A pharma firm has achieved its enrolment target for a drug trial to treat paediatric and adolescent patients with autism spectrum disorders (ASD).
Zynerba Pharmaceuticals – a developer of pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders – have started a 14-week Phase 2 BRIGHT trial.
The company expects to announce topline results from this study in the second quarter of 2020.
“We are committed to developing new treatment options to improve outcomes in patients suffering from a variety of neuropsychiatric conditions including autism spectrum disorder and achieving our enrollment target in the BRIGHT trial is an especially meaningful step toward this goal,” said Joseph M Palumbo, chief medical officer of Zynerba.
“We would like to thank everyone involved in this study to date, particularly the participating patients and their caregivers, as well as the investigators.”
The 14-week exploratory Phase 2 BRIGHT trial has enrolled 36 patients with ASD at a single clinical site in Australia.
The trial is designed to evaluate the efficacy and safety of Zygel in children and adolescents (ages four through 17) with ASD as confirmed by DSM-5 diagnostic criteria for ASD.
Enrolled patients are receiving weight-based initial doses of 250 mg daily or 500 mg daily of Zygel.
After completing dosing in the 14-week period, participants may enroll in a six-month extension trial.
Using the Autism Diagnostic Observation Schedule (ADOS-2) which is administered at baseline by a qualified clinician, 94% of enrolled patients had moderate-to-severe symptoms of ASD at baseline.
Communication and behaviour
Autism Spectrum Disorder is a developmental disorder that affects communication and behaviour in approximately one million paediatric and adolescent patients between the ages of five and 17 in the US.
It refers to a range of conditions characterised by anxiety, repetitive patterns of behavior, impairments in social communication including verbal and non-verbal communication, and deficits in developing and maintaining relationships.
Zygel is being studied in clinical trials in a number of rare and near-rare neuropsychiatric conditions.