Sodium oxybate was effective at reducing cataplexy in pediatric patients with narcolepsy, according to research presented at SLEEP 2017, the 31st Annual Meeting of the Associated Professional Sleep Societies, held in Boston, Massachusetts, June 3-7, 2017.
Giuseppe Plazzi, MD, from the Department of Biomedical and Neuromotor Sciences at the University of Bologna, Italy, and colleagues recruited 106 children and adolescents aged 7 to 16 years who had narcolepsy and cataplexy. Both those receiving sodium oxybate treatment and those who were sodium oxybate-naive were eligible.
After a stable dose period, 63 of 106 patients participated in a 2-week double-blind placebo-controlled withdrawal period and were randomly assigned 1:1 to receive either sodium oxybate at a stable dose or to placebo. Of the 63 randomized patients, 41% were aged 7-11 years, 44% were girls, and 38% were receiving sodium oxybate treatment at the beginning of the study.
A preplanned interim analysis of 35 patients showed that sodium oxybate was effective at reducing weekly cataplexy attacks (P <.005). On the advice of the Data Safety Monitoring Board, and in agreement with the US Food and Drug Administration, the double-blind randomized withdrawal period was therefore terminated early.
Key secondary end points include safety during the withdrawal period, Clinical Global Impression of Change for cataplexy severity, and change in the Epworth Sleepiness Scale for Children and Adolescents score.
Reported adverse events were similar to those found in prior adult and pediatric experiences.
Plazzi G, Ruoff C, Lecendreux M, et al. A double-blind, placebo-controlled, randomized-withdrawal, multicenter study on the efficacy and safety of sodium oxybate in pediatric subjects with narcolepsy with cataplexy. Presented at: SLEEP 2017: 31st Annual Meeting of the Associated Professional Sleep Societies; June 3-7, 2017; Boston, MA.