A Real-World Study of β-Thalassemia Major Treatment With Luspatercept in Taiwan

Taiwan β-Thalassemia Major Real-World Study for Luspatercept

This real-world study will assess the efficacy and safety of luspatercept treatment for β-thalassemia major in Taiwan as well as the impact on quality of life.

Study Overview

• Status: Active, not recruiting
• Conditions: β-Thalassemia Major
• Intervention / Treatment: Drug: Luspatercept

• Study Type: Observational
• Enrollment (Actual): 61

Contacts and Locations

Study Locations

• Taiwan
  • Kaohsiung, Taiwan, 807
    • Local Institution - 02
  • Tainan, Taiwan, 704
    • Local Institution - 04
  • Taipei, Taiwan, 100229
    • Local Institution - 01
  • Taipei, Taiwan, 10449
    • Local Institution - 03
  • Taoyuan, Taiwan, 333
    • Local Institution - 05

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Non-Probability Sample

Study Population

Adults participants in Taiwan diagnosed with β-thalassemia major and treated with luspatercept.

Description

Inclusion Criteria:

• Participants ≥18 years of age.
• Participants with the diagnosis of transfusion-dependent β-thalassemia major who are eligible to the treatment of luspatercept.
• Before luspatercept treatment, participant's transfusion burden ≥ 24 Red Blood Cell units in 24 weeks, without ≥35-day transfusion free.

Exclusion Criteria:

• Hypersensitivity to the active substance or to any of the excipients.
• Pregnancy.
• Participants requiring treatment to control the growth of extramedullary hematopoiesis (EMH) masses.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Adults participants diagnosed with β-thalassemia major	Drug: Luspatercept; As per product label

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Red Blood Cell (RBC) transfusion units received by participants	Baseline, every 3 weeks thereafter until week 96
Participant hemoglobin (Hb) level results	Baseline, every 3 weeks thereafter until week 96
Participant serum ferritin results	Baseline, week 12, 24, 36, 48, 60, 72, 84 and 96

Secondary Outcome Measures

Outcome Measure	Time Frame
Participant weight	Baseline
Participant comorbidities	Baseline
Participant demographics	Baseline
Participant platelet level results	Baseline, and every 3 weeks thereafter up to week 96
Participant white blood cell count results	Baseline, and every 3 weeks thereafter up to week 96
Participant reticulocytes/normoblast count results	Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant Hemoglobin F (HbF) levels	Baseline and weeks 6 and 12
Participant uric acid level results	Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant lactate dehydrogenase (LDH) level results	Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant aspartate aminotransferase (AST)/alanine aminotransferase (ALT) ratio	Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant creatinine level results	Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Participant bilirubin (total and direct) laboratory values	Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
Type of iron chelating therapy prescribed to participants	Baseline, every 3 weeks thereafter until week 96
Iron chelating therapy treatment dosage prescribed to participants	Baseline, every 3 weeks thereafter until week 96
Participant Adverse Events (AEs)	Baseline and every 3 weeks until week 12, then weeks 24, 36, 48, 60, 72, 84 and 96
Participant health-related quality of life as assessed by Transfusion-dependent Quality of Life (TranQoL) questionnaire	Baseline, week 12, 24, 48, 72 and 96
Participant health-related quality of life as assessed by EQ-5D-5L questionnaire	Baseline, week 12, 24, 48, 72 and 96
Luspatercept treatment dosage prescribed to participants	Baseline, every 3 weeks thereafter until week 96
Duration of luspatercept treatment	Baseline, every 3 weeks thereafter until week 96
Participant reason(s) for luspatercept treatment discontinuation as assessed by the treating clinician	Baseline, every 3 weeks thereafter until week 96
Participant β0/β0 genotype status	Baseline
Participant disease status	Baseline
Participant concomitant medication	Baseline
Participant β-thalassemia treatment history	Baseline
Participant surgical history (splenectomy and/or cholecystectomy)	Baseline

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
• FDA Safety Alerts and Recalls

Study record dates

Study Major Dates

• Study Start (Actual): August 21, 2024
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): December 17, 2026

Study Registration Dates

• First Submitted: July 29, 2024
• First Submitted That Met QC Criteria: September 17, 2024
• First Posted (Actual): September 19, 2024

Study Record Updates

• Last Update Posted (Actual): June 4, 2025
• Last Update Submitted That Met QC Criteria: June 3, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Keywords: β-Thalassemia major
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Thalassemia; beta-Thalassemia; Hematinics; Luspatercept
• Other Study ID Numbers: CA056-1132

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes