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An Open-Label, Single Dose Pharmacokinetic Study of Benefix (Recombinant Factor IX) in Male Chinese Subjects With Hemophilia B

Primary Purpose

Hemophilia B

Status

Completed

Phase

Phase 1

Locations

China

Study Type

Interventional

Intervention

BENEFIX

About this trial

This is an interventional treatment trial for Hemophilia B

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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).

Sites / Locations

Peking Union Medical College Hospital
Hematology Department,Beijing Children's Hospital, Capital Medical University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

BeneFIX

Arm Description

Outcomes

Primary Outcome Measures

Maximum Observed Plasma Concentration (Cmax)

Area Under the Concentration Time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUClast)

Area Under the Concentration Time Curve From Time 0 to Infinity (AUCinf)

Time to Reach Cmax (Tmax)

Volume of Distribution at Steady State (Vss)

Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. Vss is the apparent volume of distribution at steady-state.

Terminal Phase Rate Constant (Kel)

Linear regression of the log linear concentration time curve. Only those data points judged to describe the terminal log linear decline were used in the regression.

Mean Residence Time (MRT)

AUMCinf/AUCinf, where AUMCinf is the area under the first moment curve from time 0 extrapolated to infinite time, calculated using the linear/log trapezoidal method.

Plasma Decay Half-Life (t½)

Plasma decay half-life is the time measured for the plasma concentration to decrease by one half.

Systemic Clearance (CL)

CL is a quantitative measure of the rate at which a drug substance is removed from the body.

Incremental Recovery

Incremental recovery: Increase in circulating increase in FIX activity for every IU of BeneFIX administered per kg of body weight.

Secondary Outcome Measures

Number of Participants With Treatment-Emergent Adverse Events (TEAE), Serious Adverse Events (SAE), and Withdrawals Due to Adverse Events (AE)

An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; lifethreatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. TEAE is defined as newly occurring or worsening after first dose.

Number of Participants With Abnormal Clinical Laboratory Measurements (Without Regard to Baseline Abnormality)

Clinical laboratory analysis tests included hematology, serium chemistry, prothrombin time and urianalysis. Numbers of subjects with laboratory test abnormalities without regard to baseline abnormality were reported.

Number of Participants With Vital Signs Post-Dose Data Met Criteria of Potential Clinical Concern (Without Regard to Baseline Abnormality)

Number of Participants With Inhibitor Development

Number of Participants With Allergic Reactions

Number of Subjects With Thrombogenicity

Full Information

NCT ID

NCT02213250

First Posted

August 7, 2014

Last Updated

June 13, 2016

Sponsor

Pfizer

1. Study Identification

Unique Protocol Identification Number

NCT02213250

Brief Title

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

2. Study Status

Record Verification Date

June 2016

Overall Recruitment Status

Completed

Study Start Date

March 2015 (undefined)

Primary Completion Date

April 2015 (Actual)

Study Completion Date

April 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Pfizer

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hemophilia B

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

12 (Actual)

8. Arms, Groups, and Interventions

Arm Title

BeneFIX

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

BENEFIX

Intervention Description

Single dose of 50 IU/kg of BeneFIX by intravenous infusion within 10 minutes.

Primary Outcome Measure Information:

Title

Maximum Observed Plasma Concentration (Cmax)

Time Frame