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Myelodysplastic SyndromesPhase 3ELEMENT-MDSNCT05949684

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

A Phase 3 study testing Luspatercept and Epoetin Alfa in people with Myelodysplastic Syndromes, run by Bristol-Myers Squibb at 144 sites in 19 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial 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). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndromes
What is being tested?
Luspatercept and Epoetin Alfa
Could I receive a placebo?
No, everyone receives an active treatment
How many people
402 participants
Where
144 sitesin 19 countries
Study dates
24 Oct 2023to 30 May 2031
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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:.
  • < 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:.
  • 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.

Who cannot join

  • 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.

Trial locations and contacts

144 sites in 19 countries.

144 sites in 19 countries

United States33 sites

  • Local Institution - 0191

    Baton Rouge, Louisiana, 70808

  • Local Institution - 0007

    Bethesda, Maryland, 20817

  • Local Institution - 0272

    Chicago, Illinois, 60611

  • Local Institution - 0070

    Clovis, California, 93611

  • Local Institution - 0206

    Columbus, Ohio, 43210

  • Local Institution - 0044

    Covington, Louisiana, 70433

  • Halifax Health Medical Center

    Daytona Beach, Florida, 32114

  • Local Institution - 0270

    Dyer, Indiana, 46311

  • Local Institution - 0202

    Fort Myers, Florida, 33901

  • Local Institution - 0179

    Fountain Valley, California, 92708

  • Local Institution - 0212

    Hackensack, New Jersey, 07601

  • Local Institution - 0227

    Hartford, Connecticut, 06102

  • Local Institution - 0128

    Hattiesburg, Mississippi, 39401

  • Local Institution - 0240

    Honolulu, Hawaii, 96813

  • Local Institution - 0228

    Houston, Texas, 77030

  • Local Institution - 0180

    Jacksonville, Florida, 32207

  • Local Institution - 0203

    Kennewick, Washington, 99336

  • Local Institution - 0174

    Knoxville, Tennessee, 37920

  • Local Institution - 0211

    Los Alamitos, California, 90720

  • Local Institution - 0258

    Los Angeles, California, 90095

  • Local Institution - 0224

    Margate, Florida, 33063

  • Local Institution - 0173

    New Haven, Connecticut, 06510

  • Local Institution - 0247

    Orange, California, 92868

  • Local Institution - 0209

    Oxnard, California, 93030

  • Local Institution - 0163

    Plantation, Florida, 33322

  • Local Institution - 0236

    Richmond, Virginia, 23219

  • Local Institution - 0229

    Roanoke, Virginia, 24014

  • Local Institution - 0222

    Saint Louis Park, Minnesota, 55426

  • Local Institution - 0184

    Skokie, Illinois, 60077

  • Local Institution - 0201

    St. Petersburg, Florida, 33705

  • Local Institution - 0238

    Tamarac, Florida, 33321

  • Local Institution - 0106

    Tampa, Florida, 33612

  • Local Institution - 0183

    Walnut Creek, California, 94598

Other trials in United States

China23 sites

  • Local Institution - 0217

    Beijing, Beijing Municipality, 100044

  • Local Institution - 0030

    Beijing, Beijing Municipality, 100730

  • Local Institution - 0043

    Changchun, Jilin, 130021

  • Local Institution - 0089

    Changsha, Hunan, 410008

  • Local Institution - 0075

    Chengdu, Sichuan, 610041

  • Local Institution - 0223

    Chongqing, Chongqing Municipality, 400016

  • Local Institution - 0029

    Hangzhou, Zhejiang, 310003

  • Local Institution - 0038

    Harbin, Heilongjiang, 150010

  • Local Institution - 0109

    Hefei, Anhui, 230001

  • Local Institution - 0079

    Nanchang, Jiangxi, 330006

  • Local Institution - 0032

    Nanjing, Jiangsu, 210029

  • Local Institution - 0281

    Ningbo, Zhejiang, 315010

  • Local Institution - 0111

    Qingdao, Shandong, 266003

  • Local Institution - 0218

    Shanghai, Shanghai Municipality, 200030

  • Local Institution - 0048

    Shenyang, Liaoning, 110001

  • Local Institution - 0266

    Shijiazhuang, Hebei, 050000

  • Local Institution - 0083

    Suzhou, Jiangsu, 215006

  • Local Institution - 0103

    Tianjin, Tianjin Municipality, 300020

  • Local Institution - 0033

    Wenzhou, Zhejiang, 32500

  • Local Institution - 0108

    Wuhan, Hubei, 430058

  • Local Institution - 0216

    Xi’an, Shanxi, 710068

  • Local Institution - 0220

    Xiamen, Fujian, 361003

  • Local Institution - 0194

    Zhengzhou, Henan, 450003

Other trials in China

Germany11 sites

  • Local Institution - 0169

    Berlin, 10117

  • Local Institution - 0028

    Berlin, 14195

  • Local Institution - 0207

    Düsseldorf, 40225

  • Local Institution - 0261

    Erding, Bavaria, 85435

  • Local Institution - 0251

    Jena, Thuringia, 07747

  • Local Institution - 0168

    Koblenz, Rhineland-Palatinate, 56068

  • Local Institution - 0171

    Leipzig, Saxony, 04103

  • Local Institution - 0255

    Lübeck, 23562

  • Local Institution - 0259

    Münster, North Rhine-Westphalia, 48153

  • Local Institution - 0167

    Mutlangen, 73557

  • Local Institution - 0166

    Würzburg, 97080

Other trials in Germany

Italy10 sites

  • Local Institution - 0135

    Bologna, 40138

  • Local Institution - 0138

    Florence, Tuscany, 50134

  • Local Institution - 0137

    Milan, Lombardy, 20122

  • Local Institution - 0134

    Milan, Milano, 20162

  • Local Institution - 0076

    Reggio Calabria, 89133

  • Local Institution - 0244

    Roma, 00168

  • Local Institution - 0256

    Rome, Lazio, 00133

  • Local Institution - 0136

    Rozzano, Milano, 20089

  • Local Institution - 0085

    Turin, Piedmont, 10128

  • Local Institution - 0133

    Verona, 37134

Other trials in Italy

France9 sites

  • Local Institution - 0269

    Angers, Maine-et-Loire, 49933

  • Local Institution - 0116

    La Tronche, Isère, 38700

  • Local Institution - 0011

    Nice, Alpes-Maritimes, 06202

  • Local Institution - 0015

    Paris, 75010

  • Local Institution - 0016

    Pessac, Aquitaine, 33600

  • Local Institution - 0140

    Toulouse, 31100

  • Local Institution - 0019

    Tours, Indre-et-Loire, 37032

  • Local Institution - 0165

    Vandœuvre-lès-Nancy, Lorraine, 54511

  • Local Institution - 0248

    Villejuif, Val-de-Marne, 94805

Other trials in France

Spain8 sites

  • Local Institution - 0198

    Barcelona, Barcelona [Barcelona], 08035

  • Local Institution - 0199

    Granada, 18012

  • Local Institution - 0197

    L'Hospitalet de Llobregat, Catalunya [Cataluña], 08908

  • Local Institution - 0252

    Madrid, 28006

  • Local Institution - 0271

    Oviedo, 33011

  • Local Institution - 0200

    Salamanca, 37007

  • Local Institution - 0274

    Seville, 41013

  • Local Institution - 0196

    Valencia, Valenciana, Comunitat, 46010

Other trials in Spain

Australia6 sites

  • Local Institution - 0071

    Blacktown, New South Wales, 2148

  • Local Institution - 0113

    Camperdown, New South Wales, 2050

  • Local Institution - 0054

    Clayton, Victoria, 3168

  • Local Institution - 0254

    Coffs Harbour, New South Wales, 2450

  • Local Institution - 0246

    Gold Coast, Queensland, 4217

  • Local Institution - 0052

    Heidelberg, Victoria, 3084

Other trials in Australia

Greece5 sites

  • Local Institution - 0082

    Alexandroupoli, Anatolikí Makedonía Kai Thráki, 681 00

  • Local Institution - 0084

    Athens, Attikí, 115 27

  • Local Institution - 0245

    Athens, Attikí, 11527

  • Local Institution - 0131

    Chaïdári, Attikí, 12462

  • Local Institution - 0132

    Pátrai, Achaḯa, 26504

Other trials in Greece

India5 sites

  • Local Institution - 0243

    Ahmedabad, Gujarat, 380009

  • Local Institution - 0177

    Bengaluru, Karnataka, 560027

  • Local Institution - 0178

    Bhubaneswar, Odisha, 751003

  • Local Institution - 0193

    New Delhi, National Capital Territory of Delhi, 110029

  • Local Institution - 0235

    New Delhi, National Capital Territory of Delhi, 110085

Other trials in India

Brazil4 sites

  • Local Institution - 0059

    Fortaleza, Ceará, 60430-270

  • Local Institution - 0101

    Porto Alegre, Rio Grande do Sul, 90610-000

  • Local Institution - 0056

    Rio de Janeiro, 20211-030

  • Local Institution - 0003

    São Paulo, 05403-000

Other trials in Brazil

Canada4 sites

  • Local Institution - 0049

    Calgary, Alberta, T2N 5G2

  • Local Institution - 0050

    London, Ontario, N6A 5W9

  • Local Institution - 0017

    Toronto, Ontario, M4N 3M5

  • Local Institution - 0112

    Vancouver, British Columbia, V6Z 1Y6

Other trials in Canada

Chile4 sites

  • Local Institution - 0280

    Santiago, Santiago Metropolitan, 7500587

  • Local Institution - 0278

    Santiago, Santiago Metropolitan, 7500921

  • Local Institution - 0276

    Santiago, Santiago Metropolitan, 7580206

  • Local Institution - 0277

    Santiago, Santiago Metropolitan, 8420383

Other trials in Chile

Colombia4 sites

  • Local Institution - 0147

    Medellín, Antioquia, 05034

  • Local Institution - 0149

    Montería, 230002

  • Local Institution - 0267

    Piedecuesta, Santander Department, 681017

  • Local Institution - 0152

    Valledupar, Cesar Department, 200001

Other trials in Colombia

Mexico4 sites

  • Local Institution - 0069

    Huixquilucan, State of Mexico, 52787

  • Local Institution - 0142

    Mexico City, 14080

  • Local Institution - 0156

    Oaxaca City, Oaxaca, 68020

  • Local Institution - 0150

    Puebla City, 72424

Other trials in Mexico

Poland4 sites

  • Local Institution - 0060

    Gdansk, 80-952

  • Local Institution - 0061

    Katowice, 40-519

  • Local Institution - 0130

    Wałbrzych, Lower Silesian Voivodeship, 58-309

  • Local Institution - 0062

    Warsaw, 02-172

Other trials in Poland

Argentina3 sites

  • Local Institution - 0010

    Buenos Aires, C1425DND

  • Local Institution - 0009

    Buenos Aires, C1426ANZ

  • Local Institution - 0012

    Buenos Aires, CP1280AEB

Other trials in Argentina

Czechia3 sites

  • Local Institution - 0080

    Brno, Brno-město, 625 00

  • Local Institution - 0092

    Ostrava, Moravian-Silesian Region, 708 52

  • Local Institution - 0090

    Prague, 12808

Other trials in Czechia

Hungary3 sites

  • Local Institution - 0026

    Budapest, 1088

  • Local Institution - 0024

    Eger, Heves County, 3300

  • Local Institution - 0021

    Nyíregyháza, Szabolcs-Szatmár-Bereg, 4400

Other trials in Hungary

Puerto Rico1 site

  • Local Institution - 0219

    San Juan, 00917

Other trials in Puerto Rico

About this study

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

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).

Being tested
Luspatercept, Epoetin Alfa
Keywords
Luspatercept, BMS-986346, ACE-536, Myelodysplastic Syndrome, Epoetin alfa, Erythropoietin stimulating agent (ESA), Myelodysplastic Syndromes (MDS), Anaemia
Medical subject headings
Myelodysplastic Syndromes, Anemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LuspaterceptBiological

    Also called BMS-986346, ACE-536, Reblozyl®

    Specified dose on specified days

  • Epoetin AlfaBiological

    Also called Epogen®, PROCRIT®, BINOCRIT

    Specified dose on specified days

Study groups in NCT05949684
Study groupRoleWhat they receive
LuspaterceptExperimentalNot described
Receives: Biological: Luspatercept
Epoetin AlfaActive comparatorNot described
Receives: Biological: Epoetin Alfa

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

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

    Measured: Up to Week 96

  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

    Measured: Up to Week 48

Secondary outcomes (25)

  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

    Measured: 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

    Measured: Up to Week 96

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

    Measured: 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

    Measured: Up to Week 96

  5. Number of participants with TD by week 48

    Measured: 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

    Measured: Up to 5 years

  7. Time from first Luspatercept dose to first RBC transfusion

    Measured: 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

    mHI-E: median hematologic improvement in erythroid response

    Measured: Up to Week 96

  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)

    Measured: Up to Week 96

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

    Measured: Up to 5 years

  11. Number of transfusions

    Measured: Up to 5 years

  12. Number of transfusions visits/units

    Measured: 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)

    Measured: 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)

    Measured: Up to 5 years

  15. Number of participants with adverse events (AEs)

    Measured: Up to 42 days post last dose

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

    Measured: Up to Week 96

  17. Pharmacokinetics (PK): Serum concentration

    Measured: Up to Week 96

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

    Measured: Up to Week 96

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

    Measured: Up to Week 96

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

    Measured: Up to Week 96

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

    Measured: Up to 5 years

  22. Time to AML progression

    Measured: Up to 5 years

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

    Measured: Up to 5 years

  24. Time to high-risk MDS progression

    Measured: Up to 5 years

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

    Measured: Up to 5 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Jul 2023
  2. Study start24 Oct 2023
  3. Primary completion24 Mar 2028 (estimated)
  4. Study completion30 May 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
402 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05949684
Sponsor's protocol ID
CA056-025
Other IDs
2022-500430-29
Acronym
ELEMENT-MDS
Status last verified
Jun 2026
First submitted
10 Jul 2023
Last updated
30 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myer Squibb's data sharing policy and process can be found at https://www.bms.com/researchers-and-partners/clinical-trials-and-research.html

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Luspatercept and Epoetin Alfa in people with Myelodysplastic Syndromes, run by Bristol-Myers Squibb at 144 sites in 19 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 30 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 144 sites in United States, China, Germany, Italy, France and 14 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05949684.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jun 2026.

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