Information, Support May Persuade Parents of FXS Children to Join Trials

Parents of children with intellectual disability, in particular those affected by fragile X syndrome, may benefit from more information and dedicated support when considering the possibility of participating in clinical trials, a study shows.

The study, “Fragile X syndrome clinical trials: exploring parental decision‐making,” was published in the Journal of Intellectual Disability Research.

In order for clinical studies on the safety and efficacy of new therapies to be conducted, patient communities must embrace the challenge of participating in a venture whose risks and benefits have yet to be defined.

Deciding whether to take part in clinical trials is not easy, and can be even more challenging for parents of ill children. For pediatric clinical trials there is much at stake, and many decisions can have a major impact on both parent and child.

Given so, it is important to understand the decision-making process involved in clinical trial participation. Such information may help researchers improve trial design and ensure adequate care during the studies, so patients and parents receive all the support they need.

A team led by researchers at the National Human Genome Research Institute investigated parental decision-making for pediatric clinical trial participation within the disease context of fragile X syndrome (FXS).

“The goal of our study is to improve understanding of parental therapeutic trial decision-making when the main disorder manifestation is intellectual disability,” researchers said.

The study enrolled 31 parents of children diagnosed with fragile X, 16 of whom had enrolled their child in a prior clinical study and 15 who had refused to participate in clinical trials.

Some of the past studies children had participated in included several placebo-controlled trials of mavoglurant (NCT01253629 and NCT01357239), minocycline (NCT01053156), and NNZ-2566 (NCT01894958), among others.

The parents were interviewed by phone or in person following a predetermined set of open-ended questions with focus on the decision-making process, trial expectations and hopes, and perceptions of trial benefits and risks.

Their answers revealed three major topics: factors that facilitate participation in trials, factors that discourage participation in trials, and ranging decisional ease and lack of regret.

All parents whose child had participated in a trial believed that the participation could offer an opportunity of direct benefit. Some of them stated that their decision also had an altruistic motivation, in order to benefit the entire fragile X community.

In general, they were aware of the genetic nature of their child’s disorder. This had a direct impact on their decision-making process, as they reported to be more interested in studies that would address mechanisms known to be involved in disease progression.

“They perceived that targeted experimental medications were more likely to have a dramatic effect on FXS symptomatology than medications designed to intervene at a symptom level,” researchers stated.

Parents also noted that they were more interested in clinical trials believed to target symptoms with which their child had particularly struggled. Still, many hoped that the experimental treatment could be proven to be a cure for the disease, rather than just improve a symptom.

Among the reasons reported by parents not to participate in clinical studies were the poor match between the trial’s target outcome and their child’s symptomatology, and logistic inconveniences, such as traveling and the number of appointments.

They also reported concerns about the low likelihood of their child to be able to complete the full trial and a long-term treatment regimen, the risks of the trials, and the blood draw requirements.  Parents were particularly concerned about potential physical side effects associated with experimental treatments.

Inability of their child to communicate pain or discomfort because of age or symptoms was also noted by some parents. This would have a direct impact on the determination of treatment-related adverse effects.

“Our study produced a preliminary profile of parental drug trial participation decisional factors in the disease context of intellectual disability, from both parents whose children enrolled in trials and parents who declined,” researchers stated. “Despite difficulty involved in making the decision, parents in our study reported feeling that they made the right choice.”

Seventeen parents said they found the decision whether or not to participate in clinical trials to be easy. Many were focused on the potential benefits of the experimental treatment. Others noted a strong belief against medicating their child or were not interested in the experimental therapy’s mechanism.

Those who considered the decision difficult cited the risks and barriers associated with the studies.

“Even when the trial experience was challenging, or the drug was determined to be ineffective, trial joiners perceived benefits to their participation,” researchers said.

“Many parents explained that they made the right choice because their participation helped progress science, and they often perceived benefits of participation outside of the potential treatment effects, such as gaining access to resources and information,” they added.

Collectively, the study reveals that in contrast with what has previously been reported by parents of children with life-threatening conditions, parents of children with fragile X are more afraid of the risks associated with trial participation.

This suggests that they could benefit from a decisional intervention focused on helping them define the motivations and barriers that may contribute to their decision-making process.