Investigating the effects of efanesoctocog alfa on joint health in haemophilia A patients

A 12-month, Interventional, Open-label, Phase 4 Study in Europe (SHINE) to Investigate the Course of Synovial Hypertrophy as Detected by Joint Ultrasound and MRI in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis.

This study is testing if a new weekly treatment called efanesoctocog alfa can help improve joint health and reduce bleeding in people aged 12 and older with moderate or severe hemophilia A.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 35 (estimated)
• Ages: 12 Years and up
• Sex: All
• Sponsor: Swedish Orphan Biovitrum (Industry-sponsored)
• Drugs / interventions: emicizumab
• Locations: 10 sites (Milan and 9 other locations)
• Trial ID: NCT06752850 on ClinicalTrials.gov

What this trial studies

This open-label phase 4 study aims to evaluate the impact of once-weekly prophylaxis with efanesoctocog alfa on synovial hypertrophy and joint bleeding risk in patients aged 12 and older with moderate or severe haemophilia A. The study will involve a 12-month duration with imaging assessments to objectively monitor changes in synovial hypertrophy. Patients will undergo ultrasound examinations to confirm eligibility based on existing joint conditions, and their treatment history will be reviewed. The study is designed to provide insights into the effectiveness of this treatment regimen in improving joint health.

Who should consider this trial

Good fit: Ideal candidates are males and females aged 12 and older diagnosed with moderate or severe haemophilia A who have existing synovial hypertrophy.

Not a fit: Patients with mild haemophilia A or those without existing synovial hypertrophy may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved joint health and reduced bleeding episodes for patients with haemophilia A.

How similar studies have performed: Previous studies have shown positive effects of efanesoctocog alfa on joint health, indicating a promising approach.

Eligibility criteria

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.

Where this trial is running

Milan and 9 other locations

• Sobi Investigational Site — Milan, Italy (Recruiting)
• Sobi Investigational Site — Naples, Italy (Recruiting)
• Sobi Investigational Site — Rozzano, Italy (Not_yet_recruiting)
• Sobi Investigational Site — Oslo, Norway (Recruiting)
• Sobi Investigational Site — Barcelona, Spain (Recruiting)
• Sobi Investigational Site — Madrid, Spain (Recruiting)
• Sobi Investigational Site — Seville, Spain (Recruiting)
• Sobi Investigational Site — Valencia, Spain (Not_yet_recruiting)
• Sobi Investigational Site — Gothenburg, Sweden (Recruiting)
• Sobi Investigational Site — Malmö, Sweden (Recruiting)

Study contacts

• Study coordinator: Study Physician

Conditions Haemophilia A; Hemophilia, Classic; Haemophilia; Hemophilia; Classic Hemophilia; Synovial Hypertrophy; Deficiency, Factor VIII; Factor 8 Deficiency, Congenital