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Hemophilia A, Severe

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

Sponsored by Swedish Orphan Biovitrum

About this trial

Last updated 8 months ago

Study ID

Sobi.BIVV001-001

Status

Active, not recruiting

Type

Interventional

Phase

Phase 3

Placebo

No

Accepting

18-75 Years
12+ Years
All
All

Not accepting

Not accepting
Healthy Volunteers

Trial Timing

Started 2 years ago

What is this trial about?

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.

What are the Participation Requirements?

Key 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.

Key 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.

Locations

Location

Status