A Study Evaluating the Efficacy and Safety of Xywav Expanded Dosing vs Placebo in Participants With Narcolepsy or IH
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18-75
SponsorJazz Pharmaceuticals
About this trial
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).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Has a primary diagnosis of IH or narcolepsy Type 1 or Type 2 (NT1 or NT2)
Participants with a primary diagnosis of NT1 must have a history of at least 14 cataplexy attacks in a typical 2-week period, based on participant history at the time of diagnosis and prior to initiating treatment.
Participants with a primary diagnosis of IH must have an average total nightly sleep time of at least 7 hours, based on participant history at the time of diagnosis and prior to initiating treatment.
If not currently treated with oxybate, has clinically significant symptoms of excessive daytime sleepiness (EDS) with an Epworth Sleepiness Scale (ESS) score > 11 at screening and baseline.
Disqualifiers
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.
Has succinic semi-aldehyde dehydrogenase deficiency by medical history.
Has uncontrolled hypothyroidism as determined by central clinical laboratory test results.
Has a current seizure disorder.
Trial design
Design model
Parallel
Treatments tested in this trial
Xywav
Drug0.5 g/ml calcium, magnesium, potassium, and sodium oxybates solution taken by mouth
Placebo
Other interventionPlacebo solution taken by mouth
Treatment groups
120 Participants
are divided into 8 treatment groups8
Treatment groupsSee each treatment group below.
Group A: Narcolepsy Cohort once-nightly expanded Xywav dose groupActive comparator 1 intervention
Group B: Narcolepsy Cohort once-nightly expanded Xywav followed by placebo groupPlacebo comparator 2 interventions
Group C: Narcolepsy Cohort twice-nightly expanded Xywav dose groupActive comparator 1 intervention
Group D: Narcolepsy Cohort twice-nightly expanded Xywav followed by placebo groupPlacebo comparator 2 interventions
Group E: IH Cohort once-nightly expanded Xywav dose groupActive comparator 1 intervention
Group F: IH Cohort once-nightly expanded Xywav followed by placebo groupPlacebo comparator 2 interventions
Group G: IH Cohort twice-nightly expanded Xywav dose groupActive comparator 1 intervention
Group H: IH Cohort twice-nightly expanded Xywav followed by placebo groupPlacebo comparator 2 interventions
Trial outcomes
Primary outcomes
1
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 Period (up to Week 14), up to end of Double-Blind Randomized-Withdrawal Period (up to Week 16)Secondary outcomes
1
Percentage of participants reporting minimally, much and very much worse in their CGIc questionnaire for IH or narcolepsy symptoms
Clinical Global Impression of Change (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 period (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)2
Percentage of participants reporting minimally, much and very much worse in their PGIc questionnaire for IH or narcolepsy symptoms
Patient Global Impression of Change (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 period (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)3
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 period (up to Week 14), up to end of Double-Blind Randomized-Withdrawal Period (up to Week 16)4
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 period (up to Week 14), up to end of Double-Blind Randomized-Withdrawal Period (up to Week 16)Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Jazz Pharmaceuticals
Lead sponsor
Jazz Pharmaceuticals Ireland Limited
Collaborator
Source ClinicalTrials.gov