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

NCT06596642 | Active Not Recruiting FDA Drug

• 1 other identifier: CA056-1132
• Study Type: observational
• Target: 61
• Locations: 1 country
• Sites: 5

Brief Summary

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.

Conditions

β-Thalassemia Major

Keywords

β-Thalassemia major

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (25)

• 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

Study Arms (1)

Adults participants diagnosed with β-thalassemia major

Drug: Luspatercept

Interventions

Luspatercept DRUG

As per product label

Adults participants diagnosed with β-thalassemia major

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

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

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (5)

Local Institution - 02

Kaohsiung City, 807, Taiwan

Location

Local Institution - 04

Tainan, 704, Taiwan

Location

Local Institution - 01

Taipei, 100229, Taiwan

Location

Local Institution - 03

Taipei, 10449, Taiwan

Location

Local Institution - 05

Taoyuan District, 333, Taiwan

Location

Related Links

• BMS Clinical Trial Information
• FDA Safety Alerts and Recalls

MeSH Terms

Interventions

luspatercept

Study Officials

• Bristol-Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: July 29, 2024
• First Posted: September 19, 2024
• Study Start: August 21, 2024
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): December 17, 2026
• Last Updated: June 4, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

TW (5)