Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects

This study has been completed.
Sponsor:
Information provided by:
Wyeth is now a wholly owned subsidiary of Pfizer
ClinicalTrials.gov Identifier:
NCT00866606
First received: March 19, 2009
Last updated: April 1, 2011
Last verified: April 2011
  Purpose

This study will evaluate the safety and efficacy of on-demand treatment with BeneFIX in Chinese hemophilia B subjects.


Condition Intervention Phase
Hemophilia B
Biological: Benefix
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Evaluation Of The Safety And Efficacy Of On-Demand Treatment With BeneFIX (Nonacog Alfa, Recombinant Factor IX) In Chinese Subjects With Hemophilia B

Resource links provided by NLM:


Further study details as provided by Wyeth is now a wholly owned subsidiary of Pfizer:

Primary Outcome Measures:
  • Investigator Hemostatic Efficacy Assessment of Participants After 8 Hours Post Infusion [ Time Frame: 8 hours post infusion ] [ Designated as safety issue: No ]
    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.

  • Investigator Hemostatic Efficacy Assessment of Participants After 24 Hours Post Infusion [ Time Frame: 24 hours post infusion ] [ Designated as safety issue: No ]
    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.

  • Percentage of Participants With FIX Inhibitor Development [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]
    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.


Secondary Outcome Measures:
  • Number of Infusions Required to Treat Each Bleed [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: No ]
    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.

  • FIX Incremental Recovery [ Time Frame: Baseline (Visit 2) up to 6 months (Visit 4) ] [ Designated as safety issue: No ]
    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.

  • Percentage of Participants With Less Than Expected Therapeutic Effect (LETE) [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: No ]
    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.

  • Percentage of Participants With Allergic-Type Allergic Reactions [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]
    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.

  • Percentage of Participants With Thrombosis [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]
    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.

  • Percentage of Participants With Red Blood Cell (RBC) Agglutination [ Time Frame: Baseline up to 6 months ] [ Designated as safety issue: Yes ]
    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.


Enrollment: 35
Study Start Date: February 2009
Study Completion Date: December 2009
Primary Completion Date: December 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Benefix
Subjects received on-demand treatments with BeneFIX over a 6-month (calendar day) period.
Biological: Benefix
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.

  Eligibility

Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00866606

Locations
China, Guangzhou
Pfizer Investigational Site
Guangzhou, Guangzhou, China, 510515
China, Jiangsu
Pfizer Investigational Site
Suzhou, Jiangsu, China, 215006
China, Tianjin
Pfizer Investigational Site
Tianjin, Tianjin, China, 300020
China, Zhejiang
Pfizer Investigational Site
Hangzhou, Zhejiang, China, 310003
China
Pfizer Investigational Site
Beijing, China, 100730
Pfizer Investigational Site
Shanghai, China, 200025
Sponsors and Collaborators
Wyeth is now a wholly owned subsidiary of Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided by Wyeth is now a wholly owned subsidiary of Pfizer

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Director, Clinical Trial Disclosure Group, Pfizer, Inc
ClinicalTrials.gov Identifier: NCT00866606     History of Changes
Other Study ID Numbers: 3090A1-3305, B1821004
Study First Received: March 19, 2009
Results First Received: December 14, 2010
Last Updated: April 1, 2011
Health Authority: China: Food and Drug Administration

Keywords provided by Wyeth is now a wholly owned subsidiary of Pfizer:
On-demand treatment
Hemostatic efficacy
Factor IX activity
Factor IX recovery
Factor IX inhibitor
Chinese

Additional relevant MeSH terms:
Hemophilia B
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hematologic Diseases
Hemorrhagic Disorders

ClinicalTrials.gov processed this record on October 23, 2014