Status:
RECRUITING
Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa
Lead Sponsor:
Swedish Orphan Biovitrum
Collaborating Sponsors:
PSI CRO
Conditions:
Hemophilia A
Eligibility:
All Genders
Phase:
PHASE4
Brief Summary
The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.
Detailed Description
This is a retrospective-prospective, low-interventional study to evaluate the long-term effectiveness of real-world prophylaxis with efanesoctocog alfa on joint health in people with haemophilia A. Pa...
Eligibility Criteria
Inclusion
- Male or female patient with a diagnosis of haemophilia A.
- Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
- Prescribed with efanesoctocog alfa prophylactic treatment within 6 months prior to study enrolment or at the latest at the enrolment visit, in accordance with local regulations.
- Must have received prophylactic treatment(s) with any haemophilia product(s) for at least 12 months prior to being prescribed with efanesoctocog alfa treatment.
- Have documented pre-study treatment data on haemophilia prophylaxis prescriptions and on treated bleeding episodes from the 12-months prior to being prescribed efanesoctocog alfa treatment and until enrolment into this study.
- Willing to adhere to the visit schedule and to undergo mandatory study assessment (HJHS, PROs).
- Willingness and the ability of the patient or their legally designated representative to document all bleeding episodes, including any treatments for those episodes.
- For HEAD-US subgroup: Accept to undergo annual ultrasound assessment on the index joints (ankles, elbows, knees).
Exclusion
- Acquired haemophilia A and other blood clotting disorders than hereditary haemophilia A.
- Any positive FVIII inhibitor result (defined as inhibitor titre ≥0.6 Bethesda unit \[BU\]/mL) from the medical records in connection to the switch to efanesoctocog alfa until the enrolment visit.
- Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), including for haemophilia prophylaxis, within 3 months prior to enrolment in this study.
- Patient not suitable for participation, whatever the reason, as judged by the Investigator, e.g., patient is not able or willing to perform the study assessments.
Key Trial Info
Start Date :
May 21 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2028
Estimated Enrollment :
250 Patients enrolled
Trial Details
Trial ID
NCT06940830
Start Date
May 21 2025
End Date
December 1 2028
Last Update
December 17 2025
Active Locations (24)
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1
Sobi Investigational Site
Zagreb, Croatia
2
Sobi Investigational Site
Brno, Czechia
3
Sobi Investigational Site
Ostrava, Czechia
4
Sobi Investigational Site
Prague, Czechia