Why Some Parents of Children with SMA Hesitate to Let Them Participate in Trials

Many parents of children with neuromuscular disorders like SMA would like to see if yet-to-be-approved therapies could help them.

But a lot them hesitate to let their children participate in clinical trials because they lack information on the trials’ requirements and risks, and they worry that their children will receive a placebo instead of a treatment.

The study that generated these findings — “Barriers and facilitators to clinical trial participation among parents of children with pediatric neuromuscular disorders” — appeared in the journal Clinical Trials.

Researchers will tell you that the success of a clinical trial depends, among other things, on the ability to recruit a sufficient number of participants.

In the case of rare diseases, finding trial participants can be challenging, since there are few people with the disorder to start with. 

A common problem in research on rare children’s diseases is a trial being interrupted because researchers are unable to enroll the required number of participants. This means it’s important to understand what influences parents’ willingness to enroll their children.

To learn why parents of children with neuromuscular disorders either agreed or refused to let their children participate in clinical trials, a research team asked 203 of them to respond to a questionnaire.

The children included Duchenne or Becker muscular dystrophy (DBMD) patients between 4–12 years of age, and spinal muscular atrophy patients up to 12 years of age who had never enrolled in a trial.

Many parents wanted their children to take participate. They included  64 percent of parents whose children had DBMD and the same percentage of parents whose children had SMA.

The parents said a key reason they would hesitate to enroll their children in a trial was the possibility the children would be in a placebo group. Another reason was not having enough information about the risks and the day-to-day responsibilities of the trial.

Parents of children with SMA said lack of information about a trial was the main reason they would hesitate to enroll a child. In addition to the risks and requirements of participating, they said lack information could involve trial benefits as well.

They said they would be more willing to let their child enroll if they knew the child would receive treatment rather than a placebo. They would also be more willing if they knew a trial would improve researchers’ understanding of SMA.

Other factors that could help parents decide to have a child participate is if the child’s doctor recommended it, and if the family was assured there was no cost involved.

Another finding was that parents would be more inclined to agree to their child’s participation if family and close friends thought it was a good idea.

A child’s age and the severity of their illness did not have a bearing on a decision to allow them to participate in a trial, researchers said.

Doctors and other trusted sources can improve the chance a child will participate, the researchers emphasized.

“As the rare disease research community works toward more patient-centric protocols, adaptive trial designs, and ultimately permissive decisions about drug access, it is vital to educate parents about drug development, clinical trial design, the drug approval process, and drug access determinations, so they can make informed choices,” the researchers wrote.