News Feature | May 6, 2014

CSL Behring Starts Pediatric Phase 3 Hemophilia Trial

By Estel Grace Masangkay


CSL Behring, a biopharmaceutical company specializing in plasma protein therapeutics, announced that its pivotal pediatric Phase III study to assess its investigational hemophilia drug has begun with the enrollment of the first patient in its study site in Malaysia.

The international, multicenter, open label Phase III study will investigate the safety, efficacy, and pharmacokinetics of its novel recombinant factor VIII single chain (rVIII-Single Chain) for the treatment of previously treated children (up to age 11) with severe hemophilia A. At least 25 previously treated patients six to 11 years of age and a minimum of 25 subjects under six years of age who have been recently treated with a previous factor VIII product are expected to be involved in the study. Participants will be assigned to either an on-demand treatment or prophylaxis regimen for bleeding episodes and will be administered rVIII-Single Chain at doses determined by the trial investigator.

The company manufactured rVIII-Single Chain to use a strong, covalent bond connecting light and heavy chains unlike rFVIII chains which are not hemostatically active. In previous in-house studies, rVIII-Single Chain’s molecular integrity is shown to result in a homogenous product more stable than currently available FVIII products. In vitro studies also showed that rVIII-Single Chain has a strong affinity for von Willebrand factor (VWF), which plays a crucial role in the physiological activity and clearance of FVIII.

Hemophilia is a congenital bleeding disorder caused by deficient or defective blood coagulation proteins known as factor VIII (FVIII) or IX. The disease is characterized by spontaneous and prolonged bleeding into the muscles, joints, and internal organs. The most common form of hemophilia is classic hemophilia or hemophilia A, in which the clotting factor VIII is dysfunctional. Hemophilia A affects about 1 in 5,000 to 10,000 people. Recommended treatment for people with hemophilia deficiency is replacement factor therapy.

CSL Behring is developing rVIII-Single Chain for the treatment of hemophilia A under the AFFINITY clinical trial program and in collaboration with its parent company CLS Limited.