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AzTrials

Myelodysplastic SyndromesPhase 2NCT04477850

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 study testing Luspatercept in people with Myelodysplastic Syndromes, run by Celgene at 25 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 20 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
30 participants
Where
25 sitesin 2 countries
Study dates
30 Nov 2020to 8 Apr 2026
Phase
Phase 2
Are there results?
None posted in the registry

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 20 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

25 sites in 2 countries.

25 sites in 2 countries

China14 sites

  • Local Institution - 100

    Beijing, 100730

  • Local Institution - 107

    Chengdu, Sichuan, 610041

  • Local Institution - 105

    Guangzhou, 510060

  • Local Institution - 103

    Guangzhou, 510080

  • Local Institution - 109

    Guangzhou, 510515

  • Local Institution - 102

    Hangzhou, 310006

  • Local Institution - 112

    Nanchang, 330006

  • Local Institution - 108

    Nanjing, 210029

  • Local Institution - 114

    Shanghai, 0

  • Local Institution - 101

    Shanghai, 200233

  • Local Institution - 104

    Suzhu, 215006

  • Local Institution - 106

    Tianjin, 300020

  • Local Institution - 111

    Wenzhou, 325000

  • Local Institution - 110

    Wuhan, 430022

Other trials in China

Japan11 sites

  • Local Institution - UNK11

    Fukuoka, 810-8563

  • Local Institution - 206

    Kamogawa, 296-8602

  • Local Institution - 209

    Matsuyama, Ehime, 790-8524

  • Local Institution - 201

    Mibu-Machi, 321-0293

  • Local Institution - 203

    Nagasaki, Nagasaki, 8528511

  • Local Institution - 208

    Ōgaki, 503-8502

  • Local Institution - 205

    Osaka, 545-8585

  • Local Institution - 204

    Sagamihara, 252-0375

  • Local Institution - 210

    Sayama, Osaka, 5898511

  • Local Institution - 207

    Sendai, 980-8574

  • Local Institution - 202

    Shinagawa-ku, Tokyo, 141-8625

Other trials in Japan

About this study

Official title: A Phase 2, Multicenter, Single-Arm Bridging Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes(MDS) in Chinese and Japanese Subjects With Ring Sideroblasts Who Require Red Blood Cell Transfusions

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.

Being tested
Luspatercept
Keywords
Myelodysplastic Syndromes, MDS, ACE-536, Anemia, Luspatercept
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 ACE-536

    Specified dose on specified days

Study groups in NCT04477850
Study groupRoleWhat they receive
Luspatercept AdministrationExperimentalNot described
Receives: Drug: Luspatercept

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Measured: Week 1 through Week 24

Secondary outcomes (18)

  1. RBC-TI ≥ 12 weeks

    Measured: Week 1 through Week 24

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

    Measured: Week 9 through Week 24

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

    Measured: Week 1 through Week 24

  4. Mean hemoglobin increase ≥ 1.0 g/dL

    Measured: Week 1 through Week 24

  5. Duration of RBC-TI

    Measured: Week 1 through Week 24

  6. Mean decrease in serum ferritin compared to baseline

    Measured: Week 9 through Week 24

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

    Measured: Week 9 through Week 24

  8. Time to RBC-TI

    Measured: Week 1 through Week 24

  9. Progression to acute myeloid leukemia (AML)

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

  10. Overall survival (OS)

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

  11. Incidence of type of adverse events (AEs)

    Measured: Screening through 42 days post last dose

  12. Incidence of frequency of AEs

    Measured: Screening through 42 days post last dose

  13. Incidence of severity of AEs

    Measured: Screening through 42 days post last dose

  14. Incidence of seriousness of AEs

    Measured: Screening through 42 days post last dose

  15. Incidence of relationship of AEs to study treatment

    Measured: Screening through 42 days post last dose

  16. Pharmacokinetics - Area under the curve (AUC)

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

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

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

  18. Frequency of Anti-drug antibodies (ADA)

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Jul 2020
  2. Study start30 Nov 2020
  3. Primary completion29 Sep 2023
  4. Study completion8 Apr 2026

Resources

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
30 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04477850
Sponsor's protocol ID
ACE-536-MDS-004
Other IDs
U1111-1251-9249
Status last verified
Apr 2026
First submitted
23 Jun 2020
Last updated
5 May 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
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

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

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Luspatercept in people with Myelodysplastic Syndromes, run by Celgene at 25 sites in 2 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 4 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: finished, as of the sponsor's last update on 5 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 25 sites in China, Japan. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 weeks (time frame: Week 1 through Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04477850, which also lists 1 linked publication.

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 5 May 2026.

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