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NCT Number: NCT02213250

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

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.

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Key information

Conditions

Hemophilia B Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

6 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1

Primary location

Hematology Department,Beijing Children's Hospital, Capital Medical University, Beijing, China

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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).

Treatment and study plan

BENEFIX

Drug

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

Primary outcomes

  1. Maximum Observed Plasma Concentration (Cmax)

    Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose

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

    Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose

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

    Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose

  4. Time to Reach Cmax (Tmax)

    Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose

  5. Volume of Distribution at Steady State (Vss)

    Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose

    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.

  6. Terminal Phase Rate Constant (Kel)

    Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose

    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.

  7. Mean Residence Time (MRT)

    Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose

    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.

  8. Plasma Decay Half-Life (t½)

    Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose

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

  9. Systemic Clearance (CL)

    Time frame: Pre-dose, 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72 and 96 hours post-dose

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

  10. Incremental Recovery

    Time frame: Pre-dose, 0.25, 0.5 and 1 hour post-dose

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

Secondary outcomes

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

    Time frame: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.

    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.

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

    Time frame: Baseline up to 96 hours post-dose (Day 5 or early termination)

    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.

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

    Time frame: Baseline up to 96 hours post-dose (Day 5 or early termination)

  4. Number of Participants With Inhibitor Development

    Time frame: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.

  5. Number of Participants With Allergic Reactions

    Time frame: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.

  6. Number of Subjects With Thrombogenicity

    Time frame: From the subject provided informed consent through and including 28 calendar days after the last administration of the study drug.

Sponsors and collaborators

Lead sponsor

Pfizer

Industry

Registry information

Official study title

An Open-label, Single Dose Pharmacokinetic Study Of Benefix (Nonacog Alfa, Recombinant Factor Ix) In Male Chinese Subjects With Hemophilia B

Important dates

Study start
2015
Primary completion
2015
Study completion
2015
First posted
Aug 11, 2014
Registry last updated
Jul 25, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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