A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure
Stopped early · Phase 2
Conditions studied: Aplastic Anemia, Pure Red Cell Aplasia, Diamond Blackfan Anemia
In brief
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.
Key facts
- Study ID
- NCT00001962
- Run by
- National Heart, Lung, and Blood Institute (NHLBI)
- People needed
- 100
- Starts
- 1999-11-01
- Expected to finish
- 2010-09-01
- Last updated by the study team
- 2021-07-09
Who can join
Age: 2 and older. Sex: any. Healthy volunteers: not accepted.
You may not qualify if…
- Current diagnosis or past history of myelodysplastic syndrome or Fanconi's anemia.
- Known allergy to E.coli-derived products.
- Persistent B19 parvovirus infection.
- Evidence of uncontrolled infection.
- Chronic or current clinically significant infection, including HIV positivity or hepatitis B and C virus infection.
- Significant other diseases, congestive heart failure (greater than New York Class II), poorly controlled diabetes mellitus, uncontrolled cardiac arrhythmias.
- Subjects with cancer who are on active chemotherapeutic treatment or who take drugs with hematological effects will not be eligible
- A moribund status or concurrent hepatic, renal, cardiac, metabolic disease of such severity that death within 1-4 weeks from initiation of therapy is likely.
- Recent major surgery.
- Treatment with an investigational agent other than hematopoietic growth factors within 4 weeks of study entry.
- Psychiatric, affective, or other disorder that may compromise the ability to give informed consent or to cooperate in a research study.
- Pregnancy or lactation.
Where it is running
- National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States
Full record on ClinicalTrials.gov
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