Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia
Status:
Terminated
Trial end date:
2012-12-01
Target enrollment:
Participant gender:
Summary
This is a single-center, single arm, open-label study of oral lenalidomide monotherapy
administered to red blood cell (RBC) transfusion dependent adult subjects with
Diamond-Blackfan Anemia (DBA).
Primary Objective: To evaluate the erythroid response rate as measured by rate of red blood
cell transfusion independence [MDS International Working Group (IWG) 2000 Criteria will be
applied].
Secondary Objective: 1)To evaluate the tolerability and safety profile of lenalidomide in
patients with DBA and other inherited marrow failure syndromes 2) To correlate response to
lenalidomide with biologic surrogates of DBA including ribosomal protein mutation status, ex
vivo erythroid colony growth, and microarray gene expression