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

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ClinicalTrials.gov Identifier: NCT00229619
Recruitment Status : Completed
First Posted : September 29, 2005
Results First Posted : July 28, 2014
Last Update Posted : September 18, 2018
Sponsor:
Information provided by (Responsible Party):
Adrian Wiestner, M.D., National Heart, Lung, and Blood Institute (NHLBI)

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Anemia, Aplastic
Red-Cell Aplasia, Pure
Anemia, Diamond-Blackfan
Intervention Drug: Rituximab
Enrollment 11
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Rituximab Treated Subjects
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 11
Completed 11
Not Completed 0
Arm/Group Title Rituximab Subjects
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 11
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
<=18 years
0
   0.0%
Between 18 and 65 years
11
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
Female
8
  72.7%
Male
3
  27.3%
1.Primary Outcome
Title Response to Rituximab
Hide Description

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.

Primary endpoint will determine immune response by evaluating changes in peripheral blood counts (platelets, absolute neutrophil count, reticulocyte count, hemoglobin) and transfusion requirements at 6 months. The response wil be will be categorized as complete, partial or no response. Subjects will be categorized as complete responders if their blood counts return to normal. Subjects will categorized as partial responders if there is an improvement in 2 or 3 of the depressed baseline blood counts.

Time Frame 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Rituximab Subjects
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 11
Measure Type: Number
Unit of Measure: participants
Complete response 0
Partial response 2
No Response 9
2.Secondary Outcome
Title Response Assessment at 3 Months
Hide Description [Not Specified]
Time Frame 3 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Rituximab (Rituxan)
Hide Arm/Group Description:

This is a non-randomized, off label, pilot study of humanized anti CD20 rituximab (Rituxan®) in patients with moderate aplastic anemia, pure red cell aplasia or Diamond-Blackfan anemia who have either failed to respond to at least one prior course of immunosuppressive therapy (PRCA/DBA patients only)or who have relapsed disease after prior immunosuppressive therapy (PRCA/DBA patients only).

Rituximab: Rituximab (Rituxan) 375mg/m2 intravenous infusion. The infusion will be once every week for a total of 4 doses.

Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Complete response
0
   0.0%
Partial response
1
   9.1%
No response
10
  90.9%
3.Secondary Outcome
Title Response Rates at 12 Months (After the First Dose of Study Med)
Hide Description [Not Specified]
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Rituximab Treated Subjects
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Complete response
0
   0.0%
Partial response
3
  27.3%
Relapse after response
1
   9.1%
No response
7
  63.6%
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Rituximab Subjects
Hide Arm/Group Description 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.
All-Cause Mortality
Rituximab Subjects
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Rituximab Subjects
Affected / at Risk (%)
Total   4/11 (36.36%) 
Blood and lymphatic system disorders   
anemia * [1]  1/11 (9.09%) 
Infections and infestations   
Infection with normal ANC *  1/11 (9.09%) 
Abdominal abscess *  1/11 (9.09%) 
Cellulitis *  1/11 (9.09%) 
Metabolism and nutrition disorders   
Acidosis *  1/11 (9.09%) 
Skin and subcutaneous tissue disorders   
skin breakdown * [2]  1/11 (9.09%) 
Vascular disorders   
right popliteal artery occlusion *  1/11 (9.09%) 
*
Indicates events were collected by non-systematic assessment
[1]
symptomatic anemia
[2]
Wound complication, non-infectious
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Rituximab Subjects
Affected / at Risk (%)
Total   4/11 (36.36%) 
Blood and lymphatic system disorders   
Bleeding *  1/11 (9.09%) 
Bruising *  1/11 (9.09%) 
neutropenia *  1/11 (9.09%) 
Decreased hemoglobin *  1/11 (9.09%) 
Decreased lymphocytes *  1/11 (9.09%) 
Decreased platelets *  1/11 (9.09%) 
Cardiac disorders   
arrythmia * [1]  1/11 (9.09%) 
Mild swelling of extremity *  2/11 (18.18%) 
Palpitations *  1/11 (9.09%) 
Gastrointestinal disorders   
Mouth sores *  1/11 (9.09%) 
Gastroenteritis *  1/11 (9.09%) 
Splenomegaly *  1/11 (9.09%) 
General disorders   
Cytokine release syndrome/ acute infusion reaction *  1/11 (9.09%) 
Pain * [2]  2/11 (18.18%) 
Fatigue *  1/11 (9.09%) 
Headache *  1/11 (9.09%) 
Pallor *  1/11 (9.09%) 
fever *  1/11 (9.09%) 
Arthralgia *  1/11 (9.09%) 
tooth pain *  1/11 (9.09%) 
Hepatobiliary disorders   
urinary frequency/ dysuria *  1/11 (9.09%) 
Increased LFTs *  1/11 (9.09%) 
Immune system disorders   
Serum sickness *  1/11 (9.09%) 
Lymph node * [3]  1/11 (9.09%) 
Infections and infestations   
Urinary Tract Infection *  1/11 (9.09%) 
strep throat *  1/11 (9.09%) 
infection (Strep viridans) *  1/11 (9.09%) 
Upper respiratory tract *  1/11 (9.09%) 
Respiratory, thoracic and mediastinal disorders   
Cough *  1/11 (9.09%) 
Dyspnea on exertion *  3/11 (27.27%) 
Dyspnea on exertion *  1/11 (9.09%) 
Skin and subcutaneous tissue disorders   
dry, flaking skin temporal area *  2/11 (18.18%) 
Right foot ulcer *  1/11 (9.09%) 
Lip. Pimple *  1/11 (9.09%) 
*
Indicates events were collected by non-systematic assessment
[1]
EKG changes
[2]
Localized pain, line insertion site
[3]
anterior lymph node-supraclavicular
Study was terminated early due to slow accrual. Conclusions on efficacy limited by small sample size.
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
Responsible Party: Adrian Wiestner, M.D., National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier: NCT00229619     History of Changes
Other Study ID Numbers: 050244
05-H-0244 ( Other Identifier: NIH NHLBI )
First Submitted: September 29, 2005
First Posted: September 29, 2005
Results First Submitted: January 28, 2013
Results First Posted: July 28, 2014
Last Update Posted: September 18, 2018