Efanesoctocog Alfa for Hemophilia A

(SYNOVIIIUS Trial)

Recruiting at 12 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 4

Sponsor: Sanofi

Must be taking: Hemophilia prophylaxis

Must not be taking: Efanesoctocog alfa

Disqualifiers: Other clotting disorders, FVIII inhibitor, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

35 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called efanesoctocog alfa, administered once a week to assist people with hemophilia A, a condition where blood doesn't clot properly. The goal is to determine if this treatment can reduce joint swelling caused by synovial hypertrophy (a thickening of the joint lining) over 12 months. Individuals with moderate to severe hemophilia A who have experienced joint swelling might be suitable candidates for this study. As a Phase 4 trial, this research involves an FDA-approved treatment and aims to understand how it can benefit more patients.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, it does mention that participants should not be on efanesoctocog alfa treatment already. It's best to discuss your current medications with the study team.

What is the safety track record for Efanesoctocog Alfa?

Research has shown that efanesoctocog alfa is generally safe for people with hemophilia A. In one study, patients received four weekly doses, and researchers found no safety issues. Notably, there were no bleeding incidents during the treatment. Another study confirmed these results, indicating that efanesoctocog alfa effectively prevents bleeding.

This treatment has already received approval, demonstrating its safety. The current trial is in a late stage, suggesting strong evidence of its safety in humans. Overall, efanesoctocog alfa appears to be a safe choice for managing hemophilia A. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Efanesoctocog Alfa is unique because it offers a potentially longer-lasting treatment option for Hemophilia A. Unlike standard treatments that often require more frequent infusions, Efanesoctocog Alfa is administered once weekly, potentially improving convenience and adherence for patients. Researchers are excited because it uses a novel design to extend the half-life of factor VIII, which could lead to more stable and sustained protection against bleeding episodes. This advancement could significantly enhance the quality of life for those with Hemophilia A by reducing the treatment burden.

What is the effectiveness track record for Efanesoctocog Alfa in treating hemophilia A?

Research has shown that efanesoctocog alfa, the treatment under study in this trial, effectively treats hemophilia A by boosting factor VIII, which is crucial for blood clotting. Studies have found that 88.5% of bleeding episodes stopped with just one dose of this treatment. It is recommended for preventing and treating bleeding in individuals with hemophilia A, ages 2 and up. As a first-in-class therapy, efanesoctocog alfa works in a new or unique way compared to other treatments. It has demonstrated lasting benefits, making it an effective option for managing the condition. ^{1 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for individuals aged 12 or older with moderate to severe hemophilia A and existing synovial hypertrophy, which means they have swelling in their joints. They should have been on prophylactic treatment for the last year and not planning any major joint procedures. Women who can bear children must agree to use effective contraception.

Inclusion Criteria

I have moderate to severe hemophilia A.

I have been on hemophilia prevention treatment in the past year.

I am 12 years old or older.

Exclusion Criteria

Participant has other associated clotting disorders at the time of consent/assent

Participant has been enrolled in a concurrent clinical interventional study or exposed to other investigational drugs within 3 months prior to screening for this study

Participant is currently in an institution due to regulatory or legal order

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Efanesoctocog Alfa prophylaxis once per week to assess improvement of synovial hypertrophy

52 weeks

Weekly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Efanesoctocog Alfa

Trial Overview The trial tests efanesoctocog alfa, a medication given weekly to see if it improves joint swelling in patients with hemophilia A over a period of 12 months. The focus is on whether this treatment can reduce the thickness of the synovium (joint lining) that's often swollen due to bleeding.

How Is the Trial Designed?

Group I: Efanesoctocog Alfa Experimental Treatment 1 Intervention

Participants will receive Efanesoctocog Alfa at 50 international unit per kilogram (IU/kg) intravenous (IV) QW

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

Citations

1. nejm.org nejm.org/doi/full/10.1056/NEJMoa2209226

Efanesoctocog Alfa Prophylaxis for Patients with Severe ... Efanesoctocog alfa provides high sustained factor VIII activity by overcoming the von Willebrand factor–imposed half-life ceiling.

2. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/5495/527825/Clinical-Outcomes-over-2-Years-of-Once-Weekly

Clinical Outcomes over 2 Years of Once-Weekly ... Of 61 bleeds, 54 (88.5%) resolved with a single dose of efanesoctocog alfa 50 IU/kg, with the hemostatic response rated as excellent or good for ...

3. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK615900

Efanesoctocog alfa for treating and preventing bleeding ... Efanesoctocog alfa is recommended as an option for treating and preventing bleeding episodes in people 2 years and over with haemophilia A (congenital factor ...

4. link.springer.com link.springer.com/article/10.1007/s40273-025-01556-2

Cost Effectiveness of Efanesoctocog Alfa Versus Factor VIII ... Efanesoctocog alfa is a first-in-class high-sustained factor VIII therapy approved for prophylaxis, on-demand treatment, and peri-operative ...

5. hematologyadvisor.com hematologyadvisor.com/news/efanesoctocog-alfa-not-cost-effective-for-prophylaxis-in-hemophilia-a/

Efanesoctocog Alfa Not Cost-Effective for Prophylaxis in ... The researchers found that efanesoctocog alfa and standard-care factor VIII accrued 19.7 and 14.6 discounted lifetime quality-adjusted life ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04644575

Study Details | NCT04644575 | Long-term Safety and ... Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A (XTEND-ed). ClinicalTrials.gov ID NCT04644575.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8864644/

Efanesoctocog alfa for hemophilia A: results from a phase 1 ... 4 once-weekly doses of efanesoctocog alfa were well tolerated, no safety concerns were identified, and no bleeds were reported during the treatment period.

8. sanofi.com sanofi.com/en/media-room/press-releases/2023/2023-01-25-22-30-00-2595681

NEJM publishes once-weekly efanesoctocog alfa Phase 3 ... These data demonstrate that efanesoctocog alfa delivered normal to near-normal factor activity levels (>40%) for the majority of the week with once-weekly ...

9. sobi.com sobi.com/en/press-releases/late-breaking-efanesoctocog-alfa-data-presented-isth-demonstrates-highly-effective-bleed-protection-children-severe-haemophilia-once

Late-breaking efanesoctocog alfa data presented at ISTH ... Late-breaking efanesoctocog alfa data presented at ISTH demonstrates highly effective bleed protection in children with severe haemophilia A ...