Russian Kogenate Pediatric Study

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.

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.

Study Overview

• Status: Completed
• 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)

• Study Type: Interventional
• Enrollment (Actual): 32
• Phase: Phase 4

Contacts and Locations

Study Locations

• Russian Federation
  • Ekaterinburg, Russian Federation, 620149
  • Kirov, Russian Federation, 610 027
  • Moscow, Russian Federation, 105077
  • St. Petersburg, Russian Federation, 191186

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 12 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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

Study Plan

How is the study designed?

Design Details

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

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg qw; 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; 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)
Experimental: rFVIII-FS (Kogenate FS, BAY14-2222), biw (30 IU/kg + 40 IU/kg); 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); 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)
Experimental: rFVIII-FS (Kogenate FS, BAY14-2222), tiw (3 x 25 IU/kg); 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); 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Bayer

Publications and helpful links

General Publications

• V.V. Vdovin, T.A. Andreeva, T.A. Chernova, F.G. Perina, E.E.M. Shiller, P.V. Svirin, M. Maas Enriquez, and S. Rauchensteiner. Prophylaxis with Once, Twice or Three-Times Weekly Dosing of rFVIII-FS Prevents Joint Bleeds in a Previously Treated Pediatric Population with Moderate/Severe Hemophilia A. Journal of Coagulation Disorders 2011; 3:(1). October 2011

Helpful Links

• Click here to find information about studies related to Bayer Healthcare products conducted in Europe

Study record dates

Study Major Dates

• Study Start: June 1, 2007
• Primary Completion (Actual): September 1, 2009
• Study Completion (Actual): September 1, 2009

Study Registration Dates

• First Submitted: March 4, 2008
• First Submitted That Met QC Criteria: March 10, 2008
• First Posted (Estimate): March 11, 2008

Study Record Updates

• Last Update Posted (Estimate): July 1, 2015
• Last Update Submitted That Met QC Criteria: June 4, 2015
• Last Verified: June 1, 2015

More Information

Terms related to this study

• Keywords: Prophylactic; Children,; Hemophilia A,; Kogenate,; Factor VIII,
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Coagulants; Factor VIII
• Other Study ID Numbers: 12684; 2014-005253-39 (EudraCT Number)