written by Melissa

If you’re anything like me, hearing the term “clinical trial” generates some conflicting reactions. There’s excitement over the possibility of a game-changing treatment on the horizon, and at the same time, there’s some fear in the idea of participating in something that hasn’t been fully approved by the medical powers-at-be.         

Recently, I had the chance to weigh the risks and benefits and decided that I felt very safe participating in a drug trial for a medication that I hope one day soon will help keep my daughters, my mom, my cousins, me and everyone else with Long QT safer. 

The trial needed people with Long QT of a certain type, who were of a certain age group, had an ICD and whose QTC fell within a given range… check, check, check, check. So I decided to chat with the SADS team about it and eventually reached out to the administrator of the trial.  

In the process, I learned this trial would require me to leave my husband and two kids (ages seven and nine) for six days while I stayed in a clinical facility out of state. My schedule would be pretty full, so this also impacted my worklife. Yikes. That’s a big ask.  

But again, check, check, check, check… I fit within all of those parameters. And everyone is busy. So, why not me? 

I lost my sister to Long QT back in 2005. And I have two daughters with Long QT. I thought to myself: I can actually help usher in the future of treatment for this condition. I can make my daughters’ futures safer. 

And I have no regrets. In fact, I may be participating in another round of the trial at a future date. 

If you’ve seen the clinical trials listed in the SADS newsletter and wonder who they are for, well, they’re for people like you and me. Not everyone can make it work, and not everyone will be comfortable with the risks. It’s a personal decision. Personally, I feel proud to be one of the first ten people with Long QT to have tested a medication that could be a game-changer for us. 

It was only going through a clinical trial, though, that I realized how much I didn’t know about clinical trials to begin with. So, let’s shed some light on what it’s really like to participate in a drug trial. 

Here are four aspects about the experience that I didn’t expect:     

A strict regimen defines the days.

From 5:00am to 11:00pm, most of my time was mapped out, with no wiggle room. A shower was only possible at 5:00am, and limited to ten minutes. Then each of the next six hours included 15 minutes of laying still with 30-45 minutes to move around, when I usually checked email, read, texted with family, and walked some laps around the floor. Once lunchtime came around, activities were spaced out further and I would use that time to dive into a work project, or watch a few episodes of a show. The afternoon and evening became pretty relaxed as I settled into my routine.   

Your participation means a lot.

Rare diseases mean rare populations. The phase I was involved in would include a total of 12 people who met specific parameters around their QTC measurements and current treatment plans. It sounds tiny doesn’t it? Twelve!? But, I learned getting to 12 has been difficult. I was surprised to find the study was still recruiting for participants months after they began and felt even more fulfilled that I made the choice to participate.

It can be lonely. 

I knew I’d miss my husband and my kids, but honestly, I thought the week would pass quickly. We can all use a little alone time, right? Well, anticipation clashed with reality early as I said goodbye at the airport and broke into tears. I got over it though, and enjoyed night one in a hotel room all to myself. I thought I was good-to-go until the next day when I was brought to my room at the clinic and it hit me that I was the only non-staff member on the floor. I was surrounded by nice people, but they weren’t family, or friends, or even familiar coworkers. It took me a couple of days to adjust.    

I should have asked for a packing list. 

I have no idea why I didn’t reach out ahead of time to get more clarity on what I should bring. Turns out, underwire bras are not allowed. It seems so obvious to me now, but had I known this, I would have been better prepared. Also, the floor tended to run hot. I expected the opposite so my pajamas and outer layers weren’t ideal. A packing list would have been helpful.  

The process from start to finish

Wondering what it’s like leading up to a clinical trial and the follow-up required? Here’s a brief run-down of my experience:

I learned about the trial from the research newsletter that SADS sends out. Then, I leaned on the SADS team for their insight, and what they had heard about the trial from others who had already participated. I emailed the administrator of the study, and they called me back in a couple of weeks to learn more. 

I had a two-hour call with a nurse who went through the consent for participating and was able to answer any questions I had (and I had plenty). I learned just how many blood draws I would have (20+), how I would take the medication (in liquid form mixed with flavored Gatorade), and what I needed to do in preparation for the study (abstain from having caffeine, alcohol, grapefruit, etc. for several days before and throughout the study). 

Next came a physical exam that included the basics: height, weight, blood draw and EKG. A nurse came to me and conducted the process right in my living room.

Everything checked out, so it was time to schedule my stay. A coordinator from the clinical trial nailed down a week that worked for me, then booked my flight and hotel. I’d spend the first night in a hotel then go to the clinic at 7:00am the next day to ensure my QTC was still within range and that I officially qualified. Another woman who was there at the same time did not qualify at this last part of the process and was sent home. 

At check-in, I transferred all of my belongings to a clear bin after it was all inspected. This was my first hint at just how controlled a controlled study is. Variation is a problem, so, for example, they had to ensure I didn’t have anything in makeup products that could be problematic. 

Then began my five nights in the clinic with the structured schedule I described earlier. When it was time for meals, I mostly ate what was served (A+ for the banana bread) and ordered takeout to be delivered a couple of times instead. When I ordered takeout, I paid for food upfront and was reimbursed after.  

I woke up on my final day giddy that it was time to fly home to see my family. I had built my routine and was comfortable in the study, but I really missed home. After some final brief activities like an EKG and blood draw, I was given a generous check for participating, and hopped into an Uber to get to the airport. 

One week later, I had a brief call with a nurse, where they confirmed that I didn’t have any ailments to report, and that was it. The remainder of my compensation was then sent to me and my participation was wrapped up. Now I look forward to the possibility that this new medication will one day be available to me, my daughters and the rest of my family! I’d do it again in a heartbeat to speed up that timetable.   

Every clinical study will look different, but this seemed like a pretty standard experience. If you’re interested in learning more about how you could be involved, I encourage you to reach out to the SADS team.