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

NCT ID: NCT02336178

Last Updated: 2017-05-04

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 70 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-01-31
• Study Completion Date: 2016-08-31

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%).

Conditions

HEMOPHILIA B

Study Design

• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Benefix

This 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.

Group Type: EXPERIMENTAL

Benefix

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

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.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pfizer

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

Nanfang Hospital, Southern Medical University

Guangzhou, Guangdong, China

The Affiliated Hospital of Guizhou Medical University

Guiyang, Guizhou, China

Henan Provincial People's Hospital

Zhengzhou, Henan, China

Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology

Wuhan, Hubei, China

Xiangya Hospital of Centre-South University

Changsha, Hunan, China

Department of Hematology,The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, China

Department of Hematology,The Affiliated Hospital of Xuzhou Medical University

Xuzhou, Jiangsu, China

Department of Hematology,Jiangxi Provincial People's Hospital

Nanchang, Jiangxi, China

Blood Center of Shandong Province

Jinan, Shandong, China

Ruijin Hospital Affiliated to Shanghai JiaoTong University School of Medicine/Hematology Department

Shanghai, Shanghai Municipality, China

Shanxi Medical University Second Hospital

Taiyuan, Shanxi, China

Chengdu Women's and Children's Central Hospital

Chengdu, Sichuan, China

Blood Diseases Hospital, Chinese Academy of Medical Science (Institute of Hematology)

Tianjin, Tianjin Municipality, China

Department of Hematology,The First Affiliated Hospital of Kunming Medical University

Kunming, Yunnan, China

Hematology Department,Beijing Children's Hospital, Capital Medical University

Beijing, , China

Children's Hospital of Chongqing Medical University

Chongqing, , China

Countries

China

References

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.

Reference Type: DERIVED

PMID: 34032739 (View on PubMed)

Related Links

https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=B1821052&StudyName=Safety%20and%20Efficacy%20of%20Benefix%20%28nonacog%20Alfa%2C%20Recombinant%20%20Factor%20Ix%29%20in%20Patients%20with%20Hemophilia%20B%20in%20Usual%20Care%20Settings%20in%20

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Other Identifiers

2016-000765-22

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

B1821052

Identifier Type: -

Identifier Source: org_study_id