Hemophilia is a blood disorder that interferes with the blood clotting process. People with hemophilia either lack the clotting factors needed to stop bleeding, or possess abnormal clotting factors. The disease is usually inherited, and affects men far more often than women.Hemophiliacs bleed for longer when injured than people with normal levels of clotting factors. Internal bleeding is a serious problem, and people with severe hemophilia may bleed into muscles and joints several times a week. Despite popular belief, hemophilia does not make people bleed faster, rather the blood takes longer to clot.
Clinical Trial Research and Hemophilia. Treatments for hemophilia focus on replacing the missing clotting factors. Such treatments require intravenous doses of clotting factors, and in severe cases are required several times a week. Clinical trials are investigating gene therapy treatments for hemophilia, in the hopes of discovering longer-lasting and more effective treatments.
While gene therapy research continues, other clinical trials investigate new methods of producing clotting factors. One of the most recent hemophilia treatments, ADVATE Antihemophilic Factor (Recombinant) was approved by the FDA in 2003 after successful clinical trials. ADVATE is used to prevent bleeding, and to prepare people with hemophilia for surgery. ADVATE research led to the use of genetically altered ovaries from Chinese hamsters, which produce a clotting agent called Factor VIII. Clinical research trials proved the new treatment and its production safe for human use.
Why Participate in Hemophilia Clinical Trial Research?
One of the most obvious motives for participating in a hemophilia clinical trial is access to new treatments. Of course, the chance always exists that you’ll be part of the control group, but either way, the advice you’ll receive from hemophilia experts can be invaluable. This site provides access to a number of trials. Registration does not oblige you to proceed, but it will enable you to find out about the options.