Clinical trial NCT05875974: A Study to Investigate the Effect of XYWAV on Sleepiness, Polysomnography, and Functional Outcomes in Participants With Idiopathic Hypersomnia or Narcolepsy
Official title: A Prospective, Open-Label, Single-Arm, Multicenter Study to Evaluate the Effect of Low-Sodium Oxybate Oral Solution (XYWAV) on Sleepiness, Polysomnography, and Functional Outcomes in Adult Participants Aged 18 to 75 Years With Idiopathic Hypersomnia or Narcolepsy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Idiopathic Hypersomnia
Narcolepsy
Keywords: Idiopathic Hypersomnia
Narcolepsy
Excessive daytime sleepiness
Polysomnography
JZP258
XYWAV
Oxybate
Lead sponsor: Jazz Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2023-05-25
Enrollment: 207
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study will assess the safety and efficacy of JZP258 (XYWAV) on sleepiness, polysomnography, and functional outcomes in patients with idiopathic hypersomnia (IH) or narcolepsy.

Detailed description: This prospective, multicenter, single-arm, open-label interventional study will assess the safety and efficacy of JZP258 on sleepiness, polysomnography measurements (eg, sleep stage transitions, sleep patterns, and sleep quality), daytime and nighttime symptoms, pharmacokinetics (in narcolepsy), and patient-reported outcomes that include subjective sleep quality and quality of life in patients with IH or narcolepsy.

Eligibility criteria: Key Inclusion Criteria:

* Is 18 to 75 years of age (inclusive) at the time of signing the informed consent form (ICF)
* Has a primary diagnosis of IH meeting International Classification of Sleep Disorders, Third Edition (ICSD-3) criteria or narcolepsy (Type 1 or Type 2) meeting ICSD-3 or Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-V) criteria.
* If not currently taking oxybate medication, has clinically significant symptoms of Excessive daytime sleepiness (EDS) with an ESS score \> 10 at Screening Visit 1. If currently taking oxybate medication, has an ESS score \> 10 at the Baseline Visit 2 polysomnography (after washout period). Note: Not applicable for participants entering the Narcolepsy \>9-gram cohort (see additional criteria below for the Narcolepsy \>9-gram cohort).
* If currently treated with anticataplectics and/or alerting agents, has been taking the same dosage for at least 1 month prior to Screening Visit 1 and has no current plans to adjust the dosage during the study period. Note: Alerting agents refer to either traditional stimulants (eg, amphetamines, methylphenidates) or wake-promoting agents (eg, modafinil, pitolisant, solriamfetol).

Additional Inclusion Criteria for Participants in the Narcolepsy \>9-Gram Cohort

* Is on a current treatment regimen including XYWAV at a dosage of 9 grams

Key Exclusion Criteria:

* Shows evidence of a previous untreated or inadequately treated sleep disorder considered by the investigator to negatively impact the conduct of the study, including sleep-disordered breathing, parasomnias, circadian rhythm sleep disorders, or restless legs syndrome determined by a previous sleep-laboratory diagnosis or interview utilizing modules of the Diagnostic Interview for Sleep Patterns and Disorders.
* Shows evidence of untreated or inadequately treated sleep-disordered breathing during Baseline Visit 2 polysomnography defined as an Apnea/Hypopnea Index (AHI) \> 10, using the US Centers for Medicare and Medicaid Services rules (CMS.gov).
* Has a history or presence of any unstable or clinically significant medical condition (chronic pain condition that may impact sleep), behavioral or psychiatric disorder (including active suicidal ideation or current or past \[within 1 year\] major depressive episode), or history or presence of another neurologic disorder or surgical history that might affect the participant's safety and/or interfere with the conduct of the study, in the opinion of the investigator.
* Has recently taken (ie, within 1 month prior to screening), is taking, or plans to take any of the following:

  * A substance or medication contraindicated with XYWAV use, ie. specifically alcohol and sedative hypnotics
  * A medication with a known drug-drug interaction with XYWAV
  * A medication that may have similar EEG effects to XYWAV
  * Medications known to have clinically significant CNS sedative effects
  * Other medications, natural health products, or substances from which the participant experiences clinically significant sedation

Additional Exclusion Criterion for Participants in the Narcolepsy \>9-Gram Cohort

• Any past or current medical conditions or experience that, in the investigator's clinical judgment, would preclude further increases in the current oxybate medication dosage beyond 9 grams

Primary outcomes:
measure: Epworth Sleepiness Scale (ESS) Total Score in Participants With IH and Narcolepsy Treated With XYWAV
timeFrame: Baseline up to End of Treatment (approximately 10-36 weeks)
Secondary outcomes:
measure: Idiopathic Hypersomnia Severity Scale (IHSS) Total Score in Participants With IH Treated With XYWAV
timeFrame: Baseline up to End of Treatment (approximately 10-21 weeks)
measure: Number of Stage Shifts of Sleep in Participants With Narcolepsy Treated With XYWAV
timeFrame: Baseline up to End of Treatment (approximately 10-36 weeks)
measure: Duration of Sleep Stages in Participants With Narcolepsy Treated With XYWAV
timeFrame: Baseline up to End of Treatment (approximately 10-36 weeks)
measure: Number of Nocturnal Awakenings and Nocturnal Arousals in Participants With Narcolepsy Treated With XYWAV
timeFrame: Baseline up to End of Treatment (approximately 10-36 weeks)
measure: Percentage of Sleep Stages in Participants With Narcolepsy Treated With XYWAV
timeFrame: Baseline up to End of Treatment (approximately 10-36 weeks)
measure: Patient Global Impression of Change (PGI-C) Score in Participants With IH and Narcolepsy Treated With XYWAV (Overall, Sleep Inertia, and Fatigue)
timeFrame: End of Treatment (approximately 10-36 weeks)
measure: Level of Rested or Refreshed Upon Awakening in Participants With IH and Narcolepsy Treated With XYWAV (Sleep Diary)
timeFrame: Baseline up to End of Treatment (approximately 10-36 weeks)
measure: Patient Global Impression of Severity (PGI-S) Score in Participants With IH and Narcolepsy Treated With XYWAV (Overall, Sleep Inertia, and Fatigue)
timeFrame: Baseline up to End of Treatment (approximately 10-36 weeks)
measure: Pharmacokinetic Parameter Maximum Plasma Concentration (Cmax) In a Subset of Participants With Narcolepsy Treated With XYWAV
timeFrame: PK: Predose, 0.5 hour, 0.75 hour, 1 hour, 1.5 hour post-first dose and post-second dose, 6 hour post-first dose, 8 hour post-first dose
measure: Pharmacokinetic Parameter Area Under the Concentration-Time Curve From time 0 to Infinity (AUC0-inf) In a Subset of Participants With Narcolepsy Treated With XYWAV
timeFrame: PK: Predose, 0.5 hour, 0.75 hour, 1 hour, 1.5 hour post-first dose and post-second dose, 6 hour post-first dose, 8 hour post-first dose
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Sleep Disorders Center of Alabama
state: Alabama
city: Scottsdale
country: United States
facility: Perseverance Research Center, LLC
state: Arizona
city: Irvine
country: United States
facility: Amnova Clinical Research
state: California
city: Lakewood
country: United States
facility: Long Beach Research Institute
state: California
city: Los Angeles
country: United States
facility: Santa Monica Clinical Trials
state: California
city: Redwood City
country: United States
facility: Stanford School of Medicine
state: California
city: San Ramon
country: United States
facility: TriValley Sleep Center
state: California
city: Santa Ana
country: United States
facility: SDS Clinical Trials, Inc.
state: California
city: Brandon
country: United States
facility: Meris Clinical Research, LLC
state: Florida
city: Miami
country: United States
facility: PharmaDev Clinical Research Institute, LLC
state: Florida
city: Winter Park
country: United States
facility: Florida Hospital for Children
state: Florida
city: Atlanta
country: United States
facility: NeuroTrials Research, Inc.
state: Georgia
city: Rincon
country: United States
facility: Centricity Research Rincon - DBA IACT Health Southeast Lung Associates Research
state: Georgia
city: Newtown
country: United States
facility: Neurocare, Inc., dba Neurocare Center for Research
state: Massachusetts
city: Sterling Heights
country: United States
facility: Clinical Neurophysiology Services, P.C.
state: Michigan
city: Chesterfield
country: United States
facility: St. Lukes Hospital Medical Center
state: Missouri
city: Gastonia
country: United States
facility: Clinical Research of Gastonia
state: North Carolina
city: Huntersville
country: United States
facility: Advanced Respiratory and Sleep Medicine, PLLC
state: North Carolina
city: Cincinnati
country: United States
facility: CTI Clinical Research Center
state: Ohio
city: Cincinnati
country: United States
facility: Intrepid Research
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic Main Campus - Clinical Research Unit
state: Ohio
city: Dublin
country: United States
facility: Ohio Sleep Medicine and Neuroscience Institute
state: Ohio
city: Abington
country: United States
facility: Abington Neurological Associates, LTD
state: Pennsylvania
city: Scranton
country: United States
facility: Geisinger Clinic
state: Pennsylvania
city: Columbia
country: United States
facility: Bogan Sleep Consultants, LLC
state: South Carolina
city: Austin
country: United States
facility: FutureSearch Trials of Neurology
state: Texas
city: Houston
country: United States
facility: Comprehensive Sleep Medicine Associates
state: Texas
city: San Antonio
country: United States
facility: Sleep Therapy & Research Center
state: Texas
city: Sugar Land
country: United States
facility: Tricoastal Narcolepsy and Sleep Disorders Center
state: Texas
city: Calgary
country: Canada
facility: Heritage Medical Research Clinic, University of Calgary
state: Alberta
city: Markham
country: Canada
facility: AMNDX Inc
state: Ontario
city: Toronto
country: Canada
facility: Johda Tishon Inc
state: Ontario
Study officials:
References: