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Trial record 23 of 80 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)
Enrollment 21
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cyclophosphamide and Fludarabine
Hide Arm/Group Description 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
Period Title: Overall Study
Started 21
Completed 11
Not Completed 10
Reason Not Completed
Death             6
Relapsed             3
joined another trial             1
Arm/Group Title Cyclophosphamide and Fludarabine
Hide Arm/Group Description 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
Overall Number of Baseline Participants 21
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
<=18 years
16
  76.2%
Between 18 and 65 years
5
  23.8%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
Female
8
  38.1%
Male
13
  61.9%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 21 participants
21
1.Primary Outcome
Title Incidence & Quality of Engraftment & Hematopoietic Reconstitution
Hide Description Number of patients who engrafted
Time Frame 8 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cyclophosphamide and Fludarabine
Hide Arm/Group Description:
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
Overall Number of Participants Analyzed 21
Measure Type: Number
Unit of Measure: participants
19
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cyclophosphamide and Fludarabine
Hide Arm/Group Description 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
All-Cause Mortality
Cyclophosphamide and Fludarabine
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Cyclophosphamide and Fludarabine
Affected / at Risk (%)
Total   5/21 (23.81%) 
Blood and lymphatic system disorders   
Hemorrhage, other   1/21 (4.76%) 
Cardiac disorders   
Cardiovascular, other   1/21 (4.76%) 
Gastrointestinal disorders   
Diarrhea (PEDS/BMT)   1/21 (4.76%) 
Infections and infestations   
Infection w.out neutropenia   1/21 (4.76%) 
Infection/Feb Neut-Other   1/21 (4.76%) 
Renal and urinary disorders   
Renal failure   1/21 (4.76%) 
Respiratory, thoracic and mediastinal disorders   
Dyspnea   1/21 (4.76%) 
Hypoxia   2/21 (9.52%) 
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 5%
Cyclophosphamide and Fludarabine
Affected / at Risk (%)
Total   21/21 (100.00%) 
Blood and lymphatic system disorders   
Bilirubin   10/21 (47.62%) 
Leukocytes   21/21 (100.00%) 
Lymphopenia   21/21 (100.00%) 
Platelets   21/21 (100.00%) 
Infections and infestations   
Neutrophils/gran   21/21 (100.00%) 
Metabolism and nutrition disorders   
Alkaline phosphatase   3/21 (14.29%) 
Hemoglobin (Hgb)   21/21 (100.00%) 
Hyperglycemia   15/21 (71.43%) 
Hyperkalemia   4/21 (19.05%) 
Hypermagnesmia   2/21 (9.52%) 
Hypertriglyceridemia   2/21 (9.52%) 
Hypocalcemia   2/21 (9.52%) 
Hypokalemia   14/21 (66.67%) 
Hyponatremia   8/21 (38.10%) 
Hypophosphatemia   11/21 (52.38%) 
SGOT (AST)   9/21 (42.86%) 
SGPT (ALT)   13/21 (61.90%) 
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: Farid Boulad, MD, Attending
Organization: Memorial Sloan Kettering Cancer Center
Phone: +1212-639-2429
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