Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa

Launched by SWEDISH ORPHAN BIOVITRUM · Apr 16, 2025

Keywords

ClinConnect Summary

This clinical trial is studying the long-term effects of a treatment called efanesoctocog alfa in people with Hemophilia A, a condition that affects the blood's ability to clot. The goal is to see how well this treatment works in preventing joint bleeds when used in everyday life. Researchers want to gather information about how effective this treatment is over time, helping to improve the management of hemophilia for patients.

To participate in this study, you need to be diagnosed with Hemophilia A and have been receiving prophylactic treatment (preventative treatment) for at least a year before starting efanesoctocog alfa. You should also be willing to keep track of any bleeding episodes and attend regular study visits, including assessments of your joint health. This trial is still in the planning stage and hasn't started recruiting participants yet, so if you're interested, keep an eye out for updates!

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Male or female patient with a diagnosis of haemophilia A.
• • 2. 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.
• • 3. 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.
• • 4. Must have received prophylactic treatment(s) with any haemophilia product(s) for at least 12 months prior to being prescribed with efanesoctocog alfa treatment.
• • 5. 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.
• • 6. Willing to adhere to the visit schedule and to undergo mandatory study assessment (HJHS, PROs).
• • 7. Willingness and the ability of the patient or their legally designated representative to document all bleeding episodes, including any treatments for those episodes.
• • 8. For HEAD-US subgroup: Accept to undergo annual ultrasound assessment on the index joints (ankles, elbows, knees).
• Exclusion Criteria:
• • 1. Acquired haemophilia A and other blood clotting disorders than hereditary haemophilia A.
• • 2. 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.
• • 3. 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.
• • 4. 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.