I like to participate in clinical trials. Sometimes it seems like a small thing to do, but I know it is a big thing. FA is a rare disease. Because there are so few of us (about 5,000 in the US and 15,000 worldwide), those of us with FA who are eligible for clinical trials need to step up and populate them. As we do, the researchers and the companies behind them can help us reach a time when no one has to hear: "FA, no cure, no treatment, plan for a short life." If we don't become research subjects, a cure will never be found.
I don't mean that to sound judgmental. Being a subject for a clinical trial is just one, albeit critical, way to help. I know and love people whose work and family obligations make it difficult/impossible to participate. And some folks with FA have complicated medical issues which prohibit them from participating in or leave them ineligible for clinical trials. I am lucky enough to have a flexible schedule and no major FA complications.
Whenever I enroll in a trial, I believe that the participation benefits me by helping the body of FA knowledge grow. I don't expect to walk again. I do expect to help my community. My three major considerations for participating in any clinical trial are (first and foremost) medical risk; time and lifestyle commitment; and travel expenses. The following are how things balanced out for me in my decision to participate in TAK-831.
Medical risk: I discuss the potential risk with my neurologist. I completely trust mine, David Lynch. He explained that they had studied TAK-831 on healthy subjects (phase 1) and there had been no notable side effects. His staff also explained that I could stop participating at any time for any reason. (I try to do my homework, so I don't have to drop out because I know it is not helpful to the research if I, or anyone, drop out.) Additionally, this goes back to trusting Dr. Lynch, he will pull me out if he thinks participating is harming my health. With every clinical trial, there are risks because the medication is new and harm, if any, ten years down the road is not known. Obviously, for me, the (known!) benefit of moving the research forward outweighs the risks. This is the most important decision. Get the facts, ask every question you need to, and make the best choice you can. Do what you feel is best for your health, don't feel pressured to do anything else!
Time and lifestyle commitments: I have to go to Philadelphia five times over the course of my twelve weeks of treatment. Four of these trips are one nighters, but one is a two-night trip. Philly is a six-seven hour drive/train ride one way or an hour and a half flight one way for me. So, I have to consider travel time as well as time spent with Dr. Lynch and his staff. A really lucky bonus is I enjoy all these folks! Also, I have friends and family in Philadelphia, so I can usually add a fun dinner on my trip.
I am not good at factoring in fatigue which, given that I have an energy depleting disease, is considerable. So, giving myself time to rest after these trips should be part of my time consideration. A third of the way into it, I am better at incorporating rest into time spent taking these trips and I am still happy with my decision to participate.
Most of these trips are with my family. However, I have and don't mind traveling to Philly alone. This means I am not dependent on other folks' schedules and other peoples' schedules are not a factor in my decision-making.
Back at home participating in this trial doesn't require much of me beyond making sure I take my pills.
Paying for travel: In TAK-831 all travel expenses for me and a companion are paid for. My only out of pocket is a pet sitter for our dog (not my service dog, she comes with me). I have participated in trials for which my travel expenses were a major consideration, but this is not one.
Over a third of the way into this trial, I am glad I chose to participate. It is just one of the many things I do to help make FA a disease found only in obscure history books.