NCT06596642进行中（未招募）不适用
Taiwan Β-Thalassemia Major Real-World Study for Luspatercept
Bristol-Myers Squibb5 个研究点 分布在 1 个国家目标入组 61 人开始时间: 2024年8月21日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 61
- 试验地点
- 5
- 主要终点
- Number of Red Blood Cell (RBC) transfusion units received by participants
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •Hypersensitivity to the active substance or to any of the excipients.
- •Pregnancy.
- •Participants requiring treatment to control the growth of extramedullary hematopoiesis (EMH) masses.
研究组 & 干预措施
Adults participants diagnosed with β-thalassemia major
干预措施: Luspatercept (Drug)
结局指标
主要结局
Number of Red Blood Cell (RBC) transfusion units received by participants
时间窗: Baseline, every 3 weeks thereafter until week 96
Participant hemoglobin (Hb) level results
时间窗: Baseline, every 3 weeks thereafter until week 96
Participant serum ferritin results
时间窗: Baseline, week 12, 24, 36, 48, 60, 72, 84 and 96
次要结局
- Participant Adverse Events (AEs)(Baseline and every 3 weeks until week 12, then weeks 24, 36, 48, 60, 72, 84 and 96)
- 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 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)
研究者
研究点 (5)
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