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Cyclophosphamide and Busulfan Followed by Donor Stem Cell Transplant in Treating Patients With Myelofibrosis, Acute Myeloid Leukemia, or Myelodysplastic Syndrome

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ClinicalTrials.gov Identifier: NCT00445744
Recruitment Status : Completed
First Posted : March 9, 2007
Results First Posted : January 2, 2018
Last Update Posted : January 2, 2018
Sponsor:
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Joachim Deeg, Fred Hutchinson Cancer Research Center

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: 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(15;17)(q22;q12)
Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)
Adult Acute Myeloid Leukemia With t(8;21)(q22;q22)
Childhood Acute Myeloid Leukemia in Remission
Childhood Myelodysplastic Syndromes
de Novo Myelodysplastic Syndromes
Essential Thrombocythemia
Myelodysplastic Syndrome With Isolated Del(5q)
Polycythemia Vera
Previously Treated Myelodysplastic Syndromes
Primary Myelofibrosis
Recurrent Adult Acute Myeloid Leukemia
Recurrent Childhood Acute Myeloid Leukemia
Secondary Acute Myeloid Leukemia
Secondary Myelodysplastic Syndromes
Secondary Myelofibrosis
Untreated Adult Acute Myeloid Leukemia
Untreated Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies
Interventions: Drug: cyclophosphamide
Drug: busulfan
Drug: tacrolimus
Drug: methotrexate
Genetic: cytogenetic analysis
Other: flow cytometry
Other: pharmacological study
Other: pharmacogenomic studies
Procedure: peripheral blood stem cell transplantation
Procedure: allogeneic hematopoietic stem cell transplantation

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Treatment (Cyclophosphamide, Busulfan, Transplant)

CONDITIONING REGIMEN: Patients receive cyclophosphamide IV on days -7 and -6 and busulfan IV over 3 hours on days -5 to -2.

TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplant on day 0.

POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive tacrolimus IV or PO twice daily on days -1 to 200 with taper on day 56 and methotrexate on days 1, 3, 6, and 11.

cyclophosphamide: Given IV

busulfan: Given IV

tacrolimus: Given IV or PO

methotrexate: Given IV

cytogenetic analysis: Correlative studies

flow cytometry: Correlative studies

pharmacological study: Correlative studies

pharmacogenomic studies: Correlative studies

peripheral blood stem cell transplantation: Undergo PBPC transplantation

allogeneic hematopoietic stem cell transplantation: Undergo allogeneic transplantation


Participant Flow:   Overall Study
    Treatment (Cyclophosphamide, Busulfan, Transplant)
STARTED   52 
COMPLETED   51 
NOT COMPLETED   1 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Treatment (Cyclophosphamide, Busulfan, Transplant)

CONDITIONING REGIMEN: Patients receive cyclophosphamide IV on days -7 and -6 and busulfan IV over 3 hours on days -5 to -2.

TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplant on day 0.

POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive tacrolimus IV or PO twice daily on days -1 to 200 with taper on day 56 and methotrexate on days 1, 3, 6, and 11.

cyclophosphamide: Given IV

busulfan: Given IV

tacrolimus: Given IV or PO

methotrexate: Given IV

cytogenetic analysis: Correlative studies

flow cytometry: Correlative studies

pharmacological study: Correlative studies

pharmacogenomic studies: Correlative studies

peripheral blood stem cell transplantation: Undergo PBPC transplantation

allogeneic hematopoietic stem cell transplantation: Undergo allogeneic transplantation


Baseline Measures
   Treatment (Cyclophosphamide, Busulfan, Transplant) 
Overall Participants Analyzed 
[Units: Participants]
 51 
Age 
[Units: Years]
Median (Full Range)
 55 
 (30 to 65) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      21  41.2% 
Male      30  58.8% 


  Outcome Measures

1.  Primary:   Effectiveness of Cyclophosphamide/Busulfan Regimen in Reducing Regimen-related Liver Toxicity   [ Time Frame: Up to day +20 ]

2.  Primary:   Non-relapse Mortality (NRM) (Patients With AML/MDS)   [ Time Frame: Up to day 200 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: H. Joachim Deeg MD
Organization: FHCRC
phone: 206 667 4409
e-mail: jdeeg@fhcrc.org



Responsible Party: Joachim Deeg, Fred Hutchinson Cancer Research Center
ClinicalTrials.gov Identifier: NCT00445744     History of Changes
Other Study ID Numbers: 2130.00
NCI-2010-00270 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
P01HL036444 ( U.S. NIH Grant/Contract )
First Submitted: March 7, 2007
First Posted: March 9, 2007
Results First Submitted: April 5, 2017
Results First Posted: January 2, 2018
Last Update Posted: January 2, 2018