Paediatric epilepsy

The NSW Government has committed $3.5 million towards research into the use of cannabis medicines for children with severe, treatment-resistant refractory epilepsy. A Compassionate Access Scheme and clinical trials are underway.

Compassionate access scheme

Since July 2016, NSW children with severe, treatment-resistant refractory epilepsy have been provided with compassionate access to Epidiolex®, an oral pharmaceutical formulation of cannabis medicine containing the non-psychoactive cannabinoid Cannabidiol (CBD). Developed by the UK-based company GW Pharmaceuticals, Epidiolex® has shown promise in the treatment of epilepsy in children and young people in overseas Phase III clinical trials.

The Compassionate Access Scheme is for a small number of children who are not able to participate in paediatric epilepsy clinical trials due to the refractory nature of their epilepsy, which has proven unresponsive to all conventional treatments. Up to 66 of NSW’s sickest children at any one time will be able to gain access to this cannabis medicine under the Compassionate Access Scheme.

NSW researchers at the Sydney Children’s Hospitals Network (SCHN) are leading the Compassionate Access Scheme (see fact sheet below) and clinical trials. These are currently underway across four sites: Sydney Children’s Hospital, Randwick; The Children’s Hospital at Westmead; Royal North Shore Hospital; and John Hunter Children’s Hospital. While providing potential relief from the symptoms of severe refractory epilepsy for sick children and their families, the NSW Government’s Compassionate Access Scheme for Epidiolex® is also providing valuable safety information, which adds to the international evidence base for the treatment of childhood epilepsy.

Rett Syndrome clinical trial

Another clinical trial, funded by the NSW Government and designed by the SCHN, aims to evaluate the long-term safety and efficacy of a new cannabis medicine called Cannabidivarin (CBDV). CBDV will be used to treat seizures in 30 children with Rett Syndrome aged between the ages of one and 18 years. 

Patient enrolments for this trial commenced in early 2019. Referral to this trial should be discussed with the child’s neurologist.

International clinical trial – Tuberous Sclerosis Complex

The GW Pharmaceuticals partnership has allowed NSW children and young people with the rare disorder Tuberous Sclerosis Complex (TSC) to take part in the GW-sponsored international clinical trial evaluating the use of Epidiolex® in treating their seizures.

This Phase III global study, allows 210 patients access to Epidiolex®, in which 12 children in NSW are participating, through the SCHN, Clinical Research Centre.

International clinical trial – Dravet Syndrome

The GW Pharmaceuticals partnership has also allowed one NSW child to take part in a further international trial evaluating the use of Epidiolex® in treating Dravet Syndrome. This trial is sponsored by GW Pharmaceuticals.

Participating in a paediatric epilepsy trial

All trials are being conducted in accordance with Good Clinical Practice for clinical trials and are subject to regulatory approval, including review and approval by an appropriate Human Research Ethics Committee and Research Governance Office.

Participants are referred to the trials according to an inclusion and exclusion criteria and participant numbers are calculated based on expert statistical analysis to achieve a scientifically valid outcome. Suitability for referral to the Compassionate Access Scheme or one of these trials should be discussed with the child’s neurologist.

The Compassionate Access Scheme and clinical trial for Rett Syndrome are underway and ongoing. The clinical trials for Dravet Syndrome and Tuberous Sclerosis Complex have closed.