A Multicenter Study of the Efficacy and Safety of Xyrem With an Open- Label Pharmacokinetic Evaluation and Safety Extension in Pediatric Subjects With Narcolepsy With Cataplexy

Summary

The purpose of this trial is to assess the efficacy and safety of Xyrem in pediatrics subjects with narcolepsy that includes cataplexy.

Timeline

Start

2014-10-01

Primary completion

2017-02

Completion

2019-01-25

Outcome

Met primary endpoint

paper A preplanned interim analysis of the primary endpoint showed efficacy (p=0·0002), PMID 30169321 ↗

release “The efficacy of Xyrem for the treatment of cataplexy or EDS in pediatric patients with narcolepsy was established in the multisite Phase 2/3 EXPRESS study” jazzpharma.com ↗

Publications

• Dauvilliers Y, Lammers GJ, Lecendreux M, Maski K, Kansagra S, Black J, Parvataneni R, Chen A, Wang YG, Plazzi G. Effect of sodium oxybate on body mass index in pediatric patients with narcolepsy. J Clin Sleep Med. 2024 Mar 1;20(3):445-454. doi: 10.5664/jcsm.10912.
• Lecendreux M, Plazzi G, Dauvilliers Y, Rosen CL, Ruoff C, Black J, Parvataneni R, Guinta D, Wang YG, Mignot E. Long-term safety and maintenance of efficacy of sodium oxybate in the treatment of narcolepsy with cataplexy in pediatric patients. J Clin Sleep Med. 2022 Sep 1;18(9):2217-2227. doi: 10.5664/jcsm.10090.
• Wang YG, Menno D, Chen A, Steininger TL, Morris S, Black J, Profant J, Johns MW. Validation of the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD) questionnaire in pediatric patients with narcolepsy with cataplexy aged 7-16 years. Sleep Med. 2022 Jan;89:78-84. doi: 10.1016/j.sleep.2021.11.003. Epub 2021 Nov 11.
• Plazzi G, Ruoff C, Lecendreux M, Dauvilliers Y, Rosen CL, Black J, Parvataneni R, Guinta D, Wang YG, Mignot E. Treatment of paediatric narcolepsy with sodium oxybate: a double-blind, placebo-controlled, randomised-withdrawal multicentre study and open-label investigation. Lancet Child Adolesc Health. 2018 Jul;2(7):483-494. doi: 10.1016/S2352-4642(18)30133-0. Epub 2018 May 21.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Sodium Oxybate	Unknown	9 g	Oral

Indications