The first part of this series, Understanding the Journey of Gene Therapy Clinical Trials for Hemophilia, covers what gene therapy is designed to do and explains the aim of investigational gene therapy for hemophilia. Now let’s look at some considerations to keep in mind when deciding whether participation in a clinical trail may be right for you.
Talking with your doctor
You can start by letting your doctor know in advance that you’d like to talk about gene therapy and clinical trials at your next appointment. This will help your doctor better prepare and will ensure that you have the time necessary to discuss your options. Let’s take a look at some considerations your doctor may cover.
Time commitment
Your doctor may discuss the time commitment of participating in a clinical trial, as well as testing and follow-up requirements. Although the dosing of a gene therapy may happen on a single day, multiple appointments will be required leading up to the actual gene therapy administration. You will continue to participate in follow-ups for several years.
Trial location sites
Talk to your doctor to learn specifics about the clinical trial, such as the closest trial site. Even if your hemophilia treatment center isn’t currently a trial site, most trials are designed with the consideration for travel and assistance with travel expenses. Some testing and follow-up can be done closer to your home, helping to reduce your travel and travel expenses.
Informed consent
You will receive an informed consent form to review and sign before enrolling in a clinical trial. This is a two-way conversation between the research team and you. The research team will explain the potential benefits and risks of participating in a clinical trial, and they will answer any questions you may have. They will also explain the different parts of the clinical trial and the logistics of participation, including a visit schedule summary and safety observation. Your doctor and healthcare team will work with you throughout this process to ensure that you understand every aspect of the trial. Only with this full understanding can you decide whether to participate in the trial.
Starting the conversation
Consider the following questions when talking with your doctor:
- How do I know if I'm a good candidate for an investigational gene therapy?
- Are there any restrictions or limitations?
- Are there any short-term or long-term side effects to the investigational gene therapy?
- What type of follow-up and assessment may be required?
- Will I need to take factor after receiving the investigational gene therapy?
For additional questions to ask your doctor, check out this guide from the National Hemophilia Foundation.
Come back next month to learn what to consider before deciding to participate in a gene therapy clinical trial. Until then, visit Hemophilia Forward®, and check out frequently asked questions about gene therapy clinical trials that people in the hemophilia community are asking.
