Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China
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
2 other identifiers
interventional
70
1 country
16
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_4
Started Jan 2015
16 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 16, 2014
CompletedStudy Start
First participant enrolled
January 1, 2015
CompletedFirst Posted
Study publicly available on registry
January 12, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2016
CompletedResults Posted
Study results publicly available
May 4, 2017
CompletedMay 4, 2017
March 1, 2017
1.5 years
December 16, 2014
January 9, 2017
March 23, 2017
Conditions
Outcome Measures
Primary Outcomes (3)
Percentage of Participants Who Developed Factor IX Inhibitor
Factor IX (FIX) inhibitor development was defined as any Bethesda inhibitor titer greater than the laboratory's normal range or Bethesda inhibitor titer \>=0.6 Bethesda Unit (BU)/mL.
Up to 6 months
Number of Participants With Allergic Reactions
FIX product allergy was defined as a hypersensitivity reaction to a FIX product with symptoms such as hives, urticaria, tightness of chest, wheezing, hypotension, and anaphylaxis based on investigator's judgments.
Up to 6 months
Number of Participants With Thrombotic Events
Thrombotic event was defined as any event associated with formation of a blood clot including catheter-associated thrombi and thrombotic complications.
Up to 6 months
Secondary Outcomes (11)
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Up to 7 months (28 calendar days after end of 6-month or 50-exposure day study treatment)
Annualized Bleeding Rates (ABRs) in Participants Receiving Prophylaxis Treatment With BeneFIX During Their Prophylaxis Period
Up to 6 months or 50 exposure days whichever occurred first
Number of Spontaneous/Non-traumatic Breakthrough Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX
Up to 6 months or 50 exposure days whichever occurred first
Annualized Bleeding Rates (ABRs) in Participants Receiving On-demand Treatment With BeneFIX During Their On-demand Period
Up to 6 months or 50 exposure days whichever occurred first
Number of Infusions Resulted in the Following Response to On-demand Treatment of Bleeds: Excellent, Good, Moderate, no Response
Up to 6 months or 50 exposure days whichever occurred first
- +6 more secondary outcomes
Study Arms (1)
Benefix
EXPERIMENTALThis is a single arm study. Subjects will be treated with Benefix by the investigator according to usual care in China and in accord with the China BeneFIX Package Insert.
Interventions
Subjects will be treated by the investigator according to usual care in China and in accord with the China BeneFIX Package Insert. The treatment duration is approximately 6 months (±7 days) or approximately 50 Exposure Days (EDs) (±5 EDs) (see protocol definition on EDs) whichever occurs first.
Eligibility Criteria
You may qualify if:
- 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)
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Pfizerlead
Study Sites (16)
Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510515, China
The Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou, 550004, China
Henan Provincial People's Hospital
Zhengzhou, Henan, 450003, China
Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology
Wuhan, Hubei, 430030, China
Xiangya Hospital of Centre-South University
Changsha, Hunan, 410008, China
Department of Hematology,The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215006, China
Department of Hematology,The Affiliated Hospital of Xuzhou Medical University
Xuzhou, Jiangsu, 221006, China
Department of Hematology,Jiangxi Provincial People's Hospital
Nanchang, Jiangxi, 330006, China
Blood Center of Shandong Province
Jinan, Shandong, 250014, China
Ruijin Hospital Affiliated to Shanghai JiaoTong University School of Medicine/Hematology Department
Shanghai, Shanghai Municipality, 200025, China
Shanxi Medical University Second Hospital
Taiyuan, Shanxi, 030001, China
Chengdu Women's and Children's Central Hospital
Chengdu, Sichuan, 610073, China
Blood Diseases Hospital, Chinese Academy of Medical Science (Institute of Hematology)
Tianjin, Tianjin Municipality, 300020, China
Department of Hematology,The First Affiliated Hospital of Kunming Medical University
Kunming, Yunnan, 650032, China
Hematology Department,Beijing Children's Hospital, Capital Medical University
Beijing, 100045, China
Children's Hospital of Chongqing Medical University
Chongqing, 400014, China
Related Publications (1)
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.
PMID: 34032739DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Pfizer ClinicalTrials.gov Call Center
- Organization
- Pfizer, Inc.
Study Officials
- STUDY DIRECTOR
Pfizer CT.gov Call Center
Pfizer
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Masking
- NONE
- Purpose
- TREATMENT
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 16, 2014
First Posted
January 12, 2015
Study Start
January 1, 2015
Primary Completion
July 1, 2016
Study Completion
August 1, 2016
Last Updated
May 4, 2017
Results First Posted
May 4, 2017
Record last verified: 2017-03