WARSAW, Poland — Prolonged-release melatonin improves sleep quality in children with autism spectrum disorder (ASD) and is associated not only with improved externalizing behaviors in kids, but also better quality of life for parents, results of a phase 3 trial and extended follow-up show.
Here at the European Psychiatric Association (EPA) 2019 Congress, investigators presented the results of two studies of pediatric prolonged-release melatonin (pedPRM; Slenyto, Neurim Pharmaceuticals).
Athanasios Maras, MD, PhD, Yulius Academy, Yulius Mental Health Organization, Barendrecht, The Netherlands, presented the initial phase 3 trial results that showed that pedPRM was associated with significantly improved sleep from baseline compared with placebo.
The drug was also associated with a threefold improvement in externalizing behavior scores among the children, as well as significant improvements in parental quality of life and satisfaction with their child’s sleep.
Presenting the open-label follow-up data, Carmen Schroder, MD, PhD, Department of Child and Adolescent Psychiatry, Strasbourg University Hospital, France, said children treated continuously with pedPRM had significantly longer sleep, fell asleep faster, and had longer uninterrupted sleep compared with baseline.
The improvements in parental quality of life and satisfaction with the child’s sleep also continued after the phase 3 trial and into the open-label follow-up study.
Schroder said that the two presentations demonstrated that “pediatric prolonged release melatonin is efficient not only short term but maintains its effect long term in children and adolescents with ASD and has positive effects on their caregivers.”
Maras began his presentation by pointing out that externalizing behaviors such as hyperactivity and aggression are significantly and negatively correlated with impaired sleep quality in children with ASD.
To examine the drug’s safety and efficacy, investigators conducted a randomized phase 3 multicenter trial in children with ASD and other neurodevelopmental disorders and impaired sleep that compared the active drug with placebo.
All participants switched to melatonin for a 9-month open-label phase using three different doses of the drug.
Sleep impairment was defined as 6 or less hours of continuous sleep and/or sleep latency of a half-hour or more in 60 or more nights for more than 3 months, having failed to improve with basic sleep hygiene and behavioral interventions.
After 2-week run-in phase, 125 children from 24 sites in the US and European Union were randomly assigned in a 1:1 fashion to pedPRM 2 mg for 3 weeks followed by pedPRM 2 mg or 5 mg for 10 weeks, or an equivalent placebo.
Children from both the active and control groups were then switched to open-label follow-up, in which they were given pedPRM 2 mg or 5 mg for 18 weeks, followed by a further 78 weeks of the intervention at 2 mg, 5 mg, or 10 mg doses.