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

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

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.

Study Overview

• Status: Recruiting
• Conditions: Haemophilia A (Moderate or Severe)
• Intervention / Treatment: Drug: Efanesoctocog alfa

• Study Type: Interventional
• Enrollment (Estimated): 104
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Study Physician; Phone Number: +46 (0)8 697 20 00; Email: medical.info@sobi.com
• Study Contact Backup: Name: Clinical Program Lead; Phone Number: +46 (0)8 697 20 00; Email: medical.info@sobi.com

Study Locations

• Bulgaria
  • Plovdiv, Bulgaria
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
  • Sofia, Bulgaria
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
• France
  • Bordeaux, France
    • Withdrawn
    • Sobi Investigational Site
  • Brest, France
    • Active, not recruiting
    • Sobi Investigational Site
  • Bron, France
    • Active, not recruiting
    • Sobi Investigational Site
  • Le Kremlin-Bicêtre, France
    • Withdrawn
    • Sobi Investigational Site
  • Lille, France
    • Active, not recruiting
    • Sobi Investigational Site
  • Marseille, France
    • Withdrawn
    • Sobi Investigational Site
  • Strasbourg, France
    • Withdrawn
    • Sobi Investigational Site
• Greece
  • Athens, Greece
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
• Italy
  • Catanzaro, Italy
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
  • Florence, Italy
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
  • Milan, Italy
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
  • Naples, Italy
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
  • Parma, Italy
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
  • Rome, Italy
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
  • Rozzano, Italy
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
  • Vicenza, Italy
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
• Norway
  • Oslo, Norway
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
• Spain
  • A Coruña, Spain
    • Active, not recruiting
    • Sobi Investigational Site
  • Zaragoza, Spain
    • Active, not recruiting
    • Sobi Investigational Site
• Sweden
  • Gothenburg, Sweden
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
  • Lund, Sweden
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
  • Stockholm, Sweden
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Efanesoctocog alfa prophylaxis; 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.	Drug: Efanesoctocog alfa; 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; Altuviiio; Altuvoct; Recombinant coagulation factor VIII Fc-von-Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of injections to treat a bleeding episode		From enrollment and up to 52 weeks
Total dose 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)	Number of participants with occurrence of AEs, SAEs, and AESIs	From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for treated bleeding episodes	ABR for treated bleeding episodes during prophylactic treatment.	From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for treated bleeding episodes by type of bleed	ABR for treated bleeding episodes during prophylactic treatment by type of bleed.	From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for treated bleeding episodes by location of bleed	ABR for treated bleeding episodes during prophylactic treatment by location of bleed.	From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for all bleeding episodes	ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment.	From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for all bleeding episodes by type of bleed	ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment, by type of bleed.	From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for all bleeding episodes by location of bleed	ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment, by location of bleed.	From enrollment and up to 52 weeks

Collaborators and Investigators

• Sponsor: Swedish Orphan Biovitrum
• Collaborators: PSI CRO
• Investigators: Study Director: Medical Development Lead, Sobi AB

Study record dates

Study Major Dates

• Study Start (Actual): March 6, 2025
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027

Study Registration Dates

• First Submitted: October 2, 2024
• First Submitted That Met QC Criteria: November 29, 2024
• First Posted (Actual): December 4, 2024

Study Record Updates

• Last Update Posted (Actual): April 20, 2026
• Last Update Submitted That Met QC Criteria: April 15, 2026
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: Factor VIII; FVIII; Haemophilia A; Blood coagulation disorder; Coagulation protein disorder; Efanesoctocog alfa; Severe Haemophilia A; Haemophilia A (Moderate or Severe)
• Additional Relevant MeSH Terms: Neoplasms; Genetic Diseases, Inborn; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Lymphoma, Non-Hodgkin; Lymphoma; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Lymphoma, Follicular; Hemophilia A; Blood Coagulation Disorders; Coagulation Protein Disorders; Coagulants; BIVV001
• Other Study ID Numbers: Sobi.BIVV001-002; 2023-506537-29-00 (Ctis)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sobi data sharing criteria and process for requesting access can be found at: https:// www.sobi.com/en/policies
• IPD Sharing Time Frame: Within 1 year following completion of the trial, or at the time of CSR finalization.

IPD Sharing Access Criteria

A decision on sharing will be based on the following:

The scientific merit of the proposal - i.e. the proposal should be scientifically sound, ethical, and have the potential to contribute to the advancement of public health. The feasibility of the research proposal - i.e. the requesting research team must be scientifically qualified and have the resources to conduct the proposed project. Maintenance of personal integrity - i.e. Sobi will not consider sharing individual data if there is a risk of re-identification of individuals despite a proper anonymisation. Moreover, the patients' informed consent will always be respected. Sobi reserves the right to reject the proposal if the anonymisation process will render unusable data. Publication of results - the applicants should commit to submit their findings to a peer-reviewed scientific journal, alternatively to present the results at a congress (poster or similar), regardless of the research outcome.

• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes