Clinical trial NCT02336178: Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China
Official title: An Open-label, Single-arm, Post-authorization Pragmatic Clinical Trial On The Safety And Efficacy Of Benefix (Nonacog Alfa, Recombinant Factor Ix) In Subjects With Hemophilia B In Usual Care Settings In China
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: HEMOPHILIA B
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2015-01-12
Enrollment: 70
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Primary purpose: TREATMENT

Brief summary: The purpose of this post-approval study is to evaluate the safety and efficacy of Benefix in subjects with hemophilia B in usual care settings in China.

Detailed description: The purpose of this post-approval study is to provide supplementary information relating to the use of BeneFIX in Chinese subjects with hemophilia B, especially on the safety and efficacy in different populations of Chinese hemophilia B patients, in particular in pediatric patients \<6 years of age, pediatric patients ≥6 to ≤12 years of age, Previously Untreated Patients (PUPs) , subjects receiving prophylaxis treatment after enrollment in the study, and severe patients (FIX activity \<1%).

Eligibility criteria: Inclusion Criteria:

* Male and/or female subjects with hemophilia B.
* Subjects/parents/legal representatives must be able to comply with study procedures (informed consent/assent process, clinical visits, reporting of infusion and bleed data, reporting of adverse events, etc)

Exclusion Criteria:

* Presence of any other bleeding disorder in addition to hemophilia B. Subjects with a past history of, or current factor IX inhibitor. For laboratory-based assessments, any Bethesda inhibitor titer greater than the laboratory's normal range or ≥0.6 Bethesda Unit (BU)/mL.
* Subjects with known hypersensitivity to the active substance or to any of the excipients of BeneFIX.
* Subjects with a known hypersensitivity to Chinese Hamster Ovary cell proteins.

Primary outcomes:
measure: Percentage of Participants Who Developed Factor IX Inhibitor
timeFrame: Up to 6 months
measure: Number of Participants With Allergic Reactions
timeFrame: Up to 6 months
measure: Number of Participants With Thrombotic Events
timeFrame: Up to 6 months
Secondary outcomes:
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
timeFrame: Up to 7 months (28 calendar days after end of 6-month or 50-exposure day study treatment)
measure: Annualized Bleeding Rates (ABRs) in Participants Receiving Prophylaxis Treatment With BeneFIX During Their Prophylaxis Period
timeFrame: Up to 6 months or 50 exposure days whichever occurred first
measure: Number of Spontaneous/Non-traumatic Breakthrough Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX
timeFrame: Up to 6 months or 50 exposure days whichever occurred first
measure: Annualized Bleeding Rates (ABRs) in Participants Receiving On-demand Treatment With BeneFIX During Their On-demand Period
timeFrame: Up to 6 months or 50 exposure days whichever occurred first
measure: Number of Infusions Resulted in the Following Response to On-demand Treatment of Bleeds: Excellent, Good, Moderate, no Response
timeFrame: Up to 6 months or 50 exposure days whichever occurred first
measure: Number of BeneFIX Infusions to Treat Each New Bleed
timeFrame: Up to 6 months or 50 exposure days whichever occurred first
measure: Average Infusion Dose and Total Factor IX Consumption in On-demand Setting
timeFrame: Up to 6 months or 50 exposure days whichever occurred first
measure: Average Infusion Dose and Total Factor IX Consumption in Prophylaxis Setting
timeFrame: Up to 6 months or 50 exposure days whichever occurred first
measure: Average Infusion Dose and Total Factor IX Consumption in Recovery Setting
timeFrame: Up to 6 months or 50 exposure days whichever occurred first
measure: Percentage of Infusions With Less Than Expected Therapeutic Effects (LETEs) in On-demand Setting
timeFrame: Up to 6 months or 50 exposure days whichever occurred first
measure: Percentage of Infusions With Less Than Expected Therapeutic Effects (LETEs) in Prophylaxis Setting
timeFrame: Up to 6 months or 50 exposure days whichever occurred first
Other outcomes:
Locations:
city: Guangzhou
country: China
facility: Nanfang Hospital, Southern Medical University
state: Guangdong
city: Guiyang
country: China
facility: The Affiliated Hospital of Guizhou Medical University
state: Guizhou
city: Zhengzhou
country: China
facility: Henan Provincial People's Hospital
state: Henan
city: Wuhan
country: China
facility: Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology
state: Hubei
city: Changsha
country: China
facility: Xiangya Hospital of Centre-South University
state: Hunan
city: Suzhou
country: China
facility: Department of Hematology,The First Affiliated Hospital of Soochow University
state: Jiangsu
city: Xuzhou
country: China
facility: Department of Hematology,The Affiliated Hospital of Xuzhou Medical University
state: Jiangsu
city: Nanchang
country: China
facility: Department of Hematology,Jiangxi Provincial People's Hospital
state: Jiangxi
city: Jinan
country: China
facility: Blood Center of Shandong Province
state: Shandong
city: Shanghai
country: China
facility: Ruijin Hospital Affiliated to Shanghai JiaoTong University School of Medicine/Hematology Department
state: Shanghai
city: Taiyuan
country: China
facility: Shanxi Medical University Second Hospital
state: Shanxi
city: Chengdu
country: China
facility: Chengdu Women's and Children's Central Hospital
state: Sichuan
city: Tianjin
country: China
facility: Blood Diseases Hospital, Chinese Academy of Medical Science (Institute of Hematology)
state: Tianjin
city: Kunming
country: China
facility: Department of Hematology,The First Affiliated Hospital of Kunming Medical University
state: Yunnan
city: Beijing
country: China
facility: Hematology Department,Beijing Children's Hospital, Capital Medical University
city: Chongqing
country: China
facility: Children's Hospital of Chongqing Medical University
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: Yang R, Wu R, Sun J, Sun F, Rupon J, Huard F, Korth-Bradley JM, Xu L, Luo B, Liu YC, Rendo P. First open-label, single-arm, prospective study of real-world use of FIX replacement therapy in a predominantly pediatric hemophilia B population in China. Medicine (Baltimore). 2021 May 28;100(21):e26077. doi: 10.1097/MD.0000000000026077.