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NCT Number: NCT05567458

A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.

The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of luspatercept plus best supportive care (BSC) versus placebo plus BSC in participants who require regular red blood cell transfusions due to β-thalassemia.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Beta-Thalassemia Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Thalassemia

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Local Institution - 0003, Nanning, GX, China

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Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Participant is willing and able to adhere to the study visit schedule (for example, not scheduled to receive hematopoietic stem cell transplantation [HSCT]) and other protocol requirements.
  • Participant has documented diagnosis of β-thalassemia or Hemoglobin E/β-thalassemia (β-thalassemia with mutation and/or multiplication of alpha (α) globin is allowed).
  • Participant is regularly transfused, defined as: 6-25 RBC units in the 24 weeks prior to randomization and no transfusion-free period for >42 days during that period.
  • Participant has Eastern Cooperative Oncology Group (ECOG) score of 0 or 1.

Exclusion criteria

  • Participant has a diagnosis of Hemoglobin S/β-thalassemia or α-thalassemia (for example, Hemoglobin H).
  • Participant has active hepatitis C virus (HCV) infection as demonstrated by a positive HCVribonucleic acid (RNA) test of sufficient sensitivity, or active infectious hepatitis B virus (HBV) as demonstrated by the presence of hepatitis B surface antigen (HBsAg) and/or HBVdeoxyribonucleic acid (DNA) positive, or known positive human immunodeficiency virus (HIV).
  • Participant has a history of deep venous thrombosis or stroke or thromboembolic events (venous or arterial) requiring medical intervention ≤24 weeks prior to randomization.
  • Participant uses chronic anticoagulant therapy, unless the treatment stopped at least 28 days prior to randomization. Anticoagulant therapies used for prophylaxis for surgery or high-risk procedures as well as low-molecular-weight heparin for superficial venous thrombosis and chronic aspirin are allowed.
  • Participant who has EMH complications requiring treatment to control the growth of EMH mass(es) during the screening period.
  • Participant used immunomodulatory imide drugs (IMiDs) ≤ 24 weeks prior to randomization

Treatment and study plan

Luspatercept

Drug

Specified dose on specified days

Other names: ACE-536, BMS-986346

Placebo

Drug

Specified dose on specified days

Primary outcomes

  1. Proportion of participants with ≥ 33% reduction from baseline in red blood cell (RBC) transfusion burden over any consecutive 24 weeks

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 - Week 48

Secondary outcomes

  1. Proportion of subjects with ≥ 33% reduction from baseline in RBC transfusion burden during any rolling 24-week interval compared to the 24-week interval prior to start of IP for luspatercept plus BSC versus placebo plus BSC.

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 134

  2. Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over any consecutive 12 weeks

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 134

  3. Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over any consecutive 12 weeks

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 134

  4. Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over any consecutive 24 weeks

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 134

  5. Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 13-24

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 13-Week 24

  6. Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 37-48

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 37 to Week 48

  7. Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 1-24

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 24

  8. Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 25-48

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 25 to Week 48

  9. Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 13-24

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 13-Week 24

  10. Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 37-48

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 37 to Week 48

  11. Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 1-24

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 24

  12. Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 25-48

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 25 to Week 48

  13. Best change from baseline in total RBC units transfused in 24 weeks within the first 48-week treatment period

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 48

  14. Change from baseline in total RBC units transfused over Weeks 1-24

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 24

  15. Change from baseline in total RBC units transfused over Weeks 25-48

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 25 to Week 48

  16. Mean change from baseline in serum ferritin

    Time frame: 12 weeks prior to Dose 1 Day 1 (inclusive); Week 37 to Week 48

  17. Change from baseline in Liver Iron Concentration (LIC) (mg/g dw) by magnetic resonance imaging (MRI)

    Time frame: Up to 96 weeks

  18. Change from baseline in myocardial iron by T2-star (T2*) MRI

    Time frame: Up to 96 weeks

  19. Change from baseline in mean daily dose of iron chelation therapy (ICT)

    Time frame: 12 weeks prior to Dose 1 Day 1 (inclusive); Week 37 to Week 48

  20. Change from baseline in self-reported Health-related quality-of-life (HRQoL) assessed by TranQoL

    Time frame: Up to 48 weeks

  21. Change from baseline in self-reported HRQoL assessed by SF-36

    Time frame: Up to 48 weeks

  22. Proportion of participants who are transfusion independent for any consecutive ≥8 weeks during treatment

    Time frame: Week 1 to Week 134

  23. Proportion of participants who are transfusion independent for any consecutive ≥12 weeks during treatment

    Time frame: Week 1 to Week 134

  24. Duration of reduction in transfusion burden

    Time frame: Week 1 to Week 134

  25. Duration of RBC transfusion independence (TI)

    Time frame: Week 1 to Week 134

  26. Time to response

    Time frame: Week 1 to Week 134

  27. Least number of transfusion events in 24 weeks within the first 48-week treatment period

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 48

  28. Number of participants with Adverse Events (AEs)

    Time frame: Up to 4 years

  29. Frequency of Antidrug antibodies (ADA)

    Time frame: Up to 2 years

  30. Maximum plasma concentration (Cmax)

    Time frame: Up to 2 years

  31. Area under the curve (AUC)

    Time frame: Up to 2 years

  32. Change in spleen volume

    Time frame: Up to 96 weeks

  33. Proportion of subjects, without increase in transfusion burden and with an increase of ≥ 1.0 g/dL in pre-transfusion Hb level on at least 2 separate tests (at least 60 days apart) during any rolling 24-week interval, compared to baseline

    Time frame: 24 weeks prior to Dose 1 Day 1 (inclusive); Dose 1 Day 2 through completion of 48-week treatment for last subject

Sponsors and collaborators

Lead sponsor

Bristol-Myers Squibb

Industry

Registry information

Official study title

A Phase 2, Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Luspatercept (ACE-536) in Chinese Adult Subjects Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia

Important dates

Study start
2022
Primary completion
2025
Study completion
2026
First posted
Oct 5, 2022
Registry last updated
Aug 12, 2025

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.

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