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Long-term efficacy and safety of low-sodium oxybate in an open-label extension period of a placebo-controlled, double-blind, randomized withdrawal study in adults with idiopathic hypersomnia. 2023

Anne Marie Morse, and Yves Dauvilliers, and Isabelle Arnulf, and Michael J Thorpy, and Nancy Foldvary-Schaefer, and Patricia Chandler, and Abby Chen, and Luke Hickey, and Jed Black, and Richard K Bogan
Janet Weis Children's Hospital, Geisinger, Danville, Pennsylvania.

To evaluate 6-month efficacy and safety of low-sodium oxybate in people with idiopathic hypersomnia during an open-label extension period (OLE) of a phase 3 clinical trial. Efficacy measures included the Epworth Sleepiness Scale (ESS), Idiopathic Hypersomnia Severity Scale (IHSS), Patient Global Impression of Change (PGIc), Functional Outcomes of Sleep Questionnaire, short version (FOSQ-10), and Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP). Treatment-emergent adverse events were collected throughout the OLE. The OLE population included 106 participants. Most were female (71%) and White (83%), and the mean (SD) age was 41.0 (13.8) years. ESS scores decreased (improved) during the OLE (mean [SD], study baseline: 16.3 [2.8]; OLE week 2: 6.7 [4.7]; OLE end: 5.3 [3.7]), and IHSS total scores trended toward a decrease (study baseline: 32.6 [7.3]; OLE week 2: 16.2 [8.9]; OLE end: 14.8 [8.6]. Median (minimum, maximum) paired differences from OLE week 2 to OLE end were ESS, -1.0 (-20, 7; nominal P = .012); IHSS, -1.0 (-31, 19; nominal P = .086). The proportion of participants reporting PGIc ratings of "very much improved" increased from 36.7% at OLE week 2 to 53.8% at the OLE end. The FOSQ-10 and WPAI:SHP scores remained stable during OLE. The incidence of newly reported treatment-emergent adverse events decreased over the duration of the OLE. Efficacy and safety of low-sodium oxybate were maintained or improved during the 6-month OLE, supporting long-term treatment with low-sodium oxybate in adults with idiopathic hypersomnia. Registry: ClinicalTrials.gov; Name: A Multicenter Study of the Efficacy and Safety of JZP-258 in the Treatment of Idiopathic Hypersomnia (IH) With an Open-label Safety Extension; URL: https://clinicaltrials.gov/study/NCT03533114; Identifier: NCT03533114 and Registry: EU Clinical Trials; Name: A Double-blind, Placebo-controlled, Randomized Withdrawal, Multicenter Study of the Efficacy and Safety of JZP-258 in the Treatment of Idiopathic Hypersomnia (IH) with an Open-label Safety Extension; URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2018-001311-79/results; Identifier: 2018-001311-79. Morse AM, Dauvilliers Y, Arnulf I, et al. Long-term efficacy and safety of low-sodium oxybate in an open-label extension period of a placebo-controlled, double-blind, randomized withdrawal study in adults with idiopathic hypersomnia. J Clin Sleep Med. 2023;19(10):1811-1822.

UI MeSH Term Description Entries
D006970 Disorders of Excessive Somnolence Disorders characterized by hypersomnolence during normal waking hours that may impair cognitive functioning. Subtypes include primary hypersomnia disorders (e.g., IDIOPATHIC HYPERSOMNOLENCE; NARCOLEPSY; and KLEINE-LEVIN SYNDROME) and secondary hypersomnia disorders where excessive somnolence can be attributed to a known cause (e.g., drug affect, MENTAL DISORDERS, and SLEEP APNEA SYNDROME). (From J Neurol Sci 1998 Jan 8;153(2):192-202; Thorpy, Principles and Practice of Sleep Medicine, 2nd ed, p320) Daytime Sleepiness,Daytime Somnolence,Excessive Daytime Sleepiness,Hypersomnia,Hypersomnolence,Primary Hypersomnia Disorders,Secondary Hypersomnia Disorders,DOES (Disorders of Excessive Somnolence),Excessive Somnolence Disorders,Hypersomnia, Recurrent,Hypersomnolence Disorders,Hypersomnolence Disorders, Primary,Hypersomnolence Disorders, Secondary,Primary Hypersomnolence Disorders,Secondary Hypersomnolence Disorders,DOESs (Disorders of Excessive Somnolence),Daytime Sleepiness, Excessive,Daytime Sleepinesses,Daytime Somnolences,Excessive Daytime Sleepinesses,Excessive Somnolence Disorder,Hypersomnia Disorder, Primary,Hypersomnia Disorder, Secondary,Hypersomnias,Hypersomnolence Disorder,Hypersomnolence Disorder, Primary,Hypersomnolence Disorder, Secondary,Primary Hypersomnia Disorder,Primary Hypersomnolence Disorder,Recurrent Hypersomnia,Recurrent Hypersomnias,Secondary Hypersomnia Disorder,Secondary Hypersomnolence Disorder,Sleepiness, Daytime,Sleepiness, Excessive Daytime,Somnolence Disorder, Excessive,Somnolence, Daytime
D008297 Male Males
D004311 Double-Blind Method A method of studying a drug or procedure in which both the subjects and investigators are kept unaware of who is actually getting which specific treatment. Double-Masked Study,Double-Blind Study,Double-Masked Method,Double Blind Method,Double Blind Study,Double Masked Method,Double Masked Study,Double-Blind Methods,Double-Blind Studies,Double-Masked Methods,Double-Masked Studies,Method, Double-Blind,Method, Double-Masked,Methods, Double-Blind,Methods, Double-Masked,Studies, Double-Blind,Studies, Double-Masked,Study, Double-Blind,Study, Double-Masked
D005260 Female Females
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D000328 Adult A person having attained full growth or maturity. Adults are of 19 through 44 years of age. For a person between 19 and 24 years of age, YOUNG ADULT is available. Adults
D012890 Sleep A readily reversible suspension of sensorimotor interaction with the environment, usually associated with recumbency and immobility. Sleep Habits,Sleeping Habit,Sleeping Habits,Habit, Sleep,Habit, Sleeping,Habits, Sleep,Habits, Sleeping,Sleep Habit
D012978 Sodium Oxybate The sodium salt of 4-hydroxybutyric acid. It is used for both induction and maintenance of ANESTHESIA. 4-Hydroxybutyrate Sodium,Oxybate Sodium,Sodium Oxybutyrate,Sodium gamma-Hydroxybutyrate,gamma-Hydroxybutyrate,Somsanit,Xyrem,4 Hydroxybutyrate Sodium,Oxybate, Sodium,Oxybutyrate, Sodium,Sodium gamma Hydroxybutyrate,gamma Hydroxybutyrate
D016896 Treatment Outcome Evaluation undertaken to assess the results or consequences of management and procedures used in combating disease in order to determine the efficacy, effectiveness, safety, and practicability of these interventions in individual cases or series. Rehabilitation Outcome,Treatment Effectiveness,Clinical Effectiveness,Clinical Efficacy,Patient-Relevant Outcome,Treatment Efficacy,Effectiveness, Clinical,Effectiveness, Treatment,Efficacy, Clinical,Efficacy, Treatment,Outcome, Patient-Relevant,Outcome, Rehabilitation,Outcome, Treatment,Outcomes, Patient-Relevant,Patient Relevant Outcome,Patient-Relevant Outcomes
D020177 Idiopathic Hypersomnia A sleep disorder of central nervous system origin characterized by prolonged nocturnal sleep and periods of daytime drowsiness. Affected individuals experience difficulty with awakening in the morning and may have associated sleep drunkenness, automatic behaviors, and memory disturbances. This condition differs from narcolepsy in that daytime sleep periods are longer, there is no association with CATAPLEXY, and the multiple sleep latency onset test does not record sleep-onset rapid eye movement sleep. (From Chokroverty, Sleep Disorders Medicine, 1994, pp319-20; Psychiatry Clin Neurosci 1998 Apr:52(2):125-129) Hypersomnolence, CNS, Idiopathic,Hypersomnolence, Idiopathic,Idiopathic CNS Hypersomnolence,Idiopathic Central Nervous System Hypersomnolence,CNS Hypersomnolence, Idiopathic,CNS Hypersomnolences, Idiopathic,Hypersomnia, Idiopathic,Hypersomnias, Idiopathic,Hypersomnolence, Idiopathic CNS,Hypersomnolences, Idiopathic,Hypersomnolences, Idiopathic CNS,Idiopathic CNS Hypersomnolences,Idiopathic Hypersomnias,Idiopathic Hypersomnolence,Idiopathic Hypersomnolences

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