The study will assess the pharmacokinetics (part A) safety, tolerability, and efficacy of prophylaxis treatment (2 to 3 times a week) (part B) with BAY81-8973 over a one year period (split into two six month treatment periods). The study will compare 2 different methods (assays) for measuring the amount of study drug, the chromogenic substrate assay per European Pharmacopeia (CS/EP) with the classical assay (Chromogenic Substrate Adjusted, CS/ADJ). During one six month period patients will receive the study drug where the dose has been measured using the" (CS/EP) and during the other six months period the dose will be measured based on the Chromogenic Substrate Adjusted assay CS/ADJ)
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
74
Single dose of BAY81-8973 crossed over to single dose of Kogenate FS
Single dose of Kogenate FS crossed over to Single dose of BAY81-8973
Participants received IV injections of BAY81-8973 at 20-50 IU/kg 2-3 times per week with BAY81-8973 measured by Chromogenic Substrate Assay Potency Per European Pharmacopeia (CS/EP) for 6 months and by Chromogenic Substrate Assay/Adjusted to Label Potency (CS/ADJ) for 6 months, sequence according to randomization.
Participants received a loading dose of approximately 50 IU/kg of BAY 81-8973 before the first surgical incision followed by further treatment with BAY 81-8973 according to surgical requirements for up to 3 weeks
Unnamed facility
Orange, California, United States
Unnamed facility
Sacramento, California, United States
Unnamed facility
Tampa, Florida, United States
Unnamed facility
Boston, Massachusetts, United States
Unnamed facility
East Lansing, Michigan, United States
Unnamed facility
Part A - Area Under the Drug Concentration-time Curve (AUC)
To examine the Pharmacokinetic (PK) characteristics of BAY 81-8973 and ensure that the new drug is similar to Kogenate FS. All results are based on the chromogenic assay.
Time frame: Samples taken at pre-injection, and at 0.25, 0.5, 1, 3, 6, 8, 24, 30 and 48 hours post injection. AUC calculated from time of injection to infinity.
Part A - Half-life (t 1/2)
To examine the PK characteristics of BAY81-8973 and ensure that the new drug is similar to Kogenate FS. All results are based on the chromogenic assay.
Time frame: Samples taken at pre-injection, and at 0.25, 0.5, 1, 3, 6, 8, 24, 30 and 48 hours post injection.
Part B - Annualized Number of Total Bleeds
The annualized number of bleeds experienced by participants
Time frame: 12 months after randomization
Part B - The in Vivo Recovery Values of Human Factor VIII (FVIII)
The amount of Factor VIII found in blood samples taken after the injection of the study drug at the beginning of the CS/EP treatment period.
Time frame: 15-30 minutes after the injection
Part B - Annualized Number of Bleeds in Each 6-month Potency Assignment Period
The annualized number of bleeds experienced by participants in each of the two treatment periods
Time frame: 6 months on each potency
Part B - Control of Bleeding as Measured by the Number of Injections Required to Treat a Bleed
The number of injections needed by participants to stop a bleed
Time frame: 6 months on each potency
Part B - Changes From Baseline at 12 Months in Quality of Life (QoL) as Measured by Transformed Total Score of Haemo-QoL Questionnaire
A measure of how treatment with BAY81-8973 affected the daily life of participants. the scoring system has 100 points. 0 is the worst possible score. 100 is the best possible score. Positive changes from baseline indicate an improvement in quality of life and negative changes indicate a deterioration.
Time frame: Baseline and 12 months
Part B - Changes From Baseline at 12 Months in Utility Index as Measured by EQ-5D Questionaire
A measure of how treatment with BAY81-8973 affected the daily life of participants. 1.0 = Best possible score, -0.594 = Worst possible score. Positive changes from baseline indicate an improvement and negative changes indicate a deterioration.
Time frame: Baseline and 12 months
Part A - Number of Participants With Inhibitory Antibody Formation
A test to ensure that participants have not developed antibodies that will interfere with the action of BAY81-8973
Time frame: Up to 6 weeks after first injection of study drug
Part B - Number of Participants With Incidence of Inhibitory Antibody Formation
A test to ensure that participants have not developed antibodies that will interfere with the action of BAY81-8973
Time frame: Up to 12 months after drug administration
Part C - Number of Participants With Incidence of Inhibitory Antibody Formation
A test to ensure that participants have not developed antibodies that will interfere with the action of BAY81-8973
Time frame: before and 3 weeks after surgery
Part A - Number of Participants With Incidence of Antibody Formation to Heat-shock Protein (HSP-70)
A test to analyze the formation of antibodies to HSP-70
Time frame: Up to 6 weeks after drug administration
Part B - Number of Participants With Incidence of Antibody Formation to Heat-shock Protein (HSP-70)
A test to analyze the formation of antibodies to HSP-70
Time frame: Up to 12 months after drug administration
Part C - Number of Participants With Incidence of Antibody Formation to Heat-shock Protein (HSP-70)
A test to analyze the formation of antibodies to HSP-70
Time frame: before and 3 weeks after surgery
Part A - Number of Participants With Incidence of Antibody Formation to Host Cell Proteins (HCP)
A test to ensure that participants have not developed antibodies to HCP during the study
Time frame: Up to 4 weeks after drug administration
Part B - Number of Participants With Incidence of Antibody Formation to Host Cell Proteins (HCP)
A test to ensure that participants have not developed antibodies to HCP during the study
Time frame: Up to 12 months after drug administration
Part C - Number of Participants With Incidence of Antibody Formation to Host Cell Proteins (HCP)
A test to ensure that participants have not developed antibodies to HCP during the study
Time frame: before and 3 weeks after surgery
Part B - Number of Participants With Assessment of the Hemostasis During Major Surgery
An assessment made by surgeons of how effective BAY81-8973 was in stopping bleeding during major operations
Time frame: An average of 1 month after start of treatment
Part C - Number of Participants With Assessment of the Hemostasis During Major Surgery
An assessment made by surgeons of how effective BAY81-8973 was in stopping bleeding during major operations
Time frame: at the time of surgery
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Kansas City, Missouri, United States
Unnamed facility
Cleveland, Ohio, United States
Unnamed facility
Bahía Blanca, Buenos Aires, Argentina
Unnamed facility
Rosario, Santa Fe Province, Argentina
Unnamed facility
Vienna, State of Vienna, Austria
...and 50 more locations