Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa

Phase 3

Recruiting

• Conditions: Severe Haemophilia A
• Interventions: Drug: Efanesoctocog alfa

• Registration Number: NCT06716814
• Lead Sponsor: Swedish Orphan Biovitrum

Brief Summary

This is a multinational, prospective, open-label, roll-over study in patients with severe 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), or PK comparison study (Sobi.BIVV001-003). 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 Study 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-10-02
• Study First Submitted QC: 2024-11-29
• Study First Posted: 2024-12-04
• Study Start: 2025-03-06
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-08
• Last Update Posted: 2025-04-09
• Primary Completion: 2026-08
• Study Completion: 2026-08

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Male
• Target Recruitment: 78

Inclusion Criteria

• 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, or LTS16294, 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.

Exclusion Criteria

• Positive inhibitor result (assessed by central laboratory), defined as ≥0.6 Bethesda units (BU)/mL, at Baseline Visit.
• 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
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Efanesoctocog alfa phrophylaxis	Efanesoctocog alfa	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.

Primary Outcome Measures

Name	Time	Method
Total dose to treat a bleeding episode	From enrollment and up to 52 weeks
Number of injections to treat a bleeding episode	From enrollment and up to 52 weeks
Adverse events (AEs), including serious adverse events (SAEs) and adverse events of special interest (AESIs)	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	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	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	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	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	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	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.

Trial Locations

Locations (1)

Sobi Investigational Site; 🇪🇸 Zaragoza, Spain