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 - Sobi.BIVV001-002

Swedish Orphan Biovitrum AB (publ)0 个研究点目标入组 38 人开始时间: 2023年12月4日最近更新: 2024年8月21日

适应症

试验速览

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•Capable of giving signed informed consent. 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., Contraceptive use by patients should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. A) Males: No contraceptive measures required for this study. B) Females: A female patient is eligible to participate if she is not pregnant or breastfeeding. Women of child-bearing potential (WOCBP) shall be using an acceptable contraceptive method during the intervention period until the EoS safety phone call., 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 patient or their legal designated representative to complete training in the use of the study patient diary.

•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.

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