A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants (MAXILUS)

A Phase 3b, Open-label Study Evaluating the Efficacy and Safety of Luspatercept (BMS-986346/ACE-536) Initiated at Maximum Approved Dose in LR-MDS With IPSS-R Very Low-, Low-, or Intermediate-risk Who Require RBC Transfusions (MAXILUS)

The purpose of this study is to evaluate the efficacy and safety of Luspatercept when administered at the maximum approved dose in low-risk Myelodysplastic Syndrome participants who require red blood cell transfusions.

Study Overview

• Status: Active, not recruiting
• Conditions: Myelodysplastic Syndromes
• Intervention / Treatment: Drug: Luspatercept

• Study Type: Interventional
• Enrollment (Actual): 106
• Phase: Phase 3

Contacts and Locations

Study Locations

• Belgium
  • Vlaams Brabant
    • Leuven, Vlaams Brabant, Belgium, 3000
      • Local Institution - 0016
  • West-Vlaanderen
    • Roeselare, West-Vlaanderen, Belgium, 8800
      • Local Institution - 0008
• Czechia
  • Praha, Hlavní Mesto
    • Prague, Praha, Hlavní Mesto, Czechia, 128 20
      • Local Institution - 0023
    • Prague, Praha, Hlavní Mesto, Czechia, 128 21
      • Local Institution - 0004
• France
  • Angers, France, 49933
    • Local Institution - 0026
  • Grenoble, France, 38 38043
    • Local Institution - 0007
  • Paris, France, 75475
    • Local Institution - 0046
  • Pierre-Bénite, France, 69495
    • Local Institution - 0053
  • Tour Cedex01, France, 37044
    • Local Institution - 0044
  • Alpes-Maritimes
    • Nice, Alpes-Maritimes, France, 06202
      • Local Institution - 0041
  • Vienne
    • Poitiers, Vienne, France, 86021
      • Local Institution - 0001
• Germany
  • Bavaria
    • München, Bavaria, Germany, 81675
      • Local Institution - 0013
  • Lower Saxony
    • Hanover, Lower Saxony, Germany, 30161
      • Local Institution - 0040
  • Saxony
    • Leipzig, Saxony, Germany, 04103
      • Local Institution - 0009
• Italy
  • Calabria
    • Reggio Calabria, Calabria, Italy, 89124
      • Local Institution - 0021
  • Campania
    • Napoli, Campania, Italy, 80131
      • Local Institution - 0062
  • Lazio
    • Rome, Lazio, Italy, 00133
      • Local Institution - 0050
  • Lombardy
    • Pavia, Lombardy, Italy, 27100
      • Local Institution - 0015
  • Milano
    • Rozzano (MI), Milano, Italy, 20089
      • Local Institution - 0029
  • Piedmont
    • Novara, Piedmont, Italy, 28100
      • Local Institution - 0014
    • Turin, Piedmont, Italy, 10128
      • Local Institution - 0045
  • Tuscany
    • Florence, Tuscany, Italy, 50139
      • Local Institution - 0024
• Poland
  • Katowice, Poland, 40-519
    • Local Institution - 0034
  • Wałbrzych, Poland, 58-309
    • Local Institution - 0035
  • Lódzkie
    • Lódz, Lódzkie, Poland, 93-513
      • Local Institution - 0049
  • Masovian Voivodeship
    • Warsaw, Masovian Voivodeship, Poland, 02-172
      • Local Institution - 0010
• Puerto Rico
  • San Juan, Puerto Rico, 00917
    • Local Institution - 0047
• Spain
  • Barcelona, Spain, 08035
    • Local Institution - 0005
  • Barcelona, Spain, 08916
    • Local Institution - 0052
  • Granada, Spain, 18014
    • Local Institution - 0039
  • Madrid, Spain, 28006
    • Local Institution - 0038
  • Ourense, Spain, 32005
    • Local Institution - 0027
  • Salamanca, Spain, 37007
    • Local Institution - 0028
  • Valencia, Spain, 46010
    • Local Institution - 0018
  • Valencia, Spain, 46026
    • Local Institution - 0063
  • Barcelona
    • L'Hospitalet de Llobregat, Barcelona, Spain, 08908
      • Local Institution - 0017
• United States
  • California
    • Los Alamitos, California, United States, 90720-3309
      • Local Institution - 0051
  • Connecticut
    • New Haven, Connecticut, United States, 06510
      • Local Institution - 0033
  • Florida
    • St. Petersburg, Florida, United States, 33705
      • Local Institution - 0055
    • Wellington, Florida, United States, 33414
      • Local Institution - 0056
  • Kansas
    • Kansas City, Kansas, United States, 66160-8500
      • Local Institution - 0020
  • Kentucky
    • Paducah, Kentucky, United States, 42003-7915
      • Local Institution - 0025
  • Michigan
    • Detroit, Michigan, United States, 48202
      • Local Institution - 0011
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Local Institution - 0059
  • New Jersey
    • Morristown, New Jersey, United States, 07960-6136
      • Local Institution - 0058
  • New York
    • New York, New York, United States, 10029
      • Local Institution - 0032
  • Oregon
    • Eugene, Oregon, United States, 97401-6043
      • Local Institution - 0054
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15224
      • Local Institution - 0036
  • Texas
    • Amarillo, Texas, United States, 79106-1781
      • Local Institution - 0043
    • Huntsville, Texas, United States, 77340-4101
      • Local Institution - 0022
  • West Virginia
    • Wheeling, West Virginia, United States, 26003
      • Local Institution - 0031
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Local Institution - 0003

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Participant had documented diagnosis of MDS according to World Health Organization (WHO) classification that met Revised International Prognostic Scoring System (IPSS-R) classification of very low-, low-, or intermediate-risk disease.
• Participant has an Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2.
• Participant must have red blood cell transfusions according to study criteria.

Exclusion Criteria:

• Participant has known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding.
• Participant has had a prior allogeneic or autologous stem cell transplant.
• Participant has known history or diagnosis of AML.
• Participant has uncontrolled hypertension.

Other protocol-defined inclusion/exclusion criteria apply

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1: erythropoiesis-stimulating agents (ESA) naïve	Drug: Luspatercept; Specified dose on specified days.; Other Names: ACE-536; BMS-986346; REBLOZYL
Experimental: Cohort 2: ESA relapsed or refractory	Drug: Luspatercept; Specified dose on specified days.; Other Names: ACE-536; BMS-986346; REBLOZYL

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of participants who achieve red blood cell transfusion independence (RBC-TI) for 8 weeks with a simultaneous mean hemoglobin (Hb) increase of ≥ 1 g/dL from Week 1 to Week 24	Up to week 24

Secondary Outcome Measures

Outcome Measure	Time Frame
Number of participants with adverse events (AEs)	Up to 2 years
Number of participants with a mean change in total RBC units transfused over a fixed 16-week period from Week 9 to Week 24 and from Week 33 to Week 48	Up to week 48
Number of participants who have a time from first dose to first onset of RBC-TI ≥ 8-, 12-, and 16-weeks from Week 1 to Week 24, from Week 1 to Week 48, and from Week 1 through EOT	Up to 2 years
Number of participants who achieve RBC-TI over any consecutive 8-week period from Week 1 to Week 24, from Week 1 to Week 48, and from Week 1 to EOT	Up to 2 years
Number of participants with a maximum duration of RBC-TI for participants who achieve RBC TI ≥ 8- and 16-week period from Week 1 to Week 24 and from Week 1 to EOT	Up to 2 years
Number of participants who achieve RBC-TI over any consecutive 12-, 16-, and 24-week periods from Week 1 to Week 24, from Week 1 to Week 48 and from Week 1 through EOT	Up to 2 years
Number of participants with an increase from baseline in mean hemoglobin (Hb) values of ≥ 1.0 g/dL over any consecutive 8-week period in absence of RBC transfusions from Week 1 to Week 48 and from Week 1 through EOT	Up to 2 years
Number of participants with an increase from baseline in Hb values of ≥ 1.0 g/dL over any consecutive 16-week period in absence of RBC transfusions from Week 1 to Week 24, from Week 1 to Week 48, and from Week 1 through EOT	Up to 2 years
Number of participants with an increase from baseline in Hb values of ≥ 1.5 g/dL over any consecutive 8-, and 16-week periods in absence of RBC transfusions from Week 1 to Week 24, from Week 1 to Week 48, and from Week 1 through EOT	Up to 2 years
Number of participants who achieve Hematological Improvement Erythroid (mHI-E) per International Working Group-2018 (IWG-2018) over any consecutive 8-week period from Week 1 to Week 24, from Week 1 to Week 48, and Week 1 through EOT	Up to 2 years
Number of participants who achieve Hematological Improvement - Neutrophils (HI-N) per IWG-2018 over any consecutive 8-week period from Week 1 to Week 24, from Week 1 to Week 48, and Week 1 through EOT	Up to 2 years
Number of participants who achieve Hematological Improvement - Platelets (HI-P) per IWG-2018 over any consecutive 8-week period from Week 1 to Week 24, from Week 1 to Week 48, and Week 1 through EOT	Up to 2 years
Number of participants with change in serum ferritin (SF) over a 16-week period from Week 9 to Week 24 and from Week 33 to Week 48	Up to week 48
Number of participants with change in mean daily dose of iron chelation therapy (ICT) over a 16-week period from Week 9 to Week 24 and from Week 33 to Week 48	Up to week 48
Number of participants with acute myeloid leukemia (AML) progression	Up to 4 years
Time to AML progression	Up to 4 years
Time from treatment start date to death due to any cause	Up to 4 years
Number of participants with a change in subscale scores of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) from Week 1 to Week 48 and from baseline through EOT	Up to 2 years

Collaborators and Investigators

• Sponsor: Bristol-Myers Squibb
• Investigators: Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Publications and helpful links

Helpful Links

• BMS Clinical Trial Information
• BMS Clinical Trial Patient Recruiting

Study record dates

Study Major Dates

• Study Start (Actual): October 5, 2023
• Primary Completion (Actual): October 1, 2025
• Study Completion (Estimated): December 30, 2027

Study Registration Dates

• First Submitted: September 13, 2023
• First Submitted That Met QC Criteria: September 13, 2023
• First Posted (Actual): September 21, 2023

Study Record Updates

• Last Update Posted (Actual): November 10, 2025
• Last Update Submitted That Met QC Criteria: November 7, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Keywords: Anemia; Blood Transfusion; Luspatercept; ACE-536; Myelodysplastic Syndrome; Transfusion dependent; Red Blood Cell Transfusion
• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Anemia; Hematinics; luspatercept
• Other Study ID Numbers: CA056-1060

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes