A Multinational, Prospective, Open-label, Roll-over Study (LIBERTY) to Provide Post-trial Access to Treatment for Patients With Haemophilia A Who Have Completed a Previous Trial With Efanesoctocog Alfa
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 104
- 试验地点
- 22
- 主要终点
- Number of injections to treat a bleeding episode
研究概览
简要总结
This is a multinational, prospective, open-label, roll-over study in patients with haemophilia A, ≥6 years of age, who have completed participation in any of the parental studies with efanesoctocog alfa; XTEND-ed study (LTS16294), FREEDOM study (Sobi.BIVV001-001), PK comparison study (Sobi.BIVV001-003) or SHINE study (Sobi.BIVV001-004). The aim of the study is to provide patients with continuous benefit from efanesoctocog alfa treatment and to further continue clinical monitoring for safety and efficacy until efanesoctocog alfa is commercially available in each patient's respective country (or until March 2027, whichever comes first).
The study starts with the Baseline Visit, which will be done in connection to the end of treatment at the EoT/EoS visit (or equivalent) in the respective parent study. Subsequent study visits (on site or phone call) will be done approximately every 13 weeks until End of Treatment. An End of Study safety phone call will be done 14 (+7) days after the End of Treatment Visit.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Capable of giving signed informed consent. Parents or legally designated representatives' consent is required for patients who are below 18 years of age or unable to give consent. Patients who are below 18 years of age may provide assent in addition to the parents'/legally designated representatives' consent, if appropriate.
- •Must have completed one of the required parent studies: Sobi.BIVV001-001, Sobi.BIVV001-003, LTS16294, or Sobi.BIVV001-004, and be receiving a clinical benefit from the efanesoctocog alfa treatment, as judged by the Investigator.
- •Willingness and ability of patient or their parent or legally designated representative to complete training in the use of the study patient diary and to complete the diary throughout the study.
排除标准
- •Positive inhibitor result, defined as ≥0.6 Bethesda units (BU)/mL, present at the Baseline Visit.
- •Ongoing or planned participation in any interventional clinical study at the Baseline Visit.
- •Patient not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.
研究组 & 干预措施
Efanesoctocog alfa prophylaxis
Patients who have completed a previous study with efanesoctocog alfa may continue to be treated with a prophylactic dose of 50 IU/kg efanesoctocog alfa once weekly.
干预措施: Efanesoctocog alfa (Drug)
结局指标
主要结局
Number of injections to treat a bleeding episode
时间窗: From enrollment and up to 52 weeks
Total dose to treat a bleeding episode
时间窗: From enrollment and up to 52 weeks
Adverse events (AEs), including serious adverse events (SAEs) and adverse events of special interest (AESIs)
时间窗: From enrollment and up to 52 weeks
Number of participants with occurrence of AEs, SAEs, and AESIs
Annualized bleeding rate (ABR) for treated bleeding episodes
时间窗: From enrollment and up to 52 weeks
ABR for treated bleeding episodes during prophylactic treatment.
Annualized bleeding rate (ABR) for treated bleeding episodes by type of bleed
时间窗: From enrollment and up to 52 weeks
ABR for treated bleeding episodes during prophylactic treatment by type of bleed.
Annualized bleeding rate (ABR) for treated bleeding episodes by location of bleed
时间窗: From enrollment and up to 52 weeks
ABR for treated bleeding episodes during prophylactic treatment by location of bleed.
Annualized bleeding rate (ABR) for all bleeding episodes
时间窗: From enrollment and up to 52 weeks
ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment.
Annualized bleeding rate (ABR) for all bleeding episodes by type of bleed
时间窗: From enrollment and up to 52 weeks
ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment, by type of bleed.
Annualized bleeding rate (ABR) for all bleeding episodes by location of bleed
时间窗: From enrollment and up to 52 weeks
ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment, by location of bleed.
次要结局
未报告次要终点