efanesoctocog alfa (BIVV001) for Hemophilia A

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Hemophilia Aefanesoctocog alfa (BIVV001) - Drug
Eligibility
Any Age
All Sexes
What conditions do you have?
Select

Study 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.

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

1 Primary · 33 Secondary · Reporting Duration: Month 48

Baseline to month 48
Annual bleeding rate (ABR)
Annualized bleeding rate (ABR) by location
Annualized bleeding rate (ABR) by type of bleed
Annualized joint bleeding rate (AJBR)
Assessment of response to BIVV001 treatment of individual bleeding episodes
Change from baselin in Haemo-QoL total score and physical health score
Change from baseline in Haem-A-QoL total score and physical health score
Change from baseline in Hemophilia Joint Health Score (HJHS)
Change from baseline in PROMIS-SF Physical Function
Estimated blood loss during perioperative period for major surgery
Investigators' or Surgeons' assessment of participant's hemostatic response to BIVV001 treatment
Number and type of blood component transfusions used during perioperative period for major surgery
Number of injections and dose to maintain hemostasis during perioperative period for major surgery
Number of participants with adverse events (AEs) and serious adverse events (SAEs)
Number of participants with the occurrence of embolic and thrombotic events
Number of participants with the occurrence of inhibitor development (neutralizing antibodies detected against factor VIII [FVIII])
Percentage of patients who maintain factor VIII (FVIII) above prespecified activity levels
Physician's global assessment (PGA) of participants response to BIVV001
Total BIVV001 consumption during perioperative period for major surgery
Total annualized BIVV001 consumption
Baseline to week 26
PK parameter: Accumulation index (AI)
PK parameter: Area under the activity time curve (AUC)
PK parameter: Elimination half-life (t1/2)
PK parameter: Mean residence time (MRT)
PK parameter: Time above FVIII activity levels
PK parameter: Total clearance (CL)
PK parameter: Total clearance at steady state (CLss)
PK parameter: Volume of distribution at steady state (Vss)
Baseline to week 52
PK parameter: Incremental recovery (IR)
PK parameter: Maximum activity (Cmax)
PK parameter: Trough activity (Ctrough)
Month 48
Number of injections and dose of BIVV0001 to treat a bleeding episode
Percentage of bleeding episode treated with a single injection of BIVV001
Target joint resolution

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Trial Design

3 Treatment Groups

Arm A: Previously treated in BIVV001 study
1 of 3
Arm C: Newly initiated in BIVV001 with planned major surgery
1 of 3
Arm B: Newly initiated (China Only) in BIVV001
1 of 3

Experimental Treatment

262 Total Participants · 3 Treatment Groups

Primary Treatment: efanesoctocog alfa (BIVV001) · No Placebo Group · Phase 3

Arm A: Previously treated in BIVV001 study
Drug
Experimental Group · 1 Intervention: efanesoctocog alfa (BIVV001) · Intervention Types: Drug
Arm C: Newly initiated in BIVV001 with planned major surgery
Drug
Experimental Group · 1 Intervention: efanesoctocog alfa (BIVV001) · Intervention Types: Drug
Arm B: Newly initiated (China Only) in BIVV001
Drug
Experimental Group · 1 Intervention: efanesoctocog alfa (BIVV001) · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Efanesoctocog alfa
Not yet FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: month 48

Who is running the clinical trial?

Bioverativ, a Sanofi companyLead Sponsor
17 Previous Clinical Trials
767 Total Patients Enrolled
6 Trials studying Hemophilia A
407 Patients Enrolled for Hemophilia A
Clinical Sciences & OperationsStudy DirectorSanofi
800 Previous Clinical Trials
1,633,919 Total Patients Enrolled
7 Trials studying Hemophilia A
792 Patients Enrolled for Hemophilia A

Eligibility Criteria

Age Any Age · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
People who have severe hemophilia A, meaning they have less than 1 international unit (IU) of clotting Factor VIII circulating in their blood, as documented by a central laboratory or in their medical records
This study is for people who have never taken part in BIVV001 before
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 suppressed to <400 copies/mL.
People with hemophilia A have traditionally been treated with prophylaxis or on-demand therapy with recombinant or plasma-derived factor VIII (FVIII) products
, we found that the median age was For participants rolling over into Arm A, the median age was 42.
People who have finished studies EFC16923, EFC16925, Arm B or Arm C of the current study, or any other potential BIVV001 study.
You are male.\n
The patient's platelet count was ≥100 000 cells/μL at screening.
Arm C: planned major surgery within 6 months after Day 1.
You are a Chinese citizen.