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NCT Number: NCT05949684

ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions

The purpose of the study is to compare the efficacy and safety of Luspatercept vs epoetin alfa in the treatment of anemia in adults due to IPSS-R very low, low, intermediate-risk MDS in ESA-naïve participants who are non-transfusion dependent (NTD).

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Myelodysplastic Syndromes Anemia Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Local Institution - 0009, Buenos Aires, Argentina

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Luspatercept

Biological

Specified dose on specified days

Other names: BMS-986346, ACE-536, Reblozyl®

epoetin alfa

Biological

Specified dose on specified days

Other names: Epogen®, PROCRIT®, BINOCRIT

Primary outcomes

  1. Number of participants with lower-risk non-transfusion dependent myelodysplastic syndromes (NTD-MDS) who converted to Transfusion Dependence (TD) during any continuous 16-week interval within the 96-week treatment period

    Time frame: Up to Week 96

    TD is defined as ≥ 3 red blood cells (RBC) units/16 weeks assessed by International Working Group (IWG) 2018.

  2. Number of participants with an increase from baseline in mean Hb values of ≥ 1.5 grams/deciliter (g/dL) in any continuous 16-week interval within the 48 week Treatment Period in the absence of transfusion

    Time frame: Up to Week 48

Secondary outcomes

  1. Number of participants with an increase from baseline in mean Hb values of ≥ 1.5 g/dL in any continuous 24-week interval within the 48-week and 96-week treatment period in the absence of transfusion

    Time frame: Up to Week 96

  2. Number of participants with an increase from baseline in mean Hb values of ≥ 1.0 g/dL in any continuous 24-week interval within the 48-week and 96-week treatment period in the absence of transfusion

    Time frame: Up to Week 96

  3. Mean Hb change over fixed 24-week periods compared to the baseline Hb

    Time frame: Week 24, Week 48, Week 72, Week 96

  4. Number of participants with an increase from baseline in mean Hb values of ≥ 1.5 g/dL in any continuous 16-week interval within the 96-week treatment period in the absence of transfusion

    Time frame: Up to Week 96

  5. Number of participants with TD by week 48

    Time frame: Up to Week 48

  6. Time to TD (IWG 2018 defined as ≥ 3 RBC units/16 weeks) during any continuous 16-week interval until the end of study

    Time frame: Up to 5 years

  7. Time from first Luspatercept dose to first RBC transfusion

    Time frame: Up to 5 years

  8. Duration of mHI-E in participants with an increase from baseline in mean Hb values of ≥1.5g/dL in any continuous 16-week interval within the 48-week and 96-week treatment period in absence of transfusion

    Time frame: Up to Week 96

    mHI-E: median hematologic improvement in erythroid response

  9. Time from first dose to first day of response (increase in mean Hb values of ≥ 1.5 g/dL in any continuous 16-week interval within the 48-week and 96-week Treatment Period in the absence of transfusion)

    Time frame: Up to Week 96

  10. Number of participants with RBC transfusion independence over at least a consecutive 24-week period

    Time frame: Up to 5 years

  11. Number of transfusions

    Time frame: Up to 5 years

  12. Number of transfusions visits/units

    Time frame: Up to 5 years

  13. Change from baseline in subscales of self-reported health-related quality-of-life (HRQoL) assessed by the Functional Assessment of Cancer Therapy - Anemia (FACT-An)

    Time frame: Up to 5 years

  14. Change from baseline in self-reported HRQoL assessed by the European quality of life questionnaire 5-dimension (EQ-5D-5L)

    Time frame: Up to 5 years

  15. Number of participants with adverse events (AEs)

    Time frame: Up to 42 days post last dose

  16. Number of participants with antidrug antibody (ADA) (positive or negative)

    Time frame: Up to Week 96

  17. Pharmacokinetics (PK): Serum concentration

    Time frame: Up to Week 96

  18. PK: Area under the plasma concentration time curve (AUC)

    Time frame: Up to Week 96

  19. Number of participants with a platelet response at Week 24, Week 48 and Week 96

    Time frame: Up to Week 96

    Platelet response is defined as an increase from baseline in number of platelets to ≥ 30 × 10^9/L at Week 24, Week 48 and Week 96.

  20. Number of participants with a neutrophil response at Week 24, Week 48 and Week 96

    Time frame: Up to Week 96

    Neutrophil response is defined as an absolute increase from baseline of > 0.5 × 10^9/L neutrophils at Week 24, Week 48 and Week 96.

  21. Number of participants with acute myeloid leukemia (AML) progression

    Time frame: Up to 5 years

  22. Time to AML progression

    Time frame: Up to 5 years

  23. Number of participants with high risk myelodysplastic syndromes (MDS) progression

    Time frame: Up to 5 years

  24. Time to high-risk MDS progression

    Time frame: Up to 5 years

  25. Time from date of randomization up to death due to any cause

    Time frame: Up to 5 years

Sponsors and collaborators

Lead sponsor

Bristol-Myers Squibb

Industry

Registry information

Official study title

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

Acronym: ELEMENT-MDS

Important dates

Study start
2023
Primary completion
2028
Study completion
2031
First posted
Jul 18, 2023
Registry last updated
Jun 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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