CSL Enrolls First Patient in Study of rVIII-SingleChain
- By BSTQ Staff
CSL Behring has enrolled its first patient in the pivotal pediatric Phase III study to evaluate the efficacy, safety and pharmacokinetics of its novel investigational recombinant factor VIII single chain (rVIII-SingleChain) for the treatment of previously treated children (up to 11 years) with severe hemophilia A. The study site for the first enrollment is Malaysia, and there will be a minimum of 25 previously treated subjects from 6 years to 11 years of age and at least 25 subjects under 6 years of age who have undergone more than 50 exposure days with a previous factor VIII product. Subjects will be assigned to either an on-demand or prophylaxis treatment regimen for the treatment of bleeding episodes and will receive rVIII-SingleChain at a dose to be determined by the investigator. Hemostatic efficacy will be assessed by the subject or caregiver and the investigator, who will assess overall efficacy by a four-point scale.
In an earlier study, rVIII-SingleChain showed improved pharmacokinetics over octocog alfa, the comparator, and demonstrated a safety and efficacy profile that supported advancement to late-stage clinical development. CSL Behring, in collaboration with its parent company, CSL Limited, is developing rVIII-SingleChain for the treatment of hemophilia A as part of the AFFINITY clinical trial program.
