A Study of Luspatercept (ACE-536) to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes

Official Title

A Phase 3, Double-blind, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo for the Treatment of Anemia Due to the IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in Subjects With Ring Sideroblasts Who Require Red Blood Cell Transfusions.

Summary:

The study will be conducted in compliance with the International Council on Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements. This is a Phase 3, double-blind, randomized, placebo-controlled, multicentre study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in subjects with anemia due to IPSS-R very low, low, or intermediate MDS with ring sideroblasts who require RBC transfusions.

Trial Description

Primary Outcome:

  • Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 weeks
Secondary Outcome:
  • Red Blood Cell Transfusion Independence (RBC-TI) ≥ 12 weeks
  • Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 weeks
  • Reduction in red blood cell (RBC) units transfused over 16 weeks
  • Proportion of subjects achieving Modified hematologic improvement - erythroid (mHI-E) per International Working Group (IWG) over any consecutive 56 days
  • Mean hemoglobin increase ≥ 1.0 g/dL
  • Duration of Red Blood Cell Transfusion Independence (RBC-TI)
  • Change in EORTC QLQ-C30 score
  • Hematologic improvement - neutrophils (HI-N) per International Working Group (IWG)
  • Mean decrease in serum ferritin
  • Mean decrease in iron chelation therapy (ICT) use
  • Time to red blood cell transfusion independence (RBC-TI)
  • Number of subjects progressing to acute myeloid leukemia (AML)
  • Time to progression to acute myeloid leukemia (AML)
  • Overall survival (OS)
  • Adverse Events (AEs)
  • Frequency of anti-drug antibodies (ADA)
  • Pharmacokinetics - AUC
  • Pharmacokinetics ‐ Cmax
Anemia is considered to be one of the most prevalent cytopenias in patients who have myelodysplastic syndromes, an umbrella term used to describe disorders relating to the ineffective production of red blood cells, white blood cells, and/or platelets. Ranging in severity from mild (asymptomatic) to severe, anemia can result in patients requiring regular red blood cell (RBC) transfusions, which can lead to further complications from iron overload. The goal of this study is to assess the safety and efficacy of luspatercept versus placebo in anemic patients who are categorized as International Prognostic Scoring System-Revised (IPSS-R) very low, low, or intermediate risk Myelodysplastic syndrome (MDS), have ring sideroblasts present, and require constant RBC transfusions. The design of the study will allow a period of initial randomization of patients into either the luspatercept or placebo arm, followed by a double-blind treatment period, and then an MDS disease assessment visit. For those patients that are determined to be experiencing clinical benefit as judged from the study Investigator by this disease assessment visit, they will be permitted to enter the double-blind Extension Phase of the study. Once patients are discontinued from study treatment, they will enter a post treatment follow-up period.

View this trial on ClinicalTrials.gov

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Resources

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