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Recombinant Factor VIII

Efanesoctocog Alfa for Hemophilia A (XTEND-ed Trial)

Phase 3

Waitlist Available

Research Sponsored by Bioverativ, a Sanofi company

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participants who have severe hemophilia A, defined as <1 IU/dL (<1%) endogenous FVIII activity as documented either by central laboratory testing at screening or in historical medical records from a clinical laboratory demonstrating <1% FVIII coagulant activity (FVIII:C) or a documented genotype known to produce severe hemophilia A.

Previous treatment for hemophilia A (prophylaxis or on-demand) with any recombinant and/or plasma-derived FVIII, or cryoprecipitate for at least 150 EDs or 50 EDs for participants aged <6 years.

Must not have

Treatment with NSAIDs greater than the maximum dose specified in the regional prescribing information within 2 weeks prior to screening.

Other known coagulation disorder(s) in addition to hemophilia A.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up month 48

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is looking at the long-term safety of a drug called BIVV001 in people with hemophilia A. It will also look at how well the drug works to prevent and treat bleeding episodes, and the drug's effect on joint health and quality of life.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects are not listed here, common risks may include reactions at the injection site, potential allergic reactions to the medication components, increased risk of developing antibodies against the treatment (inhibitors), which could reduce its effectiveness.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have severe hemophilia A with less than 1% FVIII activity.

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I have been treated for hemophilia A with specific medications for a required number of days.

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I am male.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I haven't taken more NSAIDs than recommended in the last 2 weeks.

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I have a blood clotting disorder in addition to hemophilia A.

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I have not had major surgery in the last 8 weeks.

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I haven't taken aspirin or non-NSAID blood thinners in the last 2 weeks.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ month 48

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~month 48

This trial's timeline: 3 weeks for screening, Varies for treatment, and month 48 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of participants with the occurrence of inhibitor development (neutralizing antibodies detected against factor VIII [FVIII])

Secondary study objectives

Annual bleeding rate (ABR)

Annualized bleeding rate (ABR) by location

Annualized bleeding rate (ABR) by type of bleed

+12 more

Side effects data

From 2022 Phase 3 trial • 159 Patients • NCT04161495

20%

Headache

19%

Arthralgia

Fall

Back Pain

Fatigue

Haemophilic Arthropathy

Cd4 Lymphocytes Decreased

Mobility Decreased

Ulnar Tunnel Syndrome

Basal Cell Carcinoma

Combined Tibia-Fibula Fracture

Arthropathy

Blood Glucose Increased

Cubital Tunnel Syndrome

Status Epilepticus

Angina Pectoris

Traumatic Haemorrhage

Central Venous Catheter Removal

Device Breakage

100%

80%

60%

40%

20%

Study treatment Arm

Arm A: Prophylaxis

Arm B: On-demand

Arm B: Prophylaxis

BIVV001 (Efanesoctocog Alfa): All Participants

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups

Experimental Treatment

Group I: Arm C: Newly initiated in BIVV001 with planned major surgeryExperimental Treatment1 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 BIVV001Experimental Treatment1 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 studyExperimental Treatment1 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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

efanesoctocog alfa (BIVV001)

2021

Completed Phase 3

~240

0 / 7Eligibility criteria met