Long-term safety and maintenance of efficacy of sodium oxybate in the treatment of narcolepsy with cataplexy in pediatric patients. Journal of clinical sleep medicine : JCSM : official publication of the American Academy of Sleep Medicine Lecendreux, M., Plazzi, G., Dauvilliers, Y., Rosen, C. L., Ruoff, C., Black, J., Parvataneni, R., Guinta, D., Wang, Y. G., Mignot, E. 2022

Abstract

STUDY OBJECTIVES: Evaluate long-term efficacy and safety of sodium oxybate (SXB) in children and adolescents (aged 7-16 years) with narcolepsy with cataplexy.METHODS: A double-blind randomized withdrawal study was conducted. Prior to randomization, SXB-naive participants were titrated to an efficacious and tolerable dose of SXB; participants taking SXB entered on their established dose. Following a 2-week stable-dose period (SDP) and 2-week, double-blind, randomized withdrawal period (DBRWP), participants entered an open-label period (OLP; =47 weeks). Efficacy measures during the OLP included number of weekly cataplexy attacks, cataplexy-free days, and Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD). Safety outcomes included treatment-emergent adverse events (TEAEs); assessments of depression, anxiety, and suicidality; and polysomnography.RESULTS: Of 106 enrolled participants, 95 entered and 85 completed the OLP. In SXB-naive participants and participants previously taking SXB, efficacy of SXB established prior to the DBRWP was maintained throughout the OLP for number of weekly cataplexy attacks (median [Q1, Q3] change from the SDP to end of the OLP: 0.0 [-2.5, 4.9] and 0.0 [-3.4, 2.6], respectively) and ESS-CHAD scores (0.0 [-3.0, 2.5] and 1.0 [-3.0, 3.0], respectively). Median (Q1, Q3) number of cataplexy-free days per week was 2.3 (0.0, 6.0) in OLP week 1 and 3.8 (0.5, 5.5) in week 48. TEAEs (=5%) were enuresis, nausea, vomiting, headache, decreased weight, decreased appetite, nasopharyngitis, upper respiratory tract infection, and dizziness.CONCLUSIONS: SXB demonstrated long-term maintenance of efficacy in pediatric narcolepsy with cataplexy, with a safety profile consistent with that observed in adults.CLINICAL TRIAL REGISTRATION: Registry: ClinicalTrials.gov; Title: A Multicenter Study of the Efficacy and Safety of Xyrem With an Open-Label Pharmacokinetic Evaluation and Safety Extension in Pediatric Subjects With Narcolepsy With Cataplexy; Identifier: NCT02221869; URL: https://clinicaltrials.gov/ct2/show/NCT02221869.

View details for DOI 10.5664/jcsm.10090

View details for PubMedID 35689598