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
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).
Annualized Number of Total Bleeds Within 48 h
Annualized Number of Total Bleeds During Prophylaxis Treatment
Recombinant Factor VIII (Kovaltry, BAY81-8973)
Study Arms / Comparison Groups
PTPs 0-12 years
Description: Previously treated patients (PTPs) aged below 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (EDs) in main study - Part A. Participants having reached at least 50 EDs in main study - Part A were offered participation in an open label extension study (optional). Participants who transitioned from main study - Part A to the extension study received BAY81-8973, 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part A and extension study).
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
June 9, 2011
October 27, 2020
Primary Completion Date
September 9, 2019
Inclusion Criteria: - Male - PTPs (previously treated patients): aged <= 12 years - PUPs (previously untreated patients) / MTPs (minimally treated patients): aged < 6 years - Severe hemophilia A defined as < 1% FVIII concentration (FVIII:C) - PTPs: >= 50 exposure days (EDs) with any FVIII concentrate, no current evidence of inhibitory antibody, and no history of FVIII inhibitor formation - PUPs: no prior exposure to any FVIII product - MTPs: having no more than 3 EDs with any FVIII product, no current evidence of inhibitory antibody and no history of FVIII inhibitor formation Exclusion Criteria: - With another bleeding disorder that is different from Hemophilia A - With thrombocytopenia (platelet count < 100 000/mm^3) - Creatinine > 2x upper limit of normal or Aspartate aminotransferase (AST)/Alanine aminotransferase (ALT) > 5x upper limit of normal - Without a negative inhibitor testing at screening (except for PUPs) - Receiving chemotherapy, immune modulatory drugs, has received another investigational FVIII product within the last month, or received another experimental drug within the last 3 months - Requires any pre-medication to tolerate FVIII treatment - Known hypersensitivity to active substance, mouse, or hamster protein
0 Years - 12 Years
Accepts Healthy Volunteers
Bayer Study Director, ,
Bayer Study Director, Study Director, Bayer