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

NCT02213250 | Completed Phase 1 Results

• 2 other identifiers: B18210482015-003027-61
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

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.

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (10)

• Maximum Observed Plasma Concentration (Cmax)

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

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

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

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

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

• Time to Reach Cmax (Tmax)

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

• 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.

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

• 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.

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

• 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.

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

• Plasma Decay Half-Life (t½)

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

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

• Systemic Clearance (CL)

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

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

• Incremental Recovery

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

  Pre-dose, 0.25, 0.5 and 1 hour post-dose

Secondary Outcomes (6)

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

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

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

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

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

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

• Number of Participants With Inhibitor Development

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

• Number of Participants With Allergic Reactions

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

Study Arms (1)

BeneFIX

EXPERIMENTAL

Drug: BENEFIX

Interventions

BENEFIX DRUG

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

BeneFIX

Eligibility Criteria

• Age: 6 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Pfizer: lead

Study Sites (2)

Peking Union Medical College Hospital

Beijing, 100032, China

Location

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

Beijing, 100045, China

Location

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Hemophilia B

Interventions

Factor IX

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Enzyme Precursors; Enzymes and Coenzymes; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer, Inc

ClinicalTrials.gov_Inquiries@pfizer.com

(001)8007181021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 7, 2014
• First Posted: August 11, 2014
• Study Start: March 1, 2015
• Primary Completion: April 1, 2015
• Study Completion: April 1, 2015
• Last Updated: July 25, 2016
• Results First Posted: July 25, 2016

Record last verified: 2016-06

Locations

CN (2)