Octapharma USA Sponsors NHF Conference, Unveils New VWD Clinical Trials Focused on Prophylactic Treatment and Pregnancy
Octapharma USA is sponsoring the National Hemophilia Foundation’s (NHF) 71st Bleeding Disorders Conference and introducing two new clinical trials focused on von Willebrand Disease (VWD). The clinical trials are featured in poster presentations at the NHF meeting being held in Anaheim, California through Saturday.
One clinical study will assess the efficacy and safety of WILATE® during prophylaxis in previously treated patients with VWD. This study has the primary objective to determine the efficacy of von Willebrand Factor (VWF)/Factor VIII (FVIIII) concentrate (WILATE®) in the prophylactic treatment of previously treated patients with type 3, type 2 (except 2N), or severe type 1 VWD.
“Prophylactic treatment in other congenital bleeding disorders is widely accepted as the standard of care to prevent bleeding and preserve quality of life in patients,” said Octapharma USA President Flemming Nielsen. “This form of treatment in VWD is not well characterized prospectively as yet. This study is intended to provide data on the efficacy of prophylactic treatment in reducing the rate of bleeding and on the impact of prophylaxis on the quality of life in VWD patients.”
Affecting approximately 1 percent of the population, VWD is a common inherited bleeding disorder that occurs when the blood lacks a protein that helps clotting. Type 3 VWD is the rarest and most severe form of the condition, representing approximately 5% of cases, according to the National Organization for Rare Disorders.
The second clinical trial, von Willebrand Factor in Pregnancy (VIP), is a multicenter study of WILATE® use in VWD for childbirth. The primary objective is to evaluate the:
- Rate of primary postpartum hemorrhage;
- Effectiveness of targeting VWF/FVIII minimum levels of 100% to 150% for delivery; and
- Effectiveness of targeting the immediate 72-hour postpartum period in pregnant patients with VWD whose third trimester factor levels are less than 100%, while aiming to maintain 50% to 100% target levels after the immediate postpartum period for the first 5 to 7 days postpartum after normal vaginal delivery or the first 7 to 10 days postpartum after caesarean section.
For pregnant women with VWD who by the third trimester do not have VWF or FVIII levels greater than 50% to 100%, specific guidance is lacking for delivery planning in terms of how high a factor level should be achieved. Specifically, guidance is lacking on whether replacement therapy should target a VWF minimum level in the 100% to 150% range, for example, or a range closer to the 200% to 250% levels observed in normal pregnancy.
