A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure

Launched by NATIONAL HEART, LUNG, AND BLOOD INSTITUTE (NHLBI) · Jan 18, 2000

Keywords

ClinConnect Summary

Many bone marrow failure syndromes in humans are now recognized to result from immunological mechanisms. These diseases include aplastic anemia; single hematopoietic lineage failures such as pure red cell aplasia, Diamond Blackfan Anemia, agranulocytosis, and amegakaryocytic thrombocytopenic purpura; and some types of myelodysplasia. Patients with these conditions, who may suffer variable degrees of anemia, leukocytopenia, and thrombocytopenia, alone or combination, have been shown to respond to a wide variety of immunosuppressive agents, ranging from corticosteroids to cyclosporine (CSA) a...

Gender

ALL

Eligibility criteria

• -INCLUSION CRITERIA:
• • 1. 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)
• • 2. Age greater than or equal to 2 years old
• • 3. Weight greater than 12 kg
• • 4. 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.

Trial Officials