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
- 招募中
- 入组人数
- 480
- 试验地点
- 206
- 主要终点
- 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
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
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 successfully rechallenged to the full dose of etavopivat before transferring.
- •Participants on hydroxyurea (HU), crizanlizumab or l-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they have been on a stable dose in the parent study as defined at the investigator's discretion. Necessary adjustments related to weight or age are accepted. Participants with temporary dose reductions or pauses due to medical reasons may still be considered to have a stable dose, as determined by the investigator, who will assess the impact of these adjustments based on clinical context and the participant's overall health status.
排除标准
- •Any disorder, except for conditions associated with SCD or thalassaemia, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol.
- •Participant withdrew or had permanent treatment discontinuation from an etavopivat clinical study.
- •Participants on permanent dose reduction (greater than [>] 28 days or more) or ongoing temporary treatment discontinuation.
- •Use of any of the following within the timeframes prior to the transfer visit as stated:
- •Use of haemoglobin S (HbS) polymerisation inhibitors 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 for more than 4 consecutive weeks during the parent study or anticipated need of such agents for a maintenance treatment 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 study, for the duration of FLORAL.
研究组 & 干预措施
Participants ≥ 2 years to less than (<) 12 years old with sickle cell disease
Participants ≥ 12 years of age will receive an oral dose of Etavopivat A or C and participants < 12 years of age will receive an oral dose of Etavopivat B.
干预措施: Etavopivat B (Drug)
Participants ≥ 12 years old with transfusion-dependent thalassaemia
Participants will receive an oral dose of Etavopivat A or C.
干预措施: Etavopivat A (Drug)
Participants greater than or equal to (≥) 12 years old with sickle cell disease
Participants will receive an oral dose of Etavopivat A or C.
干预措施: Etavopivat C (Drug)
Participants greater than or equal to (≥) 12 years old with sickle cell disease
Participants will receive an oral dose of Etavopivat A or C.
干预措施: Etavopivat A (Drug)
Participants ≥ 12 years old with sickle cell disease transfusion dependent
Participants will receive an oral dose of Etavopivat A or C.
干预措施: Etavopivat C (Drug)
Participants ≥ 12 years old with transfusion-dependent thalassaemia
Participants will receive an oral dose of Etavopivat A or C.
干预措施: Etavopivat C (Drug)
Participants ≥ 12 years old with non-transfusion dependent thalassaemia
Participants will receive an oral dose of Etavopivat A or C.
干预措施: Etavopivat C (Drug)
Participants ≥ 2 years to less than (<) 12 years old with sickle cell disease
Participants ≥ 12 years of age will receive an oral dose of Etavopivat A or C and participants < 12 years of age will receive an oral dose of Etavopivat B.
干预措施: Etavopivat C (Drug)
Participants ≥ 2 years to less than (<) 12 years old with sickle cell disease
Participants ≥ 12 years of age will receive an oral dose of Etavopivat A or C and participants < 12 years of age will receive an oral dose of Etavopivat B.
干预措施: Etavopivat A (Drug)
Participants ≥ 12 years old with non-transfusion dependent thalassaemia
Participants will receive an oral dose of Etavopivat A or C.
干预措施: Etavopivat A (Drug)
Participants ≥ 12 years old with sickle cell disease transfusion dependent
Participants will receive an oral dose of Etavopivat A or C.
干预措施: Etavopivat A (Drug)
结局指标
主要结局
Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately
时间窗: Baseline (week 0 of FLORAL) up to end of study (up to week 316)
Measured as number of events.
Number of adverse reactions, reported for each indication and age group separately
时间窗: Baseline (week 0 of FLORAL) up to end of study (up to week 316)
Measured as number of adverse reactions.
次要结局
- Annualised vaso-occlusive crisis (VOC) rates, reported for each age group separately(Baseline (week 0 of FLORAL) up to end of treatment (up to week 312))
- Change in VOCs, reported for each age group separately(Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312))
- Change in hemoglobin (Hb) concentration, reported for each age group separately(Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312))
- Annualised number of hospitalisations, reported for each age group separately(Baseline (week 0 of FLORAL) up to end of treatment (up to week 312))
- Average length of stay of hospitalisations, reported for each age group separately(Baseline (week 0 of FLORAL) up to end of treatment (up to week 312))
- Change in Hb concentration(Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312))
- Number of red blood cell (RBC) units transfused, reported for each indication separately(Baseline (week 0 of FLORAL) up to end of treatment (up to week 312))
- Change in RBC units transfused, reported for each indication separately(Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312))