A Phase 1b/2 Study Evaluating the Safety and Efficacy of Canakinumab with Darbepoetin alfa in Patients with Lower-Risk Myelodysplastic Syndromes (MDS) who have Failed Erythropoietin Stimulating Agents (ESA)
The purpose of the study is to find a safe dose and evaluate the effectiveness of the study drug, canakinumab, in combination with a commonly used therapy, darbepoetin alfa.
Primary Objective (Phase 1b) To determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of canakinumab as determined by DLTs. Primary Objective (Phase 2) To determine the rate of hematologic improvement-erythroid (HI-E), defined as red blood cell transfusion independence (RBC-TI) of at least 8 weeks in transfusion dependent patients or a mean Hgb increase of 1.5g/dL above baseline sustained for at least 8 weeks in non-transfusion dependent patients. Secondary Objectives (Phase 1b and 2) 1. To determine the duration of HI-E response 2. To determine the degree of reduction in red blood cell transfusions 3. To determine the overall response rate (ORR) (IWG 2006) 4. To determine the duration of response by IWG 2006 criteria 5. To determine the progression free survival (PFS) 6. To determine the effect on patient s symptoms and quality of life (QoL) 7. To determine whether recurrent genetic mutations are predictive of response 8. To characterize in vivo IL-1 inhibition with canakinumab 9. To evaluate the relationship between changes in innate immune and pyroptosis biomarker indices with response to treatment. 10. To determine the prevalence of infections associated with canakinumab therapy in MDS patients.
Canakinumab (); Darbepoetin alfa ()
> Adequate organ function as defined by laboratory values per protocol
> Use of chemotherapeutic agents or experimental agents (agents that are not commercially available) for the treatment of MDS within 14 days of the first day of study drug treatment.
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