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

NCT06716814 | Recruiting Phase 3 FDA Drug

• 2 other identifiers: Sobi.BIVV001-0022023-506537-29-00
• Study Type: interventional
• Target: 104
• Locations: 7 countries
• Sites: 24

Brief Summary

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.

Conditions

Haemophilia A (Moderate or Severe)

Keywords

Haemophilia A; Blood coagulation disorder; Factor VIII; FVIII; Coagulation protein disorder; Severe Haemophilia A; Efanesoctocog alfa; Haemophilia A (Moderate or Severe)

Outcome Measures

Primary Outcomes (9)

• 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

Study Arms (1)

Efanesoctocog alfa prophylaxis

EXPERIMENTAL

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

Interventions

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.

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

Efanesoctocog alfa prophylaxis

Eligibility Criteria

• Age: 6 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead
• PSI CRO: collaborator

Study Sites (24)

Sobi Investigational Site

Plovdiv, Bulgaria

RECRUITING

Sobi Investigational Site

Sofia, Bulgaria

RECRUITING

Sobi Investigational Site

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

Athens, Greece

RECRUITING

Sobi Investigational Site

Catanzaro, Italy

RECRUITING

Sobi Investigational Site

Florence, Italy

RECRUITING

Sobi Investigational Site

Milan, Italy

RECRUITING

Sobi Investigational Site

Naples, Italy

RECRUITING

Sobi Investigational Site

Parma, Italy

RECRUITING

Sobi Investigational Site

Rome, Italy

RECRUITING

Sobi Investigational Site

Rozzano, Italy

RECRUITING

Sobi Investigational Site

Vicenza, Italy

RECRUITING

Sobi Investigational Site

Oslo, Norway

RECRUITING

Sobi Investigational Site

A Coruña, Spain

ACTIVE NOT RECRUITING

Sobi Investigational Site

Zaragoza, Spain

ACTIVE NOT RECRUITING

Sobi Investigational Site

Gothenburg, Sweden

RECRUITING

Sobi Investigational Site

Lund, Sweden

RECRUITING

Sobi Investigational Site

Stockholm, Sweden

RECRUITING

MeSH Terms

Conditions

Hemophilia A; Lymphoma, Follicular; Blood Coagulation Disorders; Coagulation Protein Disorders

Interventions

BIVV001

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lymphoma, Non-Hodgkin; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Study Officials

• Medical Development Lead

  Sobi AB

  STUDY DIRECTOR

Central Study Contacts

Study Physician

CONTACT

+46 (0)8 697 20 00; medical.info@sobi.com

Clinical Program Lead

CONTACT

+46 (0)8 697 20 00; medical.info@sobi.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Roll-over study to provide post-trial access to treatment for patients with haemophilia A who have completed a previous trial with efanesoctocog alfa.

Study Record Dates

• First Submitted: October 2, 2024
• First Posted: December 4, 2024
• Study Start: March 6, 2025
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027
• Last Updated: April 20, 2026

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Within 1 year following completion of the trial, or at the time of CSR finalization.
• 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.

Locations

IT (8); BU (2); NO (1); SE (3); GR (1); ES (2); FR (7)