“How do I determine if and when it may be a good time to discuss with my doctor the possibility of participating in an investigational gene therapy clinical trial for hemophilia?”
“How do I determine if and when it may be a good time to discuss with my doctor the possibility of participating in an investigational gene therapy clinical trial for hemophilia?
Great question! The short answer is: Any time is a good time to talk with your doctor and healthcare team about the possibility of participating in an investigational gene therapy clinical trial for hemophilia.
But first, let’s look at what “investigational” means. This is a term that refers to a potential treatment that is being studied in clinical trials but isn’t approved by regulatory agencies, such as the Food and Drug Administration, or FDA. In clinical trials, scientists are studying to see if the investigational therapy is safe and effective, how much is needed, and how it may be used for that disease. Enrolling in an investigational clinical trial is a personal decision. Please consult with your doctor if you want to know more about participating in a clinical trial.
There are many reasons people with hemophilia may or may not consider participating in a clinical trial. Some people may be interested because they are looking to change the way that they manage their hemophilia. For others, the reason may be to help advance the research for hemophilia treatment now or for future generations.
It is also important to consider when it might be a good time to participate in a clinical trial. Clinical trials often require frequent and long-term monitoring that can be challenging for some people. Other considerations may include avoiding alcohol and agreeing to use contraceptives for a period of time after the infusion if these are also required criteria of a clinical trial.
And, finally, some people may choose not to enroll in a clinical trial now or at all because they don’t feel an investigational treatment is right for them, while others may choose to wait and see the results of the clinical trials before making a decision. For those who are considering participating in a clinical trial, they will be given information by the research team so they can understand all the potential risks and, if they decide to move forward after being informed, they will sign a written consent to participate.
Whichever group you fall into, and wherever you are in your hemophilia journey, discuss your questions about clinical trials for hemophilia with your doctor so you can be informed and feel empowered.”
