A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure
Aplastic Anemia, Pure Red Cell Aplasia, Diamond Blackfan Anemia
About this trial
This is an interventional treatment trial for Aplastic Anemia focused on measuring Immunosuppression, T-cells, Hematopoiesis, Monoclonal Antibody Therapy, Immunosuppressive Therapy, Aplastic Anemia
Eligibility Criteria
-INCLUSION CRITERIA: Acquired pure red cell aplasia requiring red blood cell (RBC) transfusions defined by anemia, reticulocytopenia (reticulocyte count less than or equal to 50,000/mm(3)) and absent or decreased marrow erythroid precursors Acquired aplastic anemia of moderate severity (In October 2008, this arm was closed by the DSMB when the data was determined sufficient for making statistical inferences regarding the original hypotheses. Diamond Blackfan Anemia (DBA) (In October 2008, accrual of DBAs was closed by the DSMB for lack of accrual) Relapsed patients with severe aplastic anemia (In November 2005 this arm was closed by the DSMB for lack of efficacy) Refractory disease not responding to both horse and rabbit ATG/CsA (In November 2005 this arm was closed for lack of efficacy) Age greater than or equal to 2 years old Weight greater than 12 kg 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 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.
Sites / Locations
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Arms of the Study
Arm 1
Experimental
Daclizumab in participants with a bone marrow failure syndrome
daclizumab, 1 mg/kg, will be given for a total of 5 intravenous infusions. These subjects may be diagnosed with moderate aplastic anemia, pure red cell aplasia, Diamond Blackfan anemia, relapse and refractory severe aplastic anemia will receive treatment. The subjects will be seen and receive the daclizumab infusion biweekly during the treatment period.