From an early age: Pediatric Modulator Trial

APRIL 5, 2024

When Ashley received a call from her daughter’s cystic fibrosis clinic in Edmonton about a clinical trial that would be starting in Vancouver two weeks later, she didn’t hesitate to say yes.  

Ashley’s cystic fibrosis journey began almost two years earlier when, 10 days after Cali was born, her newborn screening came back positive for cystic fibrosis. It was a complete shock to the family. Both Ashley and Cory were blindsided and couldn’t believe the news. They didn’t know anyone on either side of the family who had cystic fibrosis.   

That numbness lasted until their first appointment at the Edmonton pediatric CF clinic. “Our Edmonton clinic brought me back to life after Cali’s diagnosis. Amanda, a nurse, was the first person I met there, and I will remember her for the rest of my life. It brought me back from the dark place. They are the best I have ever dealt with,” said Ashley.  

So, when Amanda, registered nurse at the Edmonton clinic, called the family earlier this summer with an opportunity for Cali to participate in a clinical trial for 12–24-month-olds to test the efficacy and safety of a modulator therapy, Ashley jumped at the opportunity.  “This community needs people to participate in clinical trials in order to get new treatments out there for everyone. I like that we were able to help in that sense,” said Ashley  

The clinical trial would last about 12 weeks and would require the family to travel from Alberta to Vancouver six times during the trial for visits to the clinic site. While the trial would last over two months, Cali would only need to take the medication for two weeks. The research coordinator in Vancouver made things as easy as possible for the family. Expenses were covered by the drug manufacturer and the research coordinator would book the flights and coordinate the hotel.  

The first clinic visit in Vancouver as part of the trial was the longest as there were a few extra tests that Cali needed to do, including bloodwork, a hearing test and an eye test. At each appointment, Ashley and Cali would meet with the research coordinator and the trial team in Vancouver, who would go over everything in detail and ensure all of their questions were answered. “Knowing Cali would be closely monitored by the team at the clinical trial site was a lot of comfort. It was a great opportunity,” said Ashley. 

The family loves to travel and in Cali’s two short years she has already been to Mexico, Vancouver, Kelowna and Newfoundland. Between two of the visits, the family was able to coordinate their love of travel with the clinical trial, making a mini vacation out of it, with Cory coming to Vancouver as well.  

Another bonus of participating in the clinical trial was that Cali got to be closely monitored by two care teams, at her clinic in Edmonton for typical CF care and the trial team in Vancouver. Participating in the clinical trial also gave Cali the opportunity to access a medication not yet available for her age group, and, if the medication does become available, Ashley now knows what to expect and how Cali will react. 

Amanda explains they noticed positive changes in Cali’s health almost immediately. “Cali had been starting to develop a cough, but once she started the clinical trial, she purged a lot of the built up mucous in her lungs and even now, the cough has not returned, said Amanda. “We’ve even noticed that her bowel movements became a bit more regular.” 

“We had no misconceptions about clinical trials because we went into it not knowing what to expect, but we were in great hands throughout the trial and we felt very assured,” said Ashley. “The team in Vancouver made everything so easy for us, from booking the travel and sending the itinerary, it was all so organized.” 

What advice does Ashley have for anyone considering participating in a clinical trial? “I would say absolutely go ahead and do it. You never know what kind of opportunity it might present or what doors it could open up. For me, the trial took away a lot of concern, if we had the opportunity to participate in another, it would be hard to turn down.”  

Read more about the clinical trial work being done by the CF Canada Clinical Accelerating Trials Network (CF CanACT), and stories similar to Ashley’s in the 2022-23 CF CanACT Annual Report here