ClinicalTrials.gov
ClinicalTrials.gov Menu

Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00987480
Recruitment Status : Completed
First Posted : October 1, 2009
Results First Posted : July 10, 2018
Last Update Posted : July 10, 2018
Sponsor:
Collaborators:
Boston Children’s Hospital
Children's Hospital Medical Center, Cincinnati
Children's Hospital and Health System Foundation, Wisconsin
Rockefeller University
Fred Hutchinson Cancer Research Center
Information provided by (Responsible Party):
Memorial Sloan Kettering Cancer Center

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Aplastic Anemia
Leukemia
Myelodysplastic Syndrome
Interventions: Drug: Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.
Device: CliniMACS device

  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

This is a multicenter site and MSK is the site responsible for data collection and analysis for all sites.

11 participants were enrolled at Memorial Sloan Kettering Cancer Center. The remaining 34 participants were enrolled at the participating institutions. This makes a total of 45 participants enrolled and treated on this study.


Reporting Groups
  Description
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected

This phase II trial is designed to investigate the safety and efficacy of a chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the treatment of patients with Fanconi anemia and severe hematologic disease.

Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.: There are three parts in this transplant study. 1) There will be a pre-transplant - preparation - period to see if patient qualifies for the transplant study. This will be done as an outpatient and lasts 2-4 weeks. Once this is completed, there will be 2) the transplant period itself, during which the patient will be admitted and will be an inpatient. This period usually last for 4-6 weeks. Following that, there will be a 3) post transplant period, during which the patient will be watched carefully and monitored in clinic as an out patient. The post transplant period lasts from three months to one year.


Participant Flow:   Overall Study
    Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected
STARTED   45 
COMPLETED   45 
NOT COMPLETED   0 



  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
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected

This phase II trial is designed to investigate the safety and efficacy of a chemotherapy-based cytoreductive regimen plus a CD34+ selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the treatment of patients with Fanconi anemia and severe hematologic disease.

Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.: There are three parts in this transplant study. 1) There will be a pre-transplant - preparation - period to see if patient qualifies for the transplant study. This will be done as an outpatient and lasts 2-4 weeks. Once this is completed, there will be 2) the transplant period itself, during which the patient will be admitted and will be an inpatient. This period usually last for 4-6 weeks. Following that, there will be a 3) post transplant period, during which the patient will be watched carefully and monitored in clinic as an out patient. The post transplant period lasts from three months to one year.


Baseline Measures
   Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected 
Overall Participants Analyzed 
[Units: Participants]
 45 
Age 
[Units: Years]
Median (Full Range)
 8.2 
 (4.3 to 44) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      25  55.6% 
Male      20  44.4% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
 
Hispanic or Latino      5  11.1% 
Not Hispanic or Latino      0   0.0% 
Unknown or Not Reported      40  88.9% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
 
American Indian or Alaska Native      0   0.0% 
Asian      2   4.4% 
Native Hawaiian or Other Pacific Islander      0   0.0% 
Black or African American      3   6.7% 
White      36  80.0% 
More than one race      0   0.0% 
Unknown or Not Reported      4   8.9% 
Region of Enrollment 
[Units: Participants]
Count of Participants
 
United States   45 


  Outcome Measures

1.  Primary:   Successful Neutrophil Engraftment   [ Time Frame: 2 years ]

2.  Primary:   The Incidence of Early Transplant Related Mortality   [ Time Frame: 2 years ]

3.  Primary:   The Incidence of Acute GvHD   [ Time Frame: 100 days ]

4.  Primary:   The Incidence of Chronic GvHD   [ Time Frame: 2 years ]

5.  Secondary:   Overall Survival at 3 Years   [ Time Frame: 3 years ]

6.  Secondary:   Disease-free Survival at 3 Years   [ Time Frame: 3 years ]


  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:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr. Farid Boulad
Organization: Memorial Sloan Kettering Cancer Center
phone: 212-639-2429
e-mail: bouladf@mskcc.org



Responsible Party: Memorial Sloan Kettering Cancer Center
ClinicalTrials.gov Identifier: NCT00987480     History of Changes
Obsolete Identifiers: NCT00850317
Other Study ID Numbers: 08-031
First Submitted: September 30, 2009
First Posted: October 1, 2009
Results First Submitted: March 2, 2018
Results First Posted: July 10, 2018
Last Update Posted: July 10, 2018