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

Conditions

• Anemia, Aplastic
• Anemia, Diamond-Blackfan
• Red-Cell Aplasia, Pure

Eligibility Criteria

Sex

ALL

Age

2 Years - N/A

Healthy Volunteers

Not accepted

Interventions

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

Study Details

This study will test whether the immune-suppressing drug rituximab can increase blood counts and reduce the need for transfusions in patients with moderate aplastic anemia, pure red cell aplasia, or Diamond Blackfan anemia. These are rare and serious blood disorders in which the immune system turns against bone marrow cells, causing the bone marrow to stop producing red blood cells in patients with pure red cell aplasia and Diamond Blackfan anemia, and red blood cells, white blood cells and platelets in patients with aplastic anemia. Rituximab is a laboratory-made monoclonal antibody that recognizes and destroys white blood cells called lymphocytes that are responsible for destroying bone marrow cells in these diseases. The drug is currently approved by the Food and Drug Administration for treating patients with B-cell non-Hodgkin lymphoma, a disease of white blood cells.

Key Dates

Start date

Sep 30, 2005

Status verified

Aug 2018

Primary completion

Jun 30, 2010

Completion

Jun 30, 2010

Study Design

Enrollment

11 participants (actual)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Rituximab (Rituxan)
  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).

Primary Outcome Measure

Response to Rituximab [ Time Frame: 6 months ]

Locations (1)

Find similar trials in Bethesda, MD