Efficacy and safety of calcium, magnesium, potassium, and sodium oxybates (lower-sodium oxybate [LXB]; JZP-258) in a placebo-controlled, double-blind, randomized withdrawal study in adults with narcolepsy with cataplexy

. 2021 Mar 12 ; 44 (3) : .

Jazyk angličtina Země Spojené státy americké Médium print

Typ dokumentu časopisecké články, randomizované kontrolované studie, práce podpořená grantem

Perzistentní odkaz   https://www.medvik.cz/link/pmid33184650

STUDY OBJECTIVES: Evaluate efficacy and safety of lower-sodium oxybate (LXB), a novel oxybate medication with 92% less sodium than sodium oxybate (SXB). METHODS: Adults aged 18-70 years with narcolepsy with cataplexy were eligible. The study included a ≤30-day screening period; a 12-week, open-label, optimized treatment and titration period to transition to LXB from previous medications for the treatment of cataplexy; a 2-week stable-dose period (SDP); a 2-week, double-blind, randomized withdrawal period (DBRWP); and a 2-week safety follow-up. During DBRWP, participants were randomized 1:1 to placebo or to continue LXB treatment. RESULTS: Efficacy was assessed in 134 participants who received randomized treatment, and safety was assessed in all enrolled participants (N = 201). Statistically significant worsening of symptoms was observed in participants randomized to placebo, with median (first quartile [Q1], third quartile [Q3]) change in weekly number of cataplexy attacks from SDP to DBRWP (primary efficacy endpoint) in the placebo group of 2.35 (0.00, 11.61) versus 0.00 (-0.49, 1.75) in the LXB group (p < 0.0001; mean [standard deviation, SD] change: 11.46 [24.751] vs 0.12 [5.772]), and median (Q1, Q3) change in Epworth Sleepiness Scale score (key secondary efficacy endpoint) of 2.0 (0.0, 5.0) in the placebo group versus 0.0 (-1.0, 1.0) in the LXB group (p < 0.0001; mean [SD] change: 3.0 [4.68] vs 0.0 [2.90]). The most common treatment-emergent adverse events with LXB were headache (20.4%), nausea (12.9%), and dizziness (10.4%). CONCLUSIONS: Efficacy of LXB for the treatment of cataplexy and excessive daytime sleepiness was demonstrated. The safety profile of LXB was consistent with SXB. CLINICAL TRIAL REGISTRATION: NCT03030599.

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Ohayon MM. Epidemiology of narcolepsy. In: Bassetti C, Mignot E, Billard M, eds. Narcolepsy and Hypersomnia. Milton Park, UK: Taylor and Francis; 2007:125–132.

Ruoff CM, et al. . High rates of psychiatric comorbidity in narcolepsy: findings from the Burden of Narcolepsy Disease (BOND) study of 9,312 patients in the United States. J Clin Psychiatry. 2017;78(2):171–176. PubMed

Black J, et al. . Medical comorbidity in narcolepsy: findings from the Burden of Narcolepsy Disease (BOND) study. Sleep Med. 2017;33:13–18. PubMed

American Academy of Sleep Medicine. Narcolepsy type 1. The International Classification of Sleep Disorders. 3rd ed. 2014. Available at: https://learn.aasm.org/Public/Catalog/Details.aspx?id=%2FgqQVDMQIT%2FEDy86PWgqgQ%3D%3D&returnurl=%2fUsers%2fUserOnlineCourse.aspx%3fLearningActivityID%3d%252fgqQVDMQIT%252fEDy86PWgqgQ%253d%253d. Accessed November 12, 2019.

Roth T, et al. . Disrupted nighttime sleep in narcolepsy. J Clin Sleep Med. 2013;9(9):955–965. PubMed PMC

Xyrem® (Sodium Oxybate) Oral Solution Prescribing Information. Palo Alto, CA: Jazz Pharmaceuticals; 2018.

Xyrem Summary of Product Characteristics. Berkshire: UCB Pharma; 2019.

Xyrem Product Monograph Including Patient Medication Information. Dublin: Jazz Pharmaceuticals Ireland Limited; 2018.

Morgenthaler TI, et al. . Practice parameters for the treatment of narcolepsy and other hypersomnias of central origin. Sleep. 2007;30(12):1705–1711. PubMed PMC

Billiard M, et al. . EFNS guidelines on management of narcolepsy. Eur J Neurol. 2006;13(10):1035–1048. PubMed

Barateau L, et al. . Treatment options for narcolepsy. CNS Drugs. 2016;30(5):369–379. PubMed

Lopez R, et al. . French consensus. management of patients with hypersomnia: which strategy? Rev Neurol (Paris). 2017;173(1–2):8–18. PubMed

U.S. Xyrem Multicenter Study Group. A randomized, double blind, placebo-controlled multicenter trial comparing the effects of three doses of orally administered sodium oxybate with placebo for the treatment of narcolepsy. Sleep. 2002;25(1):42–49. PubMed

U.S. Xyrem Multicenter Study Group. Sodium oxybate demonstrates long-term efficacy for the treatment of cataplexy in patients with narcolepsy. Sleep Med. 2004;5(2):119–123. PubMed

Xyrem International Study Group. Further evidence supporting the use of sodium oxybate for the treatment of cataplexy: a double-blind, placebo-controlled study in 228 patients. Sleep Med. 2005;6(5):415–421. PubMed

Black J, et al. . Sodium oxybate improves excessive daytime sleepiness in narcolepsy. Sleep. 2006;29(7):939–946. PubMed

Plazzi G, et al. . 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;2(7):483–494. PubMed

Institute of Medicine. Sodium Intake in Populations: Assessment of Evidence. Washington, DC: The National Academies Press; 2013. PubMed PMC

Gardener H, et al. . Dietary sodium and risk of stroke in the northern Manhattan study. Stroke. 2012;43(5):1200–1205. PubMed PMC

George J, et al. . Association between cardiovascular events and sodium-containing effervescent, dispersible, and soluble drugs: nested case-control study. BMJ. 2013;347:f6954. PubMed PMC

American Heart Association. Why should I limit sodium?2017. Available at: https://www.heart.org/-/media/data-import/downloadables/pe-abh-why-should-i-limit-sodium-ucm_300625.pdf. Accessed July 18, 2019.

National Academies of Sciences, Engineering, and Medicine. Dietary Reference Intakes for Sodium and Potassium. Washington, DC: The National Academies Press; 2019. PubMed

Centers for Disease Control and Prevention. Get the facts: sodium and the dietary guidelines. 2017. Available at: https://www.cdc.gov/salt/pdfs/sodium_dietary_guidelines.pdf. Accessed October 4, 2019.

European Commission. Survey on members states’ implementation of the EU salt reduction framework. 2012. Available at: https://ec.europa.eu/health/sites/health/files/nutrition_physical_activity/docs/salt_report1_en.pdf. Accessed November 12, 2019.

Perrin G, et al. . Cardiovascular risk associated with high sodium-containing drugs: a systematic review. PLoS One. 2017;12(7):e0180634. PubMed PMC

Ohayon MM. Narcolepsy is complicated by high medical and psychiatric comorbidities: a comparison with the general population. Sleep Med. 2013;14(6):488–492. PubMed

Cohen A, et al. . Comorbidities in a community sample of narcolepsy. Sleep Med. 2018;43:14–18. PubMed PMC

Dauvilliers Y, et al. . Non-dipping blood pressure profile in narcolepsy with cataplexy. PLoS One. 2012;7(6):e38977. PubMed PMC

McAlpine CS, et al. . Sleep modulates haematopoiesis and protects against atherosclerosis. Nature. 2019;566(7744):383–387. PubMed PMC

Medic G, et al. . Short- and long-term health consequences of sleep disruption. Nat Sci Sleep. 2017;9:151–161. PubMed PMC

Poli F, et al. . Body mass index-independent metabolic alterations in narcolepsy with cataplexy. Sleep. 2009;32(11):1491–1497. PubMed PMC

Grimaldi D, et al. . Abnormal sleep-cardiovascular system interaction in narcolepsy with cataplexy: effects of hypocretin deficiency in humans. Sleep. 2012;35(4):519–528. PubMed PMC

Domínguez F, et al. . Association of sleep duration and quality with subclinical atherosclerosis. J Am Coll Cardiol. 2019;73(2):134–144. PubMed

Thurston RC, et al. . Sleep characteristics and carotid atherosclerosis among midlife women. Sleep. 2017;40(2). doi: 10.1093/sleep/zsw052. PubMed DOI PMC

Honda Y, et al. . Increased frequency of non-insulin-dependent diabetes mellitus among narcoleptic patients. Sleep. 1986;9(1 Pt 2):254–259. PubMed

Ben-Dov IZ, et al. . Predictors of all-cause mortality in clinical ambulatory monitoring: unique aspects of blood pressure during sleep. Hypertension. 2007;49(6):1235–1241. PubMed

XywavTM (calcium, magnesium, potassium, and sodium oxybates) oral solution, CIII Prescribing Information. Palo Alto, CA: Jazz Pharmaceuticals; 2020.

Chen C, et al. . Pharmacokinetics, relative bioavailability, and food effect of JZP-258 and sodium oxybate: results of two phase 1, open-label, randomised crossover studies in healthy volunteers [poster]. Presented at: Biennial World Sleep Congress; September 20–25, 2019; Vancouver, Canada.

American Academy of Sleep Medicine. International Classification of Sleep Disorders. 3rd ed. Darien, IL: American Academy of Sleep Medicine; 2014.

American Psychiatric Association. Narcolepsy. Diagnostic and Statistical Manual of Mental Disorders. 5th ed. Washington, DC: American Psychiatric Association; 2013:372–378.

Johns MW. A new method for measuring daytime sleepiness: the Epworth sleepiness scale. Sleep. 1991;14(6):540–545. PubMed

Hays RD, et al. . Construct validity of MOS health measures. In: Stewart AL, Ware JE, eds. Measuring Functioning and Well-being: The Medical Outcomes Study Approach. Durham, NC: Duke University Press; 1992:325–342.

Ware JE Jr, et al. . The MOS 36-item short-form health survey (SF-36). I. Conceptual framework and item selection. Med Care. 1992;30(6):473–483. PubMed

van Reenen M, et al. . EQ-5D-5L User Guide. Rotterdam: EuroQol Research Foundation; 2015.

Posner K, et al. . The Columbia-Suicide Severity Rating Scale: initial validity and internal consistency findings from three multisite studies with adolescents and adults. Am J Psychiatry. 2011;168(12):1266–1277. PubMed PMC

Kroenke K, et al. . The PHQ-9: validity of a brief depression severity measure. J Gen Intern Med. 2001;16(9):606–613. PubMed PMC

Xyrem International Study Group. A double-blind, placebo-controlled study demonstrates sodium oxybate is effective for the treatment of excessive daytime sleepiness in narcolepsy. J Clin Sleep Med. 2005;1(4):391–397. PubMed

Ristanovic RK, et al. . Exacerbation of cataplexy following gradual withdrawal of antidepressants: manifestation of probable protracted rebound cataplexy. Sleep Med. 2009;10(4):416–421. PubMed

Daniels E, et al. . Health-related quality of life in narcolepsy. J Sleep Res. 2001;10(1):75–81. PubMed

Bogan R, et al. . Evaluation of quality of life in patients with narcolepsy treated with sodium oxybate: use of the 36-item short-form health survey in a clinical trial. Neurol Ther. 2016;5(2):203–213. PubMed PMC

Jenkinson C, et al. . Comparison of three measures of quality of life outcome in the evaluation of continuous positive airways pressure therapy for sleep apnoea. J Sleep Res. 1997;6(3):199–204. PubMed

Fava GA, et al. . Withdrawal symptoms after serotonin-noradrenaline reuptake inhibitor discontinuation: systematic review. Psychother Psychosom. 2018;87(4):195–203. PubMed

Renoir T. Selective serotonin reuptake inhibitor antidepressant treatment discontinuation syndrome: a review of the clinical evidence and the possible mechanisms involved. Front Pharmacol. 2013;4:45. PubMed PMC

American Psychiatric Association. Medication-Induced Movement Disorders and Other Adverse Effects of Medication. Diagnostic and Statistical Manual of Mental Disorders. 5th ed. Arlington, VA: American Psychiatric Association; 2013.

US Food and Drug Administration. Guidance for Industry: Enrichment Strategies for Clinical Trials to Support Determination of Effectiveness of Human Drugs and Biological Products. 2019. Available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/enrichment-strategies-clinical-trials-support-approval-human-drugs-and-biological-products. Accessed March 27, 2020.

Black J, et al. . The nightly administration of sodium oxybate results in significant reduction in the nocturnal sleep disruption of patients with narcolepsy. Sleep Med. 2009;10(8):829–835. PubMed

Black J, et al. . The nightly use of sodium oxybate is associated with a reduction in nocturnal sleep disruption: a double-blind, placebo-controlled study in patients with narcolepsy. J Clin Sleep Med. 2010;6(6):596–602. PubMed PMC

Dauvilliers Y, et al. . Effect of sodium oxybate, modafinil, and their combination on disrupted nighttime sleep in narcolepsy. Sleep Med. 2017;40:53–57. PubMed

Roth T, et al. . Effect of sodium oxybate on disrupted nighttime sleep in patients with narcolepsy. J Sleep Res. 2017;26(4):407–414. PubMed

Filardi M, et al. .. In-field assessment of sodium oxybate effect in pediatric type 1 narcolepsy: an actigraphic study. Sleep. 2018;41(6). doi: 10.1093/sleep/zxy050. PubMed DOI

Thorpy MJ, et al. . The Nexus narcolepsy registry: assessment of sodium oxybate (SXB) on functioning, productivity, and health-related quality of life in participants with narcolepsy [abstract]. Neurology. 2018;90(15 Suppl):P1.105.

Whelton PK, et al. . 2017 ACC/AHA/AAPA/ABC/ACPM/AGS/APhA/ASH/ASPC/NMA/PCNA Guideline for the prevention, detection, evaluation, and management of high blood pressure in adults: a report of the American College of Cardiology/American Heart Association task force on clinical practice guidelines. Hypertension. 2018;71(6):e13–e115. PubMed

Bibbins-Domingo K, et al. . Projected effect of dietary salt reductions on future cardiovascular disease. N Engl J Med. 2010;362(7):590–599. PubMed PMC

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