Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Sabrina Martyr, M.D., National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier:
NCT00229619
First received: September 29, 2005
Last updated: July 1, 2014
Last verified: July 2014
Results First Received: January 28, 2013  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Anemia, Aplastic
Red-Cell Aplasia, Pure
Anemia, Diamond-Blackfan
Intervention: Drug: Rituximab

  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
No text entered.

Reporting Groups
  Description
Rituximab Treated Subjects Rituximab will be given to moderate aplastic anemia (MAA), pure red cell aplasia or Diamond Blackfan anemia subjects. Rituxmiab will be given to evaluate if these bone marrow failure syndrome subjects will have an immune response to the intervention. The subjects will receive 375 mg/ meters squared of rituximab which will be infused intravenously once evey week for a total of 4 doses.

Participant Flow:   Overall Study
    Rituximab Treated Subjects  
STARTED     11  
COMPLETED     11  
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.
No text entered.

Reporting Groups
  Description
Rituximab Subjects Rituximab will be given to moderate aplastic anemia (MAA), pure red cell aplasia or Diamond Blackfan anemia subjects. Rituxmiab will be given to evaluate if these bone marrow failure syndrome subjects will have an immune response to the intervention. The subjects will receive 375 mg/ meters squared of rituximab which will be infused intravenously once evey week for a total of 4 doses.

Baseline Measures
    Rituximab Subjects  
Number of Participants  
[units: participants]
  11  
Age  
[units: participants]
 
<=18 years     0  
Between 18 and 65 years     11  
>=65 years     0  
Gender  
[units: participants]
 
Female     8  
Male     3  



  Outcome Measures

1.  Primary:   Response to Rituximab   [ Time Frame: 6 months ]

2.  Secondary:   Secondary Endpoints Include Response at 3 Months, Durability of Response, Disease Progression, Survival and the Response to a Second Course of Therapy When Indicated.   [ Time Frame: 3 months ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

3.  Secondary:   Response Rates at 3 Months (After the First Dose of Study Med)   [ Time Frame: 3 months ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

4.  Secondary:   Relapse, Disease Progression, Survival, Change in Transfusion Requirements, Response to Second Cycle of Rituximab and Compare the Efficacy and Safety Profile With a Similar Patient Population Who Are Participating on the Daclizumab Trial   [ Time Frame: 6 months ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   Yes


  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: Adrian Wiestner MD, NHLBI
Organization: NIH National Heart, Lung and Blood Institute
phone: 301-594-6855
e-mail: wiestnera@nhlbi.nih.gov


Publications:

Responsible Party: Sabrina Martyr, M.D., National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier: NCT00229619     History of Changes
Other Study ID Numbers: 050244, 05-H-0244
Study First Received: September 29, 2005
Results First Received: January 28, 2013
Last Updated: July 1, 2014
Health Authority: United States: Federal Government