A Phase 3 Open-label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Pediatric Patients <12 Years of Age With Severe Hemophilia A

EFC16295

Primary Objective: - To evaluate the safety of BIVV001 in previously treated pediatric participants 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 efficacy of BIVV001 for perioperative management. * To evaluate the safety and tolerability of BIVV001 treatment. * To assess the pharmacokinetics (PK) of BIVV001.

2021-02-19

2023-01-18

2023-01-18

Completed

Early phase I

74

Inclusion criteria : * Participant must be younger than 12 years of age, at the time of signing the informed consent. * Severe hemophilia A defined as \<1 international units per deciliter (IU/dL) (\<1 percent \[%\]) endogenous Factor VIII (FVIII) 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 exposure days (EDs) for participants aged 6 to \<12 years and above 50 EDs for participants aged \<6 years. * Weight above or equal to 10 kg. Exclusion criteria: * History of hypersensitivity or anaphylaxis associated with any FVIII product. * History of a positive inhibitor (to FVIII) test defined as greater than or equal to (\>=) 0.6 Bethesda units (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 would not exclude the participant. * Positive inhibitor test result, defined as \>=0.6 BU/mL at Screening. The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 74, 'type': 'ACTUAL'}}

2024-02-13