludarabine phosphate: Given IV
melphalan: Given IV
|
VpCyTBI
n=23 participants at risk
Patients receive etoposide IV over 26 hours beginning on day -5, cyclophosphamide IV over 2 hours on day -4, and TBI twice daily on days -3 to -1.
cyclophosphamide: Given IV
etoposide: Given IV
total-body irradiation: Given twice daily for 3 days
|
Other
n=15 participants at risk
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4, TBI twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4-8 hours on days -3 to -1. OR Patients receive cyclophosphamide IV over 2 hours on days -5 to -2 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -3. OR Patients receive cyclophosphamide IV over 24 hours, carboplatin IV over 24 hours, and thiotepa IV over 24 hours on days -7 to -4. OR Patients receive fludarabine IV over 30 minutes on days -5 to -1 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -2. OR Patients receive busulfan IV over 2 hours every 6 hours and anti-thymocyte globulin IV over 4-8 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
|
|---|---|---|---|---|---|
|
Investigations
1 yr TRM
|
20.0%
11/55 • Number of events 11 • 1 year
|
14.5%
10/69 • Number of events 10 • 1 year
|
31.2%
62/199 • Number of events 62 • 1 year
|
26.1%
6/23 • Number of events 6 • 1 year
|
26.7%
4/15 • Number of events 4 • 1 year
|
Other adverse events
| Measure |
BuCy
n=55 participants at risk
Patients receive busulfan IV over 2 hours every 6 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
busulfan: Given IV
cyclophosphamide: Given IV
|
CyTBI
n=69 participants at risk
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4 and total-body irradiation (TBI) twice daily on days -3 to -1.
cyclophosphamide: Given IV
total-body irradiation: Given twice daily for 3 days
|
FluMel
n=199 participants at risk
Patients receive fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 1 hour on days -3 and -2.
fludarabine phosphate: Given IV
melphalan: Given IV
|
VpCyTBI
n=23 participants at risk
Patients receive etoposide IV over 26 hours beginning on day -5, cyclophosphamide IV over 2 hours on day -4, and TBI twice daily on days -3 to -1.
cyclophosphamide: Given IV
etoposide: Given IV
total-body irradiation: Given twice daily for 3 days
|
Other
n=15 participants at risk
Patients receive cyclophosphamide IV over 2 hours on days -5 and -4, TBI twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4-8 hours on days -3 to -1. OR Patients receive cyclophosphamide IV over 2 hours on days -5 to -2 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -3. OR Patients receive cyclophosphamide IV over 24 hours, carboplatin IV over 24 hours, and thiotepa IV over 24 hours on days -7 to -4. OR Patients receive fludarabine IV over 30 minutes on days -5 to -1 and anti-thymocyte globulin IV over 4-8 hours on days -5 to -2. OR Patients receive busulfan IV over 2 hours every 6 hours and anti-thymocyte globulin IV over 4-8 hours on days -7 to -4 and cyclophosphamide IV over 2 hours on days -3 and -2.
|
|---|---|---|---|---|---|
|
Blood and lymphatic system disorders
acute graft-versus-host disease
|
45.5%
25/55 • Number of events 25 • 1 year
|
50.7%
35/69 • Number of events 35 • 1 year
|
50.3%
100/199 • Number of events 100 • 1 year
|
26.1%
6/23 • Number of events 6 • 1 year
|
60.0%
9/15 • Number of events 9 • 1 year
|
Additional Information
Theresa Hahn, PhD
Roswell Park Comprehensive Cancer Center
Phone: 716-845-5819
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place