title: Phase I/II, open-label, multicenter, safety, efficacy and PK study of a recombinant coagulation factor IX albumin fusion protein (rIX-FP) in subjects with hemophilia B.
abstract: Recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) is a novel recombinant albumin fusion protein designed to extend the half-life of recombinant factor IX (rFIX), which is used in the management of hemophilia B. Clinical evaluation of rIX-FP in humans is underway, including a recently completed phase I/II, open-label, multicenter, study that assessed the safety, pharmacokinetics, and efficacy of rIX-FP in patients with severe hemophilia B. A total of 17 patients received rIX-FP (25 IU/kg) as either on-demand therapy (n = 4) for 20 weeks or weekly prophylaxis (n = 13) for up to 44 weeks. Preliminary results confirm that rIX-FP has an excellent safety profile and a pharmacokinetic profile highlighted by a marked extended half-life, suggesting that weekly prophylaxis with rIX-FP at a dose of 25 IU/kg may be appropriate in patients with severe hemophilia B, and that extended dosing intervals (10-14 days) may be feasible in some patients. A phase II/III study evaluating the safety and efficacy of rIX-FP in patients with hemophilia B is underway.
authors.affiliation: Institute of Thrombosis, Hemostasis and Israel National Hemophilia Center, Chaim Sheba Medical Center, Tel Hashomer, affiliated to Sackler School of Medicine, Israel. Uriel.Martinowitz@sheba.health.gov.il
authors.forename: Uri
authors.initials: U
authors.lastname: Martinowitz
authors.forename: Aaron
authors.initials: A
authors.lastname: Lubetsky
mesh_terms: D000293:Adolescent; D000328:Adult; D000418:Albumins / Q000008:administration & dosage* / Q000009:adverse effects / Q000493:pharmacokinetics; D005164:Factor IX / Q000008:administration & dosage* / Q000009:adverse effects / Q000493:pharmacokinetics; D002836:Hemophilia B / Q000097:blood / Q000188:drug therapy* / Q000378:metabolism; D006801:Humans; D008297:Male; D008875:Middle Aged; D011993:Recombinant Fusion Proteins / Q000008:administration & dosage* / Q000009:adverse effects / Q000493:pharmacokinetics; D055815:Young Adult
chemical_list: D000418:Albumins; D011993:Recombinant Fusion Proteins; D005164:Factor IX