A multinational, prospective, open-label, roll-over study (LIBERTY) to provide post-trial access to treatment for patients with severe haemophilia A who have completed a previous trial with efanesoctocog alfa
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 90
- 试验地点
- 29
- 主要终点
- Safety Endpoint: The occurrence of adverse events (AEs), including serious adverse events (SAEs) and AESIs
研究概览
简要总结
Safety Objective To collect safety and tolerability data of efanesoctocog alfa in previously treated patients with severe haemophilia A.
Efficacy Objective To collect further data on the efficacy of efanesoctocog alfa as a prophylaxis treatment To collect further data on the efficacy of efanesoctocog alfa in the treatment of bleeding episodes
研究设计
- 分配方式
- Not Applicable
- 主要目的
- EoT Visit
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 65+ years（0-17 Years, 65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and protocol. Parents’ or legally designated representatives’ consent is required for patients who are <18 years of age or unable to give consent, or as applicable per local laws, before any study-related activities are undertaken. Patients who are <18 years of age should provide assent in addition to the parents’/legally designated representatives’ consent, if appropriate.
- •Contraceptive use by patients should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Males: No contraceptive measures required for this study.
- •Must have completed one of the required parent studies: Sobi.BIVV001-001, Sobi.BIVV001-003, or LTS16294, and be receiving a clinical benefit from the efanesoctocog alfa treatment, as judged by the Investigator. The interval between the patient’s last study dose in the parent study and Visit 1 of this study should preferably be within 7 days for the Sobi.BIVV001-001 and LTS16294 studies to ensure there is no interruption in the prophylaxis dosing with efanesoctocog alfa. Patients coming from the Sobi.BIVV001-003 study, should preferably enter this study at the EoS visit of that study.
- •Willingness and ability of the patient or parent or their 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 (assessed by central laboratory), defined as ≥0.6 Bethesda units (BU)/mL, at baseline.
- •Ongoing or planned participation in any interventional clinical study at 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.
结局指标
主要结局
Safety Endpoint: The occurrence of adverse events (AEs), including serious adverse events (SAEs) and AESIs
Safety Endpoint: The occurrence of adverse events (AEs), including serious adverse events (SAEs) and AESIs
Efficacy Endpoints: To collect further data on the efficacy of efanesoctocog alfa as a prophylaxis treatment; To collect further data on the efficacy of efanesoctocog alfa in the treatment of bleeding episodes
Efficacy Endpoints: To collect further data on the efficacy of efanesoctocog alfa as a prophylaxis treatment; To collect further data on the efficacy of efanesoctocog alfa in the treatment of bleeding episodes
次要结局
未报告次要终点
研究者
Elena Santagostino
Scientific
Swedish Orphan Biovitrum AB (publ)