Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A (XTEND-ed)

  • STATUS
    Recruiting
  • End date
    Feb 12, 2027
  • participants needed
    262
  • sponsor
    Bioverativ, a Sanofi company
Updated on 27 May 2022

Summary

Primary Objective:

  • To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A

Secondary Objectives:

  • To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
  • To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes.
  • To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes.
  • To evaluate the effect of BIVV001 prophylaxis on joint health outcomes.
  • To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes.
  • To evaluate the safety and tolerability of BIVV001 treatment.
  • To assess the PK of BIVV001 based on the one stage activated partial thromboplastin time (aPTT) and two-stage chromogenic FVIII activity assays (only applicable to Arm B).
  • To evaluate the efficacy of BIVV001 for perioperative management

Description

Participants will receive BIVV001 once weekly for a total of at least 100 exposure days to BIVV001 (including exposure during a BIVV001 parent study, if applicable). 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.

Details
Condition Hemophilia A
Treatment BIVV001, efanesoctocog alfa (BIVV001)
Clinical Study IdentifierNCT04644575
SponsorBioverativ, a Sanofi company
Last Modified on27 May 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

For participants rolling over into Arm A
Participants who have completed the studies EFC16923, EFC16925, Arm B or Arm C of the current study, or any other potential BIVV001 study
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
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
Platelet count ≥100 000 cells/μL at screening
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 of <400 000 copies/mL
Male
Only for Arm B: Chinese participants
Only for Arm C: planned major surgery within 6 months after Day 1

Exclusion Criteria

For participants rolling over into Arm A
Positive inhibitor result, defined as ≥0.6 Bethesda units (BU)/mL
Participation in another study. For participants new to BIVV001 (Arm B and Arm C)
Any concurrent clinically significant liver disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment. This may include, but is not limited to cirrhosis, portal hypertension, and acute hepatitis
Serious active bacterial, fungal, or viral infection (other than chronic hepatitis or HIV) present within 30 days of screening
Other known coagulation disorder(s) in addition to hemophilia A
History of hypersensitivity or anaphylaxis associated with any FVIII product
History of a positive inhibitor (to FVIII) test defined as ≥0.6 BU/mL, or any value greater than or equal to the lower sensitivity cut-off for laboratories with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL, or clinical signs or symptoms of decreased response to FVIII administrations. Family history of inhibitors will not exclude the participant
Positive inhibitor test (FVIII) result, defined as ≥0.6 BU/mL at screening
Treatment with acetylsalicylic acid (ASA) or antiplatelet agents that are not nonsteroidal anti-inflammatory drugs (NSAIDs) within 2 weeks prior to screening
Treatment with NSAIDs greater than the maximum dose specified in the regional prescribing information within 2 weeks prior to screening
Systemic treatment within 12 weeks prior to Screening with chemotherapy and/or other immunosuppressive drugs (except for the treatment of hepatitis C virus [HCV] or HIV)
Emicizumab use within the 20 weeks prior to screening
Major surgery within 8 weeks prior to screening
The above information is not intended to contain all considerations relevant to a patient's
potential participation in a clinical trial
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