Efficacy and Safety Study of Prophylactic Versus On-Demand Treatment With Feiba NF in Subjects With Hemophilia A or B and a High Titer Inhibitor


Tracking Information

Start Date  ICMJEMarch 2009
Estimated Primary Completion DateApril 2012   (final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE 
 (submitted: February 25, 2009)
To demonstrate that the annualized rate of all types of bleeds in subjects on the prophylaxis arm is less than that of the subjects on the on-demand arm [ Time Frame: 1 year ] [ Designated as safety issue: No ]
Original Primary Outcome Measures  ICMJESame as current
Change HistoryComplete list of historical versions of study NCT00851721 on ClinicalTrials.gov Archive Site

Descriptive Information

Brief Title  ICMJEEfficacy and Safety Study of Prophylactic Versus On-Demand Treatment With Feiba NF in Subjects With Hemophilia A or B and a High Titer Inhibitor
Official Title  ICMJEFEIBA NF: A Prospective, Open-label, Randomized, Parallel Study to Evaluate the Efficacy and Safety of Prophylactic Versus On-Demand Treatment in Subjects With Hemophilia A or B and a High Titer Inhibitor
Brief Summary

The purpose of the study is to determine the efficacy, safety, and health-related quality of life benefits with FEIBA NF prophylactic treatment as compared with on-demand treatment. The study will be conducted globally and will consist of two arms. Eligible subjects will be randomized to undergo a 12-month period of either prophylactic or on-demand therapy.

Detailed Description 
Study PhasePhase III
Study Type  ICMJEInterventional
Study Design  ICMJETreatment, Randomized, Open Label, Uncontrolled, Parallel Assignment, Safety/Efficacy Study
Condition  ICMJE
  • Hemophilia A
  • Hemophilia B
  • (High-titer) Factor VIII Inhibitor
  • (High-titer) Factor IX Inhibitor
Intervention  ICMJE
  • Biological: Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)
    85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
    Other Name: FEIBA NF
  • Biological: Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)
    Standard FEIBA NF dose and dosing interval as prescribed by the treating physician
    Other Name: FEIBA NF
Study Arms / Comparison Groups
  • Prophylaxis arm: Experimental
    Intervention: Biological: Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)
  • On-demand arm: Active Comparator
    Intervention: Biological: Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)

Recruitment Information

Estimated Enrollment  ICMJE34
Estimated Completion DateSeptember 2012
Estimated Primary Completion DateApril 2012   (final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Signed and dated informed consent form by the subject or the subject's legally authorized representative
  • The subject is >= 4 to <= 65 years of age
  • The subject has a Karnofsky performance score of >= 60
  • Hemophilia A and B of any severity, with documented history of high-titer inhibitor (> 5 BU) for at least 12 months; or, if inhibitor titer is <= 5 BU, and the subject is refractory with increased dosing of either FVIII or FIX, as demonstrated from the subject's medical history
  • Currently being treated on an on-demand basis for treatment of bleeding episodes
  • Adequate venous access, with or without central venous device
  • >= 12 bleeding episodes requiring treatment with by-passing agents in the past 12 months, based on medical history
  • Competent in-home treatment and infusion therapy
  • Currently using bypassing agents (APCCs or rFVIIa) for treatment of bleeding episodes
  • HCV-, either by antibody testing or PCR; or HCV+ with stable hepatic disease
  • HIV-, or HIV+ with stable disease and CD4 count > 200 cells/mm3 at screening

Exclusion Criteria:

  • Currently receiving immune tolerance induction (ITI)
  • Currently on regular prophylactic therapy to prevent bleeding episodes
  • Clinically symptomatic liver disease (e.g. diagnosis of cirrhosis [confirmed by liver biopsy], portal vein hypertension, ascites, prothrombin time (PT) 5 seconds above upper limit of normal)
  • Platelet count < 100,000/ml
  • Planned elective surgery during participation in this study
  • Subject is currently participating in another clinical study and has received an investigational product or device within 30 days prior to study entry
  • Planned use of pegylated or non-pegylated alpha-interferon with or without ribavirin for HCV infected subjects or planned use of a protease inhibitor for HIV infected subjects. Subjects currently taking any of these medications for a 30-day course are eligible.
  • D-dimer levels twice the upper level of normal
  • Known hypersensitivity to AICCs
  • Currently treated with a systemic immunomodulating drug
  • Prior history of thromboembolic event: acute myocardial infarction, deep vein thrombosis, or pulmonary embolism
  • Diagnosis of advanced atherosclerosis, malignancy and/or other diseases that may increase the subject's risk of thromboembolic complications
  • Clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject safety or compliance
GenderBoth
Ages4 Years to 65 Years
Accepts Healthy VolunteersNo
Contacts  ICMJE 
Location Countries  ICMJEUnited States,   Bulgaria,   Croatia,   France,   Italy,   Japan,   New Zealand,   Russian Federation

Administrative Information

NCT ID  ICMJENCT00851721
Responsible PartyBrigitt Abbuehl, MD; Study Medical Director, Baxter Healthcare Corporation
Study ID Numbers  ICMJE090701
Study Sponsor  ICMJEBaxter Healthcare Corporation
Collaborators  ICMJE 
Investigators  ICMJE
Study Director:Brigitt Abbuehl, MDBaxter Healthcare Corporation
Information Provided ByBaxter Healthcare Corporation