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

Anemia Clinical Trial

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 Participants Who Have Had Unsatisfactory Response to or Are Ineligible to Erythropoietin Based Therapy and in Participants With Transfusion Dependent Anemia Due to Beta-Thalassemia

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05891249
• Other study ID #: CA056-023
• Status: Recruiting
• Phase: Phase 4
• Start date: June 5, 2023
• Est. completion date: April 13, 2026

Study information

• Verified date: June 2024
• Source: Bristol-Myers Squibb
• Contact: BMS Study Connect Contact Center www.BMSStudyConnect.com
• Phone: 855-907-3286
• Email: Clinical.Trials[@]bms.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 60
• Est. completion date: April 13, 2026
• Est. primary completion date: April 13, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

ß-Thalassemia Cohort

• Documented diagnosis of ß-thalassemia or hemoglobin (Hb E/ß-thalassemia). (ß-thalassemia with mutation and/or multiplication of alpha [a] 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:

ß-Thalassemia Cohort

• A diagnosis of Hb S/ß-thalassemia or a-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).

Related Conditions & MeSH terms

• Anemia
• beta-Thalassemia
• Myelodysplastic Syndromes
• Preleukemia
• Thalassemia

Intervention

Biological:
• Luspatercept
Specified dose on specified days

Locations

Country	Name	City	State
India	Vedanta Institute Of Medical Sciences	Ahmedabad	Gujarat
India	Gauhati Medical College and Hospital	Assam
India	HealthCare Global Enterprises Ltd. Hospital	Bangalore
India	Local Institution - 0012	Bengaluru	Karnataka
India	Post Graduate Institute of Medical Education and Research	Chandigarh
India	Rajiv Gandhi Cancer Institute And Research Centre	Delhi
India	Local Institution - 0006	Hyderabad
India	Local Institution - 0009	Kolkata
India	Nilratan Sirkar Medical College and Hospital	Kolkatta	West Bangal
India	KEM Hospital and Seth G S Medical College	Mumbai
India	All India Institute of Medical Sciences	New Delhi	Delhi
India	Local Institution - 0011	New Delhi	Delhi
India	Local Institution - 0014	Noida	Uttar Pradesh

Sponsors (1)

Lead Sponsor	Collaborator
Bristol-Myers Squibb

Country where clinical trial is conducted

India, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	ß-Thal Cohort: Number of participants with treatment-related adverse events (AEs) of grade 3 or higher		Up to 57 weeks
Primary	MDS-Ring Sideroblasts (RS) Cohort: Number of participants with treatment-related AEs of grade 3 or higher		Up to 54 weeks
Secondary	ß-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
Secondary	ß-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
Secondary	MDS-RS Cohort: Percentage of participants who achieved RBC-TI during any consecutive 56-day period		Week 1 to week 24
Secondary	ß-Thal Cohort: Number of participants with treatment-related AEs		Up to 57 weeks
Secondary	MDS-RS Cohort: Number of participants with treatment-related AEs		Up to 54 weeks

External Links

•   BMS Clinical Trial Information