Luspatercept
DrugAs per product label
Active, Not Recruiting
NCT Number: NCT06596642
A Real-World Study of β-Thalassemia Major Treatment With Luspatercept in Taiwan
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.
Active, Not Recruiting
This study is active but is not currently recruiting participants.
Notify MeKey information
Conditions
Age range
18 year and older
Sex eligibility
All sexes
Study type
Observational
Primary location
Local Institution - 02, Kaohsiung City, Taiwan
Loading trial locations.
Who can participate
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
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.
Treatment and study plan
Primary outcomes
-
Number of Red Blood Cell (RBC) transfusion units received by participants
Time frame: Baseline, every 3 weeks thereafter until week 96
-
Participant hemoglobin (Hb) level results
Time frame: Baseline, every 3 weeks thereafter until week 96
-
Participant serum ferritin results
Time frame: Baseline, week 12, 24, 36, 48, 60, 72, 84 and 96
Secondary outcomes
-
Participant platelet level results
Time frame: Baseline, and every 3 weeks thereafter up to week 96
-
Participant white blood cell count results
Time frame: Baseline, and every 3 weeks thereafter up to week 96
-
Participant reticulocytes/normoblast count results
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
-
Participant Hemoglobin F (HbF) levels
Time frame: Baseline and weeks 6 and 12
-
Participant uric acid level results
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
-
Participant lactate dehydrogenase (LDH) level results
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
-
Participant aspartate aminotransferase (AST)/alanine aminotransferase (ALT) ratio
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
-
Participant creatinine level results
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
-
Participant bilirubin (total and direct) laboratory values
Time frame: Baseline, and weeks 12, 24, 36, 48, 60, 72, 84 and 96
-
Type of iron chelating therapy prescribed to participants
Time frame: Baseline, every 3 weeks thereafter until week 96
-
Iron chelating therapy treatment dosage prescribed to participants
Time frame: Baseline, every 3 weeks thereafter until week 96
-
Participant Adverse Events (AEs)
Time frame: 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
Time frame: Baseline, week 12, 24, 48, 72 and 96
-
Participant health-related quality of life as assessed by EQ-5D-5L questionnaire
Time frame: Baseline, week 12, 24, 48, 72 and 96
-
Luspatercept treatment dosage prescribed to participants
Time frame: Baseline, every 3 weeks thereafter until week 96
-
Duration of luspatercept treatment
Time frame: Baseline, every 3 weeks thereafter until week 96
-
Participant reason(s) for luspatercept treatment discontinuation as assessed by the treating clinician
Time frame: Baseline, every 3 weeks thereafter until week 96
-
Participant demographics
Time frame: Baseline
-
Participant weight
Time frame: Baseline
-
Participant β0/β0 genotype status
Time frame: Baseline
-
Participant disease status
Time frame: Baseline
-
Participant comorbidities
Time frame: Baseline
-
Participant concomitant medication
Time frame: Baseline
-
Participant β-thalassemia treatment history
Time frame: Baseline
-
Participant surgical history (splenectomy and/or cholecystectomy)
Time frame: Baseline
Sponsors and collaborators
Lead sponsor
Bristol-Myers Squibb
Industry
Registry information
Official study title
Taiwan β-Thalassemia Major Real-World Study for Luspatercept
Important dates
- Study start
- 2024
- Primary completion
- 2026
- Study completion
- 2026
- First posted
- Sep 19, 2024
- Registry last updated
- Jun 4, 2025
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
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