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Pharmacokinetic Comparison of Advate rAHF-PFM With Recombinate rAHF in Patients With Severe Hemophilia A

Phase 4
Completed
Conditions
Hemophilia A
Interventions
Drug: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM)
Drug: Recombinant Factor VIII (rAHF)
Registration Number
NCT00666406
Lead Sponsor
Baxalta now part of Shire
Brief Summary

The purpose of this study is to compare the pharmacokinetic parameters and safety of Advate rAHF-PFM versus Recombinate rAHF in well described previously treated patients with severe hemophilia A (factor VIII level \< 1%).

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
9
Inclusion Criteria
  • Signed informed consent obtained from participant or legally authorized representative
  • 15-60 years old
  • Factor VIII level < 1% as documented by previously measured factor VIII and genotyping
  • Previously treated with factor VIII concentrate(s) for a minimum of at least 150 exposure days (as documented by the study site investigator) prior to study entry
  • Observed decrease of efficacy by subject and/or treating physician after being switched from Recombinate rAHF to Advate rAHF-PFM
Exclusion Criteria
  • The participant has a detectable factor VIII inhibitor at screening, with a titer >= 0.4 Bethesda Unit (BU) (Nijmegen modification of the Bethesda Assay) measured at the local and the central laboratory
  • The participant has a known hypersensitivity to mouse or hamster proteins
  • The participant is participating in another investigational drug study within 30 days prior to screening
  • The participant is identified by the investigator as being unable or unwilling to cooperate with study procedures

Study & Design

Study Type
INTERVENTIONAL
Study Design
CROSSOVER
Arm && Interventions
GroupInterventionDescription
1Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM)Advate rAHF-PFM
2Recombinant Factor VIII (rAHF)Recombinate rAHF
Primary Outcome Measures
NameTimeMethod
Area Under the Plasma Concentration Versus Time Curve (AUC) From 0 to 48 Hours. Chromogenic Assay Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

AUC estimated by linear trapezoidal method. The linear trapezoidal method is a numerical method used to approximate the area under a curve.

Area Under the Plasma Concentration Versus Time Curve (AUC) From 0 to 48 Hours. FVIII Clotting Assay. Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

AUC estimated by linear trapezoidal method. The linear trapezoidal method is a numerical method used to approximate the area under a curve.

Area Under the Plasma Concentration Versus Time Curve (AUC) From 0 to 48 Hours. One-Stage Activated Partial Thromboplastin Time (aPTT) -Based Assay Performed at Central Laboratory (Medical University Vienna)0-30 minutes before infusion up to 48 hours post-infusion

AUC estimated by linear trapezoidal method. The linear trapezoidal method is a numerical method used to approximate the area under a curve.

Area Under the Plasma Concentration Versus Time Curve (AUC) From 0 to 48 Hours. FVIII One-Stage Clotting Assay (Bonn Method) Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

AUC estimated by linear trapezoidal method. The linear trapezoidal method is a numerical method used to approximate the area under a curve.

Secondary Outcome Measures
NameTimeMethod
Maximum Plasma Concentration (C-max). Chromogenic Assay Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

C-max will be calculated as the maximum concentration following infusion of either Advate or Recombinate.

Area Under the Plasma Concentration Versus Time Curve (AUC) From 0 to Infinity. Chromogenic Assay Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

AUC estimated by linear trapezoidal method. The linear trapezoidal method is a numerical method used to approximate the area under a curve.

Systemic Clearance (Cl). Chromogenic Assay Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Systemic clearance in mL/kg/h will be calculated as the dose in IU/kg divided by the total area under the curve.

Area Under the Plasma Concentration Versus Time Curve (AUC) From 0 to Infinity. FVIII Clotting Assay. Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

AUC estimated by linear trapezoidal method. The linear trapezoidal method is a numerical method used to approximate the area under a curve.

Maximum Plasma Concentration (C-max). FVIII One-Stage Clotting Assay (Bonn Method) Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

C-max will be calculated as the maximum concentration following infusion of either Advate or Recombinate.

Terminal Half-life. Chromogenic Assay Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Computed from the terminal or disposition rate constant obtained from log-linear fitting using the least squares deviation to the last five quantifiable concentrations.

Incremental Recovery. One-Stage aPTT-Based Assay Performed at Central Laboratory (Medical University Vienna)0-30 minutes before infusion up to 48 hours post-infusion

Increase in factor VIII concentration from pre- to post-infusion.

Mean Residence Time (MRT). FVIII Clotting Assay. Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

The MRT in hours will be calculated as total area under the moment curve divided by the total area under the curve.

Time to Reach the Maximum Plasma Concentration (Tmax). One-Stage aPTT-Based Assay Performed at Central Laboratory (Medical University Vienna)0-30 minutes before infusion up to 48 hours post-infusion

Tmax in hours was defined as the minimum time to reach Maximum plasma concentration (Cmax).

Volume of Distribution at Steady State (Vss). Chromogenic Assay Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Computed as weight-adjusted Clearance (CL) \* Mean Residence Time

Area Under the Plasma Concentration Versus Time Curve (AUC) From 0 to Infinity. One-Stage aPTT-Based Assay Performed at Central Laboratory (Medical University Vienna)0-30 minutes before infusion up to 48 hours post-infusion

AUC estimated by linear trapezoidal method. The linear trapezoidal method is a numerical method used to approximate the area under a curve. FVIII activity measurement

Systemic Clearance (Cl). One-Stage aPTT-Based Assay Performed at Central Laboratory (Medical University Vienna)0-30 minutes before infusion up to 48 hours post-infusion

Systemic clearance in mL/kg/h will be calculated as the dose in IU/kg divided by the total area under the curve.

Systemic Clearance (Cl). FVIII Clotting Assay. Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Systemic clearance in mL/kg/h will be calculated as the dose in IU/kg divided by the total area under the curve.

Maximum Plasma Concentration (C-max). One-Stage aPTT-Based Assay Performed at Central Laboratory (Medical University Vienna)0-30 minutes before infusion up to 48 hours post-infusion

C-max will be calculated as the maximum concentration following infusion of either Advate or Recombinate.

Systemic Clearance (Cl). FVIII One-Stage Clotting Assay (Bonn Method) Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Systemic clearance in mL/kg/h will be calculated as the dose in IU/kg divided by the total area under the curve.

Terminal Half-life. FVIII One-Stage Clotting Assay (Bonn Method) Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Computed from the terminal or disposition rate constant obtained from log-linear fitting using the least squares deviation to the last five quantifiable concentrations.

Mean Residence Time (MRT). Chromogenic Assay Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

The MRT in hours will be calculated as total area under the moment curve divided by the total area under the curve.

Mean Residence Time (MRT). FVIII One-Stage Clotting Assay (Bonn Method) Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

The MRT in hours will be calculated as total area under the moment curve divided by the total area under the curve.

Time to Reach the Maximum Plasma Concentration (Tmax). FVIII One-Stage Clotting Assay (Bonn Method) Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Tmax in hours was defined as the minimum time to reach Maximum plasma concentration (Cmax).

Volume of Distribution at Steady State (Vss). FVIII Clotting Assay. Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Computed as weight-adjusted CL \* Mean Residence Time

Area Under the Plasma Concentration Versus Time Curve (AUC) From 0 to Infinity. FVIII One-Stage Clotting Assay (Bonn Method) Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

AUC estimated by linear trapezoidal method. The linear trapezoidal method is a numerical method used to approximate the area under a curve.

Terminal Half-life. One-Stage aPTT-Based Assay Performed at Central Laboratory (Medical University Vienna)0-30 minutes before infusion up to 48 hours post-infusion

Computed from the terminal or disposition rate constant obtained from log-linear fitting using the least squares deviation to the last five quantifiable concentrations (9 to 48 hours).

Incremental Recovery. FVIII Clotting Assay. Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Increase in factor VIII concentration from pre- to post-infusion

Volume of Distribution at Steady State (Vss). One-Stage aPTT-Based Assay Performed at Central Laboratory (Medical University Vienna)0-30 minutes before infusion up to 48 hours post-infusion

Computed as weight-adjusted Clearance \* Mean Residence Time

Maximum Plasma Concentration (C-max). FVIII Clotting Assay. Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

C-max will be calculated as the maximum concentration following infusion of either Advate or Recombinate.

Terminal Half-life. FVIII Clotting Assay. Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Computed from the terminal or disposition rate constant obtained from log-linear fitting using the least squares deviation to the last five quantifiable concentrations.

Time to Reach the Maximum Plasma Concentration (Tmax). FVIII Clotting Assay. Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Tmax in hours was defined as the minimum time to reach Maximum plasma concentration (Cmax).

Volume of Distribution at Steady State (Vss). FVIII One-Stage Clotting Assay (Bonn Method) Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Computed as weight-adjusted CL \* Mean Residence Time

Incremental Recovery. Chromogenic Assay Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Computed from the terminal or disposition rate constant obtained from log_e -linear fitting using the least squares deviation to the last five quantifiable concentrations.

Incremental Recovery. FVIII One-Stage Clotting Assay (Bonn Method) Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Increase in factor VIII concentration from pre- to post-infusion

Mean Residence Time (MRT). One-Stage aPTT-Based Assay Performed at Central Laboratory (Medical University Vienna)0-30 minutes before infusion up to 48 hours post-infusion

The MRT in hours will be calculated as total area under the moment curve divided by the total area under the curve.

Time to Reach the Maximum Plasma Concentration (Tmax). Chromogenic Assay Performed at Local Laboratory (i.e., University of Bonn, the Study Site)0-30 minutes before infusion up to 48 hours post-infusion

Tmax in hours was defined as the minimum time to reach Maximum plasma concentration (Cmax).

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