An Efficacy and Safety Study of AND017 for the Treatment of Anemia Due to Lower Risk Myelodysplastic Syndromes (MDS)
This is a Phase 2, multicenter, randomized, open-lable, dose ranging study to evaluate the efficacy and safety of AND017 for the treatment of anemia due to lower risk Myelodysplastic syndromes (MDS) in patients subjects who are Red blood cell (RBC) non-transfusion dependent (NTD) and low transfusion burden (LTB).
• Diagnosed of primary myelodysplastic syndrome with a PISS-R grading of very low, low or intermediate risk and a bone marrow primitive cell count \< 5%, the time frame for this grading assessment should be at least 12 weeks prior to the first dose
• Non-5q(del)-associated myelodysplastic syndrome.
• Two non-transfused hemoglobin ≥ 6.0 g/dL and \< 10.0 g/dL, averaged over the screening period, at least one week and more apart, and with no more than 1.3 g/dL difference between the two Hb.
• Non-transfused subjects (NTD cohort) defined as no red blood cell transfusion in the 16 weeks prior to randomization or low transfusion load subjects defined as 3-7 pRBC units transfused in the 16 weeks prior to randomization and at least two different time points (LTB-1 cohort) or 1-2 pRBC units transfused at one time point in the 16 weeks prior to randomization ( LTB-2 cohort) (except in the case of transfusion for treatment of other comorbidities such as blood loss, surgery, etc.);
• Baseline EPO level ≤ 500 mU/mL
• Platelets ≥ 30,000 /mm3 and absolute neutrophil count ≥ 800/mm3
• Adequate liver function with:
‣ Total bilirubin \<2 x upper limit of normal (ULN) (subjects with Gilbert's syndrome, i.e., unconjugated hyperbilirubinemia, have a total bilirubin \<3 x ULN)
⁃ Aspartate aminotransferase (AST) \<3 x ULN
⁃ Alanine aminotransferase (ALT) \<3×ULN