severe hemophilia A MedDRA version: 20.0 Level: PT Classification code 10016080 Term: Factor VIII deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Recombinant coagulation FVIII Fc – von Willebrand factor – XTEN fusion protein
Product Code: BIVV001 (rFVIIIFc-VWF-XTEN)
Pharmaceutical Form: Powder for injection
INN or Proposed INN: Ef
Sponsors
Bioverativ Therapeutics Inc. (a Sanofi company)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Participant, male or female, must be equal to or greater than 12 years of age inclusive, at the time of signing the informed consent. 2. Severe hemophilia A, defined as =65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: -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 Safety Objective - To evaluate the safety and tolerability of BIVV001 treatment Pharmacokinetic Objective - To assess the PK of BIVV001 based on the one stage activated partial thromboplastin time (aPTT) and two-stage chromogenic FVIII activity assays Exploratory Objectives - To evaluate joint-health structural outcomes via ultrasound using the JADE protocol and/or HEAD-US - To assess the impact of BIVV001 treatment on patient reported outcome (PRO) measurements and physical activity (measures per study arm and treatment regimen);Main Objective: To evaluate the efficacy of BIVV001 as a prophylaxis treatment;Primary end point(s): Primary Efficacy Endpoint: Annualized bleeding rate (ABR) in prophylaxis treatment arm;Timepoint(s) of evaluation of this end point: baseline to 52 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary Efficacy Endpoints: -Intra-patient comparison of ABR of participants of this study to the ABR of same participants previously treated in an observational study -Annualized bleeding rate (ABR) by type of bleed -Annualized bleeding rate (ABR) by location of bleed -Annualized bleeding rate (ABR) for all bleeding episodes including untreated bleeding episodes -Intra-patient comparison of ABR during the weekly once (QW) prophylaxis treatment period versus ABR during the on-demand treatment period -Percentage of participants who maintain FVIII activity levels -Number of injection and dose of BIVV001 to treat a bleeding episode -Percentage of bleeding episodes treated with a single injection of BIVV001 -Assessment of response to BIVV001 treatment of individual bleeding episodes -Physician's global assessment of the participant's response based on BIVV001 treatment -Total annualized BIVV001 consumption -Annualized Joint Bleeding Rate (AJBR) -Target joint resolution -Change in Hemophilia Joint Health Score (HJHS) total score and domain scores -Change in PROMIS-SF Physical Function -Changes in Haem-A-QoL total score and physical health score -Investigators' or Surgeons' assessment of participant's hemostatic response to BIVV001 treatment -Number of injections and dose to maintain hemostasis during perioperative period for major surgery -Total BIVV001 consumption during perioperative period for major surgery -Number of blood component transfusions used during perioperative period for major surgery -Type of blood component transfusions used during perioperative period for major surgery -Estimated blood loss during perioperative period for major surgery -Number of participants with occurance of adverse events (AEs) and serious adverse events (SAEs) -Number of participants with inhibitor development -Number of participants with occurrence of embolic and thrombotic events -PK parameter: Maximum activity (Cmax) -PK parameter: Elimination half-life | — |
Countries
Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Colombia, France, Germany, Greece, Hungary, Italy, Japan, Korea, Republic of, Mexico, Netherlands, Spain, Taiwan, United Kingdom, United States
Contacts
Public ContactProject Management
PSI CRO AG
Outcome results
None listed