A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India
Study Details
Study Description
Brief Summary
The purpose of this study is to evaluate the safety and effectiveness of luspatercept in participants who require regular blood cell transfusions due to b-thalassemia and myelodysplastic syndromes in India
Condition or Disease | Intervention/Treatment | Phase |
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Phase 4 |
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
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Experimental: Luspatercept
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Biological: Luspatercept
Specified dose on specified days
Other Names:
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Outcome Measures
Primary Outcome Measures
- β-Thal Cohort: Number of participants with treatment-related adverse events (AEs) of grade 3 or higher [Up to 57 weeks]
- MDS-Ring Sideroblasts (RS) Cohort: Number of participants with treatment-related AEs of grade 3 or higher [Up to 54 weeks]
Secondary Outcome Measures
- β-Thal Cohort: Percentage of participants who achieved red blood cell (RBC) transfusion burden reduction (≥ 33% reduction from baseline) with a reduction of at least 2 red cell units [Week 13 to week 24]
- β-Thal Cohort: Percentage of participants who achieved RBC transfusion burden reduction of at least 33% from baseline during any 12-week interval with a reduction of at least 2 red cell units [Up to 57 weeks]
- MDS-RS Cohort: Percentage of participants who achieved RBC-TI during any consecutive 56-day period [Week 1 to week 24]
- β-Thal Cohort: Number of participants with treatment-related AEs [Up to 57 weeks]
- MDS-RS Cohort: Number of participants with treatment-related AEs [Up to 54 weeks]
Eligibility Criteria
Criteria
Inclusion Criteria:
β-Thalassemia Cohort
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Documented diagnosis of β-thalassemia or hemoglobin (Hb E/β-thalassemia). (β-thalassemia with mutation and/or multiplication of alpha [α] globin is allowed).
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Regularly transfused, defined as 6 RBC units to 20 RBC units in the 24 weeks prior to enrollment and no transfusion-free period for > 35 days during that period.
MDS-RS Cohort
- Participant has documented diagnosis of MDS according to World Health Organization (WHO) (2016)/French-American-British FAB classification that meets revised International Prognostic Scoring System (IPSS-R) classification of very low, low, or intermediate risk disease and the following criteria: i) RS ≥ 15% of erythroid precursors in bone marrow. If the SF3B1 mutation is present, RS ≥ 5% will be included.
- Less than 5% blasts in bone marrow and < 1% peripheral blood blasts. iii) Peripheral blood white blood cell (WBC) count < 13,000/ microliters (μL).
- If the participant was previously treated with erythropoiesis-stimulating agents (ESAs) or granulocyte colony-stimulating factor (G-CSF)/granulocyte-macrophage colony-stimulating factor (GM-CSF), both agents must have been discontinued ≥ 4 weeks prior to the date of enrollment.
Exclusion Criteria:
β-Thalassemia Cohort
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A diagnosis of Hb S/β-thalassemia or α-thalassemia (for exampe, Hemoglobin H).
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Deep vein thrombosis (DVT) or stroke requiring medical intervention ≤ 24 weeks prior to enrollment.
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Use of chronic anticoagulant therapy is excluded unless the treatment stopped at least 28 days prior to enrollment. Anticoagulant therapies used for prophylaxis for surgery or high-risk procedures as well as low-molecular-weight (LMW) heparin for superficial venous thrombosis and chronic aspirin are allowed.
MDS-RS Cohort
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MDS associated with del 5q cytogenetic abnormality.
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Secondary MDS, that is, MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases.
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Participant has known clinically significant anemia due to iron, vitamin B12, or folate deficiencies; autoimmune or hereditary hemolytic anemia; or gastrointestinal bleeding.
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Iron deficiency to be determined by serum ferritin ≤ 15 micrograms per liter (μg/L) and additional testing if clinically indicated (for example, calculated transferrin saturation [iron/total iron binding capacity ≤ 20%] or bone marrow aspirate [BMA] stain for iron).
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Bristol-Myers Squibb
Investigators
- Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb
Study Documents (Full-Text)
None provided.More Information
Additional Information:
- BMS Clinical Trial Information
- FDA Safety Alerts and Recalls
- Investigator Inquiry Form
- BMS Clinical Trial Patient Recruiting
Publications
None provided.- CA056-023