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

Phase 2Active Not RecruitingNCT05567458

Bristol-Myers Squibb94 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Beta-thalassemia

Interventions

LuspaterceptDRUG

Specified dose on specified days

PlaceboDRUG

Specified dose on specified days

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (10)

Local Institution - 0002

Maoming, Guangdong, China

Local Institution - 0005

Shenzhen, Guangdong, China

Local Institution - 0007

Liuchow, Guangxi, China

Local Institution - 0003

Nanning, GX, China

Outcomes

Primary Outcomes

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

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

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

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

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

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

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

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

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

Local Institution - 0006

Haikou, Hainan, China

Local Institution - 0010

Kunming, Yunnan, China

Local Institution - 0009

Guangzhou, China

Local Institution - 0004

Guangzhou, China

Local Institution - 0008

Haikou, China

Local Institution - 0001

Nanning, China

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

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

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

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

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

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

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

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

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

Mean change from baseline in serum ferritin

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

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

Time frame: Up to 96 weeks

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

Time frame: Up to 96 weeks

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

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

Time frame: Up to 48 weeks

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

Time frame: Up to 48 weeks

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