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The Caregiver’s Challenge: Making Clinical Decisions for Your Child

Deciding to participate in a clinical trial can be a difficult decision. Deciding on behalf of a child is often even more difficult.

SaJo ND Graduation-cropndra Sojka Lagedrost understands the weight of that responsibility all too well. Sandra’s daughter Joanna was only 10 when she was diagnosed with Friedreich’s Ataxia, a rare degenerative nerve disease.

“You’re always asking yourself, ‘Are we covering all the bases? Have we looked everywhere?’  You are so responsible because it is someone else. You’re very conscious of the fact that this is on behalf of your child,” she says.

Unfortunately for Sandra and her husband, John, the decision to enroll Joanna in a Phase III clinical study at The Children’s Hospital of Philadelphia was easy. “We didn’t have a lot of options,” she says. Friedreich’s Ataxia, which is caused by a genetic mutation, is incurable. There are no treatments.

Joanna was in the fourth grade when she pointed out to her parents that she seemed to stumble and fall more than other children. Her observation prompted Sandra to make an appointment with the pediatrician and, ultimately, with a neurologist who diagnosed Joanna’s condition.

Over the next few years Sandra and John sought to strike a delicate balance. They encouraged their bright, determined and independent daughter to enjoy typical childhood activities. At the same time they also took her to physical therapy, sought to learn everything they could about Joanna’s condition and added her name to a disease registry. By the time they learned about the double-blind placebo controlled trial in Philadelphia, Joanna was 14. Her mobility and coordination were deteriorating and she tired easily.

Participating in the trial wasn’t easy. Sandra and Joanna had to make bimonthly trips from their home in suburban Chicago to Philadelphia over the course of 18 months. But Sandra says the research team was accommodating and compassionate.

“It was a very good experience,” Sandra says. “Although we signed the informed consent forms, they explained everything very clearly and in an age-appropriate way to her.  Every time there was even the slightest change in protocol they talked to us about it, and we would have to sign our consent forms all over again. They were really by the book, and I found that reassuring.”

Positive though the experience may have been, the treatment turned out to be ineffective. Joanna went on to participate in a second trial in Philadelphia – a natural history study to help researchers better understand the disease — but as she moved into adulthood, she began to make her own healthcare decisions.

She wanted to focus on college more than clinical trials while she attended the University of Notre Dame.

“When she turned 18 there was a piece of me that was relieved that she was a legal adult and that I could hand off the decision making and recognize her insights. It wasn’t mom and dad any more saying, ‘You have to do this,’” says Sandra, who stepped into a more advisory role. “In some ways nothing changed, but in some ways a lot did.”

Today Joanna, now 22, lives with her parents. She works as an intern in a law firm and as a freelance editor. As an adult she has participated in heart studies in Indianapolis and New York. While Friedreich’s Ataxia’s most visible manifestation is its effect on mobility, it also results in heart disease, which is often fatal.

She says she’s now aware of the amount of behind-the-scenes work it takes to find, pursue and participate in trials, but her overall appreciation for clinical research hasn’t changed. Like her parents, she knows research holds the key.

“Human beings like attention,” she says. “When you consider the fact that someone is paying enough attention and money to put on a clinical trial to focus on your condition, it’s hard to understand how someone would not want to participate. It’s obviously for a very good long-term cause and you may enjoy positive outcomes too.”

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