Skip to main content
OpenTrials
Completed

NCT Number: NCT04477850

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Myelodysplastic Syndromes Anemia Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases

Age range

20 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Local Institution - 100, Beijing, China

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Luspatercept

Drug

Specified dose on specified days

Other names: ACE-536

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

Sponsors and collaborators

Lead sponsor

Celgene

Industry

Registry information

Official study 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

Important dates

Study start
2020
Primary completion
2023
Study completion
2026
First posted
Jul 20, 2020
Registry last updated
May 5, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.