Efanesoctocog alfa: 50 IU/kg once weekly, Intravenous injection
Study summary
The rationale for conducting this open-label phase 4 study is to assess whether once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) improves the disease course of existing synovial hypertrophy and prevents the risk of joint bleeds in patients with moderate or severe haemophilia A. The use of imaging assessments will allow for objective detection and monitoring of synovial hypertrophy, and thus expand on the previous findings demonstrating positive effects of once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) on joint health.
Eligibility
Sex
ALL
Min age
12 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
1. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. Parents' or legally designated representatives' consent is required for patients who are \<18 years of age or unable to give consent, or as applicable per local laws. Patients who are \<18 years of age should provide assent in addition to the parents'/legally designated representatives' consent, if appropriate.
2. Male or female patients who are ≥12 years of age and diagnosed with moderate or severe haemophilia A (defined as ≤5% of normal FVIII clotting activity) at the time of signing the ICF.
3. A female patient is eligible to participate if she is not pregnant at enrolment and does not plan to become pregnant during the study. A woman of child-bearing potential (WOCBP) must have a negative highly sensitive serum pregnancy test at the Screening Visit.
4. Must have received prophylactic treatment per local label with any marketed FVIII product or emicizumab for ≥12 months prior to the Baseline Visit.
5. Have at least one eligible index joint (ankle, elbow, knee).
6. Have 12 months of documented pre-study treatment data on haemophilia prescriptions and on treated bleeding episodes prior to the Baseline Visit.
7. Willingness and the ability of the patient or their legally designated representative to complete training in the use of the study patient diary and to complete the diary throughout the study.
Exclusion Criteria:
1. Blood clotting disorders other than haemophilia A
2. Already on efanesoctocog alfa treatment
3. Positive inhibitor result (assessed by local laboratory) from the Screening Visit, defined as ≥0.6 Bethesda units (BU)/mL.
4. History of inhibitors without successful immune tolerance induction (ITI)
* Successful ITI is defined as:
* Negative inhibitor titer (\<0.6 BU/mL)
* FVIII recovery \> 66% of expected
* FVIII half-life ≥ 6 hours
5. ITI performed within the last 2 years prior to the Baseline Visit.
6. Currently receiving treatment with any of the prohibited concomitant medications, as specified by the protocol.
7. Planned major orthopaedic procedure in any eligible index joint during the course of the study.
8. Patients are not eligible for participation in the study if they cannot undergo MRI assessments at the Baseline Visit.
9. Patients with known hypersensitivity to the active substance or to any of the excipients.
10. Patient not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.
11. Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), within 3 months prior to inclusion in the study.
Primary outcome measure(s)
Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovial hypertrophy domain score decrease. — Baseline to 12 months At least 1 point decrease in HEAD-US synovial hypertrophy domain score at Month 12 (for joints with a domain score of 1 or 2 at baseline).
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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