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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

Primary Purpose

Anemia

Status
Recruiting
Phase
Phase 4
Locations
India
Study Type
Interventional
Intervention
Luspatercept
Sponsored by
Bristol-Myers Squibb
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Anemia focused on measuring Luspatercept, ACE-536, Transfusion dependent, Beta-thalassemia, Myelodysplastic syndromes with ring sideroblasts, Anemia, Hemoglobinopathies, Hematologic diseases, Bone marrow diseases

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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).

Sites / Locations

  • Local Institution - 0001
  • Local Institution - 0011
  • Local Institution - 0002Recruiting
  • Local Institution - 0012
  • Local Institution - 0014
  • Local Institution - 0007Recruiting
  • Local Institution - 0005Recruiting
  • Local Institution - 0003
  • Local Institution - 0004
  • Local Institution - 0010Recruiting
  • Local Institution - 0006Recruiting
  • Local Institution - 0008Recruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Luspatercept

Arm Description

Outcomes

Primary Outcome Measures

β-Thal Cohort: Number of participants with treatment-related adverse events (AEs) of grade 3 or higher
MDS-Ring Sideroblasts (RS) Cohort: Number of participants with treatment-related AEs of grade 3 or higher

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
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
MDS-RS Cohort: Percentage of participants who achieved RBC-TI during any consecutive 56-day period
β-Thal Cohort: Number of participants with treatment-related AEs
MDS-RS Cohort: Number of participants with treatment-related AEs

Full Information

First Posted
May 26, 2023
Last Updated
October 11, 2023
Sponsor
Bristol-Myers Squibb
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1. Study Identification

Unique Protocol Identification Number
NCT05891249
Brief Title
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
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 5, 2023 (Actual)
Primary Completion Date
February 17, 2026 (Anticipated)
Study Completion Date
February 17, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bristol-Myers Squibb

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. 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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Anemia
Keywords
Luspatercept, ACE-536, Transfusion dependent, Beta-thalassemia, Myelodysplastic syndromes with ring sideroblasts, Anemia, Hemoglobinopathies, Hematologic diseases, Bone marrow diseases

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
60 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Luspatercept
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
Luspatercept
Other Intervention Name(s)
ACE-536, REBLOZYL, BMS-986346
Intervention Description
Specified dose on specified days
Primary Outcome Measure Information:
Title
β-Thal Cohort: Number of participants with treatment-related adverse events (AEs) of grade 3 or higher
Time Frame
Up to 57 weeks
Title
MDS-Ring Sideroblasts (RS) Cohort: Number of participants with treatment-related AEs of grade 3 or higher
Time Frame
Up to 54 weeks
Secondary Outcome Measure Information:
Title
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
Time Frame
Week 13 to week 24
Title
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
Time Frame
Up to 57 weeks
Title
MDS-RS Cohort: Percentage of participants who achieved RBC-TI during any consecutive 56-day period
Time Frame
Week 1 to week 24
Title
β-Thal Cohort: Number of participants with treatment-related AEs
Time Frame
Up to 57 weeks
Title
MDS-RS Cohort: Number of participants with treatment-related AEs
Time Frame
Up to 54 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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).
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
BMS Study Connect Contact Center www.BMSStudyConnect.com
Phone
855-907-3286
Email
Clinical.Trials@bms.com
First Name & Middle Initial & Last Name or Official Title & Degree
First line of the email MUST contain the NCT# and Site #.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bristol-Myers Squibb
Organizational Affiliation
Bristol-Myers Squibb
Official's Role
Study Director
Facility Information:
Facility Name
Local Institution - 0001
City
New Delhi
State/Province
Delhi
ZIP/Postal Code
110029
Country
India
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0001
Facility Name
Local Institution - 0011
City
New Delhi
State/Province
Delhi
ZIP/Postal Code
110029
Country
India
Individual Site Status
Withdrawn
Facility Name
Local Institution - 0002
City
Ahmedabad
State/Province
Gujarat
ZIP/Postal Code
380009
Country
India
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0002
Facility Name
Local Institution - 0012
City
Bengaluru
State/Province
Karnataka
ZIP/Postal Code
560027
Country
India
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0012
Facility Name
Local Institution - 0014
City
Noida
State/Province
Uttar Pradesh
ZIP/Postal Code
201303
Country
India
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0014
Facility Name
Local Institution - 0007
City
Kolkatta
State/Province
West Bangal
ZIP/Postal Code
700014
Country
India
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0007
Facility Name
Local Institution - 0005
City
Assam
ZIP/Postal Code
781032
Country
India
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0005
Facility Name
Local Institution - 0003
City
Bangalore
ZIP/Postal Code
560027
Country
India
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0003
Facility Name
Local Institution - 0004
City
Chandigarh
ZIP/Postal Code
160012
Country
India
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0004
Facility Name
Local Institution - 0010
City
Delhi
ZIP/Postal Code
110085
Country
India
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0010
Facility Name
Local Institution - 0006
City
Hyderabad
ZIP/Postal Code
500034
Country
India
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0006
Facility Name
Local Institution - 0008
City
Mumbai
ZIP/Postal Code
400012
Country
India
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0008

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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 URL
https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosure-commitment.html
Links:
URL
https://www.bms.com/researchers-and-partners/clinical-trials-and-research.html
Description
BMS Clinical Trial Information
URL
https://www.bmsstudyconnect.com/s/US/English/USenHome
Description
BMS Clinical Trial Patient Recruiting
URL
https://www.fda.gov/Safety/MedWatch/SafetyInformation/default.htm
Description
FDA Safety Alerts and Recalls

Learn more about this trial

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

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