Trial Outcomes & Findings for Reduced Intensity Donor Peripheral Blood Stem Cell Transplant in Treating Patients With De Novo or Secondary Acute Myeloid Leukemia in Remission (NCT NCT00045435)

Results Overview

Sufficient evidence will be taken to be an observed rate of DFS at one year after transplant that corresponds to a one-sided 95% confidence interval with an upper limit lower than 35%.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

17 participants

Primary outcome timeframe

By 1 year after transplant

Results posted on

2020-01-29

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Nonmyeloablative Donor PBSC Transplant) Patients receive fludarabine phosphate IV on days -4 to -2 and undergo TBI \& allogeneic PBSC transplant on day 0. Patients also receive CSP PO BID on days -3 to 56 with taper to day 77, and MMF PO BID on days 0-27. Nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant Fludarabine phosphate: Given IV Total-body irradiation: Undergo total-body irradiation Cyclosporine: Given PO Mycophenolate mofetil: Given PO Peripheral blood stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant
Overall Study STARTED	17
Overall Study COMPLETED	17
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Reduced Intensity Donor Peripheral Blood Stem Cell Transplant in Treating Patients With De Novo or Secondary Acute Myeloid Leukemia in Remission

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Nonmyeloablative Donor PBSC Transplant) n=17 Participants Patients receive fludarabine phosphate IV on days -4 to -2 and undergo TBI \& allogeneic PBSC transplant on day 0. Patients also receive CSP PO BID on days -3 to 56 with taper to day 77, and MMF PO BID on days 0-27. Nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant Fludarabine phosphate: Given IV Total-body irradiation: Undergo total-body irradiation Cyclosporine: Given PO Mycophenolate mofetil: Given PO Peripheral blood stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants
Age, Categorical >=65 years	7 Participants n=5 Participants
Age, Continuous	64.8 years n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants
Region of Enrollment United States	17 participants n=5 Participants

PRIMARY outcome

Timeframe: By 1 year after transplant

Sufficient evidence will be taken to be an observed rate of DFS at one year after transplant that corresponds to a one-sided 95% confidence interval with an upper limit lower than 35%.

Outcome measures

Outcome measures
Measure	Treatment (Nonmyeloablative Donor PBSC Transplant) n=17 Participants Patients receive fludarabine phosphate IV on days -4 to -2 and undergo TBI \& allogeneic PBSC transplant on day 0. Patients also receive CSP PO BID on days -3 to 56 with taper to day 77, and MMF PO BID on days 0-27. Nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant Fludarabine phosphate: Given IV Total-body irradiation: Undergo total-body irradiation Cyclosporine: Given PO Mycophenolate mofetil: Given PO Peripheral blood stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant
Disease-free Survival-incidence of Survival Without Relapse	47 percentage of participants

PRIMARY outcome

Timeframe: 200 days after transplant

Defined as death without morphologic evidence of disease. Sufficient evidence will be taken to be an observed rate of NRM within 200 days of transplant that corresponds to a one-sided 80% confidence interval with a lower limit greater than 15%.

Outcome measures

Outcome measures
Measure	Treatment (Nonmyeloablative Donor PBSC Transplant) n=17 Participants Patients receive fludarabine phosphate IV on days -4 to -2 and undergo TBI \& allogeneic PBSC transplant on day 0. Patients also receive CSP PO BID on days -3 to 56 with taper to day 77, and MMF PO BID on days 0-27. Nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant Fludarabine phosphate: Given IV Total-body irradiation: Undergo total-body irradiation Cyclosporine: Given PO Mycophenolate mofetil: Given PO Peripheral blood stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant
Nonrelapse Mortality (NRM)-Incidence of Nonrelapse Death	6 percentage of participants

SECONDARY outcome

Timeframe: By 1 year after transplant

Percent patients surviving.

Outcome measures

Outcome measures
Measure	Treatment (Nonmyeloablative Donor PBSC Transplant) n=17 Participants Patients receive fludarabine phosphate IV on days -4 to -2 and undergo TBI \& allogeneic PBSC transplant on day 0. Patients also receive CSP PO BID on days -3 to 56 with taper to day 77, and MMF PO BID on days 0-27. Nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant Fludarabine phosphate: Given IV Total-body irradiation: Undergo total-body irradiation Cyclosporine: Given PO Mycophenolate mofetil: Given PO Peripheral blood stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant
Overall Survival	70.6 percentage of participants

SECONDARY outcome

Timeframe: By 1 year after transplant

Percent patients with relapsed disease post-transplant.

Outcome measures

Outcome measures
Measure	Treatment (Nonmyeloablative Donor PBSC Transplant) n=17 Participants Patients receive fludarabine phosphate IV on days -4 to -2 and undergo TBI \& allogeneic PBSC transplant on day 0. Patients also receive CSP PO BID on days -3 to 56 with taper to day 77, and MMF PO BID on days 0-27. Nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant Fludarabine phosphate: Given IV Total-body irradiation: Undergo total-body irradiation Cyclosporine: Given PO Mycophenolate mofetil: Given PO Peripheral blood stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant
Incidence of Relapse	41.2 percentage of participants

SECONDARY outcome

Timeframe: By 1 year after transplant

Percent patients who developed infections post-transplant.

Outcome measures

Outcome measures
Measure	Treatment (Nonmyeloablative Donor PBSC Transplant) n=17 Participants Patients receive fludarabine phosphate IV on days -4 to -2 and undergo TBI \& allogeneic PBSC transplant on day 0. Patients also receive CSP PO BID on days -3 to 56 with taper to day 77, and MMF PO BID on days 0-27. Nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant Fludarabine phosphate: Given IV Total-body irradiation: Undergo total-body irradiation Cyclosporine: Given PO Mycophenolate mofetil: Given PO Peripheral blood stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant
Incidence of Rejection	0 percentage of participants

SECONDARY outcome

Timeframe: aGVHD: 100 days after transplant; cGVHD: 1 Year after transplant.

Percent patients with acute/chronic GVHD

Outcome measures

Outcome measures
Measure	Treatment (Nonmyeloablative Donor PBSC Transplant) n=17 Participants Patients receive fludarabine phosphate IV on days -4 to -2 and undergo TBI \& allogeneic PBSC transplant on day 0. Patients also receive CSP PO BID on days -3 to 56 with taper to day 77, and MMF PO BID on days 0-27. Nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant Fludarabine phosphate: Given IV Total-body irradiation: Undergo total-body irradiation Cyclosporine: Given PO Mycophenolate mofetil: Given PO Peripheral blood stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant
Incidence of Acute and Chronic GVHD Grade II-IV aGVHD	35.3 percentage of participants
Incidence of Acute and Chronic GVHD cGVHD	35.3 percentage of participants

Adverse Events

Treatment (Nonmyeloablative Donor PBSC Transplant)

Serious events: 5 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Nonmyeloablative Donor PBSC Transplant) n=17 participants at risk Patients receive fludarabine phosphate IV on days -4 to -2 and undergo TBI \& allogeneic PBSC transplant on day 0. Patients also receive CSP PO BID on days -3 to 56 with taper to day 77, and MMF PO BID on days 0-27. Nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant Fludarabine phosphate: Given IV Total-body irradiation: Undergo total-body irradiation Cyclosporine: Given PO Mycophenolate mofetil: Given PO Peripheral blood stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant
Musculoskeletal and connective tissue disorders Flank pain	5.9% 1/17 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
Immune system disorders GVHD	5.9% 1/17 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Secondary malignancy	5.9% 1/17 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
Infections and infestations Sepsis	11.8% 2/17 • Number of events 2 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
Cardiac disorders Ventricular arrhythmia	5.9% 1/17 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200

Other adverse events

Other adverse events
Measure	Treatment (Nonmyeloablative Donor PBSC Transplant) n=17 participants at risk Patients receive fludarabine phosphate IV on days -4 to -2 and undergo TBI \& allogeneic PBSC transplant on day 0. Patients also receive CSP PO BID on days -3 to 56 with taper to day 77, and MMF PO BID on days 0-27. Nonmyeloablative allogeneic hematopoietic stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant Fludarabine phosphate: Given IV Total-body irradiation: Undergo total-body irradiation Cyclosporine: Given PO Mycophenolate mofetil: Given PO Peripheral blood stem cell transplantation: Undergo nonmyeloablative allogeneic peripheral blood stem cell transplant
Cardiac disorders Heart failure	5.9% 1/17 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
Respiratory, thoracic and mediastinal disorders Hypoxia	5.9% 1/17 • Number of events 3 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
Investigations Neutrophil count decreased	11.8% 2/17 • Number of events 2 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
Vascular disorders Thromboembolic event	5.9% 1/17 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
Cardiac disorders Ventricular arrhythmia	5.9% 1/17 • Number of events 1 • AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200

Additional Information

Dr. Brenda M. Sandmaier

Fred Hutchinson Cancer Research Center

Phone: 206-667-4961

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place