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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Luspatercept | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Luspatercept | Drug | Specified dose on specified days |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Bristol-Myers Squibb | Bristol-Myers Squibb | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Local Institution - 0002 | Maoming | Guangdong | 525447 | China | ||
| Local Institution - 0005 |
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| Label | URL |
|---|---|
| BMS Clinical Trial Information | View source |
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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
See Plan Description
See Plan Description
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| Placebo | Drug | Specified dose on specified days |
|
| 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 |
| Proportion of participants with ≥ 33% reduction from baseline in RBC transfusion burden over Weeks 37-48 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 24 weeks prior to Dose 1 Day 1 (inclusive); Week 25 to Week 48 |
| Mean change from baseline in serum ferritin | 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) | Up to 96 weeks |
| Change from baseline in myocardial iron by T2-star (T2*) MRI | Up to 96 weeks |
| Change from baseline in mean daily dose of iron chelation therapy (ICT) | 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 | Up to 48 weeks |
| Change from baseline in self-reported HRQoL assessed by SF-36 | Up to 48 weeks |
| Proportion of participants who are transfusion independent for any consecutive ≥8 weeks during treatment | Week 1 to Week 134 |
| Proportion of participants who are transfusion independent for any consecutive ≥12 weeks during treatment | Week 1 to Week 134 |
| Duration of reduction in transfusion burden | Week 1 to Week 134 |
| Duration of RBC transfusion independence (TI) | Week 1 to Week 134 |
| Time to response | Week 1 to Week 134 |
| Least number of transfusion events in 24 weeks within the first 48-week treatment period | 24 weeks prior to Dose 1 Day 1 (inclusive); Week 1 to Week 48 |
| Number of participants with Adverse Events (AEs) | Up to 4 years |
| Frequency of Antidrug antibodies (ADA) | Up to 2 years |
| Maximum plasma concentration (Cmax) | Up to 2 years |
| Area under the curve (AUC) | Up to 2 years |
| Change in spleen volume | Up to 96 weeks |
| 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 | 24 weeks prior to Dose 1 Day 1 (inclusive); Dose 1 Day 2 through completion of 48-week treatment for last subject |
| Shenzhen |
| Guangdong |
| 518035 |
| China |
| Local Institution - 0007 | Liuzhou | Guangxi | 545006 | China |
| Local Institution - 0003 | Nanning | GX | 530012 | China |
| Local Institution - 0006 | Haikou | Hainan | 570203 | China |
| Local Institution - 0010 | Kunming | Yunnan | 650032 | China |
| Local Institution - 0009 | Guangzhou | 510120 | China |
| Local Institution - 0004 | Guangzhou | 510515 | China |
| Local Institution - 0008 | Haikou | 570311 | China |
| Local Institution - 0001 | Nanning | 530021 | China |
| ID | Term |
|---|---|
| D017086 | beta-Thalassemia |
| ID | Term |
|---|---|
| D013789 | Thalassemia |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C000621232 | luspatercept |
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