What It's Like to Participate in Clinical Trials for Cystic Fibrosis

There are currently 271,677 research studies in all 50 states and in 203 countries looking for volunteers. In a CISCRP survey, 75% of those surveyed (n=8,857) were either somewhat or very willing to consider clinical trial participation. But if you've never participated in a trial, it can be hard to imagine what the reality of taking part looks like.

Emily Schaller, founder and CEO of the Rock CF Foundation, talked with Antidote about her experience participating in several clinical trials for cystic fibrosis, a progressive lung condition that she has lived with since she was 18 months old.

Cystic fibrosis is a genetic disease that causes a thick, sticky buildup of mucus in the lungs that leads to persistent lung infections and a reduced ability to breathe. The condition doesn't have many treatment options, which made it even more important to Schaller to participate in research.

She shared advice on finding a trial, what to expect when you participate, and how it felt to see her friends taking the approved treatments she helped move forward.

Video: https://vimeo.com/268619768

Antidote: Tell us a little about yourself.

Emily Schaller: My name is Emily and I was born in 1982, and diagnosed with cystic fibrosis in 1983 when I was 18 months old. My one friend with CF calls them the dark ages because there were no real treatments out then. My parents got the news and the prognosis wasn't great, but here I am today, 36 years old, runner, biker, and founder of the Rock CF Foundation.

You mentioned that you've participated in a few trials since you were a kid. What were they?

There are only 30,000 people living with CF in the U.S. and 70,000 worldwide so clinical trials are crucial if we fit the requirements for a trial. Especially myself, I try to jump into them because it takes us to do it – we don't have a huge pool of people. Early on I was on an inhaled antibiotic clinical trial, a phase II and III clinical trial, and that drug was approved in 1995 I believe. I was on that trial for a while and it took a lot of trips to the hospital, which was my CF care center here in Detroit, to do pulmonary function testing, to test blood levels and weight and all the other stuff that goes along with CF.

I was in a few other small studies. One was zinc tablet you take once a day, and track how you feel with that. The biggest one I was on was in 2009 and 2010, phase II and then phase III of a drug that treats the underlying cause of CF. So instead of just treating the bacteria in the lungs and thinning the mucus, this drug treats the underlying cause of CF. That drug was approved in early 2012 and I've been on it since. It's really cool to go through the process of phase II and phase III and then open label and then hitting market and seeing my friends be able to take it. It's quite a special thing because there are so few drugs for CF that when one comes out and it's helping people, it's pretty special.

What was your clinical trial participation experience like? Did anything surprise you?

There are so many regulations with the FDA and trials and stuff, they have to be safe, and you read the consent form. You initial it all the time – if they change a word, like from a to an, you have to sign it all over again. My hand gets tired from signing that form so many times! There are labels like guinea pig and negative connotations to clinical trials, but my experiences have been great, they've been safe. You can drop out at any time, if you're unsure or not feeling good, this is your life, it's not like once you sign in to a clinical trial you're there until it's done. If you feel like you have to leave, you can consult with your healthcare provider and you can drop out of a clinical trial. That's one piece of advice I would give to people who are maybe a little hesitant to start.

How do you find clinical trials?

You can find open clinical trials at clinicaltrials.gov. Then with each disease state, there's a resource for each group or illness. With CF, we have a tool on the Rock CF website that will help you track clinical trials that you're eligible for, which is pretty awesome.

You see your CF care team usually every two to three months, and they're tracking your specific mutations, your lung function and weight and other things that may qualify you for a trial. So they're good about calling you and asking if you're interested in a particular trial. They're proactive, but sometimes they're seeing so many patients that they can't get to you. People in the CF community are super engaged so they're always looking for trials. The CF Foundation's website, or Rock CF, different cancers, diabetes foundations, a lot of us have search engines or search tools to find trials that you may qualify for.

Tell us about the Rock CF Foundation.

I founded the Rock CF Foundation in 2007. I was in my mid twenties, I didn't know what I wanted to do with my life, I had gone to school for a few years to study theater and production, and I thought that there was more that I could do with my life and give back somehow. I had been fundraising a little at the time, and telling my story around the Detroit area, and one day I said, I'm going to start my own thing. In 2007 I started Rock CF. It started with rock and roll concerts. We got cool bands, and got people in to see their favorite bands, then educated them a little about CF. So we started with awareness and raising funds with rock concerts. And as my life got healthier and I stopped playing drums in smoky bars, and I started running and cycling instead, and becoming an athlete, we took the direction of the foundation that way as well. So now we have a program where we donate running shoes to people with CF to either get them active, either walking their first mile or 5K or running their twentieth marathon. We want to give them the tools to thrive, to be healthy and thrive in the fight against CF. We do a lot of running events, cycling. Our goals is to help others with CF to take it by the horns and thrive.

Anything else you want to add?

There's always something going on with clinical trials, especially in the CF world it's really heating up. And if we don't do it, who's going to do it? So maybe take a look and consider jumping into a clinical trial.

Interested in finding a trial near you? Start searching below.