Stem Cell Transplantation for Fanconi Anemia

This study has been terminated.
(Treatment with thymic shielding found safe, another study started.)
Sponsor:
Information provided by:
Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:
NCT00167206
First received: September 9, 2005
Last updated: November 30, 2009
Last verified: November 2009
Results First Received: July 7, 2009  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Fanconi Anemia
Interventions: Procedure: Hematopoietic Stem Cell Transplant
Procedure: Thymic Shielding During Radiation
Procedure: Total Body Irradiation
Drug: Cyclophosphamide, Fludarabine

  Participant Flow
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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
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Reporting Groups
  Description
Intent-To-Treat All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy).

Participant Flow:   Overall Study
    Intent-To-Treat  
STARTED     16  
COMPLETED     16  
NOT COMPLETED     0  



  Baseline Characteristics
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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
Intent-To-Treat All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy).

Baseline Measures
    Intent-To-Treat  
Number of Participants  
[units: participants]
  16  
Age  
[units: participants]
 
<=18 years     16  
Between 18 and 65 years     0  
>=65 years     0  
Age  
[units: years]
Mean ± Standard Deviation
  9.8  ± 4.5  
Gender  
[units: participants]
 
Female     10  
Male     6  
Region of Enrollment  
[units: participants]
 
United States     16  



  Outcome Measures
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1.  Primary:   Number of Patients Who Exhibited Hematopoietic Recovery and Engraftment   [ Time Frame: Day 42 after hematopoietic cell transplant ]

2.  Secondary:   Number of Patients Who Exhibited Secondary Graft Failure   [ Time Frame: Day 100 after hematopoietic cell transplant ]

3.  Secondary:   Number of Patients With Acute Graft Versus-Host Disease (aGVHD)   [ Time Frame: Day 100 after hematopoietic cell transplant ]

4.  Secondary:   Number of Patients With Chronic Graft Versus-Host Disease (GVHD)   [ Time Frame: 1 year after hematopoietic cell transplant ]

5.  Secondary:   Number of Patients Who Exhibited Regimen-related Toxicity (RRT)   [ Time Frame: 1 year after hematopoietic cell transplant ]

6.  Secondary:   Immune Reconstitution - Mean Value (1 Year)   [ Time Frame: 1 year post-transplant. ]

7.  Secondary:   Immune Reconstitution - Mean Value (2 Years)   [ Time Frame: at 2 years after transplant ]

8.  Secondary:   Number of Patients Alive at 1 Year   [ Time Frame: 1 year after transplant ]

9.  Secondary:   Number of Patients Alive at 2 Years   [ Time Frame: 2 years after transplant ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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
Study ended early as thymic shielding was found to be safe. Therefore, a new study was designed using thymic shielding.


  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Margaret MacMillan, M.D.
Organization: Masonic Cancer Center, University of Minnesota
phone: 612-626-2778
e-mail: macmi002@umn.edu


No publications provided


Responsible Party: MacMillan, Margaret L., MD, Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00167206     History of Changes
Other Study ID Numbers: 0312M54991, MT2003-18
Study First Received: September 9, 2005
Results First Received: July 7, 2009
Last Updated: November 30, 2009
Health Authority: United States: Institutional Review Board