Abstract Details

Title Sodium Oxybate Treatment Effects on Sleep Architecture in Pediatric Patients With Narcolepsy With Cataplexy

Topic Sleep

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

Poster/Presentation
Number 5-010

Objective

Evaluate sodium oxybate (SXB) effects on sleep architecture in pediatric narcolepsy.

Background SXB is approved for cataplexy and excessive daytime sleepiness in patients with narcolepsy. A placebo-controlled, double-blind, randomized-withdrawal study established efficacy and safety of SXB in pediatric patients (7-17 years of age) (Part 1, ≤1 year). An open-label (OL) safety study (Part 2) provided SXB treatment for up to 2 additional years.

Design/Methods Participants with narcolepsy with cataplexy (7−16 years of age) were eligible. SXB-naive participants were titrated to an optimal SXB dose and entered a 2-week stable-dose period (SD). Participants already taking SXB entered SD at their optimal dose. After SD, participants received placebo or continued SXB treatment in a 2-week, double-blind, randomized withdrawal period (DB), then entered OL treatment for up to 47 weeks. SXB-naive participants underwent polysomnography during screening (before initiating SXB), end of SD (optimal dose), and end of Part 1 (optimal dose). Participants taking SXB at study entry underwent polysomnography at screening and end of Part 1 (taking SXB).

Results

Of 106 participants, 85 completed Part 1, and 44 entered Part 2. In SXB-naive participants, changes from screening to end of SD included arousals/night (median [Q1, Q3] change, −43.0 [−58.0, −17.0]), N1% (−4.6% [−7.5, −0.6]), and N3% (12.6% [7.1, 20.9]). In participants taking SXB, sleep architecture remained similar from screening to end of Part 1. Treatment-emergent adverse events (TEAEs) >10% during Part 1 were enuresis, nausea, vomiting, headache, and decreased weight. TEAEs with onset in Part 2 in >2 participants were upper respiratory tract infection and nasopharyngitis. Increased mean blood pressure was observed with no apparent relationship to SXB; other vital signs generally remained within normal range.

Conclusions Open-label SXB treatment in children with narcolepsy was associated with reduced arousals, reduced light sleep and increased deep sleep. Treatment effects and safety were consistent with adults.

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 .
Richard K. Bogan, MD (SleepMed)	Dr. Bogan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Jazz Pharmaceutical . Dr. Bogan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Takeda. Dr. Bogan has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Axsome . 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 $50,000-$99,999 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. Dr. Bogan has received intellectual property interests from a discovery or technology relating to health care.
Jed Black, MD (Jazz Pharmaceuticals)	Jed Black, MD has received personal compensation for serving as an employee of Jazz Pharmaceuticals. Jed Black, MD has stock in Jazz Pharmaceuticals. Jed Black, MD has stock in .
	No disclosure on file
	No disclosure on file
	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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