Queensland Children’s Hospital paediatrician Honey Heussler with Emily O’Donnell, 6, who has Fragile X syndrome.

27 June 2019

A clinical trial under way at the Queensland Children’s Hospital could improve the quality of life for children with Fragile X syndrome who struggle with anxiety.

Fragile X syndrome is a genetic condition caused by a defect in the FMR1 gene. It affects boys more severely than girls, and causes various developmental issues including cognitive impairment and learning disabilities.

The six-month trial will test the use of medicinal cannabis gel as a treatment for anxiety, following a successful smaller study delivered promising results with no serious adverse side effects.

CHQ paediatrician Honey Heussler, who is leading the research at Queensland Children’s Hospital, said more than 200 children with Fragile X syndrome are involved in the international trial.

“Anxiety and behavioural problems are not uncommon for those with Fragile X syndrome – social anxiety is a huge issue,” Dr Heussler said.

“If the medicinal cannabis gel does ease social anxiety for children with Fragile X, it would greatly improve their quality of life, and that of their families.”

A preliminary open label study involving 20 children with Fragile X in Australia in 2017-2018 found the cannabis gel had a positive impact on anxiety levels with no serious adverse side effects. This study is the next stage involving comparison of the CBD gel as opposed to a placebo gel in these children to see if the results we saw in the first trial are true.

“This larger trial is needed to confirm if the results seen in previous research were a true outcome,” Dr Heussler said.

The study is recruiting participants until the end of July. For information about participating in the trial, phone 3069 7709 or email CHQ_RD@health.qld.gov.au.