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Fludarabine-based Conditioning for Severe Aplastic Anemia (BMT CTN 0301)

This study has been completed.
Sponsor:
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
Blood and Marrow Transplant Clinical Trials Network
National Cancer Institute (NCI)
National Marrow Donor Program
Information provided by (Responsible Party):
Medical College of Wisconsin
ClinicalTrials.gov Identifier:
NCT00326417
First received: May 12, 2006
Last updated: October 3, 2016
Last verified: August 2016
Results First Received: August 10, 2016  
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: Anemia, Aplastic
Interventions: Drug: Fludarabine
Drug: Cyclophosphamide 150mg
Drug: Cyclophosphamide 100mg
Drug: Cyclophosphamide 50mg

  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
Participants were enrolled from February 2006 to August 2007 for Phase I of the trial. Phase II opened in November 2007 and closed to accrual on December 2, 2013.

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 150mg Fludarabine plus 150 mg/kg Cyclophosphamide (total dose)
Cyclophosphamide 100mg Fludarabine plus 100 mg/kg Cyclophosphamide (total dose)
Cyclophosphamide 50mg Fludarabine plus 50 mg/kg Cyclophosphamide (total dose)
Fludarabine Fludarabine only (no Cyclophosphamide administered)

Participant Flow:   Overall Study
    Cyclophosphamide 150mg   Cyclophosphamide 100mg   Cyclophosphamide 50mg   Fludarabine
STARTED   14   41   39   3 
Phase I   6   6   6   3 
Phase II   8   35   33   0 
COMPLETED   0 [1]   41   38   0 [2] 
NOT COMPLETED   14   0   1   3 
Withdrawal by Subject                0                0                1                0 
Arm Closed                7                0                0                3 
Death                7                0                0                0 
[1] Dose level was closed for excessive toxicity after 8 patients in Phase II accrued.
[2] Arm was closed to accrual after 3 patients experienced graft failure.



  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
Cyclophosphamide 100mg Fludarabine plus 100 mg/kg Cyclophosphamide (total dose)
Cyclophosphamide 50mg Fludarabine plus 50 mg/kg Cyclophosphamide (total dose)
Total Total of all reporting groups

Baseline Measures
   Cyclophosphamide 100mg   Cyclophosphamide 50mg   Total 
Overall Participants Analyzed 
[Units: Participants]
 41   38   79 
Age 
[Units: Years]
Median (Full Range)
 17.6 
 (1.9 to 63.3) 
 24.5 
 (0.5 to 65.9) 
 20.6 
 (0.5 to 65.9) 
Age, Customized 
[Units: Participants]
     
< 18   21   11   32 
18 - 40   14   18   32 
> 40   6   9   15 
Gender 
[Units: Participants]
     
Female   20   19   39 
Male   21   19   40 
Race/Ethnicity, Customized 
[Units: Participants]
     
Caucasian   34   30   64 
Hispanic or Latino   6   8   14 
Unknown   1   0   1 
Recipient Cytomegalovirus Status 
[Units: Participants]
     
Positive   26   17   43 
Negative   15   21   36 
Karnofsky Score [1] 
[Units: Participants]
     
100   18   12   30 
90   15   17   32 
80   3   7   10 
70   4   2   6 
60   1   0   1 
[1] Assesses patient self-perceived global quality of life and functioning (excellent, very good, good, fair, poor), where 100 equals perfect quality of life.
Immunosuppressive therapy prior to transplant 
[Units: Participants]
     
Yes   41   35   76 
No   0   3   3 
Donor-recipient HLA match 
[Units: Participants]
     
HLA-A, -B, -C,-DRB1 matched   27   31   58 
Single HLA-locus mismatch   14   7   21 
Type of anti-thymocyte globulin administered 
[Units: Participants]
     
Rabbit-derived   33   29   62 
Horse-derived   7   9   16 
None   1   0   1 


  Outcome Measures
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1.  Primary:   Disease-free Survival (DFS)   [ Time Frame: Day 100 ]

2.  Secondary:   Cumulative Incidence of Graft Failure   [ Time Frame: Day 365 ]

3.  Secondary:   Acute Graft vs Host Disease (GVHD)   [ Time Frame: Day 100 ]

4.  Secondary:   Chronic GVHD   [ Time Frame: Day 365 ]

5.  Secondary:   Overall Survival (OS)   [ Time Frame: Day 365 ]


  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
No text entered.


  More Information
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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: Adam Mendizabal, PhD
Organization: The Emmes Corporation
phone: 301-251-1161
e-mail: amendizabal@emmes.com


Publications of Results:
Other Publications:

Responsible Party: Medical College of Wisconsin
ClinicalTrials.gov Identifier: NCT00326417     History of Changes
Obsolete Identifiers: NCT00335608
Other Study ID Numbers: BMTCTN0301
U01HL069294 ( US NIH Grant/Contract Award Number )
5U01HL069294-05 ( US NIH Grant/Contract Award Number )
5U24CA076518 ( US NIH Grant/Contract Award Number )
Study First Received: May 12, 2006
Results First Received: August 10, 2016
Last Updated: October 3, 2016
Health Authority: United States: Federal Government
United States: Institutional Review Board