18 and older, any sex, with Myelodysplastic Syndromes. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percentage Of Participants Who Achieved Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 Weeks From Week 1 to Week 24Primary· From Week 1 through Week 24 of study treatment
RBC-TI response was defined as the absence of any Red Blood Cells (RBC) transfusion during any consecutive 56-day (8-week) period (ie, Days 1 to 56, Days 2 to 57, Days 3 to 58, etc.) during the first 24 weeks of study treatment. Participants had to have at least 56 days (≥ 8 weeks) of transfusion independence prior to (and including) the Week 24 cut-off date to qualify as a responder. Participants who failed to achieve RBC-TI at least 56 days prior to or on the cut-off date were counted as non-responders.
Group
Value
95% CI
Luspatercept
37.91
30.20 – 46.10
Placebo
13.16
6.49 – 22.87
Percentage of Participants Who Achieved Red Blood Cell Transfusion Independence (RBC-TI) ≥ 12 Weeks From Week 1 to Week 24Secondary· From Week 1 through Week 24 of study treatment
RBC-TI Response was defined as the absence of any Red Blood Cells (RBC) transfusion during any consecutive 84-day (12-week) period (ie, Days 1 to 84, Days 2 to 85, Days 3 to 86, etc.) during the first 24 weeks of treatment.
Group
Value
95% CI
Luspatercept
28.10
21.14 – 35.93
Placebo
7.89
2.95 – 16.40
Percentage of Participants Who Achieved Red Blood Cell Transfusion Independence (RBC-TI) ≥ 12 Weeks From Week 1 to Week 48Secondary· From Week 1 through Week 48 of study treatment
RBC-TI Response was defined as the absence of any Red Blood Cells (RBC) transfusion during any consecutive 84-day (12-week) period (ie, Days 1 to 84, Days 2 to 85, Days 3 to 86, etc.) during the first 48 weeks of treatment.
Group
Value
95% CI
Luspatercept
33.33
25.93 – 41.40
Placebo
11.84
5.56 – 21.29
Percentage of Participants Who Achieved Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 Weeks From Week 1 Through Week 48Secondary· From Week 1 through Week 48 of study treatment
RBC-TI response was defined as the absence of any Red Blood Cells (RBC) transfusion during any consecutive 56-day (8-week) period (ie, Days 1 to 56, Days 2 to 57, Days 3 to 58, etc.) during Week 1 through Week 48. Participants had to have at least 56 days (≥ 8 weeks) of transfusion independence prior to (and including) the Week 48 cut-off date to qualify as a responder. Participants who failed to achieve RBC-TI at least 56 days prior to Week 48 were counted as non-responders.
Group
Value
95% CI
Luspatercept
45.10
37.05 – 53.34
Placebo
15.79
8.43 – 25.96
Change From Baseline in RBC Units Transfused Over Fixed 16-Week PeriodSecondary· At Baseline (16 weeks prior to first dose of study treatment) and Weeks 9 to 24 or Weeks 33 to 48
Mean change in total number of Red Blood Cells (RBC) units transfused over a fixed 16-week period (Week 9-24 or Week 33-48) from the total number of RBC units transfused in the 16 weeks immediately on or prior to first dose of study treatment.
Weeks 9 to 24
Group
Value
95% CI
Luspatercept
-3.0
± 5.17
Placebo
0.4
± 4.25
Weeks 33 to 48
Group
Value
95% CI
Luspatercept
-4.9
± 4.22
Placebo
-3.9
± 7.14
Percentage of Participants Who Achieved a Modified Hematologic Erythroid Response (mHI-E) Over Any Consecutive 56-Day PeriodSecondary· Week 1 through 24 or Week 1 Through Week 48
A modified HI-E response was defined as the percentage of participants meeting the modified HI-E per the International Working Group (IWG) sustained over 56-day consecutive period during the Treatment period. For participants with a baseline RBC transfusion burden of ≥ 4 units/8 weeks, a mHI-E was defined as a reduction in RBC transfusion of at least 4 units/8 weeks; for participants with baseline RBC transfusion burden of \<4 units/8 weeks, mHI-E, was defined as a mean increase in hemoglobin of ≥ 1.5 g/dL for 8 weeks in the absence of RBC transfusions.
Week 1 Through Week 24
Group
Value
95% CI
Luspatercept
52.9
44.72 – 61.05
Placebo
11.8
5.56 – 21.29
Week 1 Through Week 48
Group
Value
95% CI
Luspatercept
58.8
50.59 – 66.71
Placebo
17.1
9.43 – 27.47
Percentage of Participants Who Achieved a Mean Hemoglobin (Hgb) Increase of at Least 1.0 g/dL Over Any Consecutive 56-Day Period in Absence of Red Blood Cells (RBC) TransfusionsSecondary· Week 1 though Week 24 and Week 1 through 48
A mean hgb increase of ≥ 1.0 g/dL was analyzed as the percentage of participants with a hgb increase ≥ 1.0 g/dL compared with baseline (after applying the 14/3 day rule) that was sustained over any consecutive 56-day (8-week) period in the absence of RBC transfusions during the treatment period. (Week 1 through Week 24 and Week 1 through Week 48).
Week 1 Through Week 24
Group
Value
95% CI
Luspatercept
35.3
27.75 – 43.42
Placebo
7.9
2.95 – 16.40
Week 1 Through Week 48
Group
Value
95% CI
Luspatercept
41.2
33.29 – 49.41
Placebo
10.5
4.66 – 19.69
Duration of Red Blood Cell Transfusion Independence (RBC-TI) - Week 1 Through Week 24Secondary· From start of study treatment to 16 weeks after last dose, up to approximately 93 weeks
Duration of RBC-TI was defined as the longest duration of response for participants who achieved RBC-TI of ≥ 8 weeks during the treatment period Week 1 through Week 24. Participants who maintained RBC-TI through the end of the treatment period were censored at the date of IP discontinuation or death, whichever occurred first. Median was estimated from unstratified Kaplan Meier method.
Group
Value
95% CI
Luspatercept
30.6
20.6 – 40.6
Placebo
13.6
9.1 – 54.9
Duration of Red Blood Cell Transfusion Independence (RBC-TI) - Week 1 Through Week 48Secondary· From start of study treatment to 16 weeks after last dose, up to approximately 93 weeks
Duration of RBC-TI was defined as the longest duration of response for participants who achieved RBC-TI of ≥ 8 weeks during the treatment period Week 1 through Week 48. Participants who maintained RBC-TI through the end of the treatment period were censored at the date of IP discontinuation or death, whichever occurred first. Median was estimated from unstratified Kaplan Meier method.
Group
Value
95% CI
Luspatercept
30.6
20.6 – 50.9
Placebo
18.6
10.9 – NA
Mean Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) Global Quality of Life ScoreSecondary· Baseline and Cycle 3, Day 1 (C3 D1), C5 D1, C7 D1, Week 25, every other cycle during extension phase (C1 D1, C3 D1, C5 D1, etc. up to C59 D1) and end of treatment. Each cycle is composed of 21 days.
The EORTC questionnaire is a validated health-related quality of life (HRQoL) measure applicable to participants with any cancer diagnosis. Version 3.0 of the questionnaire was used in the study.
It is composed of 30 items that address 15 domains, including one global health status, functional domains, and symptom domains. Domain scores are transformed to a 0 to 100 scale, where higher scores on the global quality of life score indicate better function. As such, a positive change from Baseline score indicates an improvement in quality of life.
Cycle 3 Day 1 (C3 D1)
Group
Value
95% CI
Luspatercept
-4.1
± 21.01
Placebo
0.1
± 15.95
C5 D1
Group
Value
95% CI
Luspatercept
-2.4
± 20.73
Placebo
2.2
± 17.13
C7 D1
Group
Value
95% CI
Luspatercept
-2.1
± 23.04
Placebo
-0.6
± 18.63
Week 25
Group
Value
95% CI
Luspatercept
-1.8
± 21.75
Placebo
0.2
± 18.88
Extension Phase C1 D1
Group
Value
95% CI
Luspatercept
0.0
± 25.32
Placebo
6.3
± 14.60
Extension Phase C3 D1
Group
Value
95% CI
Luspatercept
2.0
± 19.68
Placebo
-3.9
± 26.86
Extension Phase C5 D1
Group
Value
95% CI
Luspatercept
0.8
± 18.40
Placebo
0.6
± 20.04
Extension Phase C7 D1
Group
Value
95% CI
Luspatercept
-0.5
± 20.03
Placebo
3.8
± 20.87
Percentage of Participants Who Achieved a Hematologic Improvement in Neutrophil Response (HI-N) Over Any Consecutive 56-day PeriodSecondary· Week 1 through Week 24 or Week 1 Through Week 48 of study treatment
Percentage of participants who achieved a hematologic improvement in neutrophil response (HI-N) per IWG criteria sustained over any consecutive 56-day (8-week) period, during the treatment period (Week 1 to Week 24 and Week 1 to Week 48) HI-N was defined as at least a 100% increase and an absolute increase \> 0.5 X 10\^9/L.
Week 1 Through Week 24
Group
Value
95% CI
Luspatercept
13.3
1.66 – 40.46
Placebo
0
0.00 – 30.85
Week 1 Through Week 48
Group
Value
95% CI
Luspatercept
20.0
4.33 – 48.09
Placebo
10.0
0.25 – 44.50
Percentage of Participants Who Achieved a Hematologic Improvement in Platelet Response (HI-P) Over Any Consecutive 56-day PeriodSecondary· Week 1 through Week 24 or Week 1 Through Week 48 of study treatment
Percentage of participants who achieved a hematologic improvement platelet response (HI-P) was defined as the percentage of participants meeting the HI-P criteria per the IWG sustained over any consecutive 56-day (8-week) period (Week 1 to Week 24 and Week 1 to Week 48) during the treatment period. HI - P reponse was defined as:
* Absolute increase of ≥ 30 X 10\^9/L in platelets for participants starting with \> 20 X 10\^9/L platelets
* Increase in platelets from \< 20 X 10\^9/L to \> 20 X 10\^9/L and by at least 100%
Week 1 Through Week 24
Group
Value
95% CI
Luspatercept
50.0
15.70 – 84.30
Placebo
33.3
4.33 – 77.72
Week 1 Through Week 48
Group
Value
95% CI
Luspatercept
62.5
24.49 – 91.48
Placebo
33.3
4.33 – 77.72
Adverse events — posted to ClinicalTrials.gov
Time frame: All-cause mortality was assessed from first dose to study completion (up to approximately 57 months). SAEs and Other AEs were assessed from first dose to 100 days following last dose (up to approximately 91 weeks).
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Luspatercept
Serious: 66/153 (43%)
Deaths: 45/153
Placebo
Serious: 23/76 (30%)
Deaths: 24/76
Serious adverse events (132 terms)
Reaction
System
Luspatercept
Placebo
Pneumonia
Infections and infestations
—
—
Fall
Injury, poisoning and procedural complications
—
—
Femur fracture
Injury, poisoning and procedural complications
—
—
Sepsis
Infections and infestations
—
—
Urinary tract infection
Infections and infestations
—
—
Refractory anaemia with an excess of blasts
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
—
Anaemia
Blood and lymphatic system disorders
—
—
Angina pectoris
Cardiac disorders
—
—
Pyrexia
General disorders
—
—
Hip fracture
Injury, poisoning and procedural complications
—
—
Back pain
Musculoskeletal and connective tissue disorders
—
—
Basal cell carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
—
Squamous cell carcinoma of skin
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
—
Transformation to acute myeloid leukaemia
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
—
Syncope
Nervous system disorders
—
—
Atrial fibrillation
Cardiac disorders
—
—
Atrioventricular block
Cardiac disorders
—
—
Cardiac failure
Cardiac disorders
—
—
Bronchitis
Infections and infestations
—
—
Lower respiratory tract infection
Infections and infestations
—
—
Humerus fracture
Injury, poisoning and procedural complications
—
—
Myelodysplastic syndrome
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
The study will be conducted in compliance with the International Council on Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements.
This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in participants with anemia due to the Revised International Prognostic Scoring System (IPSS-R) very low, low, or intermediate MDS with ring sideroblasts who require red blood cell (RBC) transfusions.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT07463820 — A Trial Comparing Three Different Treatment Options for Adults With Low-Risk Myelodysplasia and Anemia (A MyeloMATCH Tre
· Phase 2
· not yet recruiting
NCT07450313 — The Efficacy and Safety of Luspatercept in Improving Early Anemia After HSCT
· Phase 2
· not yet recruiting
NCT07215975 — A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East
· recruiting
NCT07465029 — A Study of Incidence, Treatment Patterns, and Outcomes in Transfusion-dependent Lower-risk Myelodysplastic Syndromes in
· active not recruiting
NCT07362095 — Luspatercept for the Treatment of Anemia Following Allogeneic Hematopoietic Stem Cell Transplantation(Allo-HSCT)
· Phase 1, PHASE2
· recruiting
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Celgene
Last refreshed: 17 December 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02631070.