A single dose study of BeneFIX (nonacog alfa, recombinant factor IX) in male Chinese subjects with haemophilia B.

Active, not recruiting

• Conditions: Haemophilia B; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2015-003027-61-Outside-EU/EEA
• Lead Sponsor: Pfizer Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-12-23
• Last Update Posted: 2015-12-28

Recruitment & Eligibility

• Status: Active
• Sex: Male
• Target Recruitment: 12

Inclusion Criteria

Subjects must meet all of the following criteria;
•Male Chinese subjects 6 years and older (weight =20 kg) with moderate to severe hemophilia B (Factor IX activity =2%)
•Subjects should not have received infusion of any Factor IX products for at least 4 days before the administration of BeneFIX on Day 1
•Subjects must be in a non-bleeding state before BeneFIX administration on Day 1
•Evidence of personally or legally representative (legal only for pediatric subjects) signed and dated informed consent documentation indicating that the subject has been informed of all pertinent aspects of the study
•Subjects who are willing and able to comply with scheduled visits, treatment plans, laboratory tests and other trial procedures.

Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 3
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 3

Exclusion Criteria

Subjects presenting with any of the following will not be included in the study
•Evidence or history of clinically significant haematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic or allergic (including drug allergies) disease or clinical findings at Screening.
•Diagnosed with any other bleeding disorder in addition to Haemophilia B
•Documented Human Immunodeficiency Virus positive patients
•Current FIX inhibitor or history of FIX inhibitor (defined as =Upper Limit of Normal (ULN) of the reporting lab)
•Subjects anticipating elective surgery that may be planned to occur in the 1 month following study entry
•Treated with immunomodulatory therapy within 30 days
•Treated with an investigational drug within 30 days or 5 half-lives preceding the first dose of the study drug
•Subjects with known hypersensitivity to the active substance or to any of the excipients of BeneFIX
•Subjects with known hypersensitivity to Chinese Hamster Ovary cell (CHO cell) proteins
•Subjects with any of hepatic or renal impairment (ALT or AST >3x Upper Limit of Normal (ULN), total bilirubin >2x ULN, serum creatinine >2x ULN), Prothrombin Time >1.5x ULN, Platelet count <80,000 uL
•Unwilling or unable to follow the terms of the protocol
•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, in the opinion of the Investigator or Sponsor
•A positive urine drug screen
•History of regular alcohol consumption exceeding 14 drinks/week for men within 6 months of screening
•Screening supine blood pressure =140 mm Hg (systolic) or =90 mm Hg (diastolic) following at least 5 minutes rest
•12-lead ECG showing QTc >450 msec or QRS >120 msec at screening
•Blood donation of approximately 1 pint within 56 days prior to dosing
•History of sensitivity to heparin or heparin-induced thrombocytopenia
•Unwilling or unable to comply with the Lifestyle Guidelines
•Other severe acute or chronic medical or psychiatric conditions or laboratory abnormality that may increase risk associated with study participation.

Study & Design

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