Factor VIII deficiency
Conditions
Interventions
Investigational drug: efanesoctocog alfa (BIVV001)
Control drug: NA
Dosage and Administration:
- Prophylaxis arm: prophylaxis treatment regimen with once weekly injection of BIVV001 for 52 weeks
- On-
Sponsors
Sanofi K.K.
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 =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. 6. 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
Exclusion criteria: Participants are excluded from the study if any of the following criteria apply: - 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 |
|---|---|
| 1. Annualized bleeding rate (ABR) in prophylaxis treatment arm [Time frame: baseline to 52 weeks] ABR will be estimated for the weekly (QW) prophylaxis treatment arm. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Intra-patient comparison of ABR of participants of this study to the ABR of same participants previously participated in an observational study [Time frame: baseline to week 52] Intra patient comparison of ABR during the BIVV001 weekly prophylaxis treatment period versus the historical prophylaxis ABR for participants in prophylaxis treatment who participated in Study 242HA201/OBS16221, an observational study 2. ABR by type of bleed [Time frame: baseline to week 52] ABR by type of bleed such as spontaneous or traumatic per study arm 3. ABR by location of bleed [Time frame: baseline to week 52] ABR by location of bleed such as joint, muscle, internal, or skin/mucosa per study arm 4. ABR for all bleeding episodes including untreated bleeding episodes [Time frame: baseline to week 52] ABR for all bleeding episodes (including untreated bleeding episodes) for prophylaxis treatment per study arm 5. Intra-patient comparison of ABR during the weekly once (QW) prophylaxis treatment period versus ABR during the on-demand treatment period [Time frame: baseline to week 52] Intra-patient comparison of ABR during the QW prophylaxis treatment period versus the ABR during the on-demand treatment period in the On-demand then prophylaxis arm 6. Percentage of participants who maintain FVIII activity levels [Time frame: 52 weeks] Percentage of participants who maintain FVIII activity levels in prophylaxis treatment arm 7. Number of injection and dose of BIVV001 to treat a bleeding episode [Time frame: 52 weeks] Number of injections and dose of BIVV001 to treat a bleeding episode per study arm and treatment regimen 8. Percentage of bleeding episodes treated with a single injection of BIVV001 [Time frame: 52 weeks] Percentage of bleeding episodes treated with a single injection of BIVV001 per study arm and treatment regimen 9. Assessment of response to BIVV001 treatment of individual bleeding episodes [Time frame: baseline to week 52] Assessment of response to BIVV001 treatmen | — |
Countries
Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, France, Germany, Greece, Hungary, Italy, Japan, Mexico, Netherlands, Republic of Korea, Spain, Taiwan, United Kingdom, United States
Contacts
Public ContactUnit Clinical
Sanofi K.K.
Outcome results
None listed