Trial Outcomes & Findings for Safety and Efficacy of Pentostatin and Low Dose TBI With Allogenic Peripheral Blood Stem Cell Transplant (NCT NCT00571662)
NCT ID: NCT00571662
Last Updated: 2023-10-24
Results Overview
the efficacy of the regimen as determined by engraftment rate and establishment of donor hematopoietic chimerism at day +28 and day +70.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
76 participants
Primary outcome timeframe
days +28 and +70
Results posted on
2023-10-24
Participant Flow
Between November 2001 and February 2007 sixty eight patients were treated on this protocol and have been included in the analysis
Participant milestones
| Measure |
Cohort I
Pentostatin to be administered intravenously on days -10, -9, and -8 at a dose of 4mg/m2/day
|
|---|---|
|
Overall Study
STARTED
|
76
|
|
Overall Study
COMPLETED
|
68
|
|
Overall Study
NOT COMPLETED
|
8
|
Reasons for withdrawal
| Measure |
Cohort I
Pentostatin to be administered intravenously on days -10, -9, and -8 at a dose of 4mg/m2/day
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
2
|
|
Overall Study
psychiatric instability
|
1
|
|
Overall Study
Adverse Event
|
5
|
Baseline Characteristics
Safety and Efficacy of Pentostatin and Low Dose TBI With Allogenic Peripheral Blood Stem Cell Transplant
Baseline characteristics by cohort
| Measure |
Cohort I
n=76 Participants
Pentostatin to be administered intravenously on days -10, -9, and -8 at a dose of 4mg/m2/day
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
71 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
5 Participants
n=99 Participants
|
|
Age, Continuous
|
56 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
29 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
47 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
76 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: days +28 and +70the efficacy of the regimen as determined by engraftment rate and establishment of donor hematopoietic chimerism at day +28 and day +70.
Outcome measures
| Measure |
Cohort I
n=68 Participants
Pentostatin to be administered intravenously on days -10, -9, and -8 at a dose of 4mg/m2/day
|
Day + 28 Post Transplant
T-cell count (CD3, CD4 and CD8 cells) on Day + 28 post transplant. Measure of donor chimerism (donor CD3+ Cell in blood as assessed by DNA fingerprinting)
|
|---|---|---|
|
Percent of Participants With Chimerism: Full Donor Chimerism Defined as >95% Donor CD3+ Cell in Blood as Assessed by DNA Fingerprinting
Day 28
|
85 percent of participants
Interval 40.0 to 100.0
|
—
|
|
Percent of Participants With Chimerism: Full Donor Chimerism Defined as >95% Donor CD3+ Cell in Blood as Assessed by DNA Fingerprinting
Day 70
|
90 percent of participants
Interval 45.0 to 100.0
|
—
|
PRIMARY outcome
Timeframe: Conditioning regimen to count recovery (D + 28 post transplant)Outcome measures
| Measure |
Cohort I
n=68 Participants
Pentostatin to be administered intravenously on days -10, -9, and -8 at a dose of 4mg/m2/day
|
Day + 28 Post Transplant
T-cell count (CD3, CD4 and CD8 cells) on Day + 28 post transplant. Measure of donor chimerism (donor CD3+ Cell in blood as assessed by DNA fingerprinting)
|
|---|---|---|
|
Toxicity for the Combination of Pentostatin and Low Dose Total Body Irradiation (TBI)
Absolute neutrophil count < 500/mm^3
|
40 Participants
|
—
|
|
Toxicity for the Combination of Pentostatin and Low Dose Total Body Irradiation (TBI)
platelet count < 20,000/mm^3
|
29 Participants
|
—
|
|
Toxicity for the Combination of Pentostatin and Low Dose Total Body Irradiation (TBI)
Grade 3 or 4 Fever
|
2 Participants
|
—
|
|
Toxicity for the Combination of Pentostatin and Low Dose Total Body Irradiation (TBI)
Grade 3 or 4 hypokalemia
|
1 Participants
|
—
|
|
Toxicity for the Combination of Pentostatin and Low Dose Total Body Irradiation (TBI)
Grade 3 or 4 bacteremia
|
2 Participants
|
—
|
|
Toxicity for the Combination of Pentostatin and Low Dose Total Body Irradiation (TBI)
Grade 3 or 4 infection
|
6 Participants
|
—
|
|
Toxicity for the Combination of Pentostatin and Low Dose Total Body Irradiation (TBI)
Grade 3 or 4 renal toxicity
|
1 Participants
|
—
|
|
Toxicity for the Combination of Pentostatin and Low Dose Total Body Irradiation (TBI)
Grade 3 or 4 thromboembolism
|
1 Participants
|
—
|
SECONDARY outcome
Timeframe: twice weekly until day 100 up to 1 year post transplantIncidence of acute and chronic graft-versus-host disease. Acute GVHD usually occurs during the first three months following transplant. Chronic GVHD usually develops after the third month post-transplant.
Outcome measures
| Measure |
Cohort I
n=68 Participants
Pentostatin to be administered intravenously on days -10, -9, and -8 at a dose of 4mg/m2/day
|
Day + 28 Post Transplant
T-cell count (CD3, CD4 and CD8 cells) on Day + 28 post transplant. Measure of donor chimerism (donor CD3+ Cell in blood as assessed by DNA fingerprinting)
|
|---|---|---|
|
Incidence of Acute and Chronic Graft-versus-host Disease
Acute GVHD
|
31 Percent of Particpants
|
—
|
|
Incidence of Acute and Chronic Graft-versus-host Disease
Chronic GVHD
|
33 Percent of Particpants
|
—
|
SECONDARY outcome
Timeframe: every 6 mo. up to 2 yearsevent-free and overall survival at 12 months
Outcome measures
| Measure |
Cohort I
n=68 Participants
Pentostatin to be administered intravenously on days -10, -9, and -8 at a dose of 4mg/m2/day
|
Day + 28 Post Transplant
T-cell count (CD3, CD4 and CD8 cells) on Day + 28 post transplant. Measure of donor chimerism (donor CD3+ Cell in blood as assessed by DNA fingerprinting)
|
|---|---|---|
|
Responses to Therapy
Event free survival
|
52 Percent of Participants
|
—
|
|
Responses to Therapy
Overall survival
|
59 Percent of Participants
|
—
|
SECONDARY outcome
Timeframe: at day 100 post transplantationRate of return of immune cells after allogeneic transplantation
Outcome measures
| Measure |
Cohort I
n=68 Participants
Pentostatin to be administered intravenously on days -10, -9, and -8 at a dose of 4mg/m2/day
|
Day + 28 Post Transplant
n=68 Participants
T-cell count (CD3, CD4 and CD8 cells) on Day + 28 post transplant. Measure of donor chimerism (donor CD3+ Cell in blood as assessed by DNA fingerprinting)
|
|---|---|---|
|
Kinetics of Immunologic Reconstitution
CD3 cells
|
13 percentage of cells in peripheral blood
Interval 0.5 to 45.0
|
7 percentage of cells in peripheral blood
Interval 0.0 to 28.0
|
|
Kinetics of Immunologic Reconstitution
CD4 cells
|
5 percentage of cells in peripheral blood
Interval 0.01 to 25.0
|
3.5 percentage of cells in peripheral blood
Interval 0.0 to 13.0
|
|
Kinetics of Immunologic Reconstitution
CD8 cells
|
5 percentage of cells in peripheral blood
Interval 0.0 to 31.0
|
1.7 percentage of cells in peripheral blood
Interval 0.02 to 8.3
|
Adverse Events
Cohort I
Serious events: 26 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort I
n=76 participants at risk
Pentostatin to be administered intravenously on days -10, -9, and -8 at a dose of 4mg/m2/day
|
|---|---|
|
General disorders
fever
|
3.9%
3/76 • Number of events 3
|
|
Blood and lymphatic system disorders
decreased WBC
|
11.8%
9/76 • Number of events 9
|
|
Blood and lymphatic system disorders
decreased ANC
|
9.2%
7/76 • Number of events 7
|
|
Blood and lymphatic system disorders
decreased hgb
|
1.3%
1/76 • Number of events 1
|
|
Blood and lymphatic system disorders
decreased platelets
|
3.9%
3/76 • Number of events 3
|
|
Gastrointestinal disorders
hypokalemia
|
1.3%
1/76 • Number of events 1
|
|
Blood and lymphatic system disorders
decreased leukocyte
|
1.3%
1/76 • Number of events 1
|
|
Cardiac disorders
klebsiella
|
1.3%
1/76 • Number of events 1
|
|
Infections and infestations
reddened catheter site
|
1.3%
1/76 • Number of events 1
|
|
Immune system disorders
herpes simplex
|
1.3%
1/76 • Number of events 1
|
|
Renal and urinary disorders
Yeast infection
|
1.3%
1/76 • Number of events 1
|
|
Hepatobiliary disorders
increased LDH
|
1.3%
1/76 • Number of events 1
|
|
Blood and lymphatic system disorders
pulmonary embolism
|
1.3%
1/76 • Number of events 1
|
|
Renal and urinary disorders
azotemia
|
1.3%
1/76 • Number of events 1
|
|
Renal and urinary disorders
atubular necrosis
|
1.3%
1/76 • Number of events 1
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place