This is a first-in-humans gene therapy study for subjects diagnosed with severe hemophilia A. A one time infusion of the gene therapy product (BAX888) will be given with the hope of eliminating or reducing the need for prophylactic and/or on-demand use of FVIII concentrate therapy. Up to 10 subjects will be enrolled study-wide with up to two subjects enrolled at MUSC. This study will require weekly visits to the study clinic after initial infusion for the first 15 weeks followed by monthly visits for the first year. Compensation will be provided.
This trial is a first-in-human clinical trial with subcutaneous injections of turoctocog alfa pegol (N8-GP). You must be male, age 12 or older with a diagnosis of hemophilia A to participate. The purpose of the trial is to investigate the potential effect of N8-GP
on prevention of bleeds in previously treated patients with severe hemophilia A. This study would require approximately 10 visits to the research clinic over 5 months. Compensation will be provided for time and travel.
The purpose of this research trial is to find out how effective a new Factor VIII (FVIII) product called N8-GP is in prevention and treatment of bleeding episodes, and to find out if there are any unwanted effects related to its use.