Clinical Trial: Alphanate in Immune Tolerance Induction Therapy

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional



Official Title: A Multicenter Phase 2 Open-Label, Single-Arm, Prospective, Interventional Study of Plasma-Derived Factor VIII/VWF (Alphanate®) in Immune Tolerance Induction Therapy in Subjects With Congenital He

Brief Summary:

This is a multicenter, multinational, prospective, single-arm, nonrandomized, open-label study of approximately 25 male subjects with congenital hemophilia A who will receive their first (primary) immune tolerance induction (ITI) treatment with Alphanate.

The study consists of 2 phases:

  • An ITI Treatment Phase in which all eligible subjects will receive ITI treatment with Alphanate for a period of up to 33 months. Upon confirmation of complete immune tolerization, subjects will then enter a 12-month Prophylactic Phase. If, after 33 months of ITI, a subject has achieved partial immune tolerance, the subject will enter a 12-month Prophylactic Phase.
  • A 12-month Prophylactic Phase for all subjects who meet the criteria for complete or partial success to continue on a prophylactic dosing regimen of Alphanate.