Efanesoctocog Alfa for Hemophilia A

(XTEND-ed Trial)

Recruiting at 185 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

261 Participants Needed

Trial Summary

What is the purpose of this trial?

Primary Objective: - To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A Secondary Objectives: * To evaluate the efficacy of BIVV001 as a prophylaxis treatment. * To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes. * To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes. * To evaluate the effect of BIVV001 prophylaxis on joint health outcomes. * To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes. * To evaluate the safety and tolerability of BIVV001 treatment. * To assess the PK of BIVV001 based on the one stage activated partial thromboplastin time (aPTT) and two-stage chromogenic FVIII activity assays (only applicable to Arm B). * To evaluate the efficacy of BIVV001 for perioperative management

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.

What data supports the effectiveness of the drug Efanesoctocog Alfa for Hemophilia A?

Efanesoctocog Alfa has been shown to provide high sustained levels of Factor VIII, which is important for blood clotting, and it was well tolerated in studies with no safety concerns. In a study, patients who received weekly doses had normal to near-normal Factor VIII levels for 3 to 4 days, which may help reduce bleeding episodes in people with Hemophilia A. ^{1 2 3 4 5}

Is Efanesoctocog Alfa safe for humans?

In a study, Efanesoctocog Alfa was given to adults with severe hemophilia A, and it was well tolerated with no safety concerns identified. Participants received multiple doses, and no issues were reported during the treatment period. ^{1 2 3 4 5}

How is the drug Efanesoctocog Alfa different from other treatments for hemophilia A?

Efanesoctocog Alfa is unique because it is a new type of factor VIII replacement therapy that extends the time the factor stays active in the body by not relying on von Willebrand factor, which usually limits how long factor VIII can work. This means it can provide longer protection against bleeding with less frequent dosing. ^{1 2 3 4 5}

Research Team

Clinical Sciences & Operations

Principal Investigator

Sanofi

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Efanesoctocog Alfa: First Approval. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Pharmacokinetics of recombinant factor VIII in adults with severe hemophilia A: fixed-sequence single-dose study of octocog alfa, rurioctocog alfa pegol, and efanesoctocog alfa. [2023]

3. United States pubmed.ncbi.nlm.nih.gov

Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A. [2023]

4. England pubmed.ncbi.nlm.nih.gov

Efanesoctocog alfa elicits functional clot formation that is indistinguishable to that of recombinant factor VIII. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Efanesoctocog alfa for hemophilia A: results from a phase 1 repeat-dose study. [2022]