A Study to Investigate Efficacy, Safety, Tolerability, and Pharmacokinetics of JZP441 Compared With Placebo in Participants With Narcolepsy Type 1
- Registration Number
- NCT06961266
- Lead Sponsor
- Jazz Pharmaceuticals
- Brief Summary
Narcolepsy is a sleep disorder in which patients are not able to maintain wakefulness or require treatment to maintain wakefulness during the daytime. Narcolepsy is a lifelong neurologic disease for which no cure has been clinically available. JZP441 is currently being developed for the treatment of narcolepsy type 1 (NT1). This study will assess the safety of efficacy of JZP441 in adult patients with NT1.
- Detailed Description
This Phase 1b, randomized, double-blind, sponsor-unblinded, placebo-controlled 4-way crossover study will evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of a range of JZP441 doses in participants with NT1. Changes in daytime sleepiness will be assessed via objective (MWT) and subjective (KSS, VAS for sleepiness) efficacy measures.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 8
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Arm && Interventions
Group Intervention Description JZP441 Dose Level 1 JZP441 Participants with narcolepsy type 1 who are randomized to receive JZP441. JZP441 Dose Level 2 JZP441 Participants with narcolepsy type 1 who are randomized to receive JZP441. JZP441 Dose Level 3 JZP441 Participants with narcolepsy type 1 who are randomized to receive JZP441. Placebo Matching Placebo Participants with narcolepsy type 1 who are randomized to receive matching placebo.
- Primary Outcome Measures
Name Time Method Mean sleep latency of the first 4 sessions of the Maintenance of Wakefulness Test 1, 3, 5, and 7 hours after the first dose of study intervention The MWT is the standard objective measure of an individual's ability to remain awake during the daytime in a darkened quiet environment and is commonly used to assess response to treatment. The MWT will be used to compare the pharmacodynamic response of JZP441 versus placebo. Sleep latency will be reported in minutes.
- Secondary Outcome Measures
Name Time Method Number of Participants Reporting Treatment-emergent Adverse Events Baseline up to Week 11 A treatment-emergent adverse event (TEAE) is defined as an adverse event (AE) that started, or worsened in severity or seriousness, following a dose of study intervention.
Pharmacokinetic Parameter Maximum Plasma Concentration Day 1 (predose), 2, 4, 6, 8, 10, and 12 hours post first dose; Day 2 (24 hours) post first dose. Mean Score of the First 4 assessments of Karolinska Sleepiness Scale 1, 3, 5, 7, 9 and 11 hours after first dose The Karolinska Sleepiness Scale (KSS) is a single-item, 9-point, self-administered questionnaire that measures a participant's subjective level of sleepiness (from "extremely alert" to "extremely sleepy, can't keep awake"). Scores generally decrease with longer periods of wakefulness, indicating that lower scores correlate with better outcomes.
Mean VAS Score For Sleepiness 1, 3, 5, 7, 9 and 11 hours after first dose The self-reported VAS measure of sleepiness in the current study will be a retrospective measure of how sleepy the participant felt throughout the day, with anchors at each end of the line labeled as "0=not at all sleepy" to "100=very sleepy." Higher VAS scores indicate worse outcome.
Pharmacokinetic Parameter Area Under the Plasma Concentration Curve Day 1 (predose), 2, 4, 6, 8, 10, and 12 hours post first dose; Day 2 (24 hours) post first dose. Pharmacokinetic Parameter Time to Maximum Plasma Concentration Day 1 (predose), 2, 4, 6, 8, 10, and 12 hours post first dose; Day 2 (24 hours) post first dose.
Trial Locations
- Locations (1)
Clinical Trial Site
🇺🇸Columbia, South Carolina, United States
Clinical Trial Site🇺🇸Columbia, South Carolina, United States