Abstract Details

Title Cataplexy-Free Days 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) Sleep Posters (7:00 AM-5:00 PM)

Poster/Presentation
Number 014

Objective Evaluate cataplexy-free days/week, as a measure of treatment impact, in a placebo-controlled randomized withdrawal study of calcium, magnesium, potassium, and sodium oxybates (lower-sodium oxybate, LXB; Xywav^TM; previously designated as JZP-258) treatment in patients with narcolepsy.

Background Sodium oxybate (SXB; 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 At study entry, participants were taking SXB only, SXB+other anticataplectics, anticataplectics other than SXB, or were cataplexy treatment–naive. Participants (aged 18–70 years with narcolepsy with cataplexy) began LXB treatment during a 12-week, open-label, optimized treatment and titration period (OLOTTP), followed by a 2-week stable-dose period (SDP). Participants were randomized to receive placebo or continue LXB treatment during a 2-week, double-blind, randomized withdrawal period (DBRWP).

Results

Of 201 enrolled participants, 134 comprised the efficacy population (placebo, n=65; LXB, n=69). Median (Q1, Q3) cataplexy-free days/week at first week of OLOTTP (while initiating LXB) by prior treatment were SXB-only, 5.8 (2.0, 7.0); SXB+other anticataplectics, 6.4 (5.0, 7.0); other anticataplectics, 4.0 (1.8, 6.0); anticataplectic-naive, 3.5 (0, 5.8). At end of SDP (on stable dose of LXB), median (Q1, Q3) cataplexy-free days/week were 6.0 (3.5, 7.0), 6.1 (1.4, 7.0), 6.0 (2.6, 7.0), and 6.2 (4.0, 7.0), respectively. Prior to randomization, there was no difference in median cataplexy-free days/week between participants to be randomized to placebo (6.0 [3.5, 7.0]) or LXB treatment (6.0 [3.0, 7.0]); during DBRWP, median cataplexy-free days/week decreased in participants randomized to placebo (3.5 [0, 5.8]) but remained similar in participants randomized to continue LXB treatment (5.6 [2.8, 7.0]). The overall safety profile of LXB was similar to SXB.

Conclusions The number of cataplexy-free days/week increased with LXB treatment in participants previously naive to oxybate. Number of cataplexy-free days/week decreased during placebo exposure in participants randomized to placebo.

Authors/Disclosures
Yves Dauvilliers, MD, PhD (Hopital Gui De Chaulliac) PRESENTER	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.
Nancy R. Foldvary-Schaefer, DO, FAAN (Cleveland Clinic)	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.
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.
	No disclosure on file
Judi Profant, PhD	No disclosure on file
	No disclosure on file
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.

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