Patient Preference and Nocturnal Experience With Sodium Oxybate Treatment for Narcolepsy: Interim Data From RESTORE
Asim Roy1, John Harsh2, Akinyemi Ajayi3, Thomas Stern4, David Seiden5, Jordan Dubow5
1Ohio Sleep Medicine Institute, 2Alpine Clinical Research Center, 3Childrens Lung Asthma and Sleep Specialists, 4Advanced Respiratory and Sleep Medicine, PLLC, 5Avadel Pharmaceuticals
Objective:
To assess patient preference for once-nightly sodium oxybate (ON-SXB; FT218) versus twice-nightly SXB and experiences with the second nightly SXB dose in patients who switched from twice-nightly SXB to ON-SXB.
Background:
Immediate-release oxybate formulations to treat narcolepsy symptoms require patients to awaken for a second dose 2.5-4 h after the first. Extended-release ON-SXB eliminates middle-of-the-night dosing.
Design/Methods:
The ongoing, open-label extension/switch study RESTORE (NCT04451668) enrolled participants age ≥16 y with a confirmed diagnosis of narcolepsy type 1 or 2 from the phase 3 REST-ON clinical trial and eligible participants receiving stable doses of twice-nightly SXB for at least 1 month. Switch patients received initial ON-SXB doses equivalent/closest to their previous total dose of twice-nightly SXB. Switch patients completed patient preference questionnaires 3 months after switching and nocturnal adverse event (AE) questionnaire at baseline.
Results:
At an interim data cutoff date of September 7, 2021, 35 participants completed patient preference questionnaires; 94.3% (33/35) preferred ON-SXB to twice-nightly SXB. Sixty switch participants completed nocturnal AE questionnaires. Thirty-eight (63%) unintentionally missed their second twice-nightly SXB dose within the past 3 months; of these, 84% indicated that narcolepsy symptoms were worse the next day. Twenty-four participants (40%) had taken their second twice-nightly SXB dose >4 h after the first dose and 42% (10/24) felt somewhat, quite a bit, or extremely groggy or unsteady the next morning. For 73% (44/60), taking a second nighttime dose was somewhat, quite a bit, or extremely inconvenient; 54 (90%) arose from bed after the second dose, 3 reported associated falls, and 2 had injuries. Anxiety and the need for someone else to wake them were reported by 20% and 23%, respectively.
Conclusions:

Interim data indicate that patients with narcolepsy prefer ON-SXB to twice-nightly SXB. ON-SXB may ease treatment burdens associated with the second nightly SXB dose.