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

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ClinicalTrials.gov Identifier: NCT00045435
Recruitment Status : Active, not recruiting
First Posted : January 27, 2003
Results First Posted : March 16, 2017
Last Update Posted : June 20, 2018
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Brenda Sandmaier, Fred Hutchinson Cancer Research Center

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome
Adult Acute Megakaryoblastic Leukemia (M7)
Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Adult Acute Monoblastic Leukemia (M5a)
Adult Acute Monocytic Leukemia (M5b)
Adult Acute Myeloblastic Leukemia With Maturation (M2)
Adult Acute Myeloblastic Leukemia Without Maturation (M1)
Adult Acute Myeloid Leukemia in Remission
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Adult Acute Myeloid Leukemia With Del(5q)
Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)
Adult Acute Myeloid Leukemia With t(8;21)(q22;q22)
Adult Acute Myelomonocytic Leukemia (M4)
Adult Erythroleukemia (M6a)
Adult Pure Erythroid Leukemia (M6b)
Secondary Acute Myeloid Leukemia
Interventions Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation
Drug: fludarabine phosphate
Radiation: total-body irradiation
Drug: cyclosporine
Drug: mycophenolate mofetil
Procedure: peripheral blood stem cell transplantation
Enrollment 17
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Treatment (Nonmyeloablative Donor PBSC Transplant)
Hide Arm/Group Description

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

Period Title: Overall Study
Started 17
Completed 17
Not Completed 0
Arm/Group Title Treatment (Nonmyeloablative Donor PBSC Transplant)
Hide Arm/Group Description

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 Number of Baseline Participants 17
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants
<=18 years
0
   0.0%
Between 18 and 65 years
10
  58.8%
>=65 years
7
  41.2%
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 17 participants
64.8
(56 to 72.7)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants
Female
4
  23.5%
Male
13
  76.5%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 17 participants
17
1.Primary Outcome
Title Disease-free Survival-incidence of Survival Without Relapse
Hide Description 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%.
Time Frame By 1 year after transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Nonmyeloablative Donor PBSC Transplant)
Hide Arm/Group Description:

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 Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: percentage of participants
47
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Treatment (Nonmyeloablative Donor PBSC Transplant)
Comments The study was to be stopped after 20 patients if the upper bound of a 1-sided 95% confidence interval for relapse-free survival was <35%.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Percent
Estimated Value 47
Confidence Interval (1-Sided) 95%
69
Estimation Comments The criterion for stopping was not met.
2.Primary Outcome
Title Nonrelapse Mortality (NRM)-Incidence of Nonrelapse Death
Hide Description 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%.
Time Frame 200 days after transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Nonmyeloablative Donor PBSC Transplant)
Hide Arm/Group Description:

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 Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: percentage of participants
6
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Treatment (Nonmyeloablative Donor PBSC Transplant)
Comments The study was to be stopped if the lower bound of a 1-sided 80% confidence interval for NRM was greater than 15%
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter percent
Estimated Value 6
Confidence Interval (1-Sided) 80%
1
Estimation Comments The stopping criterion was not met.
3.Secondary Outcome
Title Overall Survival
Hide Description Percent patients surviving.
Time Frame By 1 year after transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Nonmyeloablative Donor PBSC Transplant)
Hide Arm/Group Description:

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 Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: percentage of participants
70.6
4.Secondary Outcome
Title Incidence of Relapse
Hide Description Percent patients with relapsed disease post-transplant.
Time Frame By 1 year after transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Nonmyeloablative Donor PBSC Transplant)
Hide Arm/Group Description:

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 Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: percentage of participants
41.2
5.Secondary Outcome
Title Incidence of Rejection
Hide Description Percent patients who developed infections post-transplant.
Time Frame By 1 year after transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Nonmyeloablative Donor PBSC Transplant)
Hide Arm/Group Description:

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 Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: percentage of participants
0
6.Secondary Outcome
Title Incidence of Acute and Chronic GVHD
Hide Description Percent patients with acute/chronic GVHD
Time Frame aGVHD: 100 days after transplant; cGVHD: 1 Year after transplant.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Nonmyeloablative Donor PBSC Transplant)
Hide Arm/Group Description:

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 Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: percentage of participants
Grade II-IV aGVHD 35.3
cGVHD 35.3
Time Frame AEs: Conditioning through Day 100; SAEs: Conditioning through Day 200
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Treatment (Nonmyeloablative Donor PBSC Transplant)
Hide Arm/Group Description

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

All-Cause Mortality
Treatment (Nonmyeloablative Donor PBSC Transplant)
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Treatment (Nonmyeloablative Donor PBSC Transplant)
Affected / at Risk (%) # Events
Total   5/17 (29.41%)    
Cardiac disorders   
Ventricular arrhythmia   1/17 (5.88%)  1
Immune system disorders   
GVHD   1/17 (5.88%)  1
Infections and infestations   
Sepsis   2/17 (11.76%)  2
Musculoskeletal and connective tissue disorders   
Flank pain   1/17 (5.88%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Secondary malignancy   1/17 (5.88%)  1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Treatment (Nonmyeloablative Donor PBSC Transplant)
Affected / at Risk (%) # Events
Total   4/17 (23.53%)    
Cardiac disorders   
Heart failure   1/17 (5.88%)  1
Ventricular arrhythmia   1/17 (5.88%)  1
Investigations   
Neutrophil count decreased   2/17 (11.76%)  2
Respiratory, thoracic and mediastinal disorders   
Hypoxia   1/17 (5.88%)  3
Vascular disorders   
Thromboembolic event   1/17 (5.88%)  1
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. Brenda M. Sandmaier
Organization: Fred Hutchinson Cancer Research Center
Phone: 206-667-4961
Responsible Party: Brenda Sandmaier, Fred Hutchinson Cancer Research Center
ClinicalTrials.gov Identifier: NCT00045435     History of Changes
Other Study ID Numbers: 1654.00
NCI-2011-01307 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
First Submitted: September 6, 2002
First Posted: January 27, 2003
Results First Submitted: January 25, 2017
Results First Posted: March 16, 2017
Last Update Posted: June 20, 2018