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

Last updated: April 8, 2025
Sponsor: Swedish Orphan Biovitrum
Overall Status: Active - Recruiting

Phase

3

Condition

Hemophilia

Treatment

Efanesoctocog alfa

Clinical Study ID

NCT06716814
Sobi.BIVV001-002
2023-506537-29-00
  • Ages > 6
  • Male

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Capable of giving signed informed consent. Parents or legally designatedrepresentatives' consent is required for patients who are below 18 years of age orunable to give consent. Patients who are below 18 years of age may provide assent inaddition to the parents'/legally designated representatives' consent, ifappropriate.

  • Must have completed one of the required parent studies: Sobi.BIVV001-001,Sobi.BIVV001-003, or LTS16294, and be receiving a clinical benefit from theefanesoctocog alfa treatment, as judged by the Investigator.

  • Willingness and ability of patient or their parent or legally designatedrepresentative to complete training in the use of the study patient diary and tocomplete the diary throughout the study.

Exclusion

Exclusion Criteria:

  • Positive inhibitor result (assessed by central laboratory), defined as ≥0.6 Bethesdaunits (BU)/mL, at Baseline Visit.

  • Ongoing or planned participation in any interventional clinical study at BaselineVisit.

  • Patient not suitable for participation, whatever the reason, as judged by theInvestigator, including medical or clinical conditions, or patients potentially atrisk of noncompliance to study procedures.

Study Design

Total Participants: 78
Treatment Group(s): 1
Primary Treatment: Efanesoctocog alfa
Phase: 3
Study Start date:
March 06, 2025
Estimated Completion Date:
August 31, 2026

Connect with a study center

  • Sobi Investigational Site

    Milan,
    Italy

    Active - Recruiting

  • Sobi Investigational Site

    Naples,
    Italy

    Active - Recruiting

  • Sobi Investigational Site

    A Coruña,
    Spain

    Active - Recruiting

  • Sobi Investigational Site

    Valencia,
    Spain

    Site Not Available

  • Sobi Investigational Site

    Zaragoza,
    Spain

    Active - Recruiting

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