Abstract Details

Title Quality of Life in a Phase 3, Placebo-Controlled, Double-Blind, Randomized Withdrawal Study of Lower-Sodium Oxybate in Adults With Narcolepsy With Cataplexy

Topic Sleep

Presentation(s) S9 - Sleep: Highlight Presentations on RBD (2:08 PM-2:16 PM)

Poster/Presentation
Number 002

Objective Establish the efficacy and safety of calcium, magnesium, potassium, and sodium oxybates (lower-sodium oxybate, LXB; Xywav^TM; previously designated as JZP-258) in a double-blind randomized withdrawal study in adults with narcolepsy with cataplexy. Health-related quality of life (HRQoL) endpoints from this study are presented.

Background Narcolepsy negatively impacts HRQoL. Sodium oxybate (Xyrem®) is a standard of care for the treatment of cataplexy and excessive daytime sleepiness in narcolepsy. LXB is an oxybate medication with 92% less sodium.

Design/Methods Participants 18–70 years of age began LXB treatment during a 12-week, open-label, optimized treatment and titration period, followed by a 2-week stable-dose period. Participants were then randomized to receive placebo or continue LXB treatment during a 2-week, double-blind, randomized withdrawal period. HRQoL assessments included the 36-Item Short Form Health Survey Version 2 (SF-36) and 5-level EuroQoL 5-Dimensions Self-Report Questionnaire (EQ-5D-5L).

Results 201 participants enrolled; 134 were randomized and received at least 1 dose of double-blind study medication (efficacy population; placebo, n=65; LXB, n=69). Decreased scores (worsening) were observed in participants randomized to placebo compared with participants randomized to continue LXB treatment for the SF-36 physical component summary (median [Q1, Q3], −1.92 [−3.46, 1.73] for placebo and −0.03 [−2.07, 2.41] for LXB; nominal P=0.02), SF-36 mental component summary (−1.92 [−6.28, 1.34] for placebo and 1.55 [−1.88, 3.78] for LXB; nominal P=0.03), and EQ-5D-5L visual analog scale (−5.0 [−10.0, 5.0] for placebo and 0.0 [0.0, 5.0] for LXB; nominal P=0.0056). No change was observed in the EQ-5D-5L crosswalk index (0.00 [−0.05, 0.03] for placebo and 0.00 [−0.01, 0.03] for LXB; nominal P=0.3918). The overall safety profile of LXB was similar to sodium oxybate.

Conclusions HRQoL worsened in those randomized to placebo during the 2-week double-blind randomized withdrawal period but remained stable in participants who continued LXB treatment.

Authors/Disclosures
Nancy R. Foldvary-Schaefer, DO, FAAN (Cleveland Clinic) PRESENTER	Dr. Foldvary-Schaefer has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Jazz. The institution of Dr. Foldvary-Schaefer has received research support from Jazz. The institution of Dr. Foldvary-Schaefer has received research support from Suven. The institution of Dr. Foldvary-Schaefer has received research support from Takeda. Dr. Foldvary-Schaefer has received publishing royalties from a publication relating to health care. Dr. Foldvary-Schaefer has received publishing royalties from a publication relating to health care.
Michael J. Thorpy, MD (Montefiore Medical Center)	Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Jazz. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avadel. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for alkermes. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Harmony. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for centessa. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Axsome. Dr. Thorpy has received publishing royalties from a publication relating to health care.
Yves Dauvilliers, MD, PhD (Hopital Gui De Chaulliac)	Yves Dauvilliers, MD, PhD has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for idorsia. Yves Dauvilliers, MD, PhD has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for JAZZ. Yves Dauvilliers, MD, PhD has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Yves Dauvilliers, MD, PhD has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avadel.
Asim Roy, MD (Ohio Sleep Medicine Institute)	Dr. Roy has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Jazz Pharma . Dr. Roy has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Harmony Bioscience. The institution of Dr. Roy has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Inspire. Dr. Roy has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Avadel. 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.
	No disclosure on file
Roman Skowronski, MD, PhD	Dr. Skowronski has nothing to disclose.
	No disclosure on file
Richard K. Bogan, MD (SleepMed)	Dr. Bogan has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Harmony. Dr. Bogan has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Jazz. Dr. Bogan has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Bogan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avadel. Dr. Bogan has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Noctrix. Dr. Bogan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Harmony. Dr. Bogan has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Jazz. Dr. Bogan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Axsome. Dr. Bogan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Idorsia. Dr. Bogan has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Jazz. The institution of Dr. Bogan has received research support from Jazz Pharmaceuticals. The institution of Dr. Bogan has received research support from Harmony . The institution of Dr. Bogan has received research support from Eisai. The institution of Dr. Bogan has received research support from Avadel. The institution of Dr. Bogan has received research support from Takeda. The institution of Dr. Bogan has received research support from Suven. The institution of Dr. Bogan has received research support from Axsome. The institution of Dr. Bogan has received research support from Merck. The institution of Dr. Bogan has received research support from Liva Nova. The institution of Dr. Bogan has received research support from Nyxoah. Dr. Bogan has received research support from Bayer. The institution of Dr. Bogan has received research support from ZIO. The institution of Dr. Bogan has received research support from NLS. Dr. Bogan has received intellectual property interests from a discovery or technology relating to health care.

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