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

NCT ID: NCT05817812

Last Updated: 2025-10-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

93 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-07-31

Study Completion Date

2026-06-12

Brief Summary

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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.

Detailed Description

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Conditions

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Hemophilia A, Severe

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Efanesoctocog alfa

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

Group Type EXPERIMENTAL

Efanesoctocog alfa

Intervention Type DRUG

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

Interventions

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Efanesoctocog alfa

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

Intervention Type DRUG

Other Intervention Names

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BIVV001

Eligibility Criteria

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Inclusion Criteria

* 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.

Exclusion Criteria

* 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.
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Syneos Health

OTHER

Sponsor Role collaborator

Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Physician, MD, PhD

Role: STUDY_DIRECTOR

Swedish Orphan Biovitrum AB

Locations

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Investigational Site

Lund, , Sweden

Site Status

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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

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Vienna, , Austria

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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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Countries

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Austria Croatia Czechia France Germany Greece Ireland Italy Netherlands Norway Slovenia Spain Sweden United Kingdom

Other Identifiers

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Sobi.BIVV001-001

Identifier Type: -

Identifier Source: org_study_id

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