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Comparing Luspatercept and Epoetin Alfa for Anemia in Myelodysplastic Syndrome

A Phase 3, Open-label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) vs Epoetin Alfa for the Treatment of Anemia Due to Revised International Prognostic Scoring System (IPSS-R) Very Low, Low, or Intermediate-Risk Myelodysplastic Syndrome (MDS) in Erythropoiesis-Stimulating Agent (ESA)-Naive Participants Who Are Non-Transfusion Dependent (NTD): The "ELEMENT-MDS" Trial

Phase 3 Interventional Bristol-Myers Squibb · NCT05949684

This study is testing whether a new drug called Luspatercept works better than Epoetin Alfa for treating anemia in adults with Myelodysplastic Syndromes who aren't getting blood transfusions.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment360 (estimated)
Ages18 Years and up
SexAll
SponsorBristol-Myers Squibb Industry-sponsored
Drugs / interventionschemotherapy, radiation
Locations169 sites (Clovis, California and 168 other locations)
Trial IDNCT05949684 on ClinicalTrials.gov

What this trial studies

This study aims to evaluate the efficacy and safety of Luspatercept compared to Epoetin Alfa in treating anemia in adults with Myelodysplastic Syndromes (MDS) who are not receiving blood transfusions. Participants must have a documented diagnosis of MDS classified as very low, low, or intermediate-risk and must be naive to erythropoiesis-stimulating agents. The study will assess the impact of these treatments on anemia symptoms and overall health outcomes in this specific patient population.

Who should consider this trial

Good fit: Ideal candidates for this study are adults diagnosed with very low, low, or intermediate-risk MDS who are not dependent on blood transfusions and have not previously received erythropoiesis-stimulating agents.

Not a fit: Patients who are transfusion dependent or have received prior erythropoiesis-stimulating agents may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new effective treatment option for patients with anemia due to MDS, potentially improving their quality of life.

How similar studies have performed: Other studies have shown promise in using Luspatercept for treating anemia in MDS, indicating a potential for success with this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria

* Participant has documented diagnosis of MDS according to World Health Organization (WHO) 2016 that meet IPSS-R classification of very low, low, or intermediate-risk disease, (intermediate-risk of ≤ 3.5 IPSS-R score) confirmed via bone marrow aspirate and:.

  i) \< 5% blasts in bone marrow and \< 1% blasts in peripheral blood.
* Participant is not transfusion dependent (NTD) based on IWG2018 criteria.
* Participant is erythropoiesis-stimulating agent naive. Participants may be randomized at the investigator's discretion if the participant received no more than 2 prior doses of epoetin alfa, epoetin alfa biosimilar, or darbepoetin alfa, with the last dose at least 8 weeks prior to randomization.
* Participant has a baseline endogenous serum erythropoietin (sEPO) level of ≤ 500 U/L.
* Participant has symptoms of anemia:.

  i) Participant records a severity score of "moderate" or greater on at least 1 PGI-S item of fatigue, weakness, shortness of breath, or dizziness performed during the screening period.
* Participant has a baseline Hb concentration prior to randomization of ≤ 9.5 g/dL. The baseline Hb will be calculated using the mean of the two lowest available Hb measurements within 16 weeks prior to randomization and must include at least one central lab Hb reading done within the screening period (no more than 35 days before randomization). The two Hb measurements must have been performed at least seven days apart. Hb levels less than 21 days following RBC transfusion should not be used. Split samples for local assessments are not required.

Exclusion Criteria

* Participant with secondary MDS (that is, MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases).
* Participant with known history of diagnosis of AML.
* Participant with history of cerebrovascular accident (including ischemic, embolic, and hemorrhagic cerebrovascular accident), transient ischemic attack, deep venous thrombosis (including proximal and distal), pulmonary or arterial embolism, arterial thrombosis, or other venous thrombosis within 6 months prior to randomization.
* Participant with a history of pure red cell aplasia and/or antibody against erythropoietin.
* Other protocol-defined Inclusion/Exclusion criteria apply.

Where this trial is running

Clovis, California and 168 other locations

+119 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Myelodysplastic SyndromesLuspaterceptBMS-986346ACE-536Myelodysplastic SyndromeEpoetin alfaErythropoietin stimulating agentAnaemia
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.