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

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ClinicalTrials.gov Identifier: NCT02213250
Recruitment Status : Completed
First Posted : August 11, 2014
Results First Posted : July 25, 2016
Last Update Posted : July 25, 2016
Sponsor:
Information provided by (Responsible Party):
Pfizer

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.

Condition or disease Intervention/treatment Phase
Hemophilia B Drug: BENEFIX Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Single Dose Pharmacokinetic Study Of Benefix (Nonacog Alfa, Recombinant Factor Ix) In Male Chinese Subjects With Hemophilia B
Study Start Date : March 2015
Actual Primary Completion Date : April 2015
Actual Study Completion Date : April 2015

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: BeneFIX Drug: BENEFIX
Single dose of 50 IU/kg of BeneFIX by intravenous infusion within 10 minutes.




Primary Outcome Measures :
  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 Outcome Measures :
  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. ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02213250


Locations
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China
Peking Union Medical College Hospital
Beijing, China, 100032
Hematology Department,Beijing Children's Hospital, Capital Medical University
Beijing, China, 100045
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer

Additional Information:
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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT02213250     History of Changes
Other Study ID Numbers: B1821048
2015-003027-61 ( EudraCT Number )
First Posted: August 11, 2014    Key Record Dates
Results First Posted: July 25, 2016
Last Update Posted: July 25, 2016
Last Verified: June 2016
Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked