NCT07215975招募中不适用
REal-World Application of Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East (RELATE): A Non-interventional Retrospective and Prospective Observational Study
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 200
- 试验地点
- 14
- 主要终点
- Change in transfusion burden
研究概览
简要总结
The purpose of this study is to evaluate luspatercept treatment in adults with transfusion-dependent beta-Thalassemia in the Middle East
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
- •Male or female participants of any race aged at least 18 years at time of initiation of luspatercept treatment
- •Participants with documented diagnosis of transfusion-dependent β-thalassemia (TDT).
- •Participants who have been initiated on treatment with luspatercept as per the product's Summary of Product Characteristics (SmPC) no longer than 12 months prior to informed consent signature, and for whom therapy is ongoing.
- •Participants for whom the decision to prescribe luspatercept treatment is clearly separated from the physician's decision to include the participant in the current study.
- •Participants who have provided signed informed consent for participating in the study and for collecting and analyzing medical data pertinent to the objectives of this study
排除标准
- •Participants that meet any of the contraindications to the administration of luspatercept as outlined in the latest version of the locally approved SmPC.
- •Participants who are currently receiving or are planned to receive treatment with any investigational drug/device/intervention or who have received any investigational product within 1 month or 5 half-lives of the investigational agent (whichever is longer) prior to luspatercept therapy initiation.
- •Participants who are currently pregnant, breastfeeding, or planning a pregnancy during the study observation period.
- •Participants who have not provided signed informed consent for participating in the study and for collecting and analysing medical data pertinent to the objectives of this study.
研究组 & 干预措施
Participants receiving luspatercept treatment
干预措施: Luspatercept (Drug)
结局指标
主要结局
Change in transfusion burden
时间窗: Baseline and up to week 144
Change in mean pre-transfusion hemoglobin level
时间窗: Baseline and up to week 144
次要结局
- Change in transfusion-related visits(Baseline and up to week 144)
- Proportion of participants achieving ≥33% reduction in red blood cell (RBC) transfusion burden (number of RBC units transfused) plus a reduction of ≥2 units(Baseline and up to week 144)
- Proportion of participants achieving ≥50% reduction in red blood cell transfusion burden plus a reduction of ≥2 units(Baseline and up to week 144)
- Proportion of participants without red blood cell transfusion during any consecutive 12-week or 24-week treatment period(Baseline and up to week 144)
- Time from first luspatercept dosing date to first onset of red blood cell-transfusion independence ≥12 weeks and ≥24 weeks(Baseline and up to week 144)
- Change in proportion of participants with mean serum ferritin <1,000, 1000-2500, and >2,500 μg/L(Baseline and up to week 144)
- Number of medical encounters (inpatient hospitalizations, emergency department attendances, hospital outpatient visits, visits at office-based physicians)(Baseline and up to week 144)
- Inpatient length of stay(Baseline and up to week 144)
- Length of time from initiation to discontinuation of luspatercept treatment(Up to week 144)
- Frequencies of reasons for discontinuation of luspatercept treatment(Up to week 144)
- Participant sociodemographics(Baseline)
- Time from first luspatercept dosing date to the first erythroid response(Baseline and up to week 144)
- Time from the date the erythroid response is first observed until the last day of response(Baseline and up to week 144)
- Participant pretransfusion hemoglobin level(Baseline and up to week 144)
- Participant baseline transfusion burden(Baseline)
- Time from the date a 12-week and 24-week red blood cell-transfusion independence is first observed until the date the participant has a subsequently documented red blood cell transfusion(Baseline and up to week 144)
- Change in mean serum ferritin (SF) level(Baseline and up to week 144)
- Types (drug formulation - mono and combination therapy) of iron chelation therapy received(Baseline and up to week 144)
- Change in mean daily dose of iron chelation therapy from baseline(Baseline and up to week 144)
- Proportion of participants remaining on luspatercept treatment(Up to week 144)
- Participant disease characteristics(Baseline)
- Participant comorbidities(Baseline)
- Concomitant treatment(s) received(Baseline)
- Number of luspatercept doses administered(Up to week 144)
- Number of luspatercept dose modifications(Up to week 144)
- Frequencies of reasons for luspatercept dose modifications(Up to week 144)
研究者
研究点 (14)
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