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

NCT05949684 | Active Not Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: CA056-0252022-500430-29-00
• Study Type: interventional
• Target: 360
• Locations: 19 countries
• Sites: 144

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).

Conditions

Myelodysplastic Syndromes

Keywords

Luspatercept; BMS-986346; ACE-536; Myelodysplastic Syndrome; Epoetin alfa; Erythropoietin stimulating agent (ESA); Myelodysplastic Syndromes (MDS); Anaemia

Outcome Measures

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

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

  Up to Week 96

Secondary Outcomes (32)

• 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

  Up to Week 48

• 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

  Up to Week 48

• 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

  From Week 49 to Week 96

• 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

  Up to Week 96

• 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

  Up to Week 48

Study Arms (2)

Luspatercept

EXPERIMENTAL

Biological: Luspatercept

Epoetin Alfa

ACTIVE COMPARATOR

Biological: Epoetin Alfa

Interventions

Luspatercept BIOLOGICAL

Specified dose on specified days

Also known as: BMS-986346, ACE-536, Reblozyl®

Luspatercept

Epoetin Alfa BIOLOGICAL

Specified dose on specified days

Also known as: Epogen®, PROCRIT®, BINOCRIT

Epoetin Alfa

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (144)

Local Institution - 0070

Clovis, California, 93611, United States

Location

Local Institution - 0179

Fountain Valley, California, 92708, United States

Location

Local Institution - 0211

Los Alamitos, California, 90720, United States

Location

Local Institution - 0258

Los Angeles, California, 90095, United States

Location

Local Institution - 0247

Orange, California, 92868, United States

Location

Local Institution - 0209

Oxnard, California, 93030, United States

Location

Local Institution - 0183

Walnut Creek, California, 94598, United States

Location

Local Institution - 0227

Hartford, Connecticut, 06102, United States

Location

Local Institution - 0173

New Haven, Connecticut, 06510, United States

Location

Halifax Health Medical Center

Daytona Beach, Florida, 32114, United States

Location

Local Institution - 0202

Fort Myers, Florida, 33901, United States

Location

Local Institution - 0180

Jacksonville, Florida, 32207, United States

Location

Local Institution - 0224

Margate, Florida, 33063, United States

Location

Local Institution - 0163

Plantation, Florida, 33322, United States

Location

Local Institution - 0201

St. Petersburg, Florida, 33705, United States

Location

Local Institution - 0238

Tamarac, Florida, 33321, United States

Location

Local Institution - 0106

Tampa, Florida, 33612, United States

Location

Local Institution - 0240

Honolulu, Hawaii, 96813, United States

Location

Local Institution - 0272

Chicago, Illinois, 60611, United States

Location

Local Institution - 0184

Skokie, Illinois, 60077, United States

Location

Local Institution - 0270

Dyer, Indiana, 46311, United States

Location

Local Institution - 0191

Baton Rouge, Louisiana, 70808, United States

Location

Local Institution - 0044

Covington, Louisiana, 70433, United States

Location

Local Institution - 0007

Bethesda, Maryland, 20817, United States

Location

Local Institution - 0222

Saint Louis Park, Minnesota, 55426, United States

Location

Local Institution - 0128

Hattiesburg, Mississippi, 39401, United States

Location

Local Institution - 0212

Hackensack, New Jersey, 07601, United States

Location

Local Institution - 0206

Columbus, Ohio, 43210, United States

Location

Local Institution - 0174

Knoxville, Tennessee, 37920, United States

Location

Local Institution - 0228

Houston, Texas, 77030, United States

Location

Local Institution - 0236

Richmond, Virginia, 23219, United States

Location

Local Institution - 0229

Roanoke, Virginia, 24014, United States

Location

Local Institution - 0203

Kennewick, Washington, 99336, United States

Location

Local Institution - 0010

Buenos Aires, C1425DND, Argentina

Location

Local Institution - 0009

Buenos Aires, C1426ANZ, Argentina

Location

Local Institution - 0012

Buenos Aires, CP1280AEB, Argentina

Location

Local Institution - 0071

Blacktown, New South Wales, 2148, Australia

Location

Local Institution - 0113

Camperdown, New South Wales, 2050, Australia

Location

Local Institution - 0254

Coffs Harbour, New South Wales, 2450, Australia

Location

Local Institution - 0246

Gold Coast, Queensland, 4217, Australia

Location

Local Institution - 0054

Clayton, Victoria, 3168, Australia

Location

Local Institution - 0052

Heidelberg, Victoria, 3084, Australia

Location

Local Institution - 0059

Fortaleza, Ceará, 60430-270, Brazil

Location

Local Institution - 0101

Porto Alegre, Rio Grande do Sul, 90610-000, Brazil

Location

Local Institution - 0056

Rio de Janeiro, 20211-030, Brazil

Location

Local Institution - 0003

São Paulo, 05403-000, Brazil

Location

Local Institution - 0049

Calgary, Alberta, T2N 5G2, Canada

Location

Local Institution - 0112

Vancouver, British Columbia, V6Z 1Y6, Canada

Location

Local Institution - 0050

London, Ontario, N6A 5W9, Canada

Location

Local Institution - 0017

Toronto, Ontario, M4N 3M5, Canada

Location

Local Institution - 0280

Santiago, Santiago Metropolitan, 7500587, Chile

Location

Local Institution - 0278

Santiago, Santiago Metropolitan, 7500921, Chile

Location

Local Institution - 0276

Santiago, Santiago Metropolitan, 7580206, Chile

Location

Local Institution - 0277

Santiago, Santiago Metropolitan, 8420383, Chile

Location

Local Institution - 0109

Hefei, Anhui, 230001, China

Location

Local Institution - 0217

Beijing, Beijing Municipality, 100044, China

Location

Local Institution - 0030

Beijing, Beijing Municipality, 100730, China

Location

Local Institution - 0223

Chongqing, Chongqing Municipality, 400016, China

Location

Local Institution - 0220

Xiamen, Fujian, 361003, China

Location

Local Institution - 0266

Shijiazhuang, Hebei, 050000, China

Location

Local Institution - 0038

Harbin, Heilongjiang, 150010, China

Location

Local Institution - 0194

Zhengzhou, Henan, 450003, China

Location

Local Institution - 0108

Wuhan, Hubei, 430058, China

Location

Local Institution - 0089

Changsha, Hunan, 410008, China

Location

Local Institution - 0032

Nanjing, Jiangsu, 210029, China

Location

Local Institution - 0083

Suzhou, Jiangsu, 215006, China

Location

Local Institution - 0079

Nanchang, Jiangxi, 330006, China

Location

Local Institution - 0043

Changchun, Jilin, 130021, China

Location

Local Institution - 0048

Shenyang, Liaoning, 110001, China

Location

Local Institution - 0111

Qingdao, Shandong, 266003, China

Location

Local Institution - 0218

Shanghai, Shanghai Municipality, 200030, China

Location

Local Institution - 0216

Xi’an, Shanxi, 710068, China

Location

Local Institution - 0075

Chengdu, Sichuan, 610041, China

Location

Local Institution - 0103

Tianjin, Tianjin Municipality, 300020, China

Location

Local Institution - 0029

Hangzhou, Zhejiang, 310003, China

Location

Local Institution - 0281

Ningbo, Zhejiang, 315010, China

Location

Local Institution - 0033

Wenzhou, Zhejiang, 32500, China

Location

Local Institution - 0147

Medellín, Antioquia, 05034, Colombia

Location

Local Institution - 0152

Valledupar, Cesar Department, 200001, Colombia

Location

Local Institution - 0267

Piedecuesta, Santander Department, 681017, Colombia

Location

Local Institution - 0149

Montería, 230002, Colombia

Location

Local Institution - 0080

Brno, Brno-město, 625 00, Czechia

Location

Local Institution - 0092

Ostrava, Moravian-Silesian Region, 708 52, Czechia

Location

Local Institution - 0090

Prague, 12808, Czechia

Location

Local Institution - 0011

Nice, Alpes-Maritimes, 06202, France

Location

Local Institution - 0016

Pessac, Aquitaine, 33600, France

Location

Local Institution - 0019

Tours, Indre-et-Loire, 37032, France

Location

Local Institution - 0116

La Tronche, Isère, 38700, France

Location

Local Institution - 0165

Vandœuvre-lès-Nancy, Lorraine, 54511, France

Location

Local Institution - 0269

Angers, Maine-et-Loire, 49933, France

Location

Local Institution - 0248

Villejuif, Val-de-Marne, 94805, France

Location

Local Institution - 0015

Paris, 75010, France

Location

Local Institution - 0140

Toulouse, 31100, France

Location

Local Institution - 0261

Erding, Bavaria, 85435, Germany

Location

Local Institution - 0259

Münster, North Rhine-Westphalia, 48153, Germany

Location

Local Institution - 0168

Koblenz, Rhineland-Palatinate, 56068, Germany

Location

Local Institution - 0171

Leipzig, Saxony, 04103, Germany

Location

Local Institution - 0251

Jena, Thuringia, 07747, Germany

Location

Local Institution - 0169

Berlin, 10117, Germany

Location

Local Institution - 0028

Berlin, 14195, Germany

Location

Local Institution - 0207

Düsseldorf, 40225, Germany

Location

Local Institution - 0255

Lübeck, 23562, Germany

Location

Local Institution - 0167

Mutlangen, 73557, Germany

Location

Local Institution - 0166

Würzburg, 97080, Germany

Location

Local Institution - 0132

Pátrai, Achaḯa, 26504, Greece

Location

Local Institution - 0082

Alexandroupoli, Anatolikí Makedonía Kai Thráki, 681 00, Greece

Location

Local Institution - 0084

Athens, Attikí, 115 27, Greece

Location

Local Institution - 0245

Athens, Attikí, 11527, Greece

Location

Local Institution - 0131

Chaïdári, Attikí, 12462, Greece

Location

Local Institution - 0024

Eger, Heves County, 3300, Hungary

Location

Local Institution - 0021

Nyíregyháza, Szabolcs-Szatmár-Bereg, 4400, Hungary

Location

Local Institution - 0026

Budapest, 1088, Hungary

Location

Local Institution - 0243

Ahmedabad, Gujarat, 380009, India

Location

Local Institution - 0177

Bengaluru, Karnataka, 560027, India

Location

Local Institution - 0193

New Delhi, National Capital Territory of Delhi, 110029, India

Location

Local Institution - 0235

New Delhi, National Capital Territory of Delhi, 110085, India

Location

Local Institution - 0178

Bhubaneswar, Odisha, 751003, India

Location

Local Institution - 0256

Rome, Lazio, 00133, Italy

Location

Local Institution - 0137

Milan, Lombardy, 20122, Italy

Location

Local Institution - 0134

Milan, Milano, 20162, Italy

Location

Local Institution - 0136

Rozzano, Milano, 20089, Italy

Location

Local Institution - 0085

Turin, Piedmont, 10128, Italy

Location

Local Institution - 0138

Florence, Tuscany, 50134, Italy

Location

Local Institution - 0135

Bologna, 40138, Italy

Location

Local Institution - 0076

Reggio Calabria, 89133, Italy

Location

Local Institution - 0244

Roma, 00168, Italy

Location

Local Institution - 0133

Verona, 37134, Italy

Location

Local Institution - 0156

Oaxaca City, Oaxaca, 68020, Mexico

Location

Local Institution - 0069

Huixquilucan, State of Mexico, 52787, Mexico

Location

Local Institution - 0142

Mexico City, 14080, Mexico

Location

Local Institution - 0150

Puebla City, 72424, Mexico

Location

Local Institution - 0130

Wałbrzych, Lower Silesian Voivodeship, 58-309, Poland

Location

Local Institution - 0060

Gdansk, 80-952, Poland

Location

Local Institution - 0061

Katowice, 40-519, Poland

Location

Local Institution - 0062

Warsaw, 02-172, Poland

Location

Local Institution - 0219

San Juan, 00917, Puerto Rico

Location

Local Institution - 0198

Barcelona, Barcelona [Barcelona], 08035, Spain

Location

Local Institution - 0197

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

Location

Local Institution - 0196

Valencia, Valenciana, Comunitat, 46010, Spain

Location

Local Institution - 0199

Granada, 18012, Spain

Location

Local Institution - 0252

Madrid, 28006, Spain

Location

Local Institution - 0271

Oviedo, 33011, Spain

Location

Local Institution - 0200

Salamanca, 37007, Spain

Location

Local Institution - 0274

Seville, 41013, Spain

Location

Related Links

• BMS Clinical Trial Information
• BMS Clinical Trial Patient Recruiting

MeSH Terms

Conditions

Myelodysplastic Syndromes; Anemia

Interventions

luspatercept; Epoetin Alfa

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Erythropoietin; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Bristol-Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 10, 2023
• First Posted: July 18, 2023
• Study Start: October 24, 2023
• Primary Completion (Estimated): June 25, 2027
• Study Completion (Estimated): March 11, 2030
• Last Updated: May 5, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: See Plan Description
• Access Criteria: See Plan Description

Locations

CN (23); PL (4); BR (4); IT (10); PR (1); CO (4); HU (3); CZ (3); FR (9); AU (6); IN (5); GR (5); ES (8); AR (3); DE (11); CL (4); ME (4); US (33); CA (4)