Russian Kogenate Pediatric Study

Study Overview
=================
Brief Summary
-----------------
A prospective study to evaluate the effect of rFVIII-FS in different prophylactic regimens on bleeding events frequency and development of arthropathy in Previously Treated and Minimally treated Hemophilia A pediatric population.

Official Title
-----------------
A Prospective Study to Evaluate the Effect of rFVIII-FS in Different Prophylactic Regimens on Bleeding Events Frequency and Development of Arthropathy in Previously Treated and Minimally Treated Hemophilia A Pediatric Population.

Conditions
-----------------
Hemophilia A

Intervention / Treatment
-----------------
* Drug: rFVIII-FS (Kogenate FS, BAY14-2222) 70 IU/kg, dosing once per week
* Drug: rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg twice per week (30 IU/kg + 40 IU/kg)
* Drug: rFVIII-FS (Kogenate FS, BAY14-2222) 75 IU/kg, dosing three times per week (3 x 25 IU/kg)


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Male Severe hemophilia A or moderate hemophilia A 1-12 years of age Requiring treatment with FVIII Exclusion Criteria: Current or prior inhibitor or familial antecedents of inhibitor Surgery required during the study (9 months) Positive for HIV

Ages Eligible for Study
-----------------
Minimum Age: 1 Year
Maximum Age: 12 Years

Sexes Eligible for Study
-----------------
Male

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw<br>rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 IU/kg, dosing by injection once per week [qw] (weekly on Day 7 + 1 after previous injection) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 35 IU/kg twice a week or further escalation to 25 IU/kg three times a week) | Drug: rFVIII-FS (Kogenate FS, BAY14-2222) 70 IU/kg, dosing once per week<br>* rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 IU/kg, dosing by injection once per week (weekly on Day 7 + 1 after previous injection) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 35 IU/kg twice a week or further escalation to 25 IU/kg three times a week)<br>|
| Experimental: rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg)<br>rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 IU/kg, dosing by injection twice per week [biw] (30 IU/kg [day 1] + 40 IU/kg [day 4]) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 25 IU/kg three times a week) | Drug: rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg twice per week (30 IU/kg + 40 IU/kg)<br>* rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 70 IU/kg, dosing by injection twice per week (30 IU/kg [day 1] + 40 IU/kg [day 4]) for 9 months. Dose escalation was permitted due to joint bleeding (escalation to 25 IU/kg three times a week)<br>|
| Experimental: rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg)<br>rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 75 IU/kg, dosing by injection three times per week [tiw] (3 x 25 IU/kg [day 1, 3, 5]) for 9 months. No escalation opportunity for participants in this group | Drug: rFVIII-FS (Kogenate FS, BAY14-2222) 75 IU/kg, dosing three times per week (3 x 25 IU/kg)<br>* rFVIII-FS (Octocog-alfa, antihemophilic factor [recombinant]) 75 IU/kg, dosing by injection three times per week (3 x 25 IU/kg [day 1, 3, 5]) for 9 months. No escalation opportunity for patients in this group<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants With Less Than 2 Joint Bleeds During the 9-month Treatment Period |  | Up to 9 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Bleeds Per Participant During the 9-month Treatment Period |  | Up to 9 months | 
| Number of Participants With Bleeding Events During the 9-month Treatment Period |  | Up to 9 months | 
| Number of Participants With Joint Bleeds During the 9-month Treatment Period |  | Up to 9 months | 
| Number of Participants in Each Group at the End of the Study |  | Up to 9 months | 
| Actual Monthly rFVIII-FS Consumption |  | Up to 9 months | 
| Change From Baseline in Stockholm Hemophilia Joint Score at 9 Months of Treatment | The assessment of joint function using Stockholm Joint Score. The minimum value is 0 (the best condition), and the maximum value is 140 (the worst condition). | baseline and 9 months | 
| Haemo-QoL Standardized Total Score at 9 Months of Treatment (Completed by Participants in the Total Group) | Quality of life (QoL) was measured by the Haemo-QoL standardized total Score, which ranged from 0 (the best condition) to 100 (the worst condition). | 9 months | 
| Haemo-QoL Standardized Total Score (Completed by Parents/Caregivers in the Total Group) at 9 Months of Treatment | Quality of life (QoL) was measured by the Haemo-QoL standardized total Score, which ranged from 0 (the best condition) to 100 (the worst condition). | 9 months | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Kogenate,, Children,, Factor VIII,, Hemophilia A,, Prophylactic