A phase IIIb, open-label, single arm study to evaluate the efficacy and safety of luspatercept in patients with lower-risk MDS and ringsideroblastic phenotype (MDS-RS)

Patients with anemia due to IPSS-R defined very low-, low-, or intermediate-risk MDS-RS who require RBC transfusions and who are refractory, intolerant or ineligible to prior erythropoiesis-stimulating agents (ESA) treatment

RBC-TI rate according to IWG 2018 modified criteria[1] from Week 1 through Week 24

RBC-TI rate according to IWG 2006 criteria from Week 1 through Week 24 and through Week 52, Median time to RBC-TI (Week 1 through Week 24 and through Week 52), Median duration of RBC-TI (Week 1 through Week 24 and through Week 52), Change in RBC units transfused over a fixed 16-weeks period (Week 9 through Week 24 and Week 37 through Week 52) compared to the 16-week period prior to screening, Proportion of subjects achieving mean Hb increase ≥ 1.0 g/dL over ≥ 8 weeks (Week 1 through Week 24 and through Week 52), Proportion of subjects achieving modified hematologic improvement - erythroids (mHI-E) per IWG 2006 criteria (Week 1 through Week 24 and through Week 52), Proportion of subjects achieving hematologic improvement - neutrophils (HI-N) per IWG 2006 criteria (Week 1 through Week 24 and through Week 52), Proportion of subjects achieving hematologic improvement - platelets (HI-P) per IWG 2006 criteria (Week 1 through Week 24 and through Week 52), Mean change in serum ferritin from Week 9 through 24 and Week 37 through Week 52 compared to baseline, Mean change in mean daily dose of ICT from Week 9 through 24 and Week 37 through Week 52 compared to baseline, Proportion of subjects with progression to AML, Overall survival (OS), Safety measures: type, frequency, severity of adverse events (AEs) and relationship to luspatercept, dose reductions and dose delays, Mean change in PRO (via EORTC QLQ-C30) and PerfO (via “Timed Up and Go test” [TUG]) from baseline (Week 1) to Week 52 and to End of Treatment (EOT)

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Austria, Germany, Spain