Your session is about to expire
← Back to Search
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.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up month 48
Awards & highlights
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 belowSelect...
I have severe hemophilia A with less than 1% FVIII activity.
Select...
I have been treated for hemophilia A with specific medications for a required number of days.
Select...
I am male.
XTEND-ed Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ month 48
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~month 48
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 moreSide effects data
From 2022 Phase 3 trial • 159 Patients • NCT0416149520%
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
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
862 Previous Clinical Trials
2,019,578 Total Patients Enrolled
9 Trials studying Hemophilia A
937 Patients Enrolled for Hemophilia A
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am scheduled for major surgery within 6 months after starting the trial.I have had a positive test for FVIII inhibitors or signs of not responding well to FVIII treatments.I have severe hemophilia A with less than 1% FVIII activity.I haven't taken more NSAIDs than recommended in the last 2 weeks.I haven't had chemotherapy or immunosuppressive drugs in the last 12 weeks, except for HCV or HIV treatment.I have been treated for hemophilia A with specific medications for a required number of days.I have not had a serious infection in the last 30 days.I have a blood clotting disorder in addition to hemophilia A.I am considering starting treatment with BIVV001.I am male.I have used Emicizumab in the last 20 weeks.I have not had major surgery in the last 8 weeks.I do not have any serious liver conditions that would prevent me from joining.I haven't taken aspirin or non-NSAID blood thinners in the last 2 weeks.You have experienced severe allergic reactions or anaphylaxis from any FVIII product in the past.
Research Study Groups:
This trial has the following groups:- Group 1: Arm B: Newly initiated (China Only) in BIVV001
- Group 2: Arm A: Previously treated in BIVV001 study
- Group 3: Arm C: Newly initiated in BIVV001 with planned major surgery
Awards:
This trial has 2 awards, including:- 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.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger