Join-us
Research type
Research Study
Full title
An 18-month low-interventional prospective, multicentre study to assess joint outcomes in patients with haemophilia A or B on prophylaxis with efmoroctocog alfa or eftrenonacog alfa
IRAS ID
1007113
Contact name
Nikola Misikova
Contact email
Sponsor organisation
Swedish Orphan Biovitrum AB
Research summary
This is a low-interventional study to prospectively describe the joint health over an 18-month period of prophylactic treatment with Efmoroctocog alfa (Elocta®) and Eftrenonacog alfa (Alprolix®) in patients with haemophilia A or haemophilia B in a real-world setting. Efmoroctocog alfa (Elocta®) and Eftrenonacog alfa (Alprolix®) are coagulation factor concentrates which are prepared by a technique called recombinant DNA technology and are used for prophylaxis treatment of haemophilia.
The study will be conducted in approximately 11 European countries and United Kingdom. The sponsor of this study is Swedish Orphan Biovitrum AB.
Haemophilia can cause spontaneous bleeds into muscles and joints causing acute pain and swelling and can result in reduced joint range of motion, long-term cartilage damage and haemophilic arthropathy. There are still limited real-world data on joint health outcomes, assessed with objective measures for the patients receiving this prophylactic treatment with Efmoroctocog alfa (Elocta®) and Eftrenonacog alfa (Alprolix®). The results from the study may help in improving the knowledge of joint disease and ultrasound assessment in patients with haemophilia, and potentially in improving the management of haemophilic arthropathy in the future.
Data output from florio HAEMO (CE marked medical device for monitoring of haemophilia therapy), and patient feedback via a patient questionnaire on physical activity levels and sense of protection while using florio HAEMO, will be analysed as exploratory objectives in a sub-study. This sub-study will explore possible correlations between the levels of physical activity, the estimated FVIII/FIX levels and bleeding occurrence.REC name
HSC REC A
REC reference
23/NI/0037
Date of REC Opinion
14 Sep 2023
REC opinion
Further Information Favourable Opinion