Brief Title
Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A
Official Title
A Phase 3 Open-label, Multicenter Study of the Long-term Safety and Efficacy of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients With Severe Hemophilia A
Brief 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
Detailed 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.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Number of participants with the occurrence of inhibitor development (neutralizing antibodies detected against factor VIII [FVIII])
Secondary Outcome
Annual bleeding rate (ABR)
Condition
Hemophilia A
Intervention
efanesoctocog alfa (BIVV001)
Study Arms / Comparison Groups
Arm A: Previously treated in BIVV001 study
Description: 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.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
261
Start Date
February 23, 2021
Completion Date
February 12, 2027
Primary Completion Date
February 12, 2027
Eligibility Criteria
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.
Gender
All
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
Clinical Sciences & Operations, ,
Location Countries
Argentina
Location Countries
Argentina
Administrative Informations
NCT ID
NCT04644575
Organization ID
LTS16294
Secondary IDs
2020-002215-22
Responsible Party
Sponsor
Study Sponsor
Bioverativ, a Sanofi company
Study Sponsor
Clinical Sciences & Operations, Study Director, Sanofi
Verification Date
March 13, 2023