Brief Title
Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A
Official Title
Advate Antihemophilic Factor (Recombinant), Plasma/Albumin Free Method (ADVATE rAHF-PFM): A Phase 4 Study Comparing Two Prophylactic Regimens in Subjects With Severe or Moderately Severe Hemophilia A
Brief Summary
The primary purpose of this randomized, two-arm parallel clinical study in 66 previously treated patients with severe or moderately severe hemophilia A is to compare the rate of bleeding episodes for standard prophylaxis (20-40 IU/kg every 48 ± 6 hours; actual dose determined by the investigator) with that of alternate prophylaxis (20-80 IU/kg every 72 + 6 hours; actual dose determined by Baxter utilizing an algorithm and the patient's pharmacokinetic data). The rates of bleeding episodes for the on-demand regimen and the prophylaxis regimens will also be compared for the cross-over portion of the study. Enrolled patients will be treated originally on demand for a period of 6 months and then they will be randomized into one of the prophylaxis arms. Prophylactic treatment will last for a period of 12 months +/- 2 weeks.
Study Phase
Phase 4
Study Type
Interventional
Primary Outcome
Mean Transformed Annualized Bleed Rate Estimates From Each of the 1-year Prophylaxis Regimens
Secondary Outcome
Mean Difference of Transformed Annualized Bleeding Rate Between On-Demand and Standard Prophylaxis Treatment Regimens
Condition
Hemophilia A
Intervention
Antihemophilic factor, recombinant, manufactured protein-free
Study Arms / Comparison Groups
1
Description: Standard prophylaxis
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
82
Start Date
January 4, 2006
Completion Date
June 16, 2010
Primary Completion Date
June 16, 2010
Eligibility Criteria
Inclusion Criteria: - The subject has severe or moderately severe hemophilia A as defined by a baseline factor VIII level <= 2% of normal, as tested at screening - The subject has a documented history of at least 150 exposure days to factor VIII concentrates (either plasma-derived or recombinant) - The subject is within 7 to 65 years of age - The subject has a Karnofsky performance score > (greater than) 60 - The subject is human immunodeficiency virus negative (HIV-) or is HIV+ with a CD4 count >= 400 cells/mm³ (CD4 count determined at screening, if necessary) - The subject has been on a documented on-demand treatment regimen for at least 12 months immediately prior to enrollment - The subject has a documented history (e.g. in medical charts or dispensing information, or signed investigator statement) of at least 8 joint hemorrhages in the 12 months immediately prior to enrollment - The subject resides within the coverage area of the mobile compliance device; coverage area will be determined at screening - The subject or the subject's legally authorized representative has provided written informed consent Exclusion Criteria: - The subject has a known hypersensitivity to factor VIII concentrates or mouse or hamster proteins - The subject has a history of factor VIII inhibitors with a titer >= 0.6 BU (by Bethesda or Nijmegen assay) at any time prior to screening - The subject has a detectable factor VIII inhibitor at screening, with a titer >= 0.4 BU (by Nijmegen Assay) in the central laboratory - The subject has severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio (INR) > 1.4, hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices. - The subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (e.g., qualitative platelet defect or von Willebrand's Disease) - The subject has been treated during the last sixty (60) days prior to or is being treated at screening/enrollment with an immunomodulating drug. - The subject has participated in another investigational study within thirty (30) days of enrollment - The subject has previously participated in a clinical study with rAHF-PFM - The subject's clinical condition may require a major surgery (defined as moderate to critical risk and perioperative blood loss ≥ 500 mL) during the period of the subject's participation in the study - The subject is female of childbearing potential with a positive pregnancy test
Gender
All
Ages
7 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Study Director, ,
Location Countries
Austria
Location Countries
Austria
Administrative Informations
NCT ID
NCT00243386
Organization ID
060201
Responsible Party
Sponsor
Study Sponsor
Baxalta now part of Shire
Study Sponsor
Study Director, Study Director, Takeda
Verification Date
April 2021