A Phase 3, Open-label Interventional Study of an Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A

• Status: Active, not recruiting
• Trial end date: 2022-01-25
• Target enrollment: 0
• Participant gender: All

Summary

Primary Objective:

• To evaluate the efficacy of BIVV001 as a prophylaxis treatment in prophylaxis treatment arm.

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 the 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 efficacy of BIVV001 for perioperative management

• To evaluate the safety and tolerability of BIVV001 treatment

• To assess the pharmacokinetics (PK) of BIVV001 based on the 1-stage activated partial thromboplastin time (aPTT) and 2-stage chromogenic FVIII activity assays

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bioverativ, a Sanofi company; Sanofi
• Treatments: Factor VIII

Criteria

Inclusion criteria :

• Participant, male or female, must be equal to or greater than 12 years of age inclusive, at the time of signing the informed consent.

• 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.

• Current regimen includes one of the following:

• Prophylactic treatment regimen with a FVIII product or prophylactic emicizumab therapy for at least 6 months during the previous 12 months. Appropriate washout time needs to be taken into account.

• On-demand regimen with a FVIII product with a history of at least 12 bleeding episodes in the previous 12 months or at least 6 bleeding episodes in the previous 6 months prior to study enrollment.

• On-demand participant is accepting to move to a prophylaxis treatment regimen after 26-week on-demand period.

• Willingness and ability of the participant or surrogate (a caregiver or a family member ≥18 years of age) to complete training in the use of the study electronic Patient Diary (ePD) and to use the ePD throughout the study.

• Ability of the participant or his or her legally authorized representative (eg., parent or legal guardian) to understand the purpose and risks of the study, willing and able to comply with study requirements and provide signed and dated informed consent or assent (as applicable) and authorization to use protected health information in accordance with national and local participant privacy regulations.

Exclusion criteria:

• Clinically significant liver disease.

• Serious active bacterial 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

• Positive inhibitor results, defined as ≥0.6 BU/mL at Screening. History of a positive inhibitor test defined as ≥0.6 BU/mL. Family history of inhibitors will not exclude the participant.

• Use of Emicizumab 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.