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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

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.

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 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
20%
Headache
19%
Arthralgia
8%
Fall
6%
Back Pain
5%
Fatigue
2%
Haemophilic Arthropathy
1%
Cd4 Lymphocytes Decreased
1%
Mobility Decreased
1%
Ulnar Tunnel Syndrome
1%
Basal Cell Carcinoma
1%
Combined Tibia-Fibula Fracture
1%
Arthropathy
1%
Blood Glucose Increased
1%
Cubital Tunnel Syndrome
1%
Status Epilepticus
1%
Angina Pectoris
1%
Traumatic Haemorrhage
1%
Central Venous Catheter Removal
1%
Device Breakage
100%
80%
60%
40%
20%
0%
Study treatment Arm
Arm A: Prophylaxis
Arm B: On-demand
Arm B: Prophylaxis
BIVV001 (Efanesoctocog Alfa): All Participants

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

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
866 Previous Clinical Trials
2,019,948 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 B: Newly initiated (China Only) in BIVV001, Arm A: Previously treated in BIVV001 study, Arm C: Newly initiated in BIVV001 with planned major surgery
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
~108 spots leftby Jan 2027
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