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Trial record 1 of 1 for:    CA056-025 | MDS | Leipzig, Germany
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ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions (ELEMENT-MDS)

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ClinicalTrials.gov Identifier: NCT05949684
Recruitment Status : Recruiting
First Posted : July 18, 2023
Last Update Posted : May 14, 2024
Sponsor:
Information provided by (Responsible Party):
Bristol-Myers Squibb

Study Description
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Brief Summary:
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).

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndromes Biological: Luspatercept Biological: Epoetin Alfa Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 360 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : October 24, 2023
Estimated Primary Completion Date : June 25, 2027
Estimated Study Completion Date : March 11, 2030

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Luspatercept Biological: Luspatercept
Specified dose on specified days
Other Names:
  • BMS-986346
  • ACE-536
  • Reblozyl®
Active Comparator: Epoetin Alfa Biological: Epoetin Alfa
Specified dose on specified days
Other Names:
  • Epogen®
  • PROCRIT®
  • BINOCRIT


Outcome Measures
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Primary Outcome Measures :
  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.


Secondary Outcome Measures :
  1. 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 ]
  2. 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 48 ]
  3. 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: From Week 49 to Week 96 ]
  4. 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 ]
  5. 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 48 ]
  6. 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: From Week 49 to Week 96 ]
  7. 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 ]
  8. Mean Hb change over fixed 24-week periods compared to the baseline Hb [ Time Frame: Baseline, Week 24, Week 48, Week 72, Week 96 ]
  9. 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 ]
  10. Number of participants with TD by week 48 [ Time Frame: Up to Week 48 ]
  11. 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 ]
  12. Time from first Luspatercept dose to first RBC transfusion [ Time Frame: Up to 5 years ]
  13. Duration of median hematologic improvement in erythroid response(mHI-E) in participants with an increase from baseline in mean Hb values of ≥1.5g/dL in any continuous 16-week interval within 48-week treatment period in absence of transfusion [ Time Frame: Up to Week 48 ]
  14. Duration of median hematologic improvement in erythroid response(mHI-E) in participants with an increase from baseline in mean Hb values of ≥1.5g/dL in any continuous 16-week interval within 96-week treatment period in absence of transfusion [ Time Frame: Up to Week 96 ]
  15. 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 Treatment Period in the absence of transfusion) [ Time Frame: Up to Week 48 ]
  16. 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 96-week Treatment Period in the absence of transfusion) [ Time Frame: Up to Week 96 ]
  17. Number of participants with RBC transfusion independence over at least a consecutive 24-week period [ Time Frame: Up to 5 years ]
  18. Number of transfusions [ Time Frame: Up to 5 years ]
  19. Number of transfusions visits/units [ Time Frame: Up to 5 years ]
  20. 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: Baseline, Up to 5 years ]
  21. Change from baseline in self-reported HRQoL assessed by the European quality of life questionnaire 5-dimension (EQ-5D-5L) [ Time Frame: Baseline, Up to 5 years ]
  22. Number of participants with adverse events (AEs) [ Time Frame: Up to Week 102 ]
  23. Number of participants with antidrug antibody (ADA) (positive or negative) [ Time Frame: Up to Week 102 ]
  24. Pharmacokinetics (PK): Serum concentration [ Time Frame: Up to Week 96 ]
  25. PK: Area under the plasma concentration time curve (AUC) [ Time Frame: Up to Week 96 ]
  26. 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.

  27. 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.

  28. Number of participants with acute myeloid leukemia (AML) progression [ Time Frame: Up to 5 years ]
  29. Time to AML progression [ Time Frame: Up to 5 years ]
  30. Number of participants with high risk myelodysplastic syndromes (MDS) progression [ Time Frame: Up to 5 years ]
  31. Time to high-risk MDS progression [ Time Frame: Up to 5 years ]
  32. Time from date of randomization up to death due to any cause [ Time Frame: Up to 5 years ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 has never received treatment with an erythropoiesis stimulating agent (ESA).
  • 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 mean baseline Hb concentration prior to randomization of ≤ 9.5 g/dL. Mean Hb is defined as the mean of all central/ local/ pretransfusion available Hb measurements during the 16 weeks prior to randomization (with a minimum of 2 measurements at least 1 week apart). Only Hb levels > 21 days following a transfusion are acceptable. The last measurement must be within 35 days of randomization.

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05949684


Contacts
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Contact: BMS Study Connect Contact Center www.BMSStudyConnect.com 855-907-3286 Clinical.Trials@bms.com
Contact: First line of the email MUST contain the NCT# and Site #.

Locations
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Sponsors and Collaborators
Bristol-Myers Squibb
Investigators
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Study Director: Bristol-Myers Squibb Bristol-Myers Squibb
More Information
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Additional Information:

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Responsible Party: Bristol-Myers Squibb
ClinicalTrials.gov Identifier: NCT05949684    
Other Study ID Numbers: CA056-025
2022-500430-29-00 ( Other Identifier: EU CTR Number )
First Posted: July 18, 2023    Key Record Dates
Last Update Posted: May 14, 2024
Last Verified: May 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myers Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosure-commitment.html
Supporting Materials: Study Protocol
Clinical Study Report (CSR)
Time Frame: See Plan Description
Access Criteria: See Plan Description
URL: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosure-commitment.html

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Bristol-Myers Squibb:
Luspatercept
BMS-986346
ACE-536
Myelodysplastic Syndrome
 Epoetin alfa
Erythropoietin stimulating agent (ESA)
Myelodysplastic Syndromes (MDS)
Anaemia
Additional relevant MeSH terms:
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Preleukemia
Myelodysplastic Syndromes
Anemia
Syndrome
Disease
Pathologic Processes
Hematologic Diseases
 Bone Marrow Diseases
Precancerous Conditions
Neoplasms
Epoetin Alfa
Luspatercept
Hematinics