Trial Outcomes & Findings for Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia (NCT NCT00229619)
NCT ID: NCT00229619
Last Updated: 2018-09-18
Results Overview
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.
COMPLETED
PHASE2
11 participants
6 months
2018-09-18
Participant Flow
Participant milestones
| Measure |
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.
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|---|---|
|
Overall Study
STARTED
|
11
|
|
Overall Study
COMPLETED
|
11
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia
Baseline characteristics by cohort
| Measure |
Rituximab Subjects
n=11 Participants
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.
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|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
11 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 6 monthsRituximab 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.
Outcome measures
| Measure |
Rituximab Subjects
n=11 Participants
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.
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|---|---|
|
Response to Rituximab
Complete response
|
0 participants
|
|
Response to Rituximab
Partial response
|
2 participants
|
|
Response to Rituximab
No Response
|
9 participants
|
SECONDARY outcome
Timeframe: 3 monthsOutcome measures
| Measure |
Rituximab Subjects
n=11 Participants
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.
|
|---|---|
|
Response Assessment at 3 Months
Complete response
|
0 Participants
|
|
Response Assessment at 3 Months
Partial response
|
1 Participants
|
|
Response Assessment at 3 Months
No response
|
10 Participants
|
SECONDARY outcome
Timeframe: 12 monthsOutcome measures
| Measure |
Rituximab Subjects
n=11 Participants
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.
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|---|---|
|
Response Rates at 12 Months (After the First Dose of Study Med)
Complete response
|
0 Participants
|
|
Response Rates at 12 Months (After the First Dose of Study Med)
Partial response
|
3 Participants
|
|
Response Rates at 12 Months (After the First Dose of Study Med)
Relapse after response
|
1 Participants
|
|
Response Rates at 12 Months (After the First Dose of Study Med)
No response
|
7 Participants
|
Adverse Events
Rituximab Subjects
Serious adverse events
| Measure |
Rituximab Subjects
n=11 participants at risk
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.
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|---|---|
|
Metabolism and nutrition disorders
Acidosis
|
9.1%
1/11
|
|
Infections and infestations
Infection with normal ANC
|
9.1%
1/11
|
|
Vascular disorders
right popliteal artery occlusion
|
9.1%
1/11
|
|
Infections and infestations
Abdominal abscess
|
9.1%
1/11
|
|
Blood and lymphatic system disorders
anemia
|
9.1%
1/11
|
|
Skin and subcutaneous tissue disorders
skin breakdown
|
9.1%
1/11
|
|
Infections and infestations
Cellulitis
|
9.1%
1/11
|
Other adverse events
| Measure |
Rituximab Subjects
n=11 participants at risk
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.
|
|---|---|
|
Blood and lymphatic system disorders
Bleeding
|
9.1%
1/11
|
|
Blood and lymphatic system disorders
Bruising
|
9.1%
1/11
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
9.1%
1/11
|
|
General disorders
Cytokine release syndrome/ acute infusion reaction
|
9.1%
1/11
|
|
General disorders
Pain
|
18.2%
2/11
|
|
Immune system disorders
Serum sickness
|
9.1%
1/11
|
|
General disorders
Fatigue
|
9.1%
1/11
|
|
Infections and infestations
Urinary Tract Infection
|
9.1%
1/11
|
|
Gastrointestinal disorders
Mouth sores
|
9.1%
1/11
|
|
General disorders
Headache
|
9.1%
1/11
|
|
Cardiac disorders
arrythmia
|
9.1%
1/11
|
|
Cardiac disorders
Mild swelling of extremity
|
18.2%
2/11
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea on exertion
|
9.1%
1/11
|
|
General disorders
Pallor
|
9.1%
1/11
|
|
Infections and infestations
strep throat
|
9.1%
1/11
|
|
Skin and subcutaneous tissue disorders
dry, flaking skin temporal area
|
18.2%
2/11
|
|
Immune system disorders
Lymph node
|
9.1%
1/11
|
|
Hepatobiliary disorders
urinary frequency/ dysuria
|
9.1%
1/11
|
|
Infections and infestations
infection (Strep viridans)
|
9.1%
1/11
|
|
General disorders
fever
|
9.1%
1/11
|
|
Blood and lymphatic system disorders
neutropenia
|
9.1%
1/11
|
|
Blood and lymphatic system disorders
Decreased hemoglobin
|
9.1%
1/11
|
|
Blood and lymphatic system disorders
Decreased lymphocytes
|
9.1%
1/11
|
|
Blood and lymphatic system disorders
Decreased platelets
|
9.1%
1/11
|
|
Cardiac disorders
Palpitations
|
9.1%
1/11
|
|
Skin and subcutaneous tissue disorders
Right foot ulcer
|
9.1%
1/11
|
|
Gastrointestinal disorders
Gastroenteritis
|
9.1%
1/11
|
|
Gastrointestinal disorders
Splenomegaly
|
9.1%
1/11
|
|
Hepatobiliary disorders
Increased LFTs
|
9.1%
1/11
|
|
Infections and infestations
Upper respiratory tract
|
9.1%
1/11
|
|
General disorders
Arthralgia
|
9.1%
1/11
|
|
General disorders
tooth pain
|
9.1%
1/11
|
|
Skin and subcutaneous tissue disorders
Lip. Pimple
|
9.1%
1/11
|
Additional Information
Adrian Wiestner MD, NHLBI
NIH National Heart, Lung and Blood Institute
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place