Back to resultsA Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.
Primary Purpose
Beta-thalassemia
Status
Recruiting
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
Luspatercept
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Beta-thalassemia focused on measuring Luspatercept, ACE-536, Transfusion burden
Eligibility Criteria
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.
Sites / Locations
- Local Institution - 0003
- Local Institution - 0001Recruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Luspatercept
Placebo
Arm Description
Outcomes
Primary Outcome Measures
Proportion of participants with ≥ 33% reduction from baseline in red blood cell (RBC) transfusion burden over any consecutive 24 weeks
Secondary Outcome Measures
Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over any consecutive 12 weeks
Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over any consecutive 12 weeks
Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over any consecutive 24 weeks
Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 13-24
Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 37-48
Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 1-24
Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 25-48
Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 13-24
Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 37-48
Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 1-24
Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 25-48
Mean change from baseline in total RBC units transfused in 24 weeks within the first 48-week treatment period
Change from baseline in total RBC units transfused over Weeks 1-24
Change from baseline in total RBC units transfused over Weeks 25-48
Mean change from baseline in serum ferritin
Change from baseline in Liver Iron Concentration (LIC) (mg/g dw) by magnetic resonance imaging (MRI)
Change from baseline in myocardial iron by T2-star (T2*) MRI
Change from baseline in mean daily dose of iron chelation therapy (ICT)
Change from baseline in self-reported Health-related quality-of-life (HRQoL) assessed by TranQoL
Change from baseline in self-reported HRQoL assessed by SF-36
Proportion of participants who are transfusion independent for any consecutive ≥8 weeks during treatment
Proportion of participants who are transfusion independent for any consecutive ≥12 weeks during treatment
Duration of reduction in transfusion burden
Duration of RBC transfusion independence (TI)
Time to response
Mean number of transfusion events in 24 weeks within the first 48-week treatment period
Number of participants with Adverse Events (AEs)
Frequency of Antidrug antibodies (ADA)
Maximum plasma concentration (Cmax)
Area under the curve (AUC)
Change in spleen volume
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05567458
Brief Title
A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.
Official 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
Study Type
Interventional
2. Study Status
Record Verification Date
January 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 17, 2022 (Actual)
Primary Completion Date
February 3, 2025 (Anticipated)
Study Completion Date
July 2, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bristol-Myers Squibb
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Beta-thalassemia
Keywords
Luspatercept, ACE-536, Transfusion burden
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
90 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Luspatercept
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Luspatercept
Other Intervention Name(s)
ACE-536
Intervention Description
Specified dose on specified days
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Specified dose on specified days
Primary Outcome Measure Information:
Title
Proportion of participants with ≥ 33% reduction from baseline in red blood cell (RBC) transfusion burden over any consecutive 24 weeks
Time Frame
Up to 48 weeks
Secondary Outcome Measure Information:
Title
Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over any consecutive 12 weeks
Time Frame
Up to 48 weeks
Title
Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over any consecutive 12 weeks
Time Frame
Up to 48 weeks
Title
Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over any consecutive 24 weeks
Time Frame
Up to 48 weeks
Title
Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 13-24
Time Frame
Weeks 13 to 24
Title
Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 37-48
Time Frame
Weeks 37 to 48
Title
Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 1-24
Time Frame
Weeks 1 to 24
Title
Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 25-48
Time Frame
Weeks 25 to 48
Title
Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 13-24
Time Frame
Weeks 13 to 24
Title
Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 37-48
Time Frame
Weeks 37 to 48
Title
Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 1-24
Time Frame
Weeks 1 to 24
Title
Proportion of participants with ≥ 50% reduction from baseline in RBC transfusion burden over Weeks 25-48
Time Frame
Weeks 25 to 48
Title
Mean change from baseline in total RBC units transfused in 24 weeks within the first 48-week treatment period
Time Frame
Baseline up to Week 48
Title
Change from baseline in total RBC units transfused over Weeks 1-24
Time Frame
Baseline up to Week 24
Title
Change from baseline in total RBC units transfused over Weeks 25-48
Time Frame
Weeks 25 to 48
Title
Mean change from baseline in serum ferritin
Time Frame
Baseline, Weeks 37 to 48
Title
Change from baseline in Liver Iron Concentration (LIC) (mg/g dw) by magnetic resonance imaging (MRI)
Time Frame
Up to 96 weeks
Title
Change from baseline in myocardial iron by T2-star (T2*) MRI
Time Frame
Up to 96 weeks
Title
Change from baseline in mean daily dose of iron chelation therapy (ICT)
Time Frame
Baseline, Weeks 37 to 48
Title
Change from baseline in self-reported Health-related quality-of-life (HRQoL) assessed by TranQoL
Time Frame
Up to 48 weeks
Title
Change from baseline in self-reported HRQoL assessed by SF-36
Time Frame
Up to 48 weeks
Title
Proportion of participants who are transfusion independent for any consecutive ≥8 weeks during treatment
Time Frame
Up to 48 weeks
Title
Proportion of participants who are transfusion independent for any consecutive ≥12 weeks during treatment
Time Frame
Up to 48 weeks
Title
Duration of reduction in transfusion burden
Time Frame
Up to 48 weeks
Title
Duration of RBC transfusion independence (TI)
Time Frame
Up to 48 weeks
Title
Time to response
Time Frame
Up to 48 weeks
Title
Mean number of transfusion events in 24 weeks within the first 48-week treatment period
Time Frame
Baseline up to Week 48
Title
Number of participants with Adverse Events (AEs)
Time Frame
Up to 48 weeks
Title
Frequency of Antidrug antibodies (ADA)
Time Frame
Up to 2 years
Title
Maximum plasma concentration (Cmax)
Time Frame
Up to 2 years
Title
Area under the curve (AUC)
Time Frame
Up to 2 years
Title
Change in spleen volume
Time Frame
Up to 96 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
BMS Study Connect Contact Center www.BMSStudyConnect.com
Phone
855-907-3286
Email
Clinical.Trials@bms.com
First Name & Middle Initial & Last Name or Official Title & Degree
First line of the email MUST contain the NCT# and Site#.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bristol-Myers Squibb
Organizational Affiliation
Bristol-Myers Squibb
Official's Role
Study Director
Facility Information:
Facility Name
Local Institution - 0003
City
Nanning
ZIP/Postal Code
530012
Country
China
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0003
Facility Name
Local Institution - 0001
City
Nanning
ZIP/Postal Code
530021
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0001
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myers Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosurecommitment.html
IPD Sharing Time Frame
See Plan Description
IPD Sharing Access Criteria
See Plan Description
IPD Sharing URL
https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosure-commitment.html
Links:
URL
https://www.bms.com/researchers-and-partners/clinical-trials-and-research.html
Description
BMS Clinical Trial Information
URL
https://www.bmsstudyconnect.com/s/US/English/USenHome
Description
BMS Clinical Trial Patient Recruiting
URL
https://www.fda.gov/Safety/MedWatch/SafetyInformation/default.htm
Description
FDA Safety Alerts and Recalls
Learn more about this trial
A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.
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