Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia
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
|Anemia Leukemia Acute Myeloid Leukemia (AML) Myelodysplastic Syndromes (MDS)||Drug: Lenalidomide||Phase 1|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||A Pilot Study of Lenalidomide in Adult Diamond-Blackfan Anemia Patients With Red Blood Cell Transfusion-Dependent Anemia|
- Red Blood Cell (RBC) Transfusion Independence [ Time Frame: 6 months ]Red blood cell (RBC) transfusion independence is reported as the number of subjects who achieve a continuous absence of the intravenous infusion of any RBC transfusion during any consecutive "rolling" 56 days during the treatment period.
- Red Blood Cell (RBC) Transfusions [ Time Frame: 6 months ]The effect on red blood cell (RBC) transfusions was assessed as the number of participants that achieved a greater than 50% decrease in RBC transfusion requirements.
- Hemoglobin Concentration [ Time Frame: 6 months ]The effect on hemoglobin concentration was assessed as the change from baseline, measured in g/dL.
- Neutrophil Response [ Time Frame: 6 months ]The effect on neutrophil levels was assessed as the change in neutrophil count from baseline.
- Platelet Response [ Time Frame: 6 months ]The effect on platelet levels as assessed as the change in platelet count from baseline.
- Duration of Response [ Time Frame: 6 months ]The response duration was measured from the last of the consecutive 56 days during which the subject was free of red blood cells (RBC) transfusions to the date of the first RBC transfusion after the 56-day RBC-transfusion-free period.
- Toxicity [ Time Frame: 6 months ]Toxicity was assessed as the number of adverse events related to lenalidomide.
|Study Start Date:||November 2009|
|Study Completion Date:||December 2012|
|Primary Completion Date:||October 2012 (Final data collection date for primary outcome measure)|
Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
2.5 mg/wk up to 5 mg 3x/wk
Please refer to this study by its ClinicalTrials.gov identifier: NCT01034592
|United States, California|
|Stanford University School of Medicine|
|Stanford, California, United States, 94305|
|Principal Investigator:||Jason Robert Gotlib||Stanford University|