Post completed efanesoctocog alfa trial treatment access study for patients with severe haemophilia A

Phase 1

• Conditions: Haemophilia A; MedDRA version: 20.0Level: LLTClassification code: 10018937Term: Haemophilia A Class: 10010331; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: CTIS2023-506537-29-00
• Lead Sponsor: Swedish Orphan Biovitrum AB (publ)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-12-04
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 38

Inclusion Criteria

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.

Exclusion Criteria

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified