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

NCT05891249 | Active Not Recruiting Phase 4

• 1 other identifier: CA056-023
• Study Type: interventional
• Target: 85
• Locations: 1 country
• Sites: 9

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

Conditions

Anemia

Keywords

Luspatercept; ACE-536; Transfusion dependent; Beta-thalassemia; Myelodysplastic syndromes with ring sideroblasts; Anemia; Hemoglobinopathies; Hematologic diseases; Bone marrow diseases

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (5)

• 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

• 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

• 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

Study Arms (1)

Luspatercept

EXPERIMENTAL

Biological: Luspatercept

Interventions

Luspatercept BIOLOGICAL

Specified dose on specified days

Also known as: ACE-536, REBLOZYL, BMS-986346, ROJUZDA

Luspatercept

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (9)

Local Institution - 0002

Ahmedabad, Gujarat, 380009, India

Location

Local Institution - 0001

New Delhi, National Capital Territory of Delhi, 110029, India

Location

Local Institution - 0007

Kolkata, West Bengal, 700014, India

Location

Local Institution - 0005

Assam, 781032, India

Location

Local Institution - 0003

Bangalore, 560027, India

Location

Local Institution - 0004

Chandigarh, 160012, India

Location

Local Institution - 0010

Delhi, 110085, India

Location

Local Institution - 0006

Hyderabad, 500034, India

Location

Local Institution - 0008

Mumbai, 400012, India

Location

Related Links

• BMS Clinical Trial Information

MeSH Terms

Conditions

Anemia; beta-Thalassemia; Hemoglobinopathies; Hematologic Diseases; Bone Marrow Diseases

Interventions

luspatercept

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases; Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Bristol-Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 26, 2023
• First Posted: June 6, 2023
• Study Start: June 5, 2023
• Primary Completion (Estimated): December 16, 2028
• Study Completion (Estimated): December 16, 2028
• Last Updated: October 20, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: See Plan Description
• Access Criteria: See Plan Description

Locations

IN (9)