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 InformationStart Date ICMJE | March 2009 |
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Estimated Primary Completion Date | April 2012 (final data collection date for primary outcome measure) |
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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 ] |
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Original Primary Outcome Measures ICMJE | Same as current |
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Change History | Complete list of historical versions of study NCT00851721 on ClinicalTrials.gov Archive Site |
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Descriptive InformationBrief Title ICMJE | 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 |
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Official Title ICMJE | FEIBA 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 |
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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. |
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Detailed Description | |
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Study Phase | Phase III |
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Study Type ICMJE | Interventional |
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Study Design ICMJE | Treatment, Randomized, Open Label, Uncontrolled, Parallel Assignment, Safety/Efficacy Study |
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Condition ICMJE | - Hemophilia A
- Hemophilia B
- (High-titer) Factor VIII Inhibitor
- (High-titer) Factor IX Inhibitor
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Intervention ICMJE | |
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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)
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Recruitment InformationEstimated Enrollment ICMJE | 34 |
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Estimated Completion Date | September 2012 |
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Estimated Primary Completion Date | April 2012 (final data collection date for primary outcome measure) |
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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
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Gender | Both |
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Ages | 4 Years to 65 Years |
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Accepts Healthy Volunteers | No |
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Contacts ICMJE | |
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Location Countries ICMJE | United States, Bulgaria, Croatia, France, Italy, Japan, New Zealand, Russian Federation |
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Administrative InformationNCT ID ICMJE | NCT00851721 |
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Responsible Party | Brigitt Abbuehl, MD; Study Medical Director, Baxter Healthcare Corporation |
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Study ID Numbers ICMJE | 090701 |
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Study Sponsor ICMJE | Baxter Healthcare Corporation |
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Collaborators ICMJE | |
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Investigators ICMJE | Study Director: | Brigitt Abbuehl, MD | Baxter Healthcare Corporation | |
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Information Provided By | Baxter Healthcare Corporation |
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