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Trial record 23 of 78 for:    "Congenital aplastic anemia"

Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine

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ClinicalTrials.gov Identifier: NCT00595127
Recruitment Status : Completed
First Posted : January 16, 2008
Results First Posted : August 10, 2016
Last Update Posted : August 10, 2016
Sponsor:
Collaborators:
National Cancer Institute (NCI)
Miltenyi Biotec, Inc.
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
Condition: Fanconi Anemia
Interventions: Drug: Cyclophosphamide
Drug: Fludarabine
Radiation: Total body irradiation (TBI)

  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
Cyclophosphamide and Fludarabine To evaluate a cytoreductive regimen using standard TBI & cyclophosphamide w/ addition of fludarabine, to prepare pts w/ Fanconi anemia for Tcell depleted, allogeneic hematopoietic stem cell transplant

Participant Flow:   Overall Study
    Cyclophosphamide and Fludarabine
STARTED   21 
COMPLETED   11 
NOT COMPLETED   10 
Death                6 
Relapsed                3 
joined another trial                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
Cyclophosphamide and Fludarabine To evaluate a cytoreductive regimen using standard TBI & cyclophosphamide w/ addition of fludarabine, to prepare pts w/ Fanconi anemia for Tcell depleted, allogeneic hematopoietic stem cell transplant

Baseline Measures
   Cyclophosphamide and Fludarabine 
Overall Participants Analyzed 
[Units: Participants]
 21 
Age 
[Units: Participants]
 
<=18 years   16 
Between 18 and 65 years   5 
>=65 years   0 
Gender 
[Units: Participants]
 
Female   8 
Male   13 
Region of Enrollment 
[Units: Participants]
 
United States   21 


  Outcome Measures

1.  Primary:   Incidence & Quality of Engraftment & Hematopoietic Reconstitution   [ Time Frame: 8 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: Farid Boulad, MD, Attending
Organization: Memorial Sloan Kettering Cancer Center
phone: +1212-639-2429
e-mail: bouladf@mskcc.org



Responsible Party: Memorial Sloan Kettering Cancer Center
ClinicalTrials.gov Identifier: NCT00595127     History of Changes
Other Study ID Numbers: 01-062
First Submitted: January 7, 2008
First Posted: January 16, 2008
Results First Submitted: December 22, 2015
Results First Posted: August 10, 2016
Last Update Posted: August 10, 2016