Hemophilia A
Conditions
Interventions
Weekly administration (intravenous) of BIVV001 for a maximum period of 4 years.
Sponsors
Sanofi BV
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
Inclusion criteria: For Arm A - Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local participant privacy regulations. Parents or legal guardians* consent is required for participants who are =18 years of age) to continue use of the study ePD throughout the study. For Arm C - Participants who have severe hemophilia A, defined as =100 000 cells/µL at screening. - Weight above or equal to 10 kg. Arm B is only applicable for China
Exclusion criteria
Exclusion criteria: - 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 result, defined as >=0.6 BU/mL at Screening. Any concurrent clinically significant liver disease. - Serious active bacterial, fungal, or viral infection. - Other known coagulation disorder(s) in addition to hemophilia A. - Abnormal renal function or significant liver disease.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The occurrence of inhibitor development (neutralizing antibodies directed against FVIII) | — |
Secondary
| Measure | Time frame |
|---|---|
| - Annualized bleeding rate (ABR), ABR by type of bleed and ABR by location of bleed - Percentage of participants who maintain FVIII activity above prespecified 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 - Changes in Haemophilia Quality of Life Questionnaire (Haemo-QoL) total score and physical health domain scores from baseline to end of study for maximum 4 years - 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 occurence of adverse events (AEs) and serious adverse events (SAEs) - Number of participants with occurrence of embolic and thrombotic events - PK parameter: Maximum activity (Cmax), elimination half-life (t1/2), total clearance (CL), total clearance at steady state (CLss), dose-normalized area under the activity-time curve (DNAUC), area under the activity time curve (AUC), volume of distribution at steady state (Vss), mean residence time (MRT), incremental recovery (IR), trough activity (Ctrough), time above predefined FVIII activity levels | — |
Countries
Netherlands
Outcome results
None listed