A global phase 3 study evaluated the pharmacokinetics, efficacy, and safety of recombinant fusion protein linking @entity4682 with albumin (rIX-FP) in 63 previously treated male @entity1 (12-61 years) with severe @entity4777 ( @entity4682 [ @entity4682 ] activity  <=2%). The study included 2 groups: group 1 @entity1 received routine prophylaxis once every 7 days for 26 weeks, followed by either 7-, 10-, or 14-day prophylaxis regimen for a mean of 50, 38, or 51 weeks, respectively; group 2 @entity1 received on-demand treatment of @entity548 episodes for 26 weeks and then switched to a 7-day prophylaxis regimen for a mean of 45 weeks. The mean terminal half-life of rIX-FP was 102 hours, 4.3-fold longer than previous @entity4682 treatment. @entity1 maintained a mean trough of 20 and 12 IU/dL @entity4682 activity on prophylaxis with rIX-FP 40 IU/kg weekly and 75 IU/kg every 2 weeks, respectively. There was 100% reduction in median annualized spontaneous @entity548 rate (AsBR) and 100% resolution of @entity2720 when subjects switched from on-demand to prophylaxis treatment with rIX-FP (P< .0001). The median AsBR was 0.00 for all prophylaxis regimens. Overall, 98.6% of @entity548 episodes were treated successfully, including 93.6% that were treated with a single injection. No @entity1 developed an inhibitor, and no safety concerns were identified. These results indicate rIX-FP is safe and effective for preventing and treating @entity548 episodes in @entity1 with @entity4777 at dosing regimens of 40 IU/kg weekly and 75 IU/kg every 2 weeks. This trial was registered atwww.clinicaltrials.govas #NCT0101496274.

Long-acting recombinant coagulation XXXX albumin fusion protein (rIX-FP) in @entity4777 : results of a phase 3 trial.

@entity1 :: ('9606', 'Species') :: ['Patients', 'patients', 'patient'] @entity548 :: ('MESH:D006470', 'Disease') :: ['bleeding'] @entity4682 :: ('2158', 'Gene') :: ['factor IX', 'coagulation factor IX', 'FIX'] @entity2720 :: ('MESH:D007592', 'Disease') :: ['target joints'] @entity4777 :: ('MESH:D002836', 'Disease') :: ['hemophilia B']
@entity4682 :: (2158,Gene) :: ['factor IX']
