Baxter International Inc. today announced initiation of a Phase III clinical trial to evaluate the safety and effectiveness of BAX 111, Baxter’s investigational recombinant von Willebrand factor (rVWF), for the treatment and prevention of bleeding episodes in patients with von Willebrand disease.
This condition is the most common type of inherited bleeding disorder, affecting both men and women, the majority of whom are undiagnosed due to mild symptoms.1 BAX 111 is the first recombinant von Willebrand product in clinical development.
“Currently available treatments that are effective for von Willebrand disease are produced from human plasma. Baxter's investigational rVWF does not contain blood-based additives,” said Bruce Ewenstein, M.D., Ph.D., vice president, Clinical Affairs in Baxter’s BioScience business. “The initiation of the BAX 111 Phase III trial represents a significant step forward as the product, if proven safe and effective, may lead to an alternative treatment choice for clinicians and their patients with von Willebrand disease.”
The Phase III multicenter, open-label clinical trial is enrolling patients with severe von Willebrand disease and will assess the safety, effectiveness and pharmacokinetics of BAX 111 for the prevention and treatment of bleeding episodes. The primary endpoint is the number of patients experiencing treatment success for treated bleeding episodes. The trial will also assess changes in health-related quality of life (HRQoL) associated with the treatment. Standardized measures will be used to assess the overall impact of six months of recombinant treatment on the physical, emotional and social functioning of patients with von Willebrand disease.
The study will assess a minimum of 36 patients in trial sites in the United States, Canada, Europe, Australia, Japan and India. Information about the trial including enrollment is available at www.clinicaltrials.gov or by calling 1-805-372-3322.
About Recombinant Von Willebrand Factor (rVWF)
Baxter’s investigational rVWF concentrate was developed using a plasma- and albumin-free manufacturing method. It is the firstrecombinant replacement protein in clinical development for von Willebrand disease. Both the European Commission and the U.S. Food and Drug Administration granted orphan designation for Baxter’s rVWF in November 2010.
About von Willebrand Disease1
Von Willebrand disease is the most common inherited bleeding disorder and affects both men and women. Patients with von Willebrand disease either produce insufficient von Willebrand factor or carry defective von Willebrand factor that result in problems with forming clots to stop bleeding, particularly in the skin and mucous membranes such as in the gastrointestinal (GI) tract. It is estimated that up to one percent of the world’s population suffers from von Willebrand disease, but because many people have only mild symptoms, they may not know they have the condition. Research has suggested that as many as nine out of 10 people with von Willebrand disease have not been diagnosed.
About Baxter International Inc.
Baxter International Inc. (NYSE: BAX), through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions. As a global, diversified healthcare company, Baxter applies a unique combination of expertise in medical devices, pharmaceuticals and biotechnology to create products that advance patient care worldwide.
This release includes forward-looking statements concerning BAX 111, the Company’s investigational recombinant von Willebrand factor. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements:satisfaction of regulatory and other requirements; additional clinical results demonstrating the safety and efficacy of BAX111; the successful completion of the Phase III trial; actions of regulatory bodies and other governmental authorities; and other risks identified in the company's most recent filing on Form 10-K and other SEC filings, all of which are available on the company’s website. The company does not undertake to update its forward-looking statements.
1. The Basic Science, Diagnosis, and Clinical Management of von Willebrand Disease (2008)