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

April 29, 2026 updated by: Celgene

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
30

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

20 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Luspatercept Administration
Drug: Luspatercept
Specified dose on specified days
Other Names:
  • ACE-536

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 weeks
Time Frame: Week 1 through Week 24
Week 1 through Week 24

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
RBC-TI ≥ 12 weeks
Time Frame: Week 1 through Week 24
Week 1 through Week 24
Reduction in Red Blood Cell (RBC) units transfused over 16 weeks compared to baseline
Time Frame: Week 9 through Week 24
Week 9 through Week 24
Modified hematologic improvement - erythroid (mHI-E) per International Working Group (IWG)
Time Frame: Week 1 through Week 24
Week 1 through Week 24
Mean hemoglobin increase ≥ 1.0 g/dL
Time Frame: Week 1 through Week 24
Week 1 through Week 24
Duration of RBC-TI
Time Frame: Week 1 through Week 24
Week 1 through Week 24
Mean decrease in serum ferritin compared to baseline
Time Frame: Week 9 through Week 24
Week 9 through Week 24
Mean decrease in iron chelation therapy (ICT) use compared to baseline
Time Frame: Week 9 through Week 24
Week 9 through Week 24
Time to RBC-TI
Time Frame: Week 1 through Week 24
Week 1 through Week 24
Progression to acute myeloid leukemia (AML)
Time Frame: Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose
Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose
Overall survival (OS)
Time Frame: Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose
Cycle1 Day1 (each cycle is 21 days) through at least 3 years post first dose
Incidence of type of adverse events (AEs)
Time Frame: Screening through 42 days post last dose
Screening through 42 days post last dose
Incidence of frequency of AEs
Time Frame: Screening through 42 days post last dose
Screening through 42 days post last dose
Incidence of severity of AEs
Time Frame: Screening through 42 days post last dose
Screening through 42 days post last dose
Incidence of seriousness of AEs
Time Frame: Screening through 42 days post last dose
Screening through 42 days post last dose
Incidence of relationship of AEs to study treatment
Time Frame: Screening through 42 days post last dose
Screening through 42 days post last dose
Pharmacokinetics - Area under the curve (AUC)
Time Frame: Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose
Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose
Pharmacokinetics - Maximum plasma concentration of the drug (Cmax)
Time Frame: Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose
Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose
Frequency of Anti-drug antibodies (ADA)
Time Frame: Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose
Cycle1 Day1 (each cycle is 21 days) through 1-year post first dose

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Celgene

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 30, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 29, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 8, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 23, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 17, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 20, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 5, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 29, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ACE-536-MDS-004
  • U1111-1251-9249 (Other Identifier: WHO)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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

IPD Sharing Time Frame

See Plan Description

IPD Sharing Access Criteria

See Plan Description

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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Conditions

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