A Study to Evaluate Luspatercept Utilization in Patients With Lower Risk Myelodysplastic Syndromes or Beta-thalassemia

NCT07681440 | Terminated FDA Drug

• 1 other identifier: CA056-1082
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to understand the patient profile, utilized treatments, treatment patterns, resulting outcomes, and associated costs relating to luspatercept treatment in patients with lower-risk myelodysplastic syndromes (LR-MDS) or beta-thalassemia in the real-world setting

Conditions

Myelodysplastic Syndromes; Beta-thalassemia

Keywords

Lower-risk myelodysplastic syndromes (LR-MDS); Beta-thalassemia

Outcome Measures

Primary Outcomes (6)

• Participant age

  Baseline

• Participant gender

  Baseline

• Year of first luspatercept prescription

  Baseline

• Number of participants by Ring Sideroblast (RS) status (positive/negative/unknown) as measured by bone marrow aspiration

  As documented in participant medical charts

  Baseline

• Participant comorbidities

  Baseline

• Length of follow-up from start of luspatercept treatment initiation until the earliest of the end of continuous enrolment, death, or end of data availability

  Baseline

Secondary Outcomes (4)

• Number of participants that achieve red blood cell (RBC) transfusion independence

  Up to 12 months

• Total number of RBC units transfused per participant, as assessed from transfusion records and medical chart review, from baseline up to 6 months

  Baseline and up to 6 months

• Time to acute myeloid leukemia transformation

  Up to 6 months

• Time to treatment discontinuation

  Up to 6 months

Study Arms (2)

Cohort 1

Participants diagnosed myelodysplastic syndromes receiving luspatercept treatment

Drug: Luspatercept

Cohort 2

Participants diagnosed beta-thalassemia receiving luspatercept treatment

Drug: Luspatercept

Interventions

Luspatercept DRUG

According to the product label

Cohort 1; Cohort 2

Eligibility Criteria

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

Study Population

The study population will include adult patients diagnosed with myelodysplastic syndromes or beta-thalassemia and treated with luspatercept between January 1, 2020, to June 30, 2022, identified from a German Statutory Health Insurance database

You may qualify if:

• Participants treated with luspatercept. The date of first treatment with luspatercept will be assigned as the index date.
• At least one Lower-risk myelodysplastic syndromes (LR-MDS) or beta-thalassemia diagnosis in inpatient or at least two MDS or beta-thalassemia diagnosis in the outpatient setting at least 30 days apart prior to or at index date.
• + years old at index date.
• Participants with a minimum of 12-months observable pre-treatment baseline/look-back prior to index date.
• Participants with a minimum of 6-months of follow-up time from the index date unless the patient is lost due to death

You may not qualify if:

• None

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (1)

ZEG Berlin, Center for Epidemiology and Health Research

Berlin, Germany

Location

Related Links

• BMS Clinical Trial Information
• FDA Safety Alerts and Recalls

MeSH Terms

Conditions

Myelodysplastic Syndromes; beta-Thalassemia

Interventions

luspatercept

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Bristol Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 10, 2025
• First Posted: July 2, 2026
• Study Start: November 16, 2023
• Primary Completion: May 1, 2025
• Study Completion: May 1, 2025
• Last Updated: July 2, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)