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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00229619
• Date of registration: 29/09/2005
• Prospective Registration: No
• Primary sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Public title: Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia
• Scientific title: A Pilot Study of Recombinant Humanized Anti- Cluster of Differentiation Antigen 20 (Anti-CD20) Antibody (Rituximab) in Patients With Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia
• Date of first enrolment: September 2005
• Target sample size: 11
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00229619
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Sabrina Martyr, MD
• Affiliation: NIH National Heart, Lung and Blood Institute

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

Diagnosis of acquired moderate aplastic anemia defined as aplastic anemia (hypocellular
bone marrow) and no evidence for an underlying disease process and depression of at least
two out of three blood counts below these values:

- Absolute neutrophil count (ANC) equal to or less than l200/mm(3)

- platelet count equal to or less than 70,000/mm(3)

- anemia with hemoglobin equal to or less than 8.5 g/dl or absolute reticulocyte count
equal to or less than 60,000/mm(3) in transfusion-dependent patients but not
fulfilling the criteria for severe disease defined by bone marrow cellularity less
than 30% (excluding lymphocytes) and depression of at least two of the three
peripheral counts:

- ANC equal to or less than 500/ul

- platelet count equal to or less than 20,000/ul

- reticulocyte count less than 60,000/ul

Or

Diagnosis of pure red cell aplasia or Diamond Blackfan anemia requiring red blood cell
(RBC) transfusions

Pure red cell aplasia is defined by

- anemia,

- reticulocytopenia (reticulocyte count equal to or less than 50,000/ mm(3))

- and absent or decreased marrow erythroid precursors

Diamond Blackfan anemia is defined by

- anemia,

- reticulocytopenia (reticulocyte count equal to or less than 50,000/ mm(3))

- and absent or decreased marrow erythroid precursors diagnosed at an early age

Because this population is prone to dry bone marrow aspirates, subjects from whom
sufficient bone marrow cannot be collected for the evaluation of cellularity will not be
excluded provided they meet all other inclusion criteria based on peripheral blood counts.

Pure Red cell Aplasia and Diamond Blackfan patients must be age greater than or equal to 2
years old and weight greater than 12 kg; Moderate Aplastic anemia patients must be age
greater than or equal to 18.

Refractory to at least 1 course of immunosuppressive therapy or relapsed disease after
prior immunosuppressive therapy (PRCA/DBA patients only).

Patients or their parent(s)/responsible guardian(s) must be able to comprehend and be
willing to sign an informed consent.

EXCLUSION CRITERIA:

Current diagnosis of Fanconi's anemia or other congenital bone marrow failure syndromes
except for DBA

History of a cytogenetic abnormality indicating myelodysplasia (MDS)

Active infection not adequately responding to appropriate therapy

HIV positivity

Positive anti- hepatitis B core antibody (antiHBc) or HBsAG

History of clinically significant arrhythmia

Known anaphylaxis or immunoglobulin E (IgE) mediated hypersensitivity to murine proteins or
to any component of this product.

Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious,
or metabolic disease of such severity that it would preclude the patient's ability to
tolerate protocol therapy, or that death within the next month is likely

Potential subjects with cancer who are on active chemotherapeutic treatment or who take
drugs with hematological effects will not be eligible.

History of recent or ongoing B19 parvovirus infection

Psychiatric, affective, or other disorder that may compromise the ability to give informed
consent or to cooperate in a research study.

Pregnancy or lactation or unwillingness to take contraceptives

Participation in any other investigational drug trial or exposure to other investigational
agents (other than hematopoietic growth factors) within 30 days of study entry. Use of low
dose immunosuppressive agents may continue at the PIs discretion provided that the patient
has been taking this drug for at least 3 months.



Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Anemia, Diamond-Blackfan

Red-Cell Aplasia, Pure

Anemia, Aplastic

Intervention(s)

Drug: Rituximab

Primary Outcome(s)

Response to Rituximab [Time Frame: 6 months]

Secondary Outcome(s)

Response Assessment at 3 Months [Time Frame: 3 months]

Response Rates at 12 Months (After the First Dose of Study Med) [Time Frame: 12 months]

Secondary ID(s)

050244

05-H-0244

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 28/07/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00229619