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CompletedNCT04477850Updated May 5, 2026

A Study to Evaluate the Efficacy, Drug Levels and Safety of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in Chinese and Japanese Participants With Ring Sideroblasts Who Require Red Blood Cell Transfusions

A Phase 2 interventional study of Luspatercept in Myelodysplastic Syndromes, sponsored by Celgene. Completed at 25 sites in 2 countries. Open to participants aged 20 Years and older. Per ClinicalTrials.gov, last updated 2026-05-05.

Sponsored by Celgene · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
30
Allocation
Not applicable
Ages
20 Years and older
Sex
All
01

Study summary

The purpose of this study is to evaluate the efficacy and safety of luspatercept (ACE-536) for the treatment of anemia due to Revised International Prognostic Scoring System (IPSS-R) very low, low, or intermediate risk myelodysplastic syndromes (MDS) in Chinese and Japanese participants with ring sideroblasts who require Red Blood Cells (RBC) transfusions.

02

Conditions studied

  • Myelodysplastic Syndromes

Keywords

  • Myelodysplastic Syndromes
  • MDS
  • ACE-536
  • Anemia
  • Luspatercept
03

In context

Myelodysplastic Syndromes

2,124 studies on the registry are indexed under Myelodysplastic Syndromes; 320 are open to participants now.

This study's enrollment of 30 is below the median of 39 across 1,740 interventional studies indexed under Myelodysplastic Syndromes.

Browse Myelodysplastic Syndromes studies →

Lead sponsor

Celgene is the lead sponsor of 419 studies on the registry; 13 are open to participants now.

Of its 100 completed or terminated interventional studies of FDA-regulated products, 29 (29%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
20 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Refractory or intolerant to, or ineligible for, prior Erythropoiesis stimulating agent (ESA) treatment as defined by any one of the following: Refractory to prior ESA treatment, Intolerant to prior ESA treatment, or ESA ineligible.
  • previously treated with an ESA or granulocyte colony-stimulating factor, granulocyte-macrophage colony-stimulating factor, both agents must have been discontinued ≥ 4 weeks prior to date of luspatercept treatment
  • Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2

Exclusion criteria

Exclusion Criteria:

  • Prior therapy with disease modifying agents for underlying MDS disease
  • Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
  • Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) or alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT) ≥ 3.0 x upper limit of normal (ULN)

Other protocol-defined inclusion/exclusion criteria apply

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
30 participants (actual)

Study arms

  • Experimental
    Luspatercept Administration

    Drug: Luspatercept

Interventions

  • DrugLuspatercept

    Specified dose on specified days

    Also known as: ACE-536

06

What researchers measure

Primary outcomes

  1. Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 weeks

    Time frame: Week 1 through Week 24

Secondary outcomes

  1. RBC-TI ≥ 12 weeks

    Time frame: Week 1 through Week 24

  2. Reduction in Red Blood Cell (RBC) units transfused over 16 weeks compared to baseline

    Time frame: Week 9 through Week 24

  3. Modified hematologic improvement - erythroid (mHI-E) per International Working Group (IWG)

    Time frame: Week 1 through Week 24

  4. Mean hemoglobin increase ≥ 1.0 g/dL

    Time frame: Week 1 through Week 24

  5. Duration of RBC-TI

    Time frame: Week 1 through Week 24

  6. Mean decrease in serum ferritin compared to baseline

    Time frame: Week 9 through Week 24

  7. Mean decrease in iron chelation therapy (ICT) use compared to baseline

    Time frame: Week 9 through Week 24

  8. Time to RBC-TI

    Time frame: Week 1 through Week 24

  9. Progression to acute myeloid leukemia (AML)

    Time frame: Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose

  10. Overall survival (OS)

    Time frame: Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose

  11. Incidence of type of adverse events (AEs)

    Time frame: Screening through 42 days post last dose

  12. Incidence of frequency of AEs

    Time frame: Screening through 42 days post last dose

  13. Incidence of severity of AEs

    Time frame: Screening through 42 days post last dose

  14. Incidence of seriousness of AEs

    Time frame: Screening through 42 days post last dose

  15. Incidence of relationship of AEs to study treatment

    Time frame: Screening through 42 days post last dose

  16. Pharmacokinetics - Area under the curve (AUC)

    Time frame: Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose

  17. Pharmacokinetics - Maximum plasma concentration of the drug (Cmax)

    Time frame: Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose

  18. Frequency of Anti-drug antibodies (ADA)

    Time frame: Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose

07

Study locations

25 sites
  • Local Institution - 100
    Beijing, 100730, China
  • Local Institution - 107
    Chengdu, Sichuan, 610041, China
  • Local Institution - 105
    Guangzhou, 510060, China
  • Local Institution - 103
    Guangzhou, 510080, China
  • Local Institution - 109
    Guangzhou, 510515, China
  • Local Institution - 102
    Hangzhou, 310006, China
  • Local Institution - 112
    Nanchang, 330006, China
  • Local Institution - 108
    Nanjing, 210029, China
  • Local Institution - 114
    Shanghai, 0, China
  • Local Institution - 101
    Shanghai, 200233, China
  • Local Institution - 104
    Suzhu, 215006, China
  • Local Institution - 106
    Tianjin, 300020, China
  • Local Institution - 111
    Wenzhou, 325000, China
  • Local Institution - 110
    Wuhan, 430022, China
  • Local Institution - 209
    Matsuyama, Ehime 790-8524, Japan
  • Local Institution - 203
    Nagasaki, Nagasaki 8528511, Japan
  • Local Institution - 210
    Sayama, Osaka 5898511, Japan
  • Local Institution - UNK11
    Fukuoka, 810-8563, Japan
  • Local Institution - 206
    Kamogawa, 296-8602, Japan
  • Local Institution - 201
    Mibu-Machi, 321-0293, Japan
  • Local Institution - 205
    Osaka, 545-8585, Japan
  • Local Institution - 208
    Ōgaki, 503-8502, Japan
  • Local Institution - 204
    Sagamihara, 252-0375, Japan
  • Local Institution - 207
    Sendai, 980-8574, Japan
  • Local Institution - 202
    Shinagawa-ku, Tokyo, 141-8625, Japan
08

References and documents

Publications

  • Chang C, Suzuki T, Liang Y, Tong H, Usuki K, Liu Q, Wu Y, Fujisaki T, Han B, Huang R, Morita Y, Miao M, Nakashima Y, Tian YO, Pu J, Aggarwal D, Pozharskaya V, Shi W, Xiao Z, Mitani K. Safety and efficacy of luspatercept in treating anemia associated with myelodysplastic syndrome with ring sideroblasts in Asian patients who require red blood cell transfusions: a phase II bridging study. Ther Adv Hematol. 2025 Feb 20;16:20406207251321715. doi: 10.1177/20406207251321715. eCollection 2025. PubMed 39991012 ↗

Individual participant data

Plan to share: 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

Supporting information: Study protocol, Sap, Csr

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on May 5, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT04477850
Lead sponsor
Celgene
Responsible party
Sponsor
First posted
Jul 20, 2020
Start date
Nov 30, 2020
Primary completion
Sep 29, 2023
Completion
Apr 8, 2026
Last update
May 5, 2026

Study contacts

Bristol-Myers Squibb
study director · Bristol-Myers Squibb

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Apr 2026. You cannot join it, but the record below documents what was studied.

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