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

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ClinicalTrials.gov Identifier: NCT01335061
Recruitment Status : Completed
First Posted : April 13, 2011
Results First Posted : May 4, 2015
Last Update Posted : June 8, 2015
Sponsor:
Information provided by (Responsible Party):
Pfizer

Tracking Information
First Submitted Date  ICMJE March 30, 2011
First Posted Date  ICMJE April 13, 2011
Results First Submitted Date  ICMJE March 18, 2015
Results First Posted Date  ICMJE May 4, 2015
Last Update Posted Date June 8, 2015
Study Start Date  ICMJE September 2011
Actual Primary Completion Date April 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 30, 2015)
Annualized Number of Bleeding Episodes. [ Time Frame: 2 years ]
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.
Original Primary Outcome Measures  ICMJE
 (submitted: April 12, 2011)
Annualized number of bleeding episodes [ Time Frame: Up to 2 years ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 30, 2015)
  • Response to On-Demand Treatment for All Bleeding Episodes. [ Time Frame: 2 years ]
    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 ]
    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 ]
    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 ]
    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 ]
    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 ]
    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.
Original Secondary Outcome Measures  ICMJE
 (submitted: April 12, 2011)
  • 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 ]
  • The number of BeneFIX infusions used to treat each bleed. [ Time Frame: Up to 2 years ]
  • The number of breakthrough (spontaneous/non-traumatic) bleeds within 48 hours of a prophylaxis dose of BeneFIX . [ Time Frame: Up to 2 years ]
  • The average infusion dose and total factor consumption. [ Time Frame: Up to 2 years ]
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B
Official Title  ICMJE 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%)
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hemophilia B
Intervention  ICMJE
  • 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
Study Arms  ICMJE BeneFIX
Interventions:
  • Biological: Nonacog alfa
  • Biological: Nonacog alfa
Publications * Tortella BJ, Carr ME, Rendo P, Korth-Bradley J, Smith LM, Kavakli K. Once-weekly prophylaxis regimen of nonacog alfa in patients with hemophilia B: an analysis of timing of bleeding event onset. Blood Coagul Fibrinolysis. 2021 Feb 18. doi: 10.1097/MBC.0000000000001012. [Epub ahead of print]

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 28, 2014)
25
Original Estimated Enrollment  ICMJE
 (submitted: April 12, 2011)
24
Actual Study Completion Date  ICMJE April 2014
Actual Primary Completion Date April 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 12 Years to 65 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Bulgaria,   Canada,   Croatia,   Korea, Republic of,   Malaysia,   Mexico,   Poland,   Singapore,   Turkey
Removed Location Countries United States
 
Administrative Information
NCT Number  ICMJE NCT01335061
Other Study ID Numbers  ICMJE B1821010
3090A1-3306 ( Other Identifier: Alias Study Number )
2011-000520-15 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Pfizer CT.gov Call Center Pfizer
PRS Account Pfizer
Verification Date May 2015

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