CTRI/2025/08/093581尚未招募3 期
An open-label, multi-centre, rollover study to characterise long-term safety and efficacy of etavopivat in adults, adolescents and children who have sickle cell disease or thalassaemia and have completed a treatment period in an etavopivat study
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 325
- 试验地点
- 6
- 主要终点
- 1. Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately.
研究概览
简要总结
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant’s country.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 盲法
- None
入排标准
- 年龄范围
- 11.00 Month(s) 至 99.00 Year(s)（—）
- 性别
- All
入选标准
- •Participant must have ongoing participation in an etavopivat parent study for treatment of sickle cell disease (SCD) or thalassaemia and have completed at least a treatment period of the parent study.
- •Participant must have derived clinical benefit from treatment with etavopivat, as determined by the investigator.
- •Any participant with dose reduction or temporary discontinuation will need to be rechallenged before transferring.
- •4.Participants on hydroxyurea (HU), crizanlizumab or l-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they: 5.Have been on a stable dose during participation in the parent study (i.e., no changes to the dose except for changes to weight or age reasons).
- •Have been compliant with the treatment regimen at the discretion of the investigator during participation of the parent study.
排除标准
- •1.Any disorder, except for conditions associated with SCD or thalassaemia, which in the investigators opinion might jeopardise participants safety or compliance with the protocol.
- •Participant withdrew or had permanent treatment discontinuation from an etavopivat clinical study.
- •Participants on permanent dose reduction or temporary treatment discontinuation.
- •Use of any of the following within the timeframes prior to the transfer visit as stated:
- •Use of voxelotor within participation of the parent study or anticipated need for this agent during this study.
- •Use of an experimental selectin antagonist (e.g., monoclonal antibody or small molecule) within the parent study or anticipated need for such agents during this study.
- •Use of erythropoietin or other haematopoietic growth factor treatment within the parent study or anticipated need for such agents during this study.
- •Receiving or use of concomitant medications that are strong inducers of cytochrome P450 (CYP) 3A4 within 2 weeks of the transfer visit or anticipated need for such agents during the study.
- •Current participation in a study that is not a designated parent study, or planned participation in any other clinical trial, for the duration of FLORAL.
结局指标
主要结局
1. Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately.
时间窗: Baseline (week 0 of | FLORAL) to end of study | (week 264, or earlier)
2.Number of adverse reactions, reported for each indication and age group separately
时间窗: Baseline (week 0 of | FLORAL) to end of study | (week 264, or earlier)
次要结局
- Annualised vaso-occlusive crisis (VOC) rates, reported for each age group separately(Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier)
- Change in VOCs, reported for each age group separately(Baseline (of parent study) to end of treatment at week 260, or earlier)
- Change in hemoglobin (Hb) concentration, reported for each age group separately(Baseline (of parent study) to end of treatment at week 260, or earlier)
- Annualised number of hospitalisations, reported for each age group separately(Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier)
- Average length of stay of hospitalisations, reported for each age group separately(Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier)
- Change in Hb concentration(Baseline (of parent study) to end of treatment at week 260, or earlier)
- Number of red blood cell (RBC) units transfused, reported for each indication separately(Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier)
- Change in RBC units transfused, reported for each indication separately(Baseline (of parent study) to end of treatment at week 260, or earlier)
研究者
研究点 (6)
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