Physical activity in severe Haemophilia A patients treated once weekly with efanesoctocog alfa
Research type
Research Study
Full title
A phase 3b open-label, multicenter study evaluating physical activity and joint health in previously treated patients ≥12 years of age with severe haemophilia A treated with intravenous recombinant coagulation FactorVIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; efanesoctocog alfa) for 24 months
IRAS ID
1005474
Contact name
Sofia Bergenstråle
Contact email
Sponsor organisation
Swedish Orphan Biovitrum AB
Research summary
The cornerstone of haemophilia treatment in patients with severe haemophilia A are medicines that prevent and control bleeding episodes and, consequently, prevent or slow down the development of joint disease. This is known as protein (FVIII) prophylaxis. Joint damage and progressive chronic joint disease are the hallmarks of haemophilia because a single or few repeated joint bleeds can lead to structural joint damage.
This is a phase 3b, single-arm, open-label, multinational, multicentre study of an investigational medicine called efanesoctocog alfa in previously prophylactic treated patients ≥12 years of age with severe haemophilia A.
The study medicine works by temporarily replacing a protein (FVIII) in the participants blood that is important to stop bleeding. The study medicine is also expected to prevent and control bleeding for a longer period of time than currently available FVIII replacement therapies, allowing less frequent injections.
This study has been designed to prospectively evaluate the patterns of physical activity and long-term joint outcomes in severe haemophilia participants receiving once-weekly prophylaxis rapid intravenous (IV; into a vein) bolus injections of efanesoctocog alfa.
Swedish Orphan Biovitrum AB (publ), or Sobi, is sponsoring this study and it will take place in approximately 37 centres (clinics) in 15 countries with about 90 participants. Participation will last for approximately 2 years.
Participants will need to come to the study centre at least 7 times. The study starts with a run-in period of 30-45 days, followed by a 24-month treatment period where the participants will receive efanesoctocog alfa. On-site study visits will occur every 6th months throughout the study.
Assessments include: Physical examination, vital signs, blood sampling, urine sampling (for pregnancy testing), ultrasound (HEAD-US), MRI scans, joint assessments, questionnaires, electronic patient diary and activity tracking.
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
22/YH/0272
Date of REC Opinion
3 May 2023
REC opinion
Further Information Favourable Opinion