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

NCT05567458 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: CA056-001
• Study Type: interventional
• Target: 94
• Locations: 1 country
• Sites: 10

Brief 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.

Conditions

Beta-thalassemia

Keywords

Luspatercept; ACE-536; Transfusion burden

Outcome Measures

Primary Outcomes (1)

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

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

Secondary Outcomes (33)

• 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.

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

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

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

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

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

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

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

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

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

Study Arms (2)

Luspatercept

EXPERIMENTAL

Drug: Luspatercept

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Luspatercept DRUG

Specified dose on specified days

Also known as: ACE-536, BMS-986346

Luspatercept

Placebo DRUG

Specified dose on specified days

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (10)

Local Institution - 0002

Maoming, Guangdong, 525447, China

Location

Local Institution - 0005

Shenzhen, Guangdong, 518035, China

Location

Local Institution - 0007

Liuchow, Guangxi, 545006, China

Location

Local Institution - 0003

Nanning, GX, 530012, China

Location

Local Institution - 0006

Haikou, Hainan, 570203, China

Location

Local Institution - 0010

Kunming, Yunnan, 650032, China

Location

Local Institution - 0009

Guangzhou, 510120, China

Location

Local Institution - 0004

Guangzhou, 510515, China

Location

Local Institution - 0008

Haikou, 570311, China

Location

Local Institution - 0001

Nanning, 530021, China

Location

Related Links

• BMS Clinical Trial Information

MeSH Terms

Conditions

beta-Thalassemia

Interventions

luspatercept

Condition Hierarchy (Ancestors)

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

Study Officials

• Bristol-Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 3, 2022
• First Posted: October 5, 2022
• Study Start: October 17, 2022
• Primary Completion: August 1, 2025
• Study Completion (Estimated): August 1, 2026
• Last Updated: August 12, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: See Plan Description
• Access Criteria: See Plan Description

Locations

CN (10)