Shire has announced a phase 3 clinical trial for ADYNOVATE®, the company’s recombinant factor VIII (FVIII) therapy designed using pegylation technology to extend FVIII time in circulation and increase FVIII exposure to enhance bleed prevention. The study is called “PROPEL,” a PROspective, randomized, multi-center study comparing the safety and efficacy of ADYNOVATE® following PK-guided prophylaxis targeting two different factor Eight trough Levels in subjects with severe hemophilia A.
PK (pharmacokinetics) are a key component of the study, as they denote the measurement of concentration and duration of a drug’s effect in the body. PK tests can be used to target factor levels that will help achieve a desired therapeutic response. Improved FVIII PK parameters are key characteristics in achieving and maintaining target FVIII levels to enhance bleed protection. Specifically, investigators will assess the safety and efficacy of ADYNOVATE® in two PK-guided prophylaxis regimens targeting trough levels of 1-3% and 8-12%, using annualized bleeding rate (ABR) as a measure of efficacy.
The data collected in this study will inform whether personalized, PK guided dosing and higher trough levels can increase the number of people achieving zero bleeds. Investigators will ultimately compare the proportion of people with an ABR of zero in the lower and higher trough groups for the final six-month period of the study. They will also be testing enrolled patients for any unwanted immune response to the therapy.
To learn more about the objectives of the study and for specific inclusion/exclusion criteria go to clinicaltrials.gov and search by trial ID#: NCT02585960.
Source: Shire
