261303 BAX 855 PK-guided Dosing Adults and Adolescents
Research type
Research Study
Full title
Phase 3, prospective, randomized, multi-center clinical study comparing the safety and efficacy of BAX 855 following PK-guided prophylaxis targeting two different FVIII trough levels in subjects with severe Hemophilia A
IRAS ID
193745
Contact name
Susan Shapiro
Contact email
Sponsor organisation
Baxalta Innovations GmbH
Eudract number
2014-005477-37
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
This study will investigate treatment of severe Haemophilia A, a genetic disorder caused by missing or defective type of blood clotting factor called Factor VIII (FVIII). Without FVIII uncontrolled bleeding can occur. Treatment of Severe Haemophilia A includes replacement of FVIII to prevent bleeding. This study will investigate two different dosing regimens to prevent bleeding (prophylactic treatment) with study drug BAX855 in people with severe haemophilia A who are 12-65 years old.
This is a Phase 3 study of BAX855. BAX855 has already been tested in humans but is being tested in a larger number of patients. Approximately 116 participants will be enrolled from countries across the world. Participation in the study will last for approximately 15-16 months. BAX855 is a laboratory made blood-clotting factor similar to ADVATE, an approved medication in many countries. BAX855 is a form of ADVATE that has been modified, by the attachment of polyethylene glycol (PEG). The aim of adding the PEG is to extend the time the medication stays in the blood so the drug can be given less often.
Participants will be randomised (allocated by chance like flipping a coin) to which dose of BAX855 they receive (via a slow injection into a vein called an infusion). Participants blood will be tested to see how much of the study drug is present in their blood (Pharmacokinetic (PK) assessment). PK measures the ups and downs of drug levels in the body, when levels of the drug are at their lowest it is called the "trough”. The two different dosing regimens will get different trough levels.
The aim of the study is to compare how well the two dosing regimens/ trough levels of the study drug BAX855 prevents bleeding by comparing the number of bleeds participants have per year (called annualised bleeding rate (ABR)).
REC name
London - Fulham Research Ethics Committee
REC reference
15/LO/2120
Date of REC Opinion
26 Jan 2016
REC opinion
Further Information Favourable Opinion