A Real-World Study of Β-Thalassemia Major Treatment with Luspatercept in Taiwan
- Registration Number
- NCT06596642
- Lead Sponsor
- Bristol-Myers Squibb
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 90
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 & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Adults participants diagnosed with β-thalassemia major Luspatercept -
- Primary Outcome Measures
Name Time Method 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
Name Time Method 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 demographics Baseline Participant weight Baseline Participant β0/β0 genotype status Baseline Participant disease status Baseline Participant comorbidities Baseline Participant concomitant medication Baseline Participant β-thalassemia treatment history Baseline Participant surgical history (splenectomy and/or cholecystectomy) Baseline
Trial Locations
- Locations (5)
Kaohsiung Medical University Chung-Ho Memorial Hospital
🇨🇳Kaohsiung, Taiwan
National Cheng Kung University Hospital
🇨🇳Tainan, Taiwan
MacKay Memorial Hospital
🇨🇳Taipei, Taiwan
Chang Gung Memorial Hospital, Linkou
🇨🇳Taoyuan, Taiwan
National Taiwan University Hospital
🇨🇳Taipei, Taiwan