Comparison of demographics and baseline narcolepsy symptoms between participants with NT1 and NT2 from the Phase 3 REST-ON clinical trial
Recommended Citation
Dauvilliers Y, Roth T, Thorpy MJ, Morse AM, Kushida CA, Harsh J, Ortiz LE, Dubow J, Gudeman J. Comparison of demographics and baseline narcolepsy symptoms between participants with NT1 and NT2 from the Phase 3 REST-ON clinical trial. Sleep Med 2024; 115:210.
Document Type
Conference Proceeding
Publication Date
2-1-2024
Publication Title
Sleep Med
Abstract
Introduction: Narcolepsy is classified into 2 subtypes: narcolepsy type 1 (NT1; with cataplexy and orexin deficiency) and narcolepsy type 2 (NT2; without cataplexy or orexin deficiency). Limited data are available regarding subtype differences in clinical characteristics and disease severity. The efficacy and safety of a once-nightly formulation of sodium oxybate (ON-SXB; FT218; LUMRYZTM) was investigated in patients with NT1 and NT2. ON-SXB demonstrated significant improvements for the 3 coprimary endpoints of change from baseline in mean sleep latency on the Maintenance of Wakefulness test (MWT), Clinical Global Impression of Improvement rating, and weekly cataplexy attacks (all P<0.001) and was well tolerated. The objective of this post hoc analysis from the REST-ON trial was to compare baseline clinical characteristics between participants with NT1 and NT2. Materials and Methods: REST-ON was a multicenter, phase 3, randomized, double-blind, placebo-controlled clinical trial (NCT02720744). Participants were ≥16 years of age with NT1 or NT2 and had excessive daytime sleepiness (sleep latency <11 min on the MWT and Epworth Sleepiness Scale [ESS] score >10) and cataplexy (average of 8 episodes per week; NT1 only). Stable concurrent alerting agent use was permitted. Randomization (1:1 to ON-SXB or placebo) was stratified by narcolepsy type; the study population was oversampled for NT1. Baseline characteristics were compared between narcolepsy types. Results: The safety analysis set included 212 participants (NT1, n=162; NT2, n=50). At baseline, characteristics of participants with NT1 vs NT2 were as follows: mean (SD) age, 32.1 (11.1) vs 28.3 (10.0) years, respectively; sex, 72.8% vs 52.0% female; race, 76.5% vs 72.0% white and 17.9% vs 14.0% Black; body mass index, 28.9 (7.5) vs 25.7 (5.6) kg/m2; and use of concurrent alerting agents, 59.2% vs 68.0%. The modified intent-to-treat population included 190 participants (NT1, n=145; NT2, n=45). Mean (SD; 95% CI) baseline clinical characteristics in patients with NT1 vs NT2 were as follows: sleep latency (MWT), 4.9 (2.9; 4.4–5.3) vs 4.9 (2.9; 4.1–5.8) minutes; Clinical Global Impression scores (CGI-Severity), 5.2 (1.1; 5.0–5.4) vs 4.7 (1.1; 4.4–5.0); ESS scores, 17.6 (4.0; 16.9–18.2) vs 15.4 (3.2; 14.4–16.3); number of sleep stage shifts to lighter stage of sleep or wake measured by polysomnography (PSG), 61.5 (22.2; 57.9–65.2) vs 55.8 (23.5; 48.8–62.9); number of nocturnal arousals by PSG, 81.5 (42.4; 74.6–88.5) vs 73.2 (35.9; 62.4–84.0); sleep quality (visual analog scale [VAS; 1 = did not sleep and 100 = slept very well]), 54.5 (22.0; 50.9–58.2) vs 55.8 (20.8; 49.5–62.1); and refreshing nature of sleep (VAS; 1 = not refreshed and 100 = refreshed), 49.9 (22.6; 46.2–53.6) vs 42.6 (21.6; 36.1–49.1). Conclusions: When comparing baseline characteristics between patients with NT1 and NT2, numerical differences were observed with respect to the proportion of female participants and concomitant use of alerting agents. At baseline, 95% CIs of mean values did not overlap for subjective measures, but did overlap for objective measures of EDS, suggesting that those with NT2 perceived themselves as more sleepy than those with NT1. Acknowledgements: Funded by Avadel Pharmaceuticals.
Volume
115
First Page
210
