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=5 Participants
Age, Categorical Between 18 and 65 years	71 Participants n=5 Participants
Age, Categorical >=65 years	5 Participants n=5 Participants
Age, Continuous	56 years n=5 Participants
Sex: Female, Male Female	29 Participants n=5 Participants
Sex: Female, Male Male	47 Participants n=5 Participants
Region of Enrollment United States	76 participants n=5 Participants

PRIMARY outcome

Timeframe: days +28 and +70

the 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 transplant

Incidence 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 years

event-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 Overall survival	59 Percent of Participants	—
Responses to Therapy Event free survival	52 Percent of Participants	—

SECONDARY outcome

Timeframe: at day 100 post transplantation

Rate 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 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 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 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

R Gregory Bociek

University of Nebraska Medical Center

Phone: 402-559-5388

Email: rgbociek@unmc.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place