Efanesoctocog Alfa for Hemophilia A

(XTEND-ed Trial)

Not currently recruiting at 197 trial locations

Overseen By Trial Transparency email recommended (Toll free number for US & Canada)

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Bioverativ, a Sanofi company

Must not be taking: Aspirin, Antiplatelets, NSAIDs, Emicizumab

Disqualifiers: Liver disease, Infections, Coagulation disorders, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called Efanesoctocog Alfa, designed to assist people with hemophilia A, a condition affecting blood clotting. Researchers aim to determine if this treatment is safe for long-term use and effective in preventing and treating bleeding episodes. Participants will also help assess whether the treatment can improve joint health and overall quality of life. Individuals with severe hemophilia A who have received prior treatment may be suitable for this trial, particularly if they experience frequent bleeding issues impacting their daily lives. As a Phase 3 trial, this represents the final step before FDA approval, offering participants an opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, such as acetylsalicylic acid (ASA) or antiplatelet agents, within 2 weeks before screening. You also need to stop using NSAIDs above the maximum dose specified in regional guidelines within 2 weeks before screening, and you cannot have used Emicizumab within 20 weeks before screening.

Is there any evidence suggesting that this trial's treatment is likely to be safe?

Research has shown that efanesoctocog alfa is generally safe for people. In one study, patients who received four weekly doses experienced no safety issues and, importantly, had no bleeding during treatment. Other studies have found that this treatment maintains good levels of clotting factors in the blood for most of the week, which is crucial for managing hemophilia A.

Although this treatment remains under study, these results suggest it is likely safe. However, participating in a clinical trial involves potential unknowns, as researchers continue to learn about the treatment's long-term effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for hemophilia A?

Efanesoctocog Alfa is unique because it offers a potentially improved prophylactic treatment for Hemophilia A by extending the duration of protection with less frequent dosing. Unlike standard treatments like factor VIII replacement therapies that require multiple infusions each week, Efanesoctocog Alfa is designed for once-weekly administration. Researchers are excited about this treatment because it could enhance convenience and adherence for patients, while still effectively preventing bleeding episodes. Its innovative design aims to maintain higher factor levels in the blood for longer periods, which is a promising advancement for those living with Hemophilia A.

What evidence suggests that this trial's treatment could be effective for hemophilia A?

Research has shown that efanesoctocog alfa, also known as BIVV001, offers promising results for people with hemophilia A. In studies, weekly doses of efanesoctocog alfa provided better protection against bleeding than older treatments. Patients tolerated it well, experiencing no major side effects. Early studies reported no bleeding during treatment. The treatment also improved joint health and overall quality of life. This trial will evaluate efanesoctocog alfa in different contexts: Arm A includes participants previously treated with BIVV001, Arm B involves newly initiated participants in China, and Arm C includes those newly initiated with planned major surgery. This evidence supports its potential effectiveness for managing hemophilia A. ^{2 3 6 7 8}

Who Is on the Research Team?

Clinical Sciences & Operations

Principal Investigator

Sanofi

Are You a Good Fit for This Trial?

This trial is for males and females with severe hemophilia A, previously treated with FVIII products. Participants must have a history of at least 150 exposure days to such treatments (50 for those under 6 years). They should not have other coagulation disorders or recent major surgery, active infections, or positive tests for inhibitors to FVIII. HIV-positive individuals can join if they meet certain health criteria.

Inclusion Criteria

I am scheduled for major surgery within 6 months after starting the trial.

For participants rolling over into Arm A: Participants who have completed the studies EFC16923, EFC16925, Arm B or Arm C of the current study, or any other potential BIVV001 study.

A participant known to be human immunodeficiency virus (HIV) antibody positive, either previously documented or identified from screening assessments, must have the following results prior to enrollment: CD4 lymphocyte count >200 cells/mm³ and viral load of <400 000 copies/mL.

Exclusion Criteria

Participation in another study. For participants new to BIVV001 (Arm B and Arm C)

I have had a positive test for FVIII inhibitors or signs of not responding well to FVIII treatments.

For participants rolling over into Arm A: Positive inhibitor result, defined as ≥0.6 Bethesda units (BU)/mL.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive BIVV001 prophylaxis treatment once weekly for a total of at least 100 exposure days

52 weeks

Weekly visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

48 months

Open-label extension

Participants may continue in the study for up to 4 years unless BIVV001 is commercially available

Up to 4 years

What Are the Treatments Tested in This Trial?

Interventions

• Efanesoctocog Alfa (BIVV001)

Trial Overview The trial is testing the long-term safety and effectiveness of efanesoctocog alfa (BIVV001) in people who've been treated before for hemophilia A. It looks at how well BIVV001 prevents bleeding episodes, manages joint health, improves quality of life, and works during surgery.

How Is the Trial Designed?

Group I: Arm C: Newly initiated in BIVV001 with planned major surgery Experimental Treatment 1 Intervention

This arm includes participants of any age who will be newly initiated on BIVV001 prophylaxis treatment once-weekly (QW) and will undergo planned major surgery after at least 6 initial EDs with BIVV001, and within 26 weeks from Day 1. After 52 weeks of treatment in arm C, participants will be able to roll into arm A.

Group II: Arm B: Newly initiated (China Only) in BIVV001 Experimental Treatment 1 Intervention

This arm includes Chinese participants of any age who will be newly initiated on BIVV001 prophylaxis treatment once-weekly (QW) for 52 weeks. After 52 weeks of treatment in this arm B, participants will be able to roll over into arm A.

Group III: Arm A: Previously treated in BIVV001 study Experimental Treatment 1 Intervention

This arm includes participants who have completed study EFC16293 or study EFC16295, participants who have completed Arm B or Arm C of this study (LTS16294) and roll over into Arm A, and participants who have completed any other potential BIVV001 study. Participants in this arm will continue receiving BIVV001 prophylaxis treatment once weekly (QW) for a total of 100 exposure days (EDs) cumulative from the parent study and this study. Participants will have the opportunity to continue in this study for up to 4 years, unless BIVV001 is commercially available in their applicable participating country.

Who Is Running the Clinical Trial?

Bioverativ, a Sanofi company

Lead Sponsor

Trials

18

Recruited

1,000+

Citations

1. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/717/530808/Clinical-Outcomes-over-3-Years-of-Once-Weekly

Clinical Outcomes over 3 Years of Once-Weekly ... Once-weekly efanesoctocog alfa demonstrated superior bleed protection over prior FVIII prophylaxis, was well tolerated, and provided FVIII activity within the ...

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

Efanesoctocog Alfa Prophylaxis for Patients with Severe ... In patients with severe hemophilia A, once-weekly efanesoctocog alfa provided superior bleeding prevention to prestudy prophylaxis, normal to near-normal ...

3. 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.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT04161495

NCT04161495 | A Phase 3 Open-label Interventional Study ... Patient Experience With Efanesoctocog Alfa for Severe Hemophilia A: Results From the XTEND-1 Phase 3 Clinical Study Exit Interviews. Clin Ther. 2024 Dec;46 ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0006497124034645

Clinical Outcomes over 3 Years of Once-Weekly ... Long-term results in adults and adolescents in XTEND-ed shows that once-weekly efanesoctocog alfa continues to provide high efficacy and is well tolerated.

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. 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 ...

8. medchemexpress.com medchemexpress.com/efanesoctocog-alfa.html?srsltid=AfmBOoo9aQ2GyztPIiv8gMSsdP4LLPVl76MLeKhN3F9S54HpfA9CE9A5

Efanesoctocog alfa (BIVV-001) | Fusion Protein Efanesoctocog alfa can be used for the study of inherited hemophilia A. For ... SAFETY DATA SHEET (SDS). Request for HNMR Report. We have received your ...