A Study Evaluating the Efficacy and Safety of Xywav Expanded Dosing vs Placebo in Participants With Narcolepsy or IH

Trial statusNot yet recruiting
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 volunteers

Qualifiers

Has a primary diagnosis of IH or narcolepsy Type 1 or Type 2 (NT1 or NT2)

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.

If currently treated with oxybate, must have documented improvement of EDS with oxybate treatment per the investigator's clinical judgement.

If currently treated with oxybate, has been taking the same stable dosing regimen at a total nightly dosage of 3 g to 9 g (inclusive) for at least 2 months at screening.

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

    Drug

    0.5 g/ml calcium, magnesium, potassium, and sodium oxybates solution taken by mouth

  • Placebo

    Other intervention

    Placebo solution taken by mouth

Treatment groups

108 Participants
are divided into 4 treatment groups
Group A: Once-nightly stable dose Xywav groupActive comparator 1 intervention
Group B: Once-nightly placebo groupPlacebo comparator 2 interventions
Group C: Twice nightly stable dose Xywav groupActive comparator 1 intervention
Group D: Twice nightly 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 visit (up to Week 14), up to end of Double-Blind Randomized-Withdrawal Period (up to Week 16)

Secondary outcomes

1

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)
2

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)
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 visit (up to Week 14), up to end of Double-Blind Randomized-Withdrawal Period (up to Week 16)
4

Change in NSS scores in participants with NT1

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)

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Jazz Pharmaceuticals

Lead sponsor

Jazz Pharmaceuticals Ireland Limited

Collaborator

This trial is not recruiting at the moment. You can still explore other options: