Patient Preference and Nocturnal Experience With Oxybate Treatment for Narcolepsy: Interim Analysis of Data From RESTORE (P5-13.004)

Abstract

<h3>Objective:</h3> This study aims to assess patient preference for once- vs twice-nightly oxybate dosing regimens and experiences with the second dose of twice-nightly, immediate-release (IR) oxybate. <h3>Background:</h3> IR oxybates require patients with narcolepsy to awaken for a second dose 2.5–4 hours after the bedtime dose. RESTORE, an ongoing open-label/switch study (NCT04451668), evaluates long-term safety/tolerability of investigational, extended-release once-nightly sodium oxybate (FT218; ON-SXB). <h3>Design/Methods:</h3> Participants aged ≥16 years with narcolepsy type 1 or 2 who completed the phase 3 REST-ON trial, were on stable-dose (≥1 month) IR oxybate, or were oxybate-naive were eligible. For those switching to ON-SXB, initial doses were equivalent/closest to the previous total IR oxybate dose/night; adjustments were allowed in 1.5-g increments/week. Switch participants completed the nocturnal adverse event (AE) questionnaire about their IR oxybate experience at baseline and preference questionnaire 3 months after switching to ON-SXB. Safety and dose titration data are presented separately. <h3>Results:</h3> RESTORE enrollment completed 30June2022. At the interim data cutoff (01July2022), 78 switch participants completed preference questionnaires; 130 completed nocturnal AE questionnaires. Preference for the ON-SXB dosing regimen was stated by 93.6% (73/78) of participants. On the nocturnal AE questionnaire, 65.4% (85/130) unintentionally missed their second IR oxybate dose in previous 3 months; of participants who intentionally and/or unintentionally missed the second dose, 80.2% (73/91) felt worse the next day. Second nightly IR oxybate doses were taken &gt;4 h after the first by 39.2% (51/130); 51.0% (26/51) were somewhat to extremely groggy/unsteady the next morning. The second dose was rated somewhat to extremely inconvenient for 70.8%. Second-dose-related anxiety (29.2%) and need for someone to wake them (23.1%) were also reported. Most (90.8% [118/130]) arose from bed when waking for the second dose; 9 had falls and 5 reported injuries. <h3>Conclusions:</h3> These data indicate patient preference for once-at-bedtime dosing and reveal the treatment burden of twice-nightly IR oxybate. <b>Disclosure:</b> Dr. Roy has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Jazz Pharma. Dr. Roy has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Harmony biosciences. Dr. Roy has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Eisai . Dr. Roy has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Inspire. The institution of Dr. Roy has received research support from Jazz . The institution of Dr. Roy has received research support from Inspire. The institution of Dr. Roy has received research support from Avadel. Dr. Harsh has nothing to disclose. Akinyemi Ajayi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Jazz Pharmaceutical. Akinyemi Ajayi has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Jazz Pharmaceutical. Dr. Stern has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avadel Pharmaceutical. Dr. Stern has received intellectual property interests from a discovery or technology relating to health care. Dr. Seiden has received personal compensation for serving as an employee of Avadel Pharmaceuticals. Dr. Seiden has received stock or an ownership interest from Avadel Pharmaceuticals. Dr. Dubow has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Paragon Biosciences. Dr. Dubow has stock in Avadel Pharmaceuticals. Miss Gudeman has received personal compensation for serving as an employee of Avadel Pharmaceuticals. Miss Gudeman has stock in Avadel Pharmaceuticals.