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

Pfizer

Status and phase: Completed; Phase 4

Conditions: HEMOPHILIA B

Treatments: Drug: Benefix

Study type: Interventional

Funder types: Industry

Identifiers

NCT02336178

B1821052

2016-000765-22 (EudraCT Number)

Details and patient eligibility

About

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.

Full 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%).

Enrollment: 70 patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

70 participants in 1 patient group

Benefix

Experimental group

Description:

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.

Treatment:

Drug: Benefix