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.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT01335061
First received: March 30, 2011
Last updated: April 30, 2015
Last verified: April 2015

March 30, 2011
April 30, 2015
September 2011
April 2014   (final data collection date for primary outcome measure)
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.
Annualized number of bleeding episodes [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT01335061 on ClinicalTrials.gov Archive Site
  • 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.
  • Assessment scores on a 4-point Response Scale for an on-demand bleeding episode, as assessed by subject/caregiver or investigator/qualified staff. The 4 point scale assessments are Excellent, Good, Moderate or No response. [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
  • The number of BeneFIX infusions used to treat each bleed. [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
  • The number of breakthrough (spontaneous/non-traumatic) bleeds within 48 hours of a prophylaxis dose of BeneFIX . [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
  • The average infusion dose and total factor consumption. [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
  • The number of subjects with incidences of less-than-expected therapeutic effect (LETE) in the absence of confounding factors. There are 3 circumstances in which LETE can occur. In the on-demand or prophylaxis settings and for low recovery of Factor IX. [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B
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%)

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.

Not Provided
Interventional
Phase 3
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Hemophilia B
  • 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
BeneFIX
Interventions:
  • Biological: Nonacog alfa
  • Biological: Nonacog alfa
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
25
April 2014
April 2014   (final data collection date for primary outcome measure)

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.
Male
12 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
Bulgaria,   Canada,   Croatia,   Korea, Republic of,   Malaysia,   Mexico,   Poland,   Singapore,   Turkey
United States
 
NCT01335061
B1821010, 3090A1-3306, 2011-000520-15
Yes
Pfizer
Pfizer
Not Provided
Study Director: Pfizer CT.gov Call Center Pfizer
Pfizer
April 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP