Considering a Clinical Trial? 4 Things to Know Before You Enroll

For people living with neuromuscular diseases, few things bring more hope than progress in research, whether it leads to a new therapy or a deeper understanding of a diagnosis. Each advancement represents years of scientific work — and the dedication of those who volunteer for clinical trials.

While exciting, enrolling in a study is a deeply personal decision — one often filled with hope, fear, courage, and uncertainty. Here, members of the neuromuscular community share their experiences and four key tips for participating in clinical research.

1. Lean on your care team

Healthcare providers often help community members begin the journey toward participating in clinical research — and are there for them throughout the process. For Shelby Herschberger, it was a non-medical MDA specialist who helped her decide to enroll her son Tyson, 15, in a clinical trial studying deflazacort (Emflaza) for Duchenne muscular dystrophy (DMD).

“She told us about the resources in our area and how to get involved in the clinical trials that were happening at Children’s Healthcare of Atlanta at that time,” says Shelby, who lives in Georgia.

Looking back, Shelby says those early conversations — and not being afraid to ask questions — made all the difference. “There’s no such thing as too many questions, especially when they are about your child,” she says. “Ask all your questions until you understand and feel comfortable.”

Darci Garcia, 46, of Texas, who lives with amyotrophic lateral sclerosis (ALS), participated in a clinical trial for an experimental therapy that regulates cells in the immune system. “Educate yourself on the trial, drug, and procedure as much as possible,” she advises. “The experts are there, so ask all the questions.”

2. Plan for the time commitment

Being involved in research demands flexibility and patience. Many participants say to budget more time than you think you’ll need for appointment days.

Chloe Crabb, 13, who lives with spinal muscular atrophy (SMA), frequently traveled five-and-a-half hours to Denver when she participated in a nusinersen (Spinraza) clinical trial about 10 years ago. Her mom, Kate, says the travel was tedious, but they made the most of it. “We turned it into a fun vacation,” she says. “We would go shopping or to the museum. For the longest time, we had Chloe believe that chocolate milk only existed in Denver, so she would be excited to go.”

In 2022, Omar Sheikh, of Portland, Oregon, who lives with Becker muscular dystrophy (BMD), took part in a local study using MRIs to observe muscle changes over time. The study required annual visits, and sometimes the tests would take most of the day. Omar learned to block off a full day for the study visits.

Ann Stanley of West Virginia, whose daughter, Winnie, 10, lives with SMA, says the upheaval to your schedule can be tough. “There’s a lot of travel, some missed school, and lots of time spent at the hospital,” she says. “Just be prepared for that.”

Many studies will reimburse travel expenses, including transportation, lodging, and food, to ease the burden. Be sure to ask about reimbursement if the information is not provided to you.

3. Prepare for a range of emotions

For many families, the physical and emotional demands can be just as challenging as the logistics.

When Winnie enrolled in a clinical trial, she had to overcome her fear of needles, as her trial required weekly shots given at home and bimonthly blood draws. What helped most was letting Winnie feel ownership over the choice. “We gave her a voice in the decision and talked it through,” says her mom, Ann. “She decided she wanted to help other kids with SMA by participating in this trial.”

Kate, whose daughter was in a placebo-controlled trial, constantly wondered whether Chloe was getting the active drug or the inactive placebo. “You’re providing your child to science but getting no feedback,” she says. She was surprised by how difficult that was, even though she knew about the possibility of a placebo going into the study.

Later, when Chloe felt exhausted after multiple surgeries, the family had to weigh whether to continue in the trial. “It takes such an emotional and physical toll to participate,” Kate says. “I think that’s a huge thing that people don’t realize.”

4. Consider the bigger purpose

Darci felt like her ALS symptoms improved while she was in a clinical trial for an investigational therapy, but, due to lack of funding, the treatment was no longer available after the trial ended. Still, she remains grateful to have been part of it.

“A positive attitude goes a long way,” she says. “You have to understand that the trial may not have the outcome you’re hoping for, and it may be a stepping stone to push research further — you have to be OK with that.”

AJ Bardzilowski, who lives with inclusion body myositis (IBM) and facioscapulohumeral muscular dystrophy (FSHD), joined a trial for an investigational drug that ultimately did not prove to be effective. Still, he loved being able to contribute to the study.

“​​Everybody’s journey is unique, and studies have side effects to consider,” he says. “But, if you’re offered the chance and it’s safe for you to do it, then I think it’s a no-brainer — 100% do it if you can. It’s the only way they’re going to make advances.”

Chloe feels proud knowing that her participation played a part in Spinraza’s approval. “As I got older, I really grasped the idea that I helped — I was part of this drug becoming approved because I participated,” she says.

Maggie Callahan is a frequent contributor to Quest Media.