Severe or moderately severe hemophilia A with a residual factor FVIII activity less than or equal 2%
Conditions
Interventions
Trade Name: Advate
Product Name: Advate
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Octocog alfa
Sponsors
Baxter AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. The subject or the subject’s legally authorized representative has provided written informed consent. 2. The subject has severe or moderately severe hemophilia A as defined by a baseline factor VIII level less than or equal 2% of normal, as tested at screening. 3. The subject has a documented history of at least 150 exposure days to factor VIII concentrates (either plasma-derived or recombinant). 4. The subject is within 7 to 65 years of age. 5. The subject has a Karnofsky performance score > 60. 6. The subject is human immunodeficiency virus negative (HIV-) or is HIV+ with a stable CD4 count more than or equal 400 cells/mm³ (CD4 count determined at screening, if necessary). 7. The subject has been on a documented on-demand treatment regimen for at least 12 months immediately prior to enrollment. 8. The subject has a documented history (e.g. in medical charts, dispensing information or signed investigator statement) of at least 8 joint hemorrhages in the 12 months immediately prior to enrollment. 9. The subject resides within the coverage area of the mobile compliance device; coverage area will be determined at screening. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. The subject has a known hypersensitivity to factor VIII concentrates or mouse or hamster proteins. 2. The subject has a history of factor VIII inhibitors with a titer more than or equal 0.6 BU (by Bethesda or Nijmegen assay) at any time prior to screening. 3. The subject has a detectable factor VIII inhibitor at screening, with a titer more than or equal 0.4 BU (by Nijmegen Assay) in the central laboratory. 4. 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. 5. 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). 6. The subject has been treated during the last sixty (60) days prior to or is being treated at screening/enrollment with an immunomodulating drug. 7. The subject has participated in another investigational study within 30 days of enrollment. 8. The subject has previously participated in a clinical study with ADVATE rAHF PFM. 9. The subject’s clinical condition may require a major surgery (defined as moderate to critical risk and perioperative blood loss more than or equal 500 mL) during the period of the subject’s participation in the study. 10. The subject is female of childbearing potential with a positive pregnancy test.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 1. To compare the rate of bleeding episodes for standard prophylactic regimen (20-40 IU/kg every 48 hours +/- 6 hours) with that of alternate prophylactic regimen (20-80 IU/kg every 72 hours +/- 6 hours) ; Secondary Objective: 1. To compare the rate of bleeding episodes between the on-demand regimen and the prophylactic regimens 2. To compare the total weight adjusted consumption of ADVATE rAHF PFM on each regimen 3. To determine the efficacy of ADVATE rAHF PFM in the control of bleeding episodes throughout the study 4. To determine the pharmacokinetic parameters for ADVATE rAHF PFM utilizing at least 3 lots of the product 5. To determine the immunogenicity of ADVATE rAHF PFM 6. To determine the safety and toxicity of ADVATE rAHF-PFM 7. To determine differences in Health-Related Quality of Life (HRQoL) between the 2 prophylactic regimens and changes between the on-demand treatment and prophylactic regimens. ;Primary end point(s): Yearly transformed rate of bleeding episodes estimated from each arm of the one year prophylactic regimen | — |
Countries
Austria, Czech Republic, Hungary, Italy, Slovenia, United Kingdom
Outcome results
None listed