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

A Phase 4 Study to Evaluate Safety and Effectiveness of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic SyndromeS (MDS) With Ring Sideroblasts Who Require Red Blood Cell Transfusions in Subjects Who Have Had Unsatisfactory Response to or Are Ineligible to Erythropoietin Based Therapy and in Subjects With Transfusion Dependent Anemia Due to Beta-Thalassemia

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

Study Overview

• Status: Recruiting
• Conditions: Anemia
• Intervention / Treatment: Biological: Luspatercept

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: BMS Study Connect Contact Center www.BMSStudyConnect.com; Phone Number: 855-907-3286; Email: Clinical.Trials@bms.com
• Study Contact Backup: Name: First line of the email MUST contain the NCT# and Site #.

Study Locations

• India
  • Assam, India, 781032
    • Recruiting
    • Gauhati Medical College and Hospital
    • Contact: Jina Bhattacharyya, Site 0005; Phone Number: +919435557491
  • Bangalore, India, 560027
    • Recruiting
    • HealthCare Global Enterprises Ltd. Hospital
    • Contact: Sachin Jadhav, Site 0003; Phone Number: +919741351357
  • Chandigarh, India, 160012
    • Recruiting
    • Post Graduate Institute of Medical Education and Research
    • Contact: Pankaj Malhotra, Site 0004; Phone Number: 911722756680
  • Delhi, India, 110085
    • Recruiting
    • Rajiv Gandhi Cancer Institute And Research Centre
    • Contact: Dinesh Bhurani, Site 0010; Phone Number: 9971500861
  • Hyderabad, India, 500034
    • Recruiting
    • Local Institution - 0006
    • Contact: Site 0006
  • Kolkata, India, 700156
    • Withdrawn
    • Local Institution - 0009
  • Mumbai, India, 400012
    • Recruiting
    • KEM Hospital and Seth G S Medical College
    • Contact: S Chandrakala, Site 0008; Phone Number: +919322042985
  • Delhi
    • New Delhi, Delhi, India, 110029
      • Not yet recruiting
      • Local Institution - 0001
      • Contact: Site 0001
    • New Delhi, Delhi, India, 110029
      • Withdrawn
      • Local Institution - 0011
  • Gujarat
    • Ahmedabad, Gujarat, India, 380009
      • Recruiting
      • Vedanta Institute Of Medical Sciences
      • Contact: Sandip A. Shah, Site 0002; Phone Number: 9824041170
  • Karnataka
    • Bengaluru, Karnataka, India, 560027
      • Withdrawn
      • Local Institution - 0012
  • Uttar Pradesh
    • Noida, Uttar Pradesh, India, 201303
      • Withdrawn
      • Local Institution - 0014
  • West Bangal
    • Kolkatta, West Bangal, India, 700014
      • Recruiting
      • Nilratan Sirkar Medical College and Hospital
      • Contact: Tuphan Dolai, Site 0007; Phone Number: +919874890275

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Luspatercept	Biological: Luspatercept; Specified dose on specified days; Other Names: ACE-536; BMS-986346; REBLOZYL

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Bristol-Myers Squibb
• Investigators: Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Publications and helpful links

Helpful Links

• BMS Clinical Trial Information

Study record dates

Study Major Dates

• Study Start (Actual): June 5, 2023
• Primary Completion (Estimated): February 17, 2026
• Study Completion (Estimated): February 17, 2026

Study Registration Dates

• First Submitted: May 26, 2023
• First Submitted That Met QC Criteria: May 26, 2023
• First Posted (Actual): June 6, 2023

Study Record Updates

• Last Update Posted (Actual): February 28, 2024
• Last Update Submitted That Met QC Criteria: February 26, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: Anemia; Luspatercept; ACE-536; Beta-thalassemia; Hemoglobinopathies; Bone marrow diseases; Transfusion dependent; Hematologic diseases; Myelodysplastic syndromes with ring sideroblasts
• Additional Relevant MeSH Terms: Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Genetic Diseases, Inborn; Precancerous Conditions; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Myelodysplastic Syndromes; Preleukemia; Anemia; Thalassemia; beta-Thalassemia; Hematinics; Luspatercept
• Other Study ID Numbers: CA056-023

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD 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 Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No