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

August 30, 2011 updated by: Pfizer

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

50

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2B7
        • Pfizer Investigational Site
      • Edmonton, Alberta, Canada, T6G 2C8
        • Pfizer Investigational Site
    • Ontario
      • Ottawa, Ontario, Canada, K1H 8L6
        • Pfizer Investigational Site
  • Croatia
      • Zagreb, Croatia, 10000
        • Pfizer Investigational Site
  • Hungary
      • Budapest, Hungary, 1134
        • Pfizer Investigational Site
  • Italy
      • Castelfranco Veneto (TV), Italy, 31033
        • Pfizer Investigational Site
      • Coppito (AQ), Italy, 67100
        • Pfizer Investigational Site
  • Romania
      • Bucuresti, Romania, 011155
        • Pfizer Investigational Site
  • Russian Federation
      • Moscow, Russian Federation, 125167
        • Pfizer Investigational Site
      • Saint Petersburg, Russian Federation, 191186
        • Pfizer Investigational Site
  • Serbia
      • Belgrade, Serbia, 11000
        • Pfizer Investigational Site
      • Nis, Serbia, 18000
        • Pfizer Investigational Site
  • Spain
      • Madrid, Spain, 28046
        • Pfizer Investigational Site
      • Sevilla, Spain, 41013
        • Pfizer Investigational Site
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Pfizer Investigational Site
    • Illinois
      • Chicago, Illinois, United States, 60612-3833
        • Pfizer Investigational Site
      • Chicago, Illinois, United States, 60614
        • Pfizer Investigational Site
    • New Jersey
      • New Brunswick, New Jersey, United States, 08903-0019
        • Pfizer Investigational Site
    • Texas
      • Houston, Texas, United States, 77030-4009
        • Pfizer Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  • Documented history of moderately severe or severe hemophilia B (FIX:C less than or equal to 2%)
  • Male subjects, aged 6 years to 65 years.
  • Subjects with a minimum of 12 bleeding episodes, 6 of which must be joint bleeds, in the 12 months before screening.

Exclusion Criteria:

  • Subjects currently utilizing FIX primary prophylaxis.
  • Subjects with HIV+ who have a CD4 count less than 200
  • Subjects with hepatic or renal impairment.
  • Prothrombin time or International Normalized Ration more than 1.5 times the upper limit of normal.
  • Major surgery or an orthopedic surgical procedure within the past 3 months or is planned within the duration of participation in this study.
  • Past history of, or current FIX inhibitor.
  • Hypersensitivity to any FIX product or hamster protein.
  • Exposure to any investigational drug, except for rFIX-R, or device within 30 days of providing consent/assent (as appropriate) for this study.
  • Bleeding disorders other than hemophilia B.
  • Concurrent inflammatory disease that in the investigator's judgment could confound the study results.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: A
Drug: Recombinant Coagulation Factor IX (BeneFIX)
100 IU/kg once weekly then crossover to 50 IU/kg twice weekly
Drug: Recombinant Coagulation Factor IX (BeneFIX)
50 IU/kg twice weekly then crossover to 100 IU/kg once weekly
Experimental: B
Drug: Recombinant Coagulation Factor IX (BeneFIX)
100 IU/kg once weekly then crossover to 50 IU/kg twice weekly
Drug: Recombinant Coagulation Factor IX (BeneFIX)
50 IU/kg twice weekly then crossover to 100 IU/kg once weekly

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualized Number of Bleeding Episodes
Time Frame: Baseline up to Week 56
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)
Baseline up to Week 56

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
24 and 48 hours post-bleed
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.
24 and 48 hours post-bleed
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).
24 and 48 hours post-bleed
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.
24 hours post-bleed
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.
48 hours post-bleed
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).
Weeks 16, 32, and 56

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Pfizer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2007

Primary Completion (Actual)

October 1, 2010

Study Completion (Actual)

October 1, 2010

Study Registration Dates

First Submitted

August 14, 2006

First Submitted That Met QC Criteria

August 14, 2006

First Posted (Estimate)

August 15, 2006

Study Record Updates

Last Update Posted (Estimate)

October 4, 2011

Last Update Submitted That Met QC Criteria

August 30, 2011

Last Verified

August 1, 2011

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 3090A1-400
  • B1821002 (Other Identifier: Pfizer)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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