Clinical Trial: Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Multicenter, Open-Label Study To Compare On-Demand Treatment With 2 Prophylaxis Regimens of Recombinant Coagulation Factor IX (BeneFIX) Refo

Brief Summary:

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.

Annualized bleed rate (ABR) or number of bleeds per year derived for each participant for each treatment regimen by using the following formula: ABR = number of bleeds / (days on treatment regimen / 365.25)



Original Primary Outcome: The primary efficacy endpoint is the annualized number of bleeding episodes. Information regarding bleeding episodes will be collected with electronic subject diaries.

Current Secondary Outcome:

  • Amount of Sleep Measured by Sleep Diary After Hemarthrosis [ Time Frame: 24 and 48 hours post-bleed ]
    For each bleeding event, a diary was filled out that night and the subsequent night. Questions included: How long do you think you slept last night? Reported as average duration of sleep during study.
  • Quality of Sleep Measured by Sleep Diary After Hemarthrosis [ Time Frame: 24 and 48 hours post-bleed ]
    For each bleeding event, a diary was filled out that night and the subsequent night. Questions included: How would you describe the quality of your sleep last night? 1=Very Good, 2=Good, 3=Fair, 4=Poor, 5=Very Poor. Reported as quality of sleep during study.
  • Acute Pain After Hemarthrosis [ Time Frame: 24 and 48 hours post-bleed ]
    For each bleeding event, a diary was filled out that night and the subsequent night and included a Brief Pain Inventory (BPI): self-reported scale that measured severity of pain experienced over the past 24 hours. Questions included How much pain right now? 0 (no pain) to 10 (pain as severe as you can imagine).
  • Health-Related Productivity Questionnaire (HRPQ) Score: Hours Lost From Work or School at 24 Hours Post-bleed [ Time Frame: 24 hours post-bleed ]
    HRPQ: self-reported scale that measured for each bleed event, hours lost from work, school and housework because of hemophilia and its treatments. Mean and standard deviations calculated from measured values.
  • HRPQ Score: Hours Lost From Work or School at 48 Hours Post-bleed [ Time Frame: 48 hours post-bleed ]
    HRPQ: self-reported scale that measured for each bleed event, hours lost from work, school and housework because of hemophilia and its treatments. Mean and standard deviations calculated from measured values.
  • 36-Item Short-Form Health Survey (SF-36): Physical Functioning Domain [ Time Frame: Weeks 16, 32, and 56 ]
    SF-36: standardized survey evaluating 8 aspects of functional health and well being: physical and social functioning, physical and emotional role limitations, bodily pain, general health, vitality, and mental health. The physical functioning domain score was an average of the individual physical functioning question scores across all time points, which was scaled 0-100 (100=highest level of functioning).


Original Secondary Outcome: Amount and quality of sleep (Sleep Diary); Acute pain scale after hemarthrosis (Brief Pain Inventory); Days lost from work or school after hemarthrosis (Health-Related Productivity Questionnaire); SF 36 Questionnaire

Information By: Pfizer

Dates:
Date Received: August 14, 2006
Date Started: May 2007
Date Completion:
Last Updated: August 30, 2011
Last Verified: August 2011