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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01187017
Recruitment Status : Completed
First Posted : August 23, 2010
Results First Posted : March 2, 2016
Last Update Posted : March 2, 2016
Information provided by (Responsible Party):
Danielle Townsley, M.D., National Institutes of Health Clinical Center (CC)

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Aplastic Anemia
Severe Aplastic Anemia
Interventions: Drug: Cyclophosphamide
Drug: Fludarabine

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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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
Refractory SAA Refractory Severe aplastic anemia (SAA) subjects

Participant Flow:   Overall Study
    Refractory SAA

  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.
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Reporting Groups
Refractory SAA Refractory Severe aplastic anemia (SAA) subjects

Baseline Measures
   Refractory SAA 
Overall Participants Analyzed 
[Units: Participants]
[Units: Participants]
<=18 years   0 
Between 18 and 65 years   0 
>=65 years   1 
[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.  

  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
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  More Information

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


Responsible Party: Danielle Townsley, M.D., National Institutes of Health Clinical Center (CC) Identifier: NCT01187017     History of Changes
Other Study ID Numbers: 100177
10-H-0177 ( Other Identifier: NIH NHLBI )
First Submitted: August 20, 2010
First Posted: August 23, 2010
Results First Submitted: June 5, 2014
Results First Posted: March 2, 2016
Last Update Posted: March 2, 2016