Clinical trial NCT02213250: An Open-Label, Single Dose Pharmacokinetic Study of Benefix (Recombinant Factor IX) in Male Chinese Subjects With Hemophilia B
Official title: An Open-label, Single Dose Pharmacokinetic Study Of Benefix (Nonacog Alfa, Recombinant Factor Ix) In Male Chinese Subjects With Hemophilia B
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Hemophilia B
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2014-08-11
Enrollment: 12
Sex: MALE
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The sample size of 12 male Chinese subjects are based on the CFDA requirement for a China PK study and to support the registration in China.


Eligibility criteria: Inclusion Criteria:

* Male Chinese subjects 6 years or older (weight ≥20kg) with moderate to severe hemophilia B (Factor IX activity ≤2%).
* Subjects should not have received an 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 the administration of BeneFIX on Day 1.

Exclusion Criteria:

* Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies at time of dosing) disease or clinical findings at Screening.
* Diagnosed with any other bleeding disorder in addition to hemophilia B.
* Current FIX inhibitor or history of FIX inhibitor (defined as \> Upper Limit of Normal (ULN) of the reporting lab).

Primary outcomes:
measure: Maximum Observed Plasma Concentration (Cmax)
timeFrame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
measure: Area Under the Concentration Time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUClast)
timeFrame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
measure: Area Under the Concentration Time Curve From Time 0 to Infinity (AUCinf)
timeFrame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
measure: Time to Reach Cmax (Tmax)
timeFrame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
measure: Volume of Distribution at Steady State (Vss)
timeFrame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
measure: Terminal Phase Rate Constant (Kel)
timeFrame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
measure: Mean Residence Time (MRT)
timeFrame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
measure: Plasma Decay Half-Life (t½)
timeFrame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
measure: Systemic Clearance (CL)
timeFrame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose
measure: Incremental Recovery
timeFrame: Pre-dose, 0.25, 0.5 and 1 hour post-dose
Secondary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAE), Serious Adverse Events (SAE), and Withdrawals Due to Adverse Events (AE)
timeFrame: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.
measure: Number of Participants With Abnormal Clinical Laboratory Measurements (Without Regard to Baseline Abnormality)
timeFrame: Baseline up to 96 hours post-dose (Day 5 or early termination)
measure: Number of Participants With Vital Signs Post-Dose Data Met Criteria of Potential Clinical Concern (Without Regard to Baseline Abnormality)
timeFrame: Baseline up to 96 hours post-dose (Day 5 or early termination)
measure: Number of Participants With Inhibitor Development
timeFrame: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.
measure: Number of Participants With Allergic Reactions
timeFrame: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.
measure: Number of Subjects With Thrombogenicity
timeFrame: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking Union Medical College Hospital
city: Beijing
country: China
facility: Hematology Department,Beijing Children's Hospital, Capital Medical University
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: