A Study of the Efficacy and Safety of JZP-258 in Subjects With Narcolepsy With Cataplexy
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|ClinicalTrials.gov Identifier: NCT03030599|
Recruitment Status : Completed
First Posted : January 25, 2017
Results First Posted : November 12, 2020
Last Update Posted : November 12, 2020
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|Condition or disease||Intervention/treatment||Phase|
|Narcolepsy With Cataplexy||Drug: JZP-258 Other: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||201 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||A Double-Blind, Placebo-Controlled, Randomized-Withdrawal, Multicenter Study of the Efficacy and Safety of JZP-258 in Subjects With Narcolepsy With Cataplexy|
|Actual Study Start Date :||March 14, 2017|
|Actual Primary Completion Date :||January 24, 2019|
|Actual Study Completion Date :||July 10, 2019|
Placebo Comparator: Placebo
Matching placebo solution (aqueous solution containing sodium citrate, malic acid, and sucralose; all ingredients were compendial [United States Pharmacopeia/ National Formulary])
JZP-258 oral solution 0.5 g/mL, which is equivalent to 0.413 g/mL of oxybate
Other Name: Xyrem®
- Change in Weekly Number of Cataplexy Attacks [ Time Frame: Change from baseline (2 weeks of the Stable Dose Period) to the 2 weeks of the Double Blind Randomized Withdrawal Period (DB RWP) ]Participants completed a daily Cataplexy Frequency Diary each night prior to bedtime. Participants were to record the number of cataplexy attacks that they had each day.
- Change in the Epworth Sleepiness Scale (ESS) Score [ Time Frame: From the end of the Stable Dose Period to the end of the Double Blind Randomized Withdrawal Period ]This is the key secondary endpoint. The Epworth Sleepiness Scale (ESS) was a self-administered questionnaire with 8 questions. Participants were asked to rate, on a 4-point scale (0-3), their usual chances of dozing off or falling asleep while engaged in eight different activities. Most participants engaged in those activities at least occasionally, although not necessarily every day. The ESS score (the sum of 8 item scores, 0-3) can range from 0 to 24. The higher the ESS score, the higher that participants average sleep propensity in daily life (ASP), or their 'daytime sleepiness'.
- Number of Participants With Worsening Patient Global Impression of Change (PGIc) for Narcolepsy Overall [ Time Frame: At the end of the Double Blind Randomized Withdrawal Period ]At the end of the Double Blind Randomized Withdrawal Period (DB RWP), participants rated the change in their condition on a 7-point scale ranging from 1 = "very much improved" to 7 = "very much worse" since the last visit. This endpoint measures the percentage of participants with worsening PGIc scores for narcolepsy overall (defined as scores of Much Worse or Very Much Worse).
- Number of Participants With Worsening Clinical Global Impression of Change (CGIc) for Narcolepsy Overall [ Time Frame: At the end of the Double Blind Randomized Withdrawal Period ]At the end of the Double Blind Randomized Withdrawal Period, Investigators rated their impression of any change in the severity of the participant's narcolepsy overall condition since the start of the Double Blind Randomized Withdrawal Period on a 7-point scale ranging from 1 = "very much improved" to 7 = "very much worse". This endpoint measures the percentage of participants with worsening CGIc scores for narcolepsy overall, defined as scores of Much Worse or Very Much Worse.
- Change in 36-Item Short Form Health Survey Version 2 (SF-36v2) Scores [ Time Frame: At the End of the Stable Dose Period to the End of the Double Blind Randomized Withdrawal Period ]The SF-36v2 is a multi-purpose, short-form health survey with 36 questions/ items. It yields an 8-scale profile of functional health and well-being scores as well as a psychometrically-based physical and mental overall component summary measures. Two summary scores were derived using the SF-36v2. Physical Component Summary measures dimensions of functional health that are meaningful to respondents, including the impact of health and health-related changes on physical function, pain, and the ability to carry out daily roles. The Mental Component Summary component scale measures the impact of health and health-related changes on well-being, including vitality, social function, and emotional well-being. Participants self-report on items in a summary that have between 2-6 choices per item (e.g. none of the time, some of the time, etc.). Summations of item scores were transformed into a range from 0 to 100; zero= worst HRQL, 100=best HRQL. Higher scores indicate better health status.
- Change in 5-level EQ-5D (EQ-5D-5L) Crosswalk Index Score and Visual Analog Scale [ Time Frame: At the End of the Stable Dose Period to the End of the Double Blind Randomized Withdrawal Period ]The EQ-5D-5L is a measure of health outcome that includes a descriptive system consisting of 5 dimensions (mobility, self-care, usual activities, pain/ discomfort, and anxiety/ depression). The EQ-5D-5L includes 5 levels of severity for each of the 5 dimensions of the descriptive system (1= no problems, 2= slight problems, 3= moderate problems, 4= severe problems, and 5= extreme problems) that reflect increasing levels of difficulty. The 5 digit health states for each dimension are converted into a single value per country (0= equivalent to death, 1= equivalent to best imaginable health and values below 0= health states rated worse than death capped at -1), using the EQ-5D-5L crosswalk index value calculator as recommended by EuroQol group. A visual analogue scale (VAS) used within this scale recorded the participants self-rated health on a VAS and the endpoints resulted in a numeric value set ranging from 0 (= worst imaginable health state) up to 100 (= best imaginable health state).
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|Ages Eligible for Study:||18 Years to 70 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Male or female subjects between 18 and 70 years of age, inclusive.
- Have a primary diagnosis of narcolepsy with cataplexy that meets ICSD-3 criteria or DSM-5 criteria, and currently untreated or treated with or without anticataplectics.
- If applicable, treated with a stimulant or alerting agent at unchanged doses for at least 2 months prior to dosing or not treated with a stimulant or alerting agent.
- Willing and able to comply with the study design schedule and other requirements.
- Willing and able to provide written informed consent.
- Narcolepsy secondary to another medical condition (e.g., CNS injury or lesion)
- History or presence of any unstable or clinically significant medical condition, behavioral or psychiatric disorder (including active suicidal ideation), or history or presence of another neurological disorder or surgical history that might affect the subject's safety and/or interfere with the conduct of the study in the opinion of the Investigator.
- Treatment with any central nervous system sedating agents, including but not limited to benzodiazepines, nonbenzodiazepine anxiolytics/ hypnotics/sedatives, neuroleptics, opioids, barbiturates, phenytoin, ethosuximide, or MCT inhibitors, e.g. diclofenac, valproate, ibuprofen, within 2 weeks prior to enrollment (discontinuation for the purpose of study enrollment is permitted only if considered safe by the Investigator and approved by the Medical Monitor).
- Treatment with an antidepressant for cataplexy, if the withdrawal of the antidepressant during cross-titration with JZP-258 might be unsafe due to prior history of depression.
- Unsafe for the subject to receive placebo treatment for 2 weeks, in the opinion of the Investigator.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03030599
|Study Director:||Director Clinical Trial Disclosure & Transparency||Jazz Pharmaceuticals|
Documents provided by Jazz Pharmaceuticals:
|Responsible Party:||Jazz Pharmaceuticals|
|Other Study ID Numbers:||
2016-000426-20 ( EudraCT Number )
|First Posted:||January 25, 2017 Key Record Dates|
|Results First Posted:||November 12, 2020|
|Last Update Posted:||November 12, 2020|
|Last Verified:||November 2020|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Disorders of Excessive Somnolence
Sleep Disorders, Intrinsic
Sleep Wake Disorders
Nervous System Diseases