Safety and efficacy of BeneFIX, nonacog Alfa (Recombinant Factor IX), in Chinese subjects with Hemophilia B

Phase 1

• Conditions: Haemophilia B; MedDRA version: 19.1Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2016-000765-22-Outside-EU/EEA
• Lead Sponsor: Pfizer, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-01-11
• Last Update Posted: 16 January 2017

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

1. Male and/or female subjects with hemophilia B.
2. 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).
3. Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative, parent(s)/legal guardian) has been informed of all pertinent aspects of the study.

Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Presence of any other bleeding disorder in addition to haemophilia 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.
2. Subjects with known hypersensitivity to the active substance or to any of the excipients of BeneFIX.
3. Subjects with a known hypersensitivity to Chinese Hamster Ovary cell proteins.
4. Unwilling or unable to follow the terms of the protocol.
5. Any condition which may compromise the subject’s ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; expectation of poor compliance in provision of observations for study-related documentation), in the opinion of the Investigator.
6. Participation in other studies involving investigational drug(s) (Phases 1-4) within 30 days before the current study begins (exception for studies on BeneFIX) and/or during study participation.
7. Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, or subjects who are Pfizer employees directly involved in the conduct of the study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary endpoint is to evaluate the product medically important events (FIX inhibitor development, allergic reactions, thrombotic events) in Chinese haemophilia B subjects during treatment with BeneFIX;Secondary Objective: To evaluate the overall safety of BeneFIX, including the occurrence of AEs and SAEs and the efficacy endpoints.<br>;Primary end point(s): To evaluate the product medically important events (FIX inhibitor development, allergic reactions, thrombotic events) in Chinese haemophilia B subjects during treatment with BeneFIX. ;Timepoint(s) of evaluation of this end point: During the course of the study i.e. from day 1 to the last visit.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): To evaluate the overall safety of BeneFIX, including the occurrence of AEs and SAEs and the efficacy endpoints.;Timepoint(s) of evaluation of this end point: During the course of the study i.e. from day 1 to the last visit