Trial Outcomes & Findings for Phase I/II Study of Pentostatin Combined With Tacrolimus and Mini-Methotrexate for GVHD Prevention After MUD BMT (NCT NCT00506922)
NCT ID: NCT00506922
Last Updated: 2015-04-28
Results Overview
The primary efficacy endpoint of escalating doses Pentostatin with Tacrolimus + Methotrexate is success, defined to be that the patient is alive, engrafted, and without acute graft-versus-host disease (GVHD) at 100 days.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
150 participants
Primary outcome timeframe
100 days
Results posted on
2015-04-28
Participant Flow
Recruitment Period: September 15, 2000 to July 26, 2007; All recruitment done at UT MD Anderson Cancer Center
A total of 150 patients were enrolled, and 147 were available for analysis. Two patients assigned to the 1.5 mg/m\^2 arm were removed without receiving treatment (due to ineligibility, and poor donor cell collection). A patient assigned to arm 2.0 mg/m\^2 withdrew consent before being treated.
Participant milestones
| Measure |
No Pentostatin
Group 1: No Pentostatin
|
Pentostatin 0.5
Group 2: Pentostatin 0.5 mg/m\^2
|
Pentostatin 1
Group 3: Pentostatin 1 mg/m\^2
|
Pentostatin 1.5
Group 4: Pentostatin 1.5 mg/m\^2
|
Pentostatin 2
Group 5: Pentostatin 2 mg/m\^2
|
|---|---|---|---|---|---|
|
Overall Study
STARTED
|
37
|
10
|
29
|
63
|
11
|
|
Overall Study
COMPLETED
|
37
|
10
|
29
|
61
|
10
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
2
|
1
|
Reasons for withdrawal
| Measure |
No Pentostatin
Group 1: No Pentostatin
|
Pentostatin 0.5
Group 2: Pentostatin 0.5 mg/m\^2
|
Pentostatin 1
Group 3: Pentostatin 1 mg/m\^2
|
Pentostatin 1.5
Group 4: Pentostatin 1.5 mg/m\^2
|
Pentostatin 2
Group 5: Pentostatin 2 mg/m\^2
|
|---|---|---|---|---|---|
|
Overall Study
Physician Decision
|
0
|
0
|
0
|
2
|
0
|
|
Overall Study
Withdrawal by Subject
|
0
|
0
|
0
|
0
|
1
|
Baseline Characteristics
Phase I/II Study of Pentostatin Combined With Tacrolimus and Mini-Methotrexate for GVHD Prevention After MUD BMT
Baseline characteristics by cohort
| Measure |
No Pentostatin
n=37 Participants
Group 1: No Pentostatin
|
Pentostatin 0.5
n=10 Participants
Group 2: Pentostatin 0.5 mg/m\^2
|
Pentostatin 1
n=29 Participants
Group 3: Pentostatin 1 mg/m\^2
|
Pentostatin 1.5
n=63 Participants
Group 4: Pentostatin 1.5 mg/m\^2
|
Pentostatin 2
n=11 Participants
Group 5: Pentostatin 2 mg/m\^2
|
Total
n=150 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
36 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
28 Participants
n=5 Participants
|
61 Participants
n=4 Participants
|
11 Participants
n=21 Participants
|
146 Participants
n=8 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
4 Participants
n=8 Participants
|
|
Age, Continuous
|
50 years
STANDARD_DEVIATION 19 • n=5 Participants
|
42 years
STANDARD_DEVIATION 23 • n=7 Participants
|
47 years
STANDARD_DEVIATION 20 • n=5 Participants
|
50 years
STANDARD_DEVIATION 22 • n=4 Participants
|
45 years
STANDARD_DEVIATION 32 • n=21 Participants
|
46.8 years
STANDARD_DEVIATION 23.2 • n=8 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
30 Participants
n=4 Participants
|
7 Participants
n=21 Participants
|
66 Participants
n=8 Participants
|
|
Sex: Female, Male
Male
|
22 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
33 Participants
n=4 Participants
|
4 Participants
n=21 Participants
|
84 Participants
n=8 Participants
|
|
Region of Enrollment
United States
|
37 participants
n=5 Participants
|
10 participants
n=7 Participants
|
29 participants
n=5 Participants
|
63 participants
n=4 Participants
|
11 participants
n=21 Participants
|
150 participants
n=8 Participants
|
PRIMARY outcome
Timeframe: 100 daysPopulation: All analysis was intention to treat (ITT).
The primary efficacy endpoint of escalating doses Pentostatin with Tacrolimus + Methotrexate is success, defined to be that the patient is alive, engrafted, and without acute graft-versus-host disease (GVHD) at 100 days.
Outcome measures
| Measure |
Pentostatin
n=147 Participants
150 patients were enrolled, 2 patients not treated, 38 patients were Group 1 (no Pentostatin), the remaining Groups 2,3,4 and 5, 110 patients were analysed that received the Pentostatin.
|
|---|---|
|
Number of Patients Without GVHD at 100 Days
|
100 participants
|
Adverse Events
No Pentostatin
Serious events: 0 serious events
Other events: 28 other events
Deaths: 0 deaths
Pentostatin 0.5
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Pentostatin 1
Serious events: 0 serious events
Other events: 17 other events
Deaths: 0 deaths
Pentostatin 1.5
Serious events: 0 serious events
Other events: 40 other events
Deaths: 0 deaths
Pentostatin 2
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
No Pentostatin
n=37 participants at risk；n=27 participants at risk
Group 1: No Pentostatin
|
Pentostatin 0.5
n=10 participants at risk
Group 2: Pentostatin 0.5 mg/m\^2
|
Pentostatin 1
n=29 participants at risk
Group 3: Pentostatin 1 mg/m\^2
|
Pentostatin 1.5
n=61 participants at risk
Group 4: Pentostatin 1.5 mg/m\^2
|
Pentostatin 2
n=10 participants at risk
Group 5: Pentostatin 2 mg/m\^2
|
|---|---|---|---|---|---|
|
Renal and urinary disorders
Increased Creatinine
|
29.7%
11/37 • Number of events 11 • 7 Years
|
60.0%
6/10 • Number of events 6 • 7 Years
|
13.8%
4/29 • Number of events 4 • 7 Years
|
34.4%
21/61 • Number of events 21 • 7 Years
|
40.0%
4/10 • Number of events 4 • 7 Years
|
|
Renal and urinary disorders
TTP/HUS
|
0.00%
0/37 • 7 Years
|
0.00%
0/10 • 7 Years
|
0.00%
0/29 • 7 Years
|
8.2%
5/61 • Number of events 5 • 7 Years
|
70.0%
7/10 • Number of events 7 • 7 Years
|
|
Infections and infestations
CMV
|
56.8%
21/37 • Number of events 21 • 7 Years
|
30.0%
3/10 • Number of events 3 • 7 Years
|
41.4%
12/29 • Number of events 12 • 7 Years
|
44.3%
27/61 • Number of events 27 • 7 Years
|
50.0%
5/10 • Number of events 5 • 7 Years
|
|
Infections and infestations
bacterial
|
64.9%
24/37 • Number of events 24 • 7 Years
|
60.0%
6/10 • Number of events 6 • 7 Years
|
58.6%
17/29 • Number of events 17 • 7 Years
|
62.3%
38/61 • Number of events 38 • 7 Years
|
70.0%
7/10 • Number of events 7 • 7 Years
|
|
Infections and infestations
viral
|
13.5%
5/37 • Number of events 5 • 7 Years
|
20.0%
2/10 • Number of events 2 • 7 Years
|
24.1%
7/29 • Number of events 7 • 7 Years
|
39.3%
24/61 • Number of events 24 • 7 Years
|
10.0%
1/10 • Number of events 1 • 7 Years
|
|
Infections and infestations
parasite
|
0.00%
0/37 • 7 Years
|
0.00%
0/10 • 7 Years
|
3.4%
1/29 • Number of events 1 • 7 Years
|
4.9%
3/61 • Number of events 3 • 7 Years
|
0.00%
0/10 • 7 Years
|
|
Infections and infestations
fungal
|
13.5%
5/37 • Number of events 5 • 7 Years
|
20.0%
2/10 • Number of events 2 • 7 Years
|
24.1%
7/29 • Number of events 7 • 7 Years
|
39.3%
24/61 • Number of events 24 • 7 Years
|
10.0%
1/10 • Number of events 1 • 7 Years
|
Additional Information
Marcos de Lima, MD / Professor
UT MD Anderson Cancer Center
Phone: 713-745-3219
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place