A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa

NCT05817812 | Active Not Recruiting Phase 3 FDA Drug

• 1 other identifier: Sobi.BIVV001-001
• Study Type: interventional
• Target: 93
• Locations: 14 countries
• Sites: 30

Brief Summary

FREEDOM is a multicentre, open-label, single-arm, phase 3b study in Europe that aims to enrol approximately 90 previously treated severe haemophilia A patients aged ≥12 years, currently on prophylaxis. After a run-in period of 30-45 days, patients will receive efanesoctocog alfa prophylaxis, 50 IU/kg once-weekly for 24 months (additional preventive dose not permitted). An activity tracker and an electronic patient diary will be used to collect data on physical activity, bleeds, factor dosing, pain, and injuries from screening throughout the study. The primary objective is to describe changes in physical activities over 24 months on efanesoctocog alfa prophylaxis, with a primary endpoint of change from baseline in International Physical Activity Questionnaire (IPAQ) at month 24. Secondary objectives include relationship between physical activity and other variables (bleeds, joint status, pain, injuries, and quality of life); changes in joint status as assessed by HEAD-US, HJHS and MRI; occurrence of bleeds, injuries, pain. Safety and tolerability of efanesoctocog alfa will also be evaluated.

Conditions

Hemophilia A, Severe

Keywords

Blood coagulation disorder; Coagulation protein disorder; Haemophilia; FVIII; Efanesoctocog alfa

Outcome Measures

Primary Outcomes (1)

• Change from baseline in International Physical Activity Questionnaire (IPAQ) scoring

  The IPAQ is a validated, 7-day recall questionnaire measuring current levels of physical activity. Scoring of the IPAQ results in a continuous variable in the form of total MET-minutes per week. Higher scores means higher physical activity level.

  Baseline and month 24

Secondary Outcomes (42)

• Change from baseline in International Physical Activity Questionnaire (IPAQ) scoring

  Baseline and month 12

• International Physical Activity Questionnaire (IPAQ) score

  Baseline, month 6, 12, 18 and 24

• Change in mean daily minutes in physical activity

  Run-in month, month 12 and 24

• Mean daily minutes of physical activity

  Per 6-month period

• Change in type of workouts

  Run-in month, month 12 and 24

Study Arms (1)

Efanesoctocog alfa

EXPERIMENTAL

All patients will be treated intravenously once weekly with 50 IU/kg efanesoctocog alfa

Drug: Efanesoctocog alfa

Interventions

Efanesoctocog alfa DRUG

Recombinant coagulation factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN)

Also known as: BIVV001

Efanesoctocog alfa

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Previous treatment for haemophilia A with any marketed recombinant and/or plasma-derived FVIII for at least 150 EDs.
• Having received prophylactic treatment with any marketed recombinant and/or plasma FVIII or emicizumab per local label for at least 12 months preceding enrolment.
• Having 12 months documented pre-study treatment data regarding prophylactic treatment prescriptions and 6 months data on bleeding episodes prior to baseline visit.
• Willingness and ability to complete training in the use of the study ePD and to use the ePD in their own smartphone throughout the study.
• Willingness and ability to use the activity tracker provided by the sponsor to measure physical activity and heart rate.
• Be able and willing to administer efanesoctocog alfa intravenously at home.

You may not qualify if:

• Serious musculoskeletal and/or neurological impairment limiting the mobility and the physical ability to a degree that makes the patient unsuitable for the study as judged by the investigator.
• Other known coagulation disorder(s) in addition to haemophilia A.
• History and/or current positive inhibitor test defined as ≥0.6 BU/mL.
• Treatment with NSAIDs above the maximum dose specified in the prescribing information within 2 weeks prior to screening.
• Systematic treatment within 12 weeks prior to screening with chemotherapy and/or other immunosuppressive drugs.
• Treatment with an investigational product within 30 days or 5.5 half-lives prior to screening, whichever is longer.
• Major surgery within 12 weeks prior to screening or planned major orthopaedic surgery to occur during the study.
• At baseline visit, patients who have not been compliant in using the activity tracker.

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead
• Syneos Health: collaborator

Study Sites (30)

Investigational Site

Vienna, Austria

Location

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Zagreb, Croatia

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Brno, Czechia

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Prague, Czechia

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Bordeaux, France

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Le Kremlin-Bicêtre, France

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Marseille, France

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Strasbourg, France

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Berlin, Germany

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Frankfurt, Germany

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Giessen, Germany

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Hamburg, Germany

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Athens, Greece

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Dublin, Ireland

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Catanzaro, Italy

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Florence, Italy

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Naples, Italy

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Parma, Italy

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Rome, Italy

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Utrecht, Netherlands

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Oslo, Norway

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Ljubljana, Slovenia

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Barcelona, Spain

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Madrid, Spain

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Oviedo, Spain

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Lund, Sweden

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Solna, Sweden

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Canterbury, United Kingdom

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Newcastle upon Tyne, United Kingdom

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Oxford, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia A; 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

Study Officials

• Study Physician, MD, PhD

  Swedish Orphan Biovitrum AB

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: March 21, 2023
• First Posted: April 18, 2023
• Study Start: July 31, 2023
• Primary Completion (Estimated): May 22, 2026
• Study Completion (Estimated): June 12, 2026
• Last Updated: October 9, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR

Locations

NL (1); ES (3); FR (4); IE (1); SL (1); CR (1); GR (1); CZ (2); SE (2); NO (1); GB (3); DE (4); IT (5); AU (1)