Clinical trial NCT01335061: Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B
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%)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hemophilia B
Keywords: Hemophilia B
on-demand
prophylaxis
nonacog alfa
BeneFIX
FIX
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2011-04-13
Enrollment: 25
Sex: MALE
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Annualized Number of Bleeding Episodes.
timeFrame: 2 years
Secondary outcomes:
measure: Response to On-Demand Treatment for All Bleeding Episodes.
timeFrame: 2 years
measure: Number of Nonacog Alfa, Recombinant Factor IX (BeneFIX) Infusions Used to Treat Each Bleeding Episode.
timeFrame: 2 years
measure: Number of Breakthrough (Spontaneous/Non-Traumatic) Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX.
timeFrame: 2 years
measure: Average Infusion Dose.
timeFrame: 2 years
measure: Total Factor Consumption.
timeFrame: 2 years
measure: Incidence of Less Than Expected Therapeutic Effect (LETE)
timeFrame: 2 years
Other outcomes:
Locations:
city: Plovdiv
country: Bulgaria
facility: UMBAL Sveti Georgi, Klinika po hematologia
city: Ottawa
country: Canada
facility: The Ottawa Hospital
state: Ontario
city: Zagreb
country: Croatia
facility: University Hospital Center Zagreb
city: Daejeon
country: Korea, Republic of
facility: Eulji University Hospital
city: Kuantan
country: Malaysia
facility: Hospital Tengku Ampuan Afzan
state: Pahang
city: Kuala Lumpur
country: Malaysia
facility: National Blood Centre
city: Monterrey
country: Mexico
facility: Hospital y Clinica OCA
state: Nuevo Leon
city: Aguascalientes
country: Mexico
facility: Instituto Biomedico de Investigacion A.C.
city: Warszawa
country: Poland
facility: Nzoz Triclinium
city: Wroclaw
country: Poland
facility: Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku
city: Singapore
country: Singapore
facility: Singapore General Hospital
city: Fatih
country: Turkey
facility: Istanbul Universitesi Cerrahpasa Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bilim Dali
state: Istanbul
city: Ankara
country: Turkey
facility: Hacettepe Universitesi Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bolumu
city: Bornova/Izmir
country: Turkey
facility: Ege Universitesi Tip Fakultesi
city: Gaziantep
country: Turkey
facility: Gaziantep Universitesi Tip Fakultesi Sahinbey Arastirma ve Uygulama Hastanesi Hematoloji Poliklinigi
city: KAYSERİ, Erciyes
country: Turkey
facility: Erciyes Universitesi Tip Fakultesi Hastaneleri M. Kemal Dedeman Onkoloji Hastanesi
city: Kayseri
country: Turkey
facility: Erciyes Universitesi Tip Fakultesi
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: 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 Apr 1;32(3):180-185. doi: 10.1097/MBC.0000000000001012.