Recombinant factor IX (BAX326) in previously treated paediatric @entity1 with XXXX : a prospective clinical trial.

@entity1 @entity548 @entity4682 @entity2720 @entity17049 @entity715 @entity4777
UNASSIGNED: A newly developed recombinant factor IX (BAX326(1) ) was investigated for prophylactic use in paediatric @entity1 aged <12  years with severe ( @entity4682 level <1%) or moderately severe ( @entity4682 level 1-2%) @entity4777 . The aim of this prospective clinical trial was to assess the safety, haemostatic efficacy and pharmacokinetic profile of BAX326 in previously treated paediatric @entity1 . BAX326 was administered as prophylaxis twice a week for a period of 6  months, and on demand for treatment of @entity548 . Safety was assessed by the occurrence of related AEs, @entity715 events and immunologic assessments. Efficacy was evaluated by annualized bleeding rate (ABR), and by treatment response rating (excellent, good, fair, none). PK was assessed over 72  h. None of the 23 treated paediatric subjects had treatment-related SAEs or AEs. There were no @entity715 events, inhibitory or specific binding antibodies against @entity4682 , @entity17049 or CHO protein. @entity548 (19 @entity2720 ) occurred (mean ABR 2.7      3.14, median 2.0), of which 23 were injury-related. Twenty subjects (87%) did not experience any @entity548 of spontaneous aetiology. Haemostatic efficacy of BAX326 was excellent or good for >96% of @entity548 (100% of minor, 88.9% of moderate and 100% of major @entity548 ); the majority (88.5%) resolved after 1-2 infusions. Longer T1/2 and lower IR were observed in younger @entity1 (<6  years) compared to those aged 6 to 12  years. BAX326 administered as prophylactic treatment as well as for controlling @entity548 is efficacious and safe in paediatric @entity1 aged <12  years with @entity4777 .
