Octapharma announced the launch of the PREVAIL clinical study (NCT03344003), which will investigate the use of Wilate for treatment of hemophilia A patients in Canada who recently have developed inhibitors to a factor VIII (FVIII) concentrate, or have had long-term inhibitors against FVIII.
Hemophilia A is a bleeding disorder that develops due to low levels of a coagulation factor known as factor VIII. The first line treatment of patients involves administration of clotting factor concentrates.
The development of neutralizing antibodies (inhibitors) against factor VIII is the most serious side effect in the treatment of hemophilia A. It also is very common, occurring in up to 30 to 40 percent of severe hemophilia A patients who start the treatment.
The standard approach for eliminating inhibitors is to make patients more tolerant to FVIII through prolonged, daily exposures to high-doses of FVIII; this process is called immune tolerance induction (ITI).
“The presence of a FVIII inhibitor in the body will rapidly inactivate the infused factor VIII concentrate, thus making bleed control and prevention of joint damage a clinical challenge in these patients. ITI can result in inhibitor resolution, but can take several years to succeed,” Paul Moorehead, MD, from the Janeway Children’s Health and Rehabilitation Centre in St. John’s, Newfoundland and Labrador, said in a press release.
According to previous studies, Wilate, which contains FVIII and the FVIII stabilizing protein, von Willebrand Factor (VWF), has been successful in achieving ITI. Wilate comes as a lyophilized (freeze-dried) powder that can be reconstituted and administered an injection.
PREVAIL is a 10-year study that will follow hemophilia A patients with inhibitors as they are treated with Wilate for either first-time inhibitors, inhibitors that won’t respond to previous ITI treatment, or re-occurrence of inhibitors to FVIII.
The aim of PREVAIL is to better understand the patient- and therapy-related variables that affect the course and outcome of ITI in Canadia.
“Although several treatment regimens are used routinely, optimal regimen for ITI is yet to be defined. Persons living with FVIII inhibitors have high morbidity, mortality and associated increases in cost of care and this study is an important step to improve treatment and patient outcomes in Canada,” said Mark Belletrutti, MD, from Stollery Children’s Hospital in Edmonton.
The PREVAIL study will recruit moderate and severe hemophilia A patients of all ages living with inhibitors who are eligible. Patient enrollment for the study is now open.
Researchers will record extensive data including ITI outcomes, bleeding during ITI, joint health, adherence to treatment, and relapse rates. The study also will evaluate quality of life during the therapy and give further insight into the biology of inhibitor formation.
Data from the PREVAIL study will build on currently available data and provide important data to help determine the optimal management of hemophilia A patients who develop inhibitors.
”Given the huge impact inhibitors can have on a person’s life and ability to manage their hemophilia, the Canadian Hemophilia Society (CHS) is supportive of studies on inhibitors and pleased to see the start of this study to collect long term ITI data in Canada to help improve approaches to treatment,” said Paul Wilton, CHS President.