- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00866606
Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects
April 1, 2011 updated by: Wyeth is now a wholly owned subsidiary of Pfizer
An Evaluation Of The Safety And Efficacy Of On-Demand Treatment With BeneFIX (Nonacog Alfa, Recombinant Factor IX) In Chinese Subjects With Hemophilia B
This study will evaluate the safety and efficacy of on-demand treatment with BeneFIX in Chinese hemophilia B subjects.
Study Overview
Study Type
Interventional
Enrollment (Actual)
35
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Beijing, China, 100730
- Pfizer Investigational Site
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Shanghai, China, 200025
- Pfizer Investigational Site
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Guangzhou
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Guangzhou, Guangzhou, China, 510515
- Pfizer Investigational Site
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Jiangsu
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Suzhou, Jiangsu, China, 215006
- Pfizer Investigational Site
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Tianjin
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Tianjin, Tianjin, China, 300020
- Pfizer Investigational Site
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Zhejiang
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Hangzhou, Zhejiang, China, 310003
- Pfizer Investigational Site
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
6 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Subjects equal or more than 6 years of age with mild, moderate or severe hemophilia B (FIX activity: more than 5%, 1-5%, or less than 1%, respectively)
- Subjects with previous exposure to FIX replacement therapy
- If HIV positive, documented CD4 count more than 200/µL within 6 months of study entry
Exclusion Criteria:
- Diagnosed with any bleeding disorder in addition to hemophilia B
- Current FIX inhibitor or history of FIX inhibitor (defined as >ULN of the reporting laboratory)
- Subject has no history of exposure to FIX products (previously untreated patient [PUP])
- Subject is currently utilizing primary FIX prophylaxis
- Subjects anticipating elective surgery that may be planned to occur in the 6 months following study entry
- Treated with immunomodulatory therapy within 30 days prior to study entry or planned use for the duration of their study participation
- Participated in another investigational drug or device study within 30 days prior to study entry or planned participation for the duration of their study participation
- Subjects with a known hypersensitivity to hamster protein
- Significant hepatic or renal impairment (ALT and AST >5 x ULN, bilirubin >2 mg/dL or serum creatinine >1.25 x ULN)
- Prothrombin Time >1.5 x ULN
- Platelet count <80,000/µL
- Pregnant or breastfeeding women
- Unwilling or unable to follow the terms of the protocol
- Any condition which may compromise the subject's ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation, in the opinion of the Investigator or Sponsor
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Benefix
Subjects received on-demand treatments with BeneFIX over a 6-month (calendar day) period.
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BeneFIX for on-demand treatment of bleeding episodes were according to investigator prescription.
FIX recovery was assessed by determining the FIX activity (FIX:C) levels in individual subjects.
BeneFIX dosage for recovery assessments: single 75 IU/kg (±5 IU/kg) IV bolus infusion.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Investigator Hemostatic Efficacy Assessment of Participants After 8 Hours Post Infusion
Time Frame: 8 hours post infusion
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Investigator Hemostatic Efficacy Assessment was based on response of bleeding episodes to BeneFIX treatment on 4-point rating scale: Excellent(1): definite pain relief or improvement in signs of bleeding starting within 8 hrs after infusion, with no additional infusion; Good(2): definite pain relief or improvement in signs of bleeding starting within 8 hrs or following infusion; Moderate(3): probable or slight improvement starting after 8 hours following infusion; No Response(4): no improvement at all between infusions or during 24 hour interval following an infusion, or condition worsens.
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8 hours post infusion
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Investigator Hemostatic Efficacy Assessment of Participants After 24 Hours Post Infusion
Time Frame: 24 hours post infusion
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Investigator Hemostatic Efficacy Assessment was based on response of bleeding episodes to BeneFIX treatment on 4-point rating scale: Excellent(1): definite pain relief or improvement in signs of bleeding starting within 8 hrs after infusion, with no additional infusion; Good(2): definite pain relief or improvement in signs of bleeding starting within 8 hrs or following infusion; Moderate(3): probable or slight improvement starting after 8 hours following infusion; No Response(4): no improvement at all between infusions or during 24 hour interval following an infusion, or condition worsens.
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24 hours post infusion
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Percentage of Participants With FIX Inhibitor Development
Time Frame: Baseline up to 6 months
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Incidence of FIX inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory with Nijmegen assay result >=0.6 Bethesda Unit (BU).
Incidence was stratified by participant exposure history - Minimally Treated Patients (MTPs): those who had received at least one prior FIX infusion, and <= 100 documented Exposure Days (EDs); while Previously Treated Patients (PTPs): those who had received >100 documented prior EDs.
When number of prior EDs for an individual was not known to be at least 100, participants were included in the MTP population.
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Baseline up to 6 months
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Number of Infusions Required to Treat Each Bleed
Time Frame: Baseline up to 6 months
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The number of BeneFIX infusions required to treat each bleeding episode were analyzed.
The average frequency of BeneFIX infusions per hemorrhage incidence to treat every hemorrhage was equal to the total number of injections throughout the study divided by total number of hemorrhagic events.
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Baseline up to 6 months
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FIX Incremental Recovery
Time Frame: Baseline (Visit 2) up to 6 months (Visit 4)
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FIX recovery was assessed by evaluating FIX:C after initial exposure and following 6 months of repeated exposures to BeneFIX.
A modified FIX recovery study was performed at Day 1 (Visit 2) and Month 6/Final/Early Termination visits (Visit 4) and when clinically indicated at the applicable on-demand visits.
Blood samples for determination of FIX:C were collected immediately before BeneFIX infusion and at 30 minutes (±5 minutes) after the start of infusion.
Post-infusion blood samples were collected via venipuncture in arm contralateral to arm used for infusion.
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Baseline (Visit 2) up to 6 months (Visit 4)
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Percentage of Participants With Less Than Expected Therapeutic Effect (LETE)
Time Frame: Baseline up to 6 months
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The incidence of LETE for on-demand treatment was defined as no response after each of 2 successive infusions within 24 hours for the same bleeding event in the absence of confounding factors.
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Baseline up to 6 months
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Percentage of Participants With Allergic-Type Allergic Reactions
Time Frame: Baseline up to 6 months
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Hypersensitivity to undesirable (damaging, discomfort-producing and sometimes fatal) reactions produced by the normal immune system.
Hypersensitivity reactions require a pre-sensitized (immune) state of the host.
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Baseline up to 6 months
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Percentage of Participants With Thrombosis
Time Frame: Baseline up to 6 months
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Thrombosis is the formation of a blood clot (thrombus) inside a blood vessel, obstructing the flow of blood through the circulatory system.
When a blood vessel is injured, the body uses platelets and fibrin to form a blood clot to prevent blood loss.
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Baseline up to 6 months
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Percentage of Participants With Red Blood Cell (RBC) Agglutination
Time Frame: Baseline up to 6 months
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RBC Agglutination is the clumping of red blood cells in the presence of an antibody.
The antibody or other molecule bonded multiple particles and joined them, creating a large complex.
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Baseline up to 6 months
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
February 1, 2009
Primary Completion (Actual)
December 1, 2009
Study Completion (Actual)
December 1, 2009
Study Registration Dates
First Submitted
March 19, 2009
First Submitted That Met QC Criteria
March 19, 2009
First Posted (Estimate)
March 20, 2009
Study Record Updates
Last Update Posted (Estimate)
April 5, 2011
Last Update Submitted That Met QC Criteria
April 1, 2011
Last Verified
April 1, 2011
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 3090A1-3305
- B1821004
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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