Myelodysplastic SyndromesPhase 3MAXILUSNCT06045689
A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants
A Phase 3 study testing Luspatercept in people with Myelodysplastic Syndromes, run by Bristol-Myers Squibb at 52 sites in 9 countries.
Trial summary
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. 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
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 106 participants
- Where
- 52 sitesin 9 countries
- Study dates
- 5 Oct 2023to 29 Sep 2027
- 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 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.
Who cannot join
- 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
Trial locations and contacts
52 sites in 9 countries.
52 sites in 9 countries
United States16 sites
Local Institution - 0043
Amarillo, Texas, 79106-1781
Local Institution - 0011
Detroit, Michigan, 48202
Local Institution - 0054
Eugene, Oregon, 97401-6043
Local Institution - 0022
Huntsville, Texas, 77340-4101
Local Institution - 0020
Kansas City, Kansas, 66160-8500
Local Institution - 0051
Los Alamitos, California, 90720-3309
Local Institution - 0003
Milwaukee, Wisconsin, 53226
Local Institution - 0058
Morristown, New Jersey, 07960-6136
Local Institution - 0033
New Haven, Connecticut, 06510
Local Institution - 0032
New York, New York, 10029
Local Institution - 0025
Paducah, Kentucky, 42003-7915
Local Institution - 0036
Pittsburgh, Pennsylvania, 15224
Local Institution - 0059
St Louis, Missouri, 63110
Local Institution - 0055
St. Petersburg, Florida, 33705
Local Institution - 0056
Wellington, Florida, 33414
Local Institution - 0031
Wheeling, West Virginia, 26003
Spain9 sites
Local Institution - 0005
Barcelona, 08035
Local Institution - 0052
Barcelona, 08916
Local Institution - 0039
Granada, 18014
Local Institution - 0017
L'Hospitalet de Llobregat, Barcelona, 08908
Local Institution - 0038
Madrid, 28006
Local Institution - 0027
Ourense, 32005
Local Institution - 0028
Salamanca, 37007
Local Institution - 0018
Valencia, 46010
Local Institution - 0063
Valencia, 46026
Italy8 sites
Local Institution - 0024
Florence, Tuscany, 50139
Local Institution - 0062
Naples, Campania, 80131
Local Institution - 0014
Novara, Piedmont, 28100
Local Institution - 0015
Pavia, Lombardy, 27100
Local Institution - 0021
Reggio Calabria, Calabria, 89124
Local Institution - 0050
Rome, Lazio, 00133
Local Institution - 0029
Rozzano (MI), Milano, 20089
Local Institution - 0045
Turin, Piedmont, 10128
France7 sites
Local Institution - 0026
Angers, 49933
Local Institution - 0007
Grenoble, 38 38043
Local Institution - 0041
Nice, Alpes-Maritimes, 06202
Local Institution - 0046
Paris, 75475
Local Institution - 0053
Pierre-Bénite, 69495
Local Institution - 0001
Poitiers, Vienne, 86021
Local Institution - 0044
Tour Cedex01, 37044
Poland4 sites
Local Institution - 0034
Katowice, 40-519
Local Institution - 0049
Lódz, Lódzkie, 93-513
Local Institution - 0035
Wałbrzych, 58-309
Local Institution - 0010
Warsaw, Masovian Voivodeship, 02-172
Germany3 sites
Local Institution - 0040
Hanover, Lower Saxony, 30161
Local Institution - 0009
Leipzig, Saxony, 04103
Local Institution - 0013
München, Bavaria, 81675
Belgium2 sites
Local Institution - 0016
Leuven, Vlaams Brabant, 3000
Local Institution - 0008
Roeselare, West-Vlaanderen, 8800
Czechia2 sites
Local Institution - 0023
Prague, Praha, Hlavní Mesto, 128 20
Local Institution - 0004
Prague, Praha, Hlavní Mesto, 128 21
About this study
Official title: 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.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Luspatercept
- Keywords
- Luspatercept, Transfusion dependent, ACE-536, Anemia, Blood Transfusion, Red Blood Cell Transfusion, Myelodysplastic Syndrome
- 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.
LuspaterceptDrug
Also called BMS-986346, ACE-536, REBLOZYL
Specified dose on specified days.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: erythropoiesis-stimulating agents (ESA) naïve | Experimental | Not described Receives: Drug: Luspatercept |
| Cohort 2: ESA relapsed or refractory | Experimental | Not described Receives: Drug: Luspatercept |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Up to week 24
Secondary outcomes (18)
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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: Up to week 48
Number of participants with adverse events (AEs)
Measured: Up to 2 years
Number of participants with acute myeloid leukemia (AML) progression
Measured: Up to 4 years
Time to AML progression
Measured: Up to 4 years
Time from treatment start date to death due to any cause
Measured: 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
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2023
- Study start5 Oct 2023
- Primary completion1 Oct 2025
- Study completion29 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Bristol-Myers SquibbThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 106 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bristol-Myers Squibb
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bristol-Myers Squibb, Study director, Bristol-Myers Squibb
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06045689
- Sponsor's protocol ID
- CA056-1060
- Acronym
- MAXILUS
- Status last verified
- Jun 2026
- First submitted
- 13 Sep 2023
- Last updated
- 15 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- 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
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT06045689
- Acronym / sponsor ID
- MAXILUS / CA056-1060
- Conditions
- Myelodysplastic Syndromes
- Keywords
- Luspatercept, Transfusion dependent, ACE-536, Anemia, Blood Transfusion, Red Blood Cell Transfusion, Myelodysplastic Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 106 participants (actual)
- Lead sponsor
- Bristol-Myers SquibbIndustry (pharma, biotech, devices)
- Study start
- 5 Oct 2023 (actual)
- Primary completion
- 1 Oct 2025 (actual)
- Study completion
- 29 Sep 2027 (estimated)
- First posted
- 21 Sep 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: erythropoiesis-stimulating agents (ESA) naïve | Experimental | Drug: Luspatercept |
| Cohort 2: ESA relapsed or refractory | Experimental | Drug: Luspatercept |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LuspaterceptAlso: BMS-986346, ACE-536, REBLOZYLSpecified dose on specified days. | Drug | Cohort 1: erythropoiesis-stimulating agents (ESA) naïve, Cohort 2: ESA relapsed or refractory |
Primary outcome
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
Time frame: Up to week 24
18 secondary outcomes
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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Up to week 48
Number of participants with adverse events (AEs)
Time frame: Up to 2 years
Number of participants with acute myeloid leukemia (AML) progression
Time frame: Up to 4 years
Time to AML progression
Time frame: Up to 4 years
Time from treatment start date to death due to any cause
Time frame: 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
Time frame: Up to 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2026.
- BMS Clinical Trial InformationLink provided by the sponsor.
- BMS Clinical Trial Patient RecruitingLink provided by the sponsor.
- Individual participant dataSharing planned: 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bristol-Myers Squibb study director
Bristol-Myers Squibb
52 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Luspatercept in people with Myelodysplastic Syndromes, run by Bristol-Myers Squibb at 52 sites in 9 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 3 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 15 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 52 sites in United States, Spain, Italy, France, Poland and 4 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 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 (time frame: Up to week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Jun 2026.