A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Danielle Townsley, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier:
NCT01187017
First received: August 20, 2010
Last updated: February 3, 2016
Last verified: February 2016
Results First Received: June 5, 2014  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Aplastic Anemia
Neutropenia
Pancytopenia
Severe Aplastic Anemia
Interventions: Drug: Cyclophosphamide
Drug: 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
Refractory SAA Refractory Severe aplastic anemia (SAA) subjects

Participant Flow:   Overall Study
    Refractory SAA  
STARTED     1  
COMPLETED     1  
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.
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Reporting Groups
  Description
Refractory SAA Refractory Severe aplastic anemia (SAA) subjects

Baseline Measures
    Refractory SAA  
Number of Participants  
[units: participants]
  1  
Age  
[units: participants]
 
<=18 years     0  
Between 18 and 65 years     0  
>=65 years     1  
Gender  
[units: participants]
 
Female     1  
Male     0  
Region of Enrollment  
[units: participants]
 
United States     1  



  Outcome Measures

1.  Primary:   Response Rate at 6 Months   [ Time Frame: 6 months ]

2.  Secondary:   Secondary Endpoints Will Evaluated for the Study to Include: (a) Hematologic Response at 3 and 12 Months and Yearly Thereafter; (b) Relapse (c) Clonal Evolution to Paroxysmal Nocturnal Hemoglobinuria (PNH), Myelodysplasia or Acute Leukemia; (e) Survival.   [ Time Frame: 12 months ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No


  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
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  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: Danielle Townsley, M.D.
Organization: NIHNHLBI
phone: 301-402-3477
e-mail: townsleydm@nhlbi.nih.gov


Publications:

Responsible Party: Danielle Townsley, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT01187017     History of Changes
Other Study ID Numbers: 100177
10-H-0177 ( Other Identifier: NIH NHLBI )
Study First Received: August 20, 2010
Results First Received: June 5, 2014
Last Updated: February 3, 2016
Health Authority: United States: Food and Drug Administration