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Efficacy and safety of prophylaxis with once-weekly BAY 79-4980 compared with thrice-weekly rFVIII-FS in haemophilia A patients. A randomised, active-controlled, double-blind study.

Abstract
The benefits of prophylaxis of haemophilia A patients regarding joint health and quality-of-life are well established. However, adherence to an up to every-other-day infusion regimen is a barrier to widespread adoption of prophylaxis. BAY 79-4980 is an investigational drug consisting of rFVIII-FS (sucrose-formulated recombinant FVIII) reconstituted with liposome solvent. Previous clinical studies showed extended protection from bleeding after a single injection of BAY 79-4980 (13.3 ± 6.2 days) compared with rFVIII-FS (7.2 ± 1.7 days). The effect of once-a-week prophylaxis with BAY 79-4980 (35 IU/kg) compared with three times-per-week rFVIII-FS (25 IU/kg) in previously treated, severe haemophilia A patients was evaluated in a 52-week, double-blind, two-arm, randomised, controlled study. The primary and secondary endpoints were protection from total bleeds and joint bleeds, respectively. Short- and long-term safety and tolerability of BAY 79-4980 including effects on lipid levels were assessed. A total of 139 and 131 subjects were evaluable for safety and efficacy analyses, respectively. A large difference in efficacy between treatment groups was observed with 72.1% (49/68) in the rFVIII-FS control group demonstrating <9 bleeds/year compared with 38.1% (24/63) of BAY 79-4980-treated subjects. A similar difference was seen in annualised joint bleeds, with 43 subjects (63.2%) in the control group demonstrating <5 joint bleeds/year compared with 24 subjects (38.1%) treated with BAY 79-4980. The distribution of bleeds seven days post-prophylactic treatment with BAY 79-4980 showed that 61% of bleeds occurred after day 4 post dosing. There were no safety concerns identified. The investigational treatment arm was prematurely discontinued due to failure to achieve the primary endpoint.
AuthorsJerry Powell, Uri Martinowitz, Jerzy Windyga, Giovanni Di Minno, Andrzej Hellmann, Ingrid Pabinger, Monika Maas Enriquez, Lawrence Schwartz, Jørgen Ingerslev, LipLong Study Investigators
JournalThrombosis and haemostasis (Thromb Haemost) Vol. 108 Issue 5 Pg. 913-22 (Nov 2012) ISSN: 2567-689X [Electronic] Germany
PMID23014711 (Publication Type: Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
Chemical References
  • Delayed-Action Preparations
  • Liposomes
  • Solvents
  • recombinant FVIII, sugar formulated
  • Polyethylene Glycols
  • Sucrose
  • Factor VIII
Topics
  • Adolescent
  • Adult
  • Chemistry, Pharmaceutical
  • Delayed-Action Preparations
  • Double-Blind Method
  • Drug Administration Schedule
  • Factor VIII (administration & dosage, adverse effects)
  • Hemophilia A (drug therapy)
  • Hemorrhage (prevention & control)
  • Humans
  • Liposomes (administration & dosage)
  • Male
  • Middle Aged
  • Polyethylene Glycols (administration & dosage)
  • Solvents
  • Sucrose (administration & dosage, adverse effects)
  • Treatment Outcome
  • Young Adult

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