Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B

This study has been completed.
Information provided by (Responsible Party):
Pfizer Identifier:
First received: March 30, 2011
Last updated: May 12, 2015
Last verified: May 2015
The purpose of this study will be to determine if a once-weekly prophylaxis regimen of BeneFIX in subjects with moderately severe to severe Hemophilia B is safe and effective.

Condition Intervention Phase
Hemophilia B
Biological: Nonacog alfa
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicenter, Open-label Study To Compare On-demand Treatment To A Prophylaxis Regimen Of Nonacog-alfa (Benefix) In Subjects With Moderately Severe To Severe Hemophilia B (Fix:c</=2%)

Resource links provided by NLM:

Further study details as provided by Pfizer:

Primary Outcome Measures:
  • Annualized Number of Bleeding Episodes. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    The annualized bleed rate (ABR) or the annualized number of bleeding episodes per year, will be derived for each participant for each treatment period by using the following formula: ABR = number of bleeds / (Days on treatment period / 365.25) The number of bleeds for the ABR calculation includes all bleeds requiring treatment with factor IX product during the time on treatment.

Secondary Outcome Measures:
  • Response to On-Demand Treatment for All Bleeding Episodes. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Assessment scores on a 4-point Response Scale for an on-demand bleeding episode, as assessed by participant/caregiver or investigator/qualified staff. The 4-point scale assessments are Excellent, Good, Moderate or No response. Responses to number of observations were noted.

  • Number of Nonacog Alfa, Recombinant Factor IX (BeneFIX) Infusions Used to Treat Each Bleeding Episode. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    The number of study drug infusions administered to treat a bleed will be calculated by adding the initial (on-demand) infusion to any subsequent (on-demand) infusions for the same bleed (same bleed start date/time). The number of infusions needed to treat a bleed will be classified into the following categories: 1, 2, 3, 4 and >4 infusions. If there were more than one bleed location (e.g., ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence.

  • Number of Breakthrough (Spontaneous/Non-Traumatic) Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    The number of spontaneous, non-traumatic breakthrough bleeds within 48 hours following a prophylaxis dose of BeneFIX were summarized. If there was more than one bleed location (eg, ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence.

  • Average Infusion Dose. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    The mean dose by per infusion by weight (IU/kg) was reported for both prophylaxis and on demand infusions

  • Total Factor Consumption. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    The total amount (IU) infused for each infusion recorded were summed to calculate the total factor consumption for each participant. For each infusion, IU/kg was calculated, using the most recently recorded weight measurement and the total factor consumption, divided by number of infusions, and was summarized similarly to average infusion dose (IU). Annualized TFC by weight was reported. Annualized TFC by weight = (Total IU/kg / treatment interval duration)*365.25.

  • Incidence of Less Than Expected Therapeutic Effect (LETE) [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    The following criteria are the definitions for LETE in this study: 1. LETE in the On-Demand Setting: LETE occurs in the on-demand setting if 2 successive "No Response" ratings are recorded after 2 successive BeneFIX drug infusions in the absence of confounding factors. 2. LETE in the Prophylaxis Setting: LETE occurs in the prophylaxis setting if there is a spontaneous bleed within 48 hours (≤ 48 hours) after a regularly scheduled prophylactic dose of BeneFIX in the absence of confounding factors. 3. LETE (Low Recovery): LETE can also be lower than expected recovery of FIX in the opinion of the investigator following infusion of BeneFIX in the absence of confounding factors. Each reported occurrence of low recovery LETE was listed.

Enrollment: 25
Study Start Date: September 2011
Study Completion Date: April 2014
Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
BeneFIX Biological: Nonacog alfa
Period 1: During on-demand period, dosing at the discretion of investigator.
Other Name: BeneFIX
Biological: Nonacog alfa
Period 2: During the prophylaxis period, 100 IU/kg once weekly
Other Name: BeneFIX


Ages Eligible for Study:   12 Years to 65 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Documented history of moderately-severe to severe hemophilia B (FIX activity </=2%).
  • Male subjects, aged 12 years to 65 years.
  • Subjects with at least 100 exposure days (EDs) to factor IX products.
  • Subjects with a minimum of 12 bleeding episodes, 6 of which must be joint bleeds, in the 12-month period before the Screening visit.

Exclusion Criteria:

  • Subjects who have received FIX as a primary or secondary prophylaxis regimen within the last 12 months prior to the Screening visit.
  • Subjects who have had major surgery or an orthopedic surgical procedure within the past 3 months prior to Screening visit.
  • Subjects for which major surgery or orthopedic surgery is planned within the duration of study participation.
  • Subjects with a past history of, or current FIX inhibitor, defined as >ULN (upper limit of normal) of the reporting laboratory.
  • Subjects with a known hypersensitivity to any FIX product or hamster protein.
  Contacts and Locations
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Please refer to this study by its identifier: NCT01335061

UMBAL Sveti Georgi, Klinika po hematologia
Plovdiv, Bulgaria, 4002
Canada, Ontario
The Ottawa Hospital
Ottawa, Ontario, Canada, K1H 8L6
University Hospital Center Zagreb
Zagreb, Croatia, 10000
Korea, Republic of
Eulji University Hospital
Daejeon, Korea, Republic of, 302-799
Hospital Tengku Ampuan Afzan
Kuantan, Pahang, Malaysia, 25200
National Blood Centre
Kuala Lumpur, Malaysia, 50400
Hospital y Clinica OCA
Monterrey, Nuevo Leon, Mexico, 64000
Instituto Biomedico de Investigacion A.C.
Aguascalientes, Mexico, 20127
Nzoz Triclinium
Warszawa, Poland, 02-797
Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku
Wroclaw, Poland, 50-367
Singapore General Hospital
Singapore, Singapore, 169608
Istanbul Universitesi Cerrahpasa Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bilim Dali
Fatih, Istanbul, Turkey, 34098
Hacettepe Universitesi Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bolumu
Ankara, Turkey, 06100
Ege Universitesi Tip Fakultesi
Bornova/Izmir, Turkey, 35100
Gaziantep Universitesi Tip Fakultesi Sahinbey Arastirma ve Uygulama Hastanesi Hematoloji Poliklinigi
Gaziantep, Turkey, 27300
Erciyes Universitesi Tip Fakultesi
Kayseri, Turkey, 38039
Erciyes Universitesi Tip Fakultesi Hastaneleri M. Kemal Dedeman Onkoloji Hastanesi
KAYSERİ, Erciyes, Turkey, 38039
Sponsors and Collaborators
Study Director: Pfizer Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Pfizer Identifier: NCT01335061     History of Changes
Other Study ID Numbers: B1821010, 3090A1-3306, 2011-000520-15
Study First Received: March 30, 2011
Results First Received: March 18, 2015
Last Updated: May 12, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
Hemophilia B
nonacog alfa

Additional relevant MeSH terms:
Hemophilia B
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hematologic Diseases
Hemorrhagic Disorders processed this record on November 30, 2015