NCT05817812

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

A Phase 3b Open-label Study Evaluating Physical Activity and Joint Health in Previously Treated Patients ≥12 Years With Severe Haemophilia A Treated With Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (Efanesoctocog Alfa) for 24 Months

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 93 (actual)
• Sponsor: Swedish Orphan Biovitrum · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
DRUG	Efanesoctocog alfa	Recombinant coagulation factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN)

Timeline

• Start date: 2023-07-31
• Primary completion: 2026-05-22
• Completion: 2026-06-12
• First posted: 2023-04-18
• Last updated: 2025-10-09

Locations

30 sites across 14 countries: Austria, Croatia, Czechia, France, Germany, Greece, Ireland, Italy, Netherlands, Norway, Slovenia, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study