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A Phase 1b/2 Study Evaluating the Safety and Efficacy of Intravenous LB-100 in Patients with Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) who had Disease Progression or are Intolerant to Prior Therapy
The purpose of this study is to test the safety and efficacy (benefits) of an investigational drug LB-100, for treatment of myelodysplastic syndromes. LB-100 has previously been administered to patients with various solid tumors. In this study, LB-100 will be administered as an intravenous infusion over 120 minutes. This study will be conducted in 2 phases. In phase Ib, escalating doses of LB-100 will be administered to patients to study the safety and to determine a safe dose of LB-100. In phase 2, patients will be administered LB-100 at the dose that was found to be safe in phase Ib. The efficacy (benefits) and safety of LB-100 will be determined in this phase of the study.
Primary Objective (Phase1b): 1.To determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of LB-100 as determined by dose-limiting toxicities (DLTs) Primary Objective (Phase 2): 2.To estimate overall best response rates of LB-100 using standard international working group (IWG) 2006 response criteria Secondary Objectives (Phase 1b and 2): 3.To characterize the plasma pharmacokinetics (PK) of LB-100 (Phase 1b only) 4.To evaluate the effect of LB-100 on the hematologic and cytogenetic response in patients with deletion 5q (del(5q)) MDS 5.To estimate the duration of response 6.To estimate the time to AML transformation of subjects on LB-100 7.To characterize in vivo LB-100 target inhibition 8.To characterize the effect of LB-100 treatment on erythropoietin signaling 9.To determine whether recurrent genetic mutations are predictive of LB-100 response