Abstract Details

Title Cataplexy-Free Days With Sodium Oxybate Treatment in Children/Adolescents With Narcolepsy With Cataplexy

Topic Sleep

Presentation(s) P3 - Poster Session 3 (12:00 PM-1:00 PM)

Poster/Presentation
Number 5-012

Objective

To determine the number of cataplexy-free days/week experienced by participants treated with sodium oxybate (SXB) in a placebo-controlled, randomized withdrawal study in children and adolescents with narcolepsy with cataplexy.

Background

Cataplexy resolves in some patients with narcolepsy when treated with SXB.

Design/Methods SXB-naive participants were titrated to an optimal dose of SXB and then entered a stable-dose period (SD) for 2 weeks; participants already taking SXB entered SD on their usual dose of SXB for 3 weeks. After a 2-week, placebo-controlled, double-blind, randomized withdrawal period (DB), participants entered an open-label safety period (OL) for a total duration of ≤1 year. Cataplexy-free days/week were calculated from daily participant diaries.

Results Of 106 participants, 74 (69.8%) were SXB naive and 32 (30.2%) were taking SXB at enrollment. In SXB-naive participants, median (Q1, Q3) cataplexy-free days/week increased during titration: week 1 (0.0 [0.0, 2.0]), week 2 (1.0 [0.0, 3.0]), and last 7 days (4.0 [1.0, 6.0]); n=71. Sixty-seven SXB-naive participants entered SD. During the last 14 days of SD, cataplexy-free days/week remained stable and were similar in participants who were SXB naive or taking SXB at study entry: 4.3 (1.0, 5.8), n=66, and 4.8 (0.8, 6.5); n=32, respectively. During the last week of DB, cataplexy-free days/week decreased to 0.0 (0.0, 2.7) in participants randomized to placebo (n=32) but remained stable at 4.0 (1.0, 6.0) in participants continuing SXB (n=31). During the last week of observation for each participant in the OL period, median cataplexy-free days/week was 5.0, in both participants who were SXB naive (n=63) and participants taking SXB at study entry (n=32). Common adverse events (>10%) in the safety population (n=104) were enuresis, nausea, vomiting, headache, and decreased weight.

Conclusions SXB treatment increased cataplexy-free days/week in children/adolescents with narcolepsy with cataplexy. The safety profile was consistent with previous adult and pediatric narcolepsy studies.

Authors/Disclosures
Emmanuel Mignot, MD, PhD (Stanford University) PRESENTER	Dr. Mignot has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Takeda. Dr. Mignot has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Jazz Pharmaceutical. Dr. Mignot has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eisai Pharmaceuticals, Inc. . Dr. Mignot has received personal compensation in the range of $0-$499 for serving as a Consultant for EcoR1. Dr. Mignot has received personal compensation in the range of $0-$499 for serving as a Consultant for ApneaCo. Dr. Mignot has received personal compensation in the range of $0-$499 for serving as a Consultant for Eisai Pharmaceuticals. Dr. Mignot has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Centessa .
	No disclosure on file
	No disclosure on file
	No disclosure on file
Judi Profant, PhD	No disclosure on file
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. 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 Alkermes. 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 Centessa. 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 Bioprojet. 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 Harmony Bioscience.

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