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

Last updated: May 30, 2025
Sponsor: Bristol-Myers Squibb
Overall Status: Active - Not Recruiting

Phase

2

Condition

Thalassemia

Treatment

Placebo

Luspatercept

Clinical Study ID

NCT05567458
CA056-001
  • Ages > 18
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

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 otherprotocol 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 weeksprior to randomization and no transfusion-free period for >42 days during thatperiod.

  • Participant has Eastern Cooperative Oncology Group (ECOG) score of 0 or 1.

Exclusion

Exclusion Criteria:

  • Participant has a diagnosis of Hemoglobin S/β-thalassemia or α-thalassemia (forexample, Hemoglobin H).

  • Participant has active hepatitis C virus (HCV) infection as demonstrated by apositive HCVribonucleic acid (RNA) test of sufficient sensitivity, or activeinfectious hepatitis B virus (HBV) as demonstrated by the presence of hepatitis Bsurface antigen (HBsAg) and/or HBVdeoxyribonucleic acid (DNA) positive, or knownpositive human immunodeficiency virus (HIV).

  • Participant has a history of deep venous thrombosis or stroke or thromboembolicevents (venous or arterial) requiring medical intervention ≤24 weeks prior torandomization.

  • Participant uses chronic anticoagulant therapy, unless the treatment stopped atleast 28 days prior to randomization. Anticoagulant therapies used for prophylaxisfor surgery or high-risk procedures as well as low-molecular-weight heparin forsuperficial venous thrombosis and chronic aspirin are allowed.

  • Participant who has EMH complications requiring treatment to control the growth ofEMH mass(es) during the screening period.

  • Participant used immunomodulatory imide drugs (IMiDs) ≤ 24 weeks prior torandomization

Study Design

Total Participants: 90
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 2
Study Start date:
October 17, 2022
Estimated Completion Date:
August 01, 2026

Connect with a study center

  • Local Institution - 0002

    Maoming, Guangdong 525447
    China

    Site Not Available

  • Local Institution - 0005

    Shenzhen, Guangdong 518035
    China

    Site Not Available

  • Shenzhen Second People's Hospital

    Shenzhen, Guangdong 518035
    China

    Site Not Available

  • Local Institution - 0007

    Liuzhou, Guangxi 545006
    China

    Site Not Available

  • Local Institution - 0003

    Nanning, Guangxi 530012
    China

    Site Not Available

  • People's Liberation Army The 923rd Hospital

    Nanning, Guangxi 530012
    China

    Active - Recruiting

  • Hainan Medical College - First Affiliated Hospital

    Haikou, Hainan 570203
    China

    Active - Recruiting

  • Local Institution - 0006

    Haikou, Hainan 570203
    China

    Site Not Available

  • First People's Hospital of Yunnan Province

    Kunming, Yunnan 650032
    China

    Site Not Available

  • Local Institution - 0010

    Kunming, Yunnan 650032
    China

    Site Not Available

  • Local Institution - 0011

    Kunming, Yunnan 650032
    China

    Site Not Available

  • Local Institution - 0004

    Guangzhou, 510515
    China

    Site Not Available

  • Local Institution - 0009

    Guangzhou, 510120
    China

    Site Not Available

  • Nanfang Hospital of Southern Medical University

    Guangzhou, 510515
    China

    Active - Recruiting

  • Sun Yat-sen Memorial Hospital, Sun Yat-Sen University

    Guangzhou, 510120
    China

    Active - Recruiting

  • Hainan General Hospital

    Haikou, 570311
    China

    Active - Recruiting

  • Local Institution - 0008

    Haikou, 570311
    China

    Site Not Available

  • Liuzhou General Hospital

    Liuzhou, 545006
    China

    Active - Recruiting

  • Maoming People's Hospital

    Maoming, 525447
    China

    Active - Recruiting

  • Local Institution - 0001

    Nanning, 530021
    China

    Site Not Available

  • The First Affiliated Hospital of Guangxi Medical University

    Nanning, 530021
    China

    Active - Recruiting

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