The purpose of this study is to evaluate the efficacy and safety of expanded Xywav dosing regimens in adult participants with narcolepsy or idiopathic hypersomnia (IH).
The trial has 2 treatment periods: titration and optimization period and the randomized withdrawal period. Once eligibility to participate in the trial is confirmed, eligible participants will begin the titration and optimization treatment period where they will be assigned to either cohort 1 or cohort 2. Participants assigned to Cohort 1 will receive once-nightly dosing of Xywav and participants assigned to Cohort 2 will receive twice-nightly dosing of Xywav. Assignment is dependent on participant's standard oxybate treatment and the treating investigator's decision. During the titration and optimization treatment period, participants' Xywav dosing will be adjusted until they achieve a stable dose in this period. This period will last up to 14 weeks. After a stable dose is achieved, the participants will begin the randomized withdrawal treatment period. During the withdrawal treatment period, participants assigned in both cohorts will be randomized to either continue on their stable dose of Xywav or receive placebo for 2 additional weeks of treatment in the trial.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
108
Intrepid Research
Cincinnati, Ohio, United States
Change in Epworth Sleepiness Scale (ESS) scores
ESS is a self-administered questionnaire with 8 questions. Each question is scored on a scale ranging from 0 (would never fall asleep) to 3 (high chance of falling asleep). It has a total score ranging from 0 to 24, with a higher score representing increased daytime sleepiness.
Time frame: End of stable dose visit (up to Week 14), up to end of Double-Blind Randomized-Withdrawal Period (up to Week 16)
Clinical Global Impression of Change (CGIc) scores
CGIc is a 7-point questionnaire completed by the treating physician that evaluates how the physician thinks the participant is responding to treatment since the end of stable dose visit (up to week 14). Responses are graded from 1 (very much improved) to 7 (very much worse)
Time frame: At the end of Double-Blind Randomized-Withdrawal Period (up to Week 16)
Patient Global Impression of Change (PGIc) scores
PGIc is a 7-point questionnaire completed by the participant evaluating how they think they are responding to treatment since the end of stable dose visit (up to week 14). Responses are graded from 1 (very much improved) to 7 (very much worse)
Time frame: At the end of Double-Blind Randomized-Withdrawal Period (up to Week 16)
Change in IHSS scores in participants with IH
Idiopathic Hypersomnia Severity Scare (IHSS) is a 14-item self-reported questionnaire that assesses the severity and functional consequences of IH symptoms. Questions capture symptoms of excessive sleepiness, sleep inertia, and long sleep duration. The total score for the IHSS ranges from 0 to 50, with higher scores reflecting greater symptom severity.
Time frame: End of stable dose visit (up to Week 14), up to end of Double-Blind Randomized-Withdrawal Period (up to Week 16)
Change in NSS scores in participants with NT1
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Narcolepsy Severity Scale (NSS) is a 15-item self-administered questionnaire that assesses the severity and consequences of the 5 major narcolepsy symptoms such as daytime sleepiness, cataplexy, hallucinations, sleep paralysis, and disrupted nighttime sleep. The total score for NSS ranges from 0 to 57, with the higher score indicating greater symptom severity
Time frame: End of stable dose visit (up to Week 14), up to end of Double-Blind Randomized-Withdrawal Period (up to Week 16)
Change in NSS-2 scores in participants with NT2
NSS-2 is a modified NSS self-administered questionnaire with only 12 items (omits questions regarding cataplexy). The total score for NSS-2 ranges from 0 to 44, with the higher score indicating greater symptom severity
Time frame: End of stable dose visit (up to Week 14), up to end of Double-Blind Randomized-Withdrawal Period (up to Week 16)
Change in weekly rate of cataplexy (WRC) in participants with NT1
WRC will be assessed for participants with NT1 using a cataplexy frequency electronic diary. Participants will be recording the number of daily cataplexy attacks experienced.
Time frame: End of stable dose visit (up to Week 14), up to end of Double-Blind Randomized-Withdrawal Period (up to Week 16)
Number of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
Time frame: Up to the end of the Safety follow up visit (Up to Week 18)