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Continued access to efanesoctocog alfa for patients with severe haemophilia A

A Multinational, Prospective, Open-label, Roll-over Study (LIBERTY) to Provide Post-trial Access to Treatment for Patients With Haemophilia A Who Have Completed a Previous Trial With Efanesoctocog Alfa

PHASE3 · Swedish Orphan Biovitrum · NCT06716814

This study is for people aged 6 and older with severe hemophilia A to see if they can keep using the treatment efanesoctocog alfa safely until it becomes available for everyone.

Quick facts

PhasePHASE3
Study typeInterventional
Enrollment104 (estimated)
Ages6 Years and up
SexMale
SponsorSwedish Orphan Biovitrum (industry)
Locations24 sites (Plovdiv and 23 other locations)
Trial IDNCT06716814 on ClinicalTrials.gov

What this trial studies

This is a multinational, open-label study designed for patients aged 6 years and older with severe haemophilia A who have previously participated in studies involving efanesoctocog alfa. The study aims to provide ongoing access to this treatment while monitoring its safety and efficacy until it becomes commercially available. Patients will have regular visits approximately every 13 weeks, either in person or via phone, to assess their health and treatment progress. The study will continue until March 2027 or until the treatment is available in their country.

Who should consider this trial

Good fit: Ideal candidates are patients aged 6 years and older who have completed a prior study with efanesoctocog alfa and are benefiting from the treatment.

Not a fit: Patients with a positive inhibitor result or those currently participating in another interventional clinical study may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could ensure continuous treatment access for patients with severe haemophilia A, improving their health outcomes.

How similar studies have performed: Other studies involving efanesoctocog alfa have shown promising results, indicating that this approach is based on previously successful methodologies.

Eligibility criteria

Show full inclusion / exclusion criteria 
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, LTS16294, or Sobi.BIVV001-004, 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, defined as ≥0.6 Bethesda units (BU)/mL, present at the Baseline Visit.
* Ongoing or planned participation in any interventional clinical study at the 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.

Where this trial is running

Plovdiv and 23 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Haemophilia A, Blood coagulation disorder, Factor VIII, FVIII, Coagulation protein disorder, Severe Haemophilia A, Efanesoctocog alfa

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.