Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China

HEMOPHILIA B Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02336178
• Other study ID #: B1821052
• Secondary ID: 2016-000765-22
• Status: Completed
• Phase: Phase 4
• First received: December 16, 2014
• Last updated: November 28, 2016
• Start date: January 2015
• Est. completion date: August 2016

Study information

• Verified date: November 2016
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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%).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 70
• Est. completion date: August 2016
• Est. primary completion date: July 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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.

Study Design

Endpoint Classification: Safety/Efficacy Study, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Drug:
• Benefix
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.

Locations

Country	Name	City	State
China	Hematology Department,Beijing Children's Hospital, Capital Medical University	Beijing
China	Xiangya Hospital of Centre-South University	Changsha	Hunan
China	Chengdu Women's and Children's Central Hospital	Chengdu	Sichuan
China	Children's Hospital of Chongqing Medical University	Chongqing
China	Nanfang Hospital, Southern Medical University	Guangzhou	Guangdong
China	Affiliated Hospital of Guiyang Medical College/Hematology Department	Guiyang	Guizhou
China	Blood Center of Shandong Province	Jinan	Shandong
China	Department of Hematology,The First Affiliated Hospital of Kunming Medical University	Kunming	Yunnan
China	Department of Hematology,Jiangxi Provincial People's Hospital	Nanchang	Jiangxi
China	Ruijin Hospital Affiliated to Shanghai JiaoTong University School of Medicine/Hematology Department	Shanghai	Shanghai
China	Department of Hematology,The First Affiliated Hospital of Soochow University	Suzhou	Jiangsu
China	Shanxi Medical University Second Hospital	Taiyuan	Shanxi
China	Blood Diseases Hospital, Chinese Academy of Medical Science (Institute of Hematology)	Tianjin	Tianjin
China	Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology	Wuhan	Hubei
China	Department of Hematology,The Affiliated Hospital of Xuzhou Medical College	Xuzhou	Jiangsu
China	Henan Provincial People's Hospital	Zhengzhou	Henan

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	FIX inhibitor development		up to approximately 7months	Yes
Primary	allergic reactions		up to approximately 7months	Yes
Primary	thrombotic events		up to approximately 7months	Yes
Secondary	the overall safety of BeneFIX, including the occurrence of Adverse Events (AEs) and Serious Adverse Events (SAEs)		up to approximately 7months	Yes
Secondary	Annualized bleeding rates (ABR) in subjects receiving prophylaxis treatment with BeneFIX		up to 6months or 50 exposure days which occurs first	No
Secondary	Number of spontaneous/non traumatic breakthrough bleeds within 48 hours of a prophylaxis dose of BeneFIX.		up to 6months or 50 exposure days which occurs first	No
Secondary	The response to on-demand treatment with BeneFIX for all bleeds (4-point scale of assessment, one per infusion)		up to 6months or 50 exposure days which occurs first	No
Secondary	Number of BeneFIX infusions to treat each new bleed.		up to 6months or 50 exposure days which occurs first	No
Secondary	The average infusion dose and total factor IX consumption		up to 6months or 50 exposure days which occurs first	No
Secondary	• The incidence of less than expected therapeutic effect (LETE) in the on-demand, prophylaxis, and low recovery (when available) settings		up to 6months or 50 exposure days which occurs first	No

External Links

•   To obtain contact information for a study center near you, click here.