A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve

Official Title

A Phase 3, Open-label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alpha for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Due to Myelodysplastic Syndrome (MDS) ESA in Native Subjects Who Require Red Blood Cell Transfusions

Summary:

The purpose of this study is to determine the effectiveness of luspatercept (ACE-536) compared to epoetin alfa on red blood cell (RBC) transfusion independence (for at least 12 weeks) with a concurrent hemoglobin increase of at least 1.5 g/dL in participants with anemia due to revised international prognostic scoring system (IPSS-R) very low, low, or intermediate risk myelodysplastic syndromes (MDS) who require RBC transfusions and have never been exposed to erythropoiesis stimulating agent (ESA).

Trial Description

Primary Outcome:

• Red blood cell transfusion independence (RBC-TI) for 12 weeks (84 days) with a mean hemoglobin increase ≥ 1.5 g/dL

• RBC-TI for 24 weeks
• Mean hemoglobin change over 24 weeks
• Hematologic improvement - erythroid response (HI-E) per International Working Group (IWG)
• Time to HI-E
• RBC-TI for ≥ 12 weeks (84 days)
• Duration of RBC-TI ≥ 12 weeks (84 days)
• Time to RBC-TI ≥ 12 weeks (84 days)
• Time to first red blood cell (RBC) transfusion
• RBC transfusion burden on treatment
• RBC-TI for ≥ 56 days (8 weeks)
• RBC-TI for a consecutive 24-week period
• The European Organization for Research and Treatment of Cancer Quality-of-Life questionnaire (EORTC QLQ-C30)
• The Functional Assessment of Cancer Therapy-Anemia Version 4 (FACT-An) questionnaire
• Number of participants with Adverse Events (AEs)
• Pharmacokinetic - area under the concentration-time curve (AUC)
• Pharmacokinetic - maximum plasma concentration of drug (Cmax)
• Frequency of antidrug antibodies (ADA)
• Number of participants progressing to acute myeloid leukemia (AML)
• Percentage of participants progressing to AML
• Time to AML progression
• Overall survival

View this trial on ClinicalTrials.gov