Hemophilia B Clinical Trial
Official title:
A Multicenter, Open-Label Study To Compare On-Demand Treatment With 2 Prophylaxis Regimens of Recombinant Coagulation Factor IX (BeneFIX) Reformulated Drug Product (rFIX-R) In Subjects With Severe Hemophilia B
This study is designed to evaluate BeneFIX infused as prophylaxis regimens, compared with
BeneFIX administered as an on-demand regimen only. In the trial, subjects will participate
in 4 study periods. The first period is a 16-week on-demand treatment, during which subjects
will utilize BeneFIX to treat bleeding events episodically only as they occur (i.e.,
on-demand treatment). At the end of this period, subjects will be randomly assigned to 1 of
2 BeneFIX prophylaxis regimens: either 100 IU/kg once weekly or 50 IU/kg twice weekly, and
followed for 16 weeks. At the end of this period, subjects will use on-demand treatment for
8 weeks. The purpose of this 8-week period is to mitigate the potential carry-over effect of
the prior prophylaxis regimen on bleeding frequency. Following this 8-week on-demand
treatment, subjects will cross-over and receive the alternate study prophylactic regimen for
16 weeks.
The primary endpoint is annualized number of bleeding episodes, compared between the first
on-demand period and each of the prophylaxis regimens. A comparison of annualized bleeding
episodes between the two prophylaxis regimens will also be performed. Subject-reported
outcomes will also be collected using a patient diary. At 24 and 48 hours following the
onset of each joint bleeding episode, information on pain, sleep, and work will be
collected. Additional data on physical functioning will be recorded on the diary at the end
of the 16-week on-demand period, and at the end of each prophylaxis period. Information on
safety will also be collected.
Each subject will participate in this study for approximately 59 weeks (15 months),
including a screening period of up to 3 weeks, an initial 16-week on-demand period, two
prophylaxis treatment periods of 16 weeks each, separated by an 8-week on-demand treatment
period.
A modified FIX recovery study will be performed once during each prophylaxis period.
The study diary will also be used by subjects to collect secondary endpoints. These
endpoints, which are subject-reported outcomes, will be recorded in the diary at 24 and 48
hours following the onset of each joint bleeding episode. Additional data on physical
functioning will be recorded on the diary at the end of the 16-week on-demand period, and at
the end of each prophylaxis period.
Patients will be recruited in the United States, Canada, Europe and Russia.
n/a
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Prevention
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