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

Phase 4

Active, not recruiting

• Conditions: Anemia
• Interventions: Biological: Luspatercept

• Registration Number: NCT05891249
• Lead Sponsor: Bristol-Myers Squibb

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-05-26
• Study First Submitted QC: 2023-05-26
• Study Start: 2023-06-05
• Study First Posted: 2023-06-06
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-15
• Last Update Posted: 2025-04-16
• Primary Completion: 2026-04-16
• Study Completion: 2026-04-16

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

β-Thalassemia Cohort

• Documented diagnosis of β-thalassemia or hemoglobin (Hb E/β-thalassemia). (β-thalassemia with mutation and/or multiplication of alpha [α] globin is allowed).
• 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.

ii) 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

• A diagnosis of Hb S/β-thalassemia or α-thalassemia (for exampe, Hemoglobin H).
• Deep vein thrombosis (DVT) or stroke requiring medical intervention ≤ 24 weeks prior to enrollment.
• 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.
• Cytotoxic agents or immunosuppressants or immunomodulatory drugs (IMiDs) ≤ 28 days prior to enrollment (ie, antithymocite globulin or cyclosporine or thalidomide).

MDS-RS Cohort

• MDS associated with del 5q cytogenetic abnormality.
• 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.
• Participant has known clinically significant anemia due to iron, vitamin B12, or folate deficiencies; autoimmune or hereditary hemolytic anemia; or gastrointestinal bleeding.
• 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).

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Luspatercept	Luspatercept	-

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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 compared to the 12-week interval prior to enrollment	Week 13 to week 24
MDS-RS Cohort: Percentage of participants who achieved RBC-TI during any consecutive 56-day period	Week 1 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 compared to the 12-week interval prior to enrollment	Up to 57 weeks
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

Trial Locations

Locations (9)

Local Institution - 0001; 🇮🇳 New Delhi, Delhi, India

Local Institution - 0002; 🇮🇳 Ahmedabad, Gujarat, India

Local Institution - 0007; 🇮🇳 Kolkata, West Bengal, India

Local Institution - 0005; 🇮🇳 Assam, India

Local Institution - 0003; 🇮🇳 Bangalore, India

Local Institution - 0004; 🇮🇳 Chandigarh, India

Local Institution - 0010; 🇮🇳 Delhi, India

Local Institution - 0008; 🇮🇳 Mumbai, India

Local Institution - 0006; 🇮🇳 Hyderabad, India