Hemophilia A MedDRA version: 20.0 Level: LLT Classification code 10060612 Term: Hemophilia A System Organ Class: 100000004850
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: Re
Sponsors
Bioverativ Therapeutics Inc.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: - Participant must be younger than 12 years of age, at the time of signing the informed consent - Severe hemophilia A defined as =65 years) no F.1.3.1 Number of subjects for this age range
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: - To evaluate the safety of BIVV001 in previously treated pediatric subjects with hemophilia A;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 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 ;Primary end point(s): Occurrence of inhibitor development ;Timepoint(s) of evaluation of this end point: baseline to 52 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1 - Annualized bleeding rate (ABR) 2 - Annualized bleeding rate (ABR) by type of bleed 3 - Annualized bleeding rate (ABR) by location of bleed 4 - Percentage of participants who maintain FVIII activity above prespecified levels 5 - Number of injection and dose of BIVV001 to treat a bleeding episode 6 - Percentage of bleeding episodes treated with a single injection of BIVV001 7 - Assessment of response to BIVV001 treatment of individual bleeding episodes 8 - Physician’s global assessment of the participant’s response based on BIVV001 treatment 9 - Total annualized BIVV001 consumption 10 - Annualized Joint Bleeding Rate (AJBR) 11 - Target joint resolution 12 - Change in Hemophilia Joint Health Score (HJHS) total score and domain scores 13 - Changes in Haemophilia Quality of Life Questionnaire for Children (Haemo-QoL) total score 14 - Changes in Haemophilia Quality of Life Questionnaire for Children (Haemo-QoL) physical health domain scores 15 - Investigators’ or Surgeons’ assessment of participant’s hemostatic response to BIVV001 treatment 16-- Number of injections and dose to maintain hemostasis during perioperative period for major surgery 17 - Total BIVV001 consumption during perioperative period for major surgery 18 - Number of blood component transfusions used during perioperative period for major surgery 19 - Type of blood component transfusions used during perioperative period for major surgery 20 - Estimated blood loss during perioperative period for major surgery 21 - Number of participants with occurence of adverse events (AEs) and serious adverse events (SAEs) 22 - Number of participants with occurrence of embolic and thrombotic events 23 - PK parameter: Maximum activity (Cmax) 24 - PK parameter: Elimination half-life (t1/2) 25 - PK parameter: Total clearance (CL) 26 - PK parameter: Total clearance at steady state (CLss) 27 - PK parameter:dose-normalized area under the activity-time curve ( | — |
Countries
Australia, Belgium, Canada, France, Germany, Hungary, Ireland, Italy, Netherlands, Spain, Sweden, Switzerland, Taiwan, Turkey, United Kingdom, United States
Outcome results
None listed