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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
Male or Female For participants new to BIVV001 (Arm B and C)
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.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up month 48
Awards & highlights
No Placebo-Only Group
Pivotal Trial

XTEND-ed 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.Check my eligibility
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.See study design
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.

XTEND-ed Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am considering starting treatment with BIVV001.
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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.
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I am scheduled for major surgery within 6 months after starting the trial.

XTEND-ed Trial Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of participants with the occurrence of inhibitor development (neutralizing antibodies detected against factor VIII [FVIII])
Secondary outcome measures
Annual bleeding rate (ABR)
Annualized bleeding rate (ABR) by location
Annualized bleeding rate (ABR) by type of bleed
+30 more

Side effects data

From 2022 Phase 3 trial • 159 Patients • NCT04161495
4%
Headache
100%
80%
60%
40%
20%
0%
Study treatment Arm
Arm B: Prophylaxis
BIVV001 (Efanesoctocog Alfa): All Participants
Arm A: Prophylaxis
Arm B: On-demand

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.

XTEND-ed 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 into arm A.
Group III: Arm A: Previously treated in BIVV001 studyExperimental Treatment1 Intervention
This arm includes all participants who have completed the previous phase 3 studies on BIVV001, as well as participants who have completed Arm B or Arm C of this study rolling over in Arm A, and participants who will have completed any future BIVV001 study who will be proposed to continue BIVV001 treatment. 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

Find a Location

Who is running the clinical trial?

Bioverativ, a Sanofi companyLead Sponsor
17 Previous Clinical Trials
775 Total Patients Enrolled
6 Trials studying Hemophilia A
406 Patients Enrolled for Hemophilia A
Clinical Sciences & OperationsStudy DirectorSanofi
855 Previous Clinical Trials
2,019,986 Total Patients Enrolled
9 Trials studying Hemophilia A
937 Patients Enrolled for Hemophilia A

Media Library

Efanesoctocog Alfa (BIVV001) (Recombinant Factor VIII) Clinical Trial Eligibility Overview. Trial Name: NCT04644575 — Phase 3
Hemophilia A Research Study Groups: Arm C: Newly initiated in BIVV001 with planned major surgery, Arm A: Previously treated in BIVV001 study, Arm B: Newly initiated (China Only) in BIVV001
Hemophilia A Clinical Trial 2023: Efanesoctocog Alfa (BIVV001) Highlights & Side Effects. Trial Name: NCT04644575 — Phase 3
Efanesoctocog Alfa (BIVV001) (Recombinant Factor VIII) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04644575 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Have similar research projects been conducted in the past?

"3 clinical trials involving efanesoctocog alfa (BIVV001) are ongoing as of now in 23 different countries and 41 cities. The first one was sponsored by Bioverativ, a subsidiary of Sanofi, and completed its Phase 3 in 2021. It had 75 participants."

Answered by AI

What is the most recent update on efanesoctocog alfa's (BIVV001) FDA status?

"There is some efficacy data and extensive safety data from multiple rounds of testing, thus our team has given efanesoctocog alfa (BIVV001) a score of 3."

Answered by AI

Are there precedents for BI VV001's efanesoctocog alfa in human trials?

"Efanesoctocog alfa (BIVV001) is the focus of 3 live clinical trials, 2 of which are in Phase 3. Although a handful of the efanesoctocog alfa (BIVV001) trials are located in Leuven, Illinois, there are a total of 171 research sites for this medication."

Answered by AI
~127 spots leftby Jan 2027