Active — Not RecruitingNot Applicable

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

Taiwan61 participantsStarted 2024-08-21

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

Number of Red Blood Cell (RBC) transfusion units received by participants

Timeframe: Baseline, every 3 weeks thereafter until week 96

Participant hemoglobin (Hb) level results

Timeframe: Baseline, every 3 weeks thereafter until week 96

Participant serum ferritin results

Timeframe: Baseline, week 12, 24, 36, 48, 60, 72, 84 and 96

Trial details

NCT IDNCT06596642

SponsorBristol-Myers Squibb

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2026-10-01

View on ClinicalTrials.gov More β-Thalassemia Major trials