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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Adults participants diagnosed with β-thalassemia major |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Luspatercept | Drug | As per product label |
|
| Measure | Description | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | |
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Inclusion Criteria:
Exclusion Criteria:
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Adults participants in Taiwan diagnosed with β-thalassemia major and treated with luspatercept.
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| Name | Affiliation | Role |
|---|---|---|
| Bristol-Myers Squibb | Bristol-Myers Squibb | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Local Institution - 02 | Kaohsiung City | 807 | Taiwan | |||
| Local Institution - 04 |
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| Label | URL |
|---|---|
| BMS Clinical Trial Information | View source |
| FDA Safety Alerts and Recalls | View source |
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| ID | Term |
|---|---|
| C000621232 | luspatercept |
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| 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 |
| Tainan |
| 704 |
| Taiwan |
| Local Institution - 01 | Taipei | 100229 | Taiwan |
| Local Institution - 03 | Taipei | 10449 | Taiwan |
| Local Institution - 05 | Taoyuan | 333 | Taiwan |