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NCT Number: NCT06716814

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

This is a multinational, prospective, open-label, roll-over study in patients with 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), PK comparison study (Sobi.BIVV001-003) or SHINE study (Sobi.BIVV001-004). 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 treatment at the EoT/EoS 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.

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Key information

Conditions

Haemophilia A (Moderate or Severe) Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia A Hemorrhagic Disorders Immune System Diseases Immunoproliferative Disorders Lymphatic Diseases Lymphoma Lymphoma, Follicular Lymphoma, Non-Hodgkin Lymphoproliferative Disorders Neoplasms Neoplasms by Histologic Type

Age range

6 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Sobi Investigational Site, Plovdiv, Bulgaria

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Efanesoctocog alfa

Drug

Treatment starts when the patient completes the parent study and continues until efanesoctocog alfa is commercially available in each patient's respective country, or until March 2027, whichever comes first.

Other names: BIVV001, Altuvoct, Altuviiio, Recombinant coagulation factor VIII Fc-von-Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN)

Primary outcomes

  1. Number of injections to treat a bleeding episode

    Time frame: From enrollment and up to 52 weeks

  2. Total dose to treat a bleeding episode

    Time frame: From enrollment and up to 52 weeks

  3. Adverse events (AEs), including serious adverse events (SAEs) and adverse events of special interest (AESIs)

    Time frame: From enrollment and up to 52 weeks

    Number of participants with occurrence of AEs, SAEs, and AESIs

  4. Annualized bleeding rate (ABR) for treated bleeding episodes

    Time frame: From enrollment and up to 52 weeks

    ABR for treated bleeding episodes during prophylactic treatment.

  5. Annualized bleeding rate (ABR) for treated bleeding episodes by type of bleed

    Time frame: From enrollment and up to 52 weeks

    ABR for treated bleeding episodes during prophylactic treatment by type of bleed.

  6. Annualized bleeding rate (ABR) for treated bleeding episodes by location of bleed

    Time frame: From enrollment and up to 52 weeks

    ABR for treated bleeding episodes during prophylactic treatment by location of bleed.

  7. Annualized bleeding rate (ABR) for all bleeding episodes

    Time frame: From enrollment and up to 52 weeks

    ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment.

  8. Annualized bleeding rate (ABR) for all bleeding episodes by type of bleed

    Time frame: 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.

  9. Annualized bleeding rate (ABR) for all bleeding episodes by location of bleed

    Time frame: 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Program Lead

CONTACT

[email protected]

+46 (0)8 697 20 00

Study Physician

CONTACT

[email protected]

+46 (0)8 697 20 00

Sponsors and collaborators

Lead sponsor

Swedish Orphan Biovitrum

Industry

Collaborators

  • PSI CRO

Registry information

Official study title

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

Acronym: LIBERTY

Important dates

Study start
2025
Primary completion
2027
Study completion
2027
First posted
Dec 4, 2024
Registry last updated
Jun 26, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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