A Patient-Centric, Prospective, Observational, Non-Interventional Switch Study of XYWAV in Narcolepsy

Transition Experience of Persons With Narcolepsy Taking Oxybate in the Real-world (TENOR): A Patient-Centric, Prospective, Observational, Non-Interventional Study to Assess Dosing Regimen in Persons Switching From Xyrem to Xywav for the Treatment of Narcolepsy

The rationale for the patient-centric, prospective, observational, non-interventional study design of JZP258-402 is to evaluate the transition experience of participants with narcolepsy treated with oxybate and to examine the impact of transitioning from Xyrem to Xywav in a real-world setting.

Study Overview

• Status: Completed
• Conditions: Narcolepsy
• Intervention / Treatment: Drug: Transition from Xyrem to Xywav

• Study Type: Observational
• Enrollment (Actual): 110

Contacts and Locations

Study Locations

• United States
  • California
    • Palo Alto, California, United States, 94304
      • Jazz Pharmaceuticals Clinical Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Male or female asges 18 years and older with diagnosis of type 1 or type 2 narcolepsy. On an active prescription for Xyrem with stable treatment for at least 2 consecutive months.

Description

Inclusion Criteria:

1. Male or female, aged 18 years or older
2. Diagnosis of type 1 or type 2 narcolepsy by a physician
3. Active prescription for Xyrem with a stable treatment regimen for at least 2 consecutive months
4. Transitioning from Xyrem to Xywav within the next or past 7 days (- 7 days or + 7 days from the first dose of Xywav)
5. Able to read and understand English
6. Able to access to a computer/smart phone with internet connection
7. Not currently a Jazz Pharmaceuticals employees or an immediate family member of a Jazz employee
8. Willing and able to comply with the study schedule
9. Willing and able to provide electronically written informed consent

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Transition from Xyrem to Xywav

Drug: Transition from Xyrem to Xywav; This is a single-group non-interventional study where participant's transitioning from Xyrem to Xywav will be observed. Investigational product (IP) or drug support or requested changes to their medication will not be provided to participants by Jazz Pharmaceuticals. The Xyrem and Xywav that the participants will take will be provided by the participant's health care provider.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants with Increased/Decreased/Same Dose Transitioning from Xyrem to Xywav	• Difference between usual Xyrem total nightly dose and initial prescribed total nightly Xywav dose.	Baseline to 21 Weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number Prescribed Dose Adjustments of Xywav	The number of dosing adjustments per participants and number of participants with one or more dosing adjustments during the Xywav Treatment Period (Period 2 to 5) by treating physician (dosing amount or number of nightly doses), details of adjustment, reason for adjustment and reason for unequal dosing will be summarized.	Baseline to 21 Weeks
Duration of Time Between the Last Meal Relative to Dosing	Timing and type of last meal prior to the first dose will be collected using both Daily Diary and Weekly Lookback of Daily Diary. The timing will be collected as actual time (HH:MM) from Daily Diary and as categorical data of usual number of hours relative to first dose (<0.5 hours, ≥0.5 but <1 hours, ≥1 but <1.5 hours, ≥1.5 but <2 hours, ≥2 hours) from Weekly Lookback of Daily Diary. In order to evaluate the timing of last meal across all study periods, the Daily Diary entry will be converted into categorical data using the same value scale as Weekly Lookback of Daily Diary. The type of last meal prior to the first dose will be collected as categorical data (regular meal, snack, and beverage). For both timing and type of last meal prior to the first dose, the number of participants with each response will be summarized.	Baseline to 21 Weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to Achieve Stable Dose of Xywav	Stable dose is defined as the dose and regimen which remain unchanged for at least 2 weeks. Compliance is not considered in the derivation of the time to stable dose. The time to achieve stable dose will be summarized.	Baseline to 21 Weeks
Number of Participants Achieving Treatment Adherence	Skipped doses will be collected using both the Daily Diary and Weekly Lookback Daily Diary. Skipped doses will be collected daily (yes/no) from the Daily Diary and as a weekly categorical variable (0, 1-2, 3-4, 5-6, or 7 days) from the Weekly Lookback of Daily Diary. In order to summarize skipped doses during the Xywav Treatment Period, the Daily Diary data will be converted to categorical data using the same categories as in the Weekly Lookback of Daily Diary. Skipped doses per week (0, 1-2, 3-4, 5-6, 7 days), most common dosing change (did not take Xywav, skipped first dose, skipped second dose, skipped third dose) and most common reasons for dosing change will be summarized.	Baseline to 21 Weeks
Number of Participants Who Discontinued Xywav Administration	The number of participants who discontinued Xywav administration during the Xywav Treatment Period (Period 2 to 5).	Baseline to 21 Weeks
Change in Short-term and Long-term Nausea Using Patient Global Impression of Change (PGI-C)	The PGI-C is a series of self-administered questions designed to measure the meaningfulness of change in symptoms following an intervention. The PGI-C is used to assess the change in excessive daytime sleepiness, cataplexy, and short- and long-term nausea since the transition from Xyrem to Xywav. It is a 7-point Likert-type rating scale from 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, to 7 = very much worse. The score is the sum of responses to each of the answer choices.	Baseline to 21 Weeks
Changes in Sleep Pattern and Quality	Sleep pattern (how long usually took to fall asleep after the first dose, how long usually spent awake during the night) will be summarized. Sleep pattern and quality will be collected using both Daily Diary and Weekly Lookback of Daily Diary. The number of participants with each response to sleep quality (very poor ~ very good) and feeling on how rested/refreshed when woke-up for the day (not at all rested ~ very well rested) will be summarized by day and by week. In order to analyze the data together the intention is to combine the daily by deriving the categorical frequency for the responses so that it can be combined with the categorical data collected in the weekly diary. Which will allow for a full analysis of the change from the Baseline period (Xyrem) to Xywav Treatment Period (Period 2 to 5) will be summarized for weekly data collected during the Xywav Treatment Period (Period 2 to 5) using Weekly Lookback of Daily Diary.	Baseline to 21 Weeks
Change in Epworth Sleepiness Scale (ESS)	The ESS is a self-administered questionnaire with 8 questions (Johns 1991, Johns 1992). Respondents rate on a 4-point scale (0-3) their usual chances of dozing off or falling asleep while engaged in eight different activities. Most people engage in those activities at least occasionally, although not necessarily every day. The ESS total score (the sum of 8 item scores, 0-3) can range from 0 to 24. Higher ESS total scores are associated with higher sleep propensity in daily life, also referred to as 'daytime sleepiness'. The ESS has been validated in several conditions, including narcolepsy (Johns 1994).	Baseline to 21 Weeks
Change in Functional Outcomes of Sleep Questionnaire (FOSQ-10) Total Score	The FOSQ-10 (Chasens 2009) is a self-administered questionnaire designed to assess the impact of daytime sleepiness on activities of daily living. It consists in 10 items measuring the participant's general productivity, activity level, vigilance, and intimacy and sexual relationships. For each item, participants are asked to rate the level of difficulty in completing an activity on a 4-point Likert scale, from 1 "Yes, extreme" to 4 "No"). From the FOSQ-10 a total score is calculated: a higher score indicates better functional status. The FOSQ-10 is a reduced version of the original FOSQ-30. The FOSQ-10 was tested and found to be a psychometrically strong instrument performing similarly to the long version.	Baseline to 21 Weeks
Change in British Columbia Cognitive Complaint Inventory (BC-CCI) Total Score	The BC-CCI was developed to measure cognitive complaints in individuals with Major Depressive Disorder (MDD). It is a 6-item self-report measure that asks respondents to rate problems with specific cognitive symptoms over the past 7 days. A four point scale (0 "Not at all" to 3 "Very much") is used to rate each item. The total score (ranges from 0 to 18) that is generated assesses domains of memory, concentration, trouble expressing thoughts, word-finding, and problem solving. Higher scores indicate greater severity of cognitive impairment, with four established classification ranges for the BC-CCI total score: 0 to 4 = "broadly normal"; 5 to 8 = "mild" cognitive complaints; 9 to 14 = "moderate" cognitive complaints; and 15 to 18 = "severe" cognitive complaints. Three additional items that ask about how these symptoms impact work, relationships and social/recreational activities are also included to provide further information on the nature of functional impairment.	Baseline to 21 Weeks
Change in Cataplexy using PGI-c	The PGI-C is a series of self-administered questions designed to measure the meaningfulness of change in symptoms following an intervention. The PGI-C is used to assess the change in excessive daytime sleepiness, cataplexy, and short- and long-term nausea since the transition from Xyrem to Xywav. It is a 7-point Likert-type rating scale from 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, to 7 = very much worse. The score is the sum of responses to each of the answer choices.	Baseline to 21 Weeks

Collaborators and Investigators

• Sponsor: Jazz Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): April 9, 2021
• Primary Completion (Actual): February 24, 2022
• Study Completion (Actual): February 24, 2022

Study Registration Dates

• First Submitted: March 9, 2021
• First Submitted That Met QC Criteria: March 16, 2021
• First Posted (Actual): March 18, 2021

Study Record Updates

• Last Update Posted (Actual): June 30, 2022
• Last Update Submitted That Met QC Criteria: June 27, 2022
• Last Verified: June 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Nervous System Diseases; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Disorders of Excessive Somnolence; Narcolepsy
• Other Study ID Numbers: JZP258-402

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No