NCT01029340

Trial to Evaluate the Efficacy and Safety of a New Full Length Recombinant Human FVIII for Hemophilia A

A Two Part Randomized Cross-Over Trial to Evaluate the Pharmacokinetics, Efficacy, and Safety Profile of Plasma Protein-Free Recombinant FVIII Formulated With Sucrose (BAY81-8973) in Previously Treated Subjects With Severe Hemophilia A Under Prophylaxis Therapy

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 74 (actual)
• Sponsor: Bayer · Industry
• Sex: Male
• Age: 12 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

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)

Conditions

• Blood Coagulation Disorders
• Hemophilia A

Interventions

Type	Name	Description
BIOLOGICAL	Recombinant Factor VIII (BAY81-8973)	Single dose of BAY81-8973 crossed over to single dose of Kogenate FS
BIOLOGICAL	Recombinant Factor VIII (Kogenate FS, BAY14-2222)	Single dose of Kogenate FS crossed over to Single dose of BAY81-8973
BIOLOGICAL	Recombinant Factor VIII (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.
BIOLOGICAL	Recombinant Factor VIII (BAY81-8973)	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

Timeline

• Start date: 2009-12-01
• Primary completion: 2012-06-01
• Completion: 2013-03-01
• First posted: 2009-12-09
• Last updated: 2016-11-28
• Results posted: 2013-11-18

Locations

60 sites across 22 countries: United States, Argentina, Austria, Croatia, Denmark, Germany, Hong Kong, India, Indonesia, Israel, Italy, Norway, Pakistan, Poland, Serbia, South Africa, Spain, Sweden, Taiwan, Thailand, Turkey (Türkiye), United Kingdom