A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure
Status:
Terminated
Trial end date:
2010-09-01
Target enrollment:
Participant gender:
Summary
Participants in this study are suffering from rare and serious blood disorders. In aplastic
anemia, the bone marrow stops producing red blood cells, platelets, and white blood cells. In
pure red cell aplasia, the bone marrow stops producing red cells, and in amegakaryocytic
thrombocytopenic purpura, the bone marrow stops producing platelets.
Current treatment approaches for these disorders include bone marrow transplant and/or
immunosuppression. However, bone marrow transplant is not always possible, and
immunosuppression has serious side effects.
This study will investigate whether daclizumab can be used to treat these disorders.
Daclizumab is a genetically engineered human antibody that blocks the interleukin-2 receptor
on immune cells. It has been used successfully in many transplant patients to reduce the rate
of organ rejection.
Participants will undergo a complete history and physical examination. A bone marrow
aspiration and biopsy will be performed to confirm the type of bone marrow failure. About 5
tablespoons of blood will be drawn for baseline tests and research purposes. Daclizumab will
be administered every 2 weeks by vein in a 30-minute infusion. The first dose will be given
at NIH and the next four may be given at NIH or by the participant's primary hematologist.
The treatment will last 8 weeks. Participants must also see their referring physician or NIH
physicians every 2 weeks for blood counts. In the fourth and eighth weeks of the study and at
the 3-month follow-up visit, 2 tablespoons of blood will be drawn at NIH. At the 1-month
follow-up visit to NIH, 5 tablespoons of blood will be drawn and another bone marrow
aspiration and biopsy performed.
Risks from bone marrow aspiration and biopsy and blood draws include discomfort. Daclizumab
is usually well-tolerated; however, it may weaken immunity against certain bacteria and
viruses.
Phase:
Phase 2
Details
Lead Sponsor:
National Heart, Lung, and Blood Institute (NHLBI) Neal Young, M.D.