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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
25
Period 1: During on-demand period, dosing at the discretion of investigator.
Period 2: During the prophylaxis period, 100 IU/kg once weekly
UMBAL Sveti Georgi, Klinika po hematologia
Plovdiv, Bulgaria
The Ottawa Hospital
Ottawa, Ontario, Canada
Annualized Number of Bleeding Episodes.
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.
Time frame: 2 years
Response to On-Demand Treatment for All Bleeding Episodes.
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.
Time frame: 2 years
Number of Nonacog Alfa, Recombinant Factor IX (BeneFIX) Infusions Used to Treat Each Bleeding Episode.
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.
Time frame: 2 years
Number of Breakthrough (Spontaneous/Non-Traumatic) Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX.
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.
Time frame: 2 years
Average Infusion Dose.
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University Hospital Center Zagreb
Zagreb, Croatia
National Blood Centre
Kuala Lumpur, Kuala Lumpur, Malaysia
Hospital Tengku Ampuan Afzan
Kuantan, Pahang, Malaysia
Instituto Biomedico de Investigacion A.C.
Aguascalientes, Aguascalientes, Mexico
Hospital y Clinica OCA
Monterrey, Nuevo León, Mexico
Nzoz Triclinium
Warsaw, Poland
Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku
Wroclaw, Poland
Singapore General Hospital
Singapore, Singapore
...and 7 more locations
The mean dose by per infusion by weight (IU/kg) was reported for both prophylaxis and on demand infusions
Time frame: 2 years
Total Factor Consumption.
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.
Time frame: 2 years
Incidence of Less Than Expected Therapeutic Effect (LETE)
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.
Time frame: 2 years