NCT01311648

BAY81-8973 Pediatric Safety and Efficacy Trial

A Multicenter Phase III Uncontrolled Open-label Trial to Evaluate Safety and Efficacy of BAY81-8973 in Children With Severe Hemophilia A Under Prophylaxis Therapy

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 94 (actual)
• Sponsor: Bayer · Industry
• Sex: Male
• Age: 0 Years – 12 Years
• Healthy volunteers: Not accepted

Summary

The primary objective was to evaluate the safety and efficacy of the treatment with BAY81-8973 for prophylaxis and treatment of breakthrough bleeds in children with severe hemophilia A. The secondary objectives were * To assess the safety and efficacy of BAY81-8973 during surgeries. * To assess incremental recovery of BAY81-8973. * To assess pharmacokinetic (PK) parameters in a subset of children (Previously treated patients \[PTPs\] and previously untreated patients \[PUPs\] / minimally treated patients \[MTPs\] - participation in PK sampling was voluntary and required consent).

Conditions

• Haemophilia A

Interventions

Type	Name	Description
BIOLOGICAL	Recombinant Factor VIII (Kovaltry, BAY81-8973)	Main study: 25-50 IU/kg at least 2x/week for 6 months and at least 50 EDs, IV infusion; Extension study: 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part A and extension study), IV infusion. Exposure day (ED): An ED is a unit of time (1 day) in which replacement treatment of Hemophilia is given to a patient.
BIOLOGICAL	Recombinant Factor VIII (Kovaltry, BAY81-8973)	Main study: 15-50 IU/kg at least 1x/week for at least 50 EDs or until inhibitor development, IV infusion; Extension study: For participants having reached at least 50 EDs in main study - Part B: 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part B and extension study), IV infusion. For participants who developed an inhibitor in main study - Part B: up to 200 IU/kg per day or 100 IU/kg twice a day at the discretion of the investigator and coordinating investigator until successful eradication of the inhibitor, or until failure, for up to18 months (treatment beyond 18 months required an agreement with the sponsor and coordinating investigator), IV infusion

Timeline

• Start date: 2011-06-09
• Primary completion: 2019-09-09
• Completion: 2020-10-27
• First posted: 2011-03-09
• Last updated: 2023-12-05
• Results posted: 2020-11-03

Locations

43 sites across 17 countries: United States, Argentina, Bulgaria, Canada, Denmark, Hungary, Ireland, Israel, Italy, Latvia, Lithuania, Mexico, Norway, Poland, Romania, Russia, Spain

Regulatory

• FDA-regulated drug study