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An Interventional Safety Switch Study (Segue Study) of XYWAV in Narcolepsy

Primary Purpose

Narcolepsy

Status
Completed
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
JZP-258
Sponsored by
Jazz Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Narcolepsy

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Age

  1. Participant must be 18 to 80 years of age (inclusive), at the time of signing the informed consent. Type of Participant and Disease Characteristics
  2. Participants who have a primary diagnosis of Type 1 or Type 2 narcolepsy that meets ICSD-3 criteria or DSM-5 criteria (Ruoff and Rye 2016), and are being currently treated with Xyrem, with or without additional anticataplectics or stimulants.
  3. Participants who have been taking Xyrem (with or without additional anticataplectics or stimulants eg, TCA, SNRI, SSRI, atomoxetine) in a stable dose and regimen for at least two months prior to screening, with evidence of clinical improvement on their current regimen, per the investigator's judgement. Only Xyrem will be substituted with XYWAV, with dose and regimen of any concomitant anticataplectics or stimulants remaining unchanged throughout the study.

    Sex and Contraceptive/Barrier Requirements

  4. Participant is male or female

    • A female participant is eligible to participate if she is not pregnant or breastfeeding, and one of the following conditions applies:

      • Is a woman of non-childbearing potential (WONCBP) as defined in Appendix 3 OR
      • Is a WOCBP and using a contraceptive method that is highly effective (with a failure rate of < 1% per year), preferably with low user dependency, as described in Appendix 3, during the study Intervention period and for at least 7 days after the last dose of study intervention. The investigator should evaluate the potential for contraceptive method failure (eg, noncompliance, recently initiated) in relationship to the first dose of study intervention.
    • A WOCBP must have a negative highly sensitive pregnancy test (serum) during screening.
    • The investigator is responsible for review of medical history, menstrual history, and recent sexual activity to decrease the risk for inclusion of a woman with an early undetected pregnancy.

    Informed Consent

  5. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

Exclusion Criteria:

Medical Conditions

  1. Have a diagnosis of narcolepsy, secondary to another medical condition (eg, central nervous system injury or lesion)
  2. Are currently prescribed a Xyrem regimen exceeding a dose of 9 grams nightly, or any single dose in excess of 6 grams.
  3. Have been diagnosed with restless leg syndrome (RLS) requiring treatment other than iron supplements
  4. Exhibit succinic semi-aldehyde dehydrogenase deficiency (SSADH)
  5. Have uncontrolled hypothyroidism
  6. Have a history of seizures, excluding early childhood non-pathological febrile seizures
  7. Have a history of head trauma associated with loss of consciousness in the past 5 years, or if the event occurred more than 5 years prior to screening and the participant experiences sequelae due to the event
  8. Show evidence of untreated or inadequately treated sleep-disordered breathing including:

    1. Presence of clinically significant and untreated obstructive or central sleep apnea (as determined by the investigator or documented previously); or one of the following:
    2. Apnea index (AI) >10 if on Obstructive Sleep Apnea (OSA) treatment or untreated, or
    3. Clinically significant hypoventilation, or
    4. Noncompliance with primary OSA therapy Note: "Non-compliance" is defined as positive airway pressure use of <4 hours per night on <70% of nights (<5 of 7 nights/week) per historical report (with investigator concurrence) of use of an oral appliance on <70% of nights (≥5 of 7 nights/week), or receipt of an effective surgical intervention for OSA symptoms.
  9. Experience parasomnias (eg, sleep walking, REM Sleep Behavior Disorder, etc.) considered by the investigator to negatively impact the conduct of the study. Parasomnia events associated with physical injury to the participant (or others) shall be discussed with the sponsor Medical Monitor.
  10. Meet criteria for current major depression based on clinical interview
  11. Have any clinically relevant medical, behavioral, or psychiatric disorder (other than narcolepsy) that is associated with excessive sleepiness
  12. Have a history or presence of bipolar disorder, bipolar related disorders, schizophrenia, schizophrenia spectrum disorders, or other psychotic disorders according to DSM-5 criteria
  13. Have a 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 participant's safety and/or interfere with the conduct of the study, in the opinion of the investigator
  14. Display relevant suicidality as indicated by Columbia Suicide Severity Rating Scale (C-SSRS) evaluation at screening
  15. Display moderate to severe depression as indicated by the Participant Health Questionnaire - 9 (PHQ-9) at screening
  16. Are a female participant who is pregnant or breastfeeding

    Prior/Concomitant Therapy

  17. Have undergone treatment with any prohibited central nervous system (CNS) agents, including but not limited to benzodiazepines, non-benzodiazepine anxiolytics/ hypnotics/sedatives, neuroleptics, opioids, barbiturates, phenytoin, ethosuximide, or MCT inhibitors, eg, 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 Monitors.

    Prior/Concurrent Clinical Study Experience

  18. Received any other investigational drug within 30 days or five half-lives (whichever is longer) prior to screening, or plan to use an investigational drug (other than the study intervention) during the study.

Sites / Locations

  • Wright Clinical Research, LLC
  • Sleep Disorders Center of Alabama
  • Pulmonary Associates of the Southeast, PC
  • Santa Monica Clinical Trials
  • Southern California Institute For Respiratory Diseases, Inc./ Tower Sleep Medicine
  • Southern California Institute For Respiratory Diseases, Inc.
  • Stanford University- Sleep Medicine
  • SDS Clinical Trials, Inc
  • Delta Waves, Inc.
  • Pulmonary Disease Specialists, PA d/b/a PDS Research
  • Clinical Research of West Florida, Inc
  • Florida Pediatric Research Institute, LLC
  • Sleep Practitioners LLC
  • Clinical Research Institute
  • The Center for Sleep & Wake Disorders
  • WMed Center for Clinical Research
  • Minnesota Lung Center
  • Clayton Sleep Institute, LLC
  • Clinical Research of Gastonia
  • Intrepid Research LLC
  • Cleveland Clinic, Sleep Disorders Center
  • Ohio Sleep Medicine Institute
  • Geisinger Clinic
  • Bogan Sleep Consultants, LLC
  • Medical University of South Carolina, Department of Psychiatry and Behavioral Sciences
  • Clinical Research of Rock Hill
  • FutureSearch Trials of Neurology
  • Sleep Therapy & Research Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Open-Label Conversion and Treatment Optimization

Arm Description

Conversion from Xyrem to XYWAV, maintaining the dose and regimen of any concomitant anticataplectics or stimulants unchanged throughout study.

Outcomes

Primary Outcome Measures

Number of Participants Converted from Xyrem to XYWAV

Secondary Outcome Measures

Change in Epworth Sleepiness Scale (ESS)
Changes in ESS scores between the Baseline period and ET or E/D, as applicable
Number of Participants with Participant Global Impression of Change (PGIc) Values
PGIc values will be measured at the ET or E/D, as applicable. The number of participants with each response will be summarized.
Time to Achieve Optimized Dose and Regimen
Defined as the time from the first dose and regimen to the optimized dose and regimen of XYWAV, where the optimized dose and regimen indicates the final dose and regimen that remains unchanged throughout the remainder of the Intervention period.
Number of Changes from the First Dose and Regimen to Optimized Dose and Regimen
Number of Participants Dosing Fasted Versus Dosing Without Consideration of Food
Duration of time between the last meal relative to dosing
The difference between the time of day that participants ate their last meal and the time of day participants take their first dose.
Characterization of Meals Relative to Dosing
Change in Weekly Rate of Cataplexy Attacks
Change in the Nausea Visual Analog Scale (NVAS)
Tolerability associated with Xyrem and XYWAV will be measured based on an NVAS assessment administered electronically.

Full Information

First Posted
February 25, 2021
Last Updated
November 30, 2022
Sponsor
Jazz Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT04794491
Brief Title
An Interventional Safety Switch Study (Segue Study) of XYWAV in Narcolepsy
Official Title
A Phase 4 Multicenter, Open-label, Single-arm Study of Safety, Tolerability, Effectiveness and Treatment Optimization in Participants Switching From Xyrem to XYWAV for the Treatment of Narcolepsy
Study Type
Interventional

2. Study Status

Record Verification Date
November 2022
Overall Recruitment Status
Completed
Study Start Date
April 9, 2021 (Actual)
Primary Completion Date
October 31, 2022 (Actual)
Study Completion Date
November 15, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Jazz Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

Brief Summary
The rationale for the interventional, open-label, single-arm design of JZP258-401 is to evaluate the clinical experience in participants with narcolepsy transitioning treatment from Xyrem to XYWAV.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Narcolepsy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
62 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Open-Label Conversion and Treatment Optimization
Arm Type
Experimental
Arm Description
Conversion from Xyrem to XYWAV, maintaining the dose and regimen of any concomitant anticataplectics or stimulants unchanged throughout study.
Intervention Type
Drug
Intervention Name(s)
JZP-258
Other Intervention Name(s)
XYWAV
Intervention Description
Maximum nightly dosage of 9 grams, administered once, twice or thrice nightly, with no single dose > 6 g
Primary Outcome Measure Information:
Title
Number of Participants Converted from Xyrem to XYWAV
Time Frame
Baseline to Week 8
Secondary Outcome Measure Information:
Title
Change in Epworth Sleepiness Scale (ESS)
Description
Changes in ESS scores between the Baseline period and ET or E/D, as applicable
Time Frame
Baseline to Week 8
Title
Number of Participants with Participant Global Impression of Change (PGIc) Values
Description
PGIc values will be measured at the ET or E/D, as applicable. The number of participants with each response will be summarized.
Time Frame
Week 8
Title
Time to Achieve Optimized Dose and Regimen
Description
Defined as the time from the first dose and regimen to the optimized dose and regimen of XYWAV, where the optimized dose and regimen indicates the final dose and regimen that remains unchanged throughout the remainder of the Intervention period.
Time Frame
Baseline to Week 8
Title
Number of Changes from the First Dose and Regimen to Optimized Dose and Regimen
Time Frame
Baseline to Week 8
Title
Number of Participants Dosing Fasted Versus Dosing Without Consideration of Food
Time Frame
Baseline to Week 8
Title
Duration of time between the last meal relative to dosing
Description
The difference between the time of day that participants ate their last meal and the time of day participants take their first dose.
Time Frame
Baseline to Week 8
Title
Characterization of Meals Relative to Dosing
Time Frame
Baseline to Week 8
Title
Change in Weekly Rate of Cataplexy Attacks
Time Frame
Baseline to Week 8
Title
Change in the Nausea Visual Analog Scale (NVAS)
Description
Tolerability associated with Xyrem and XYWAV will be measured based on an NVAS assessment administered electronically.
Time Frame
Baseline to Week 8

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Age Participant must be 18 to 80 years of age (inclusive), at the time of signing the informed consent. Type of Participant and Disease Characteristics Participants who have a primary diagnosis of Type 1 or Type 2 narcolepsy that meets ICSD-3 criteria or DSM-5 criteria (Ruoff and Rye 2016), and are being currently treated with Xyrem, with or without additional anticataplectics or stimulants. Participants who have been taking Xyrem (with or without additional anticataplectics or stimulants eg, TCA, SNRI, SSRI, atomoxetine) in a stable dose and regimen for at least two months prior to screening, with evidence of clinical improvement on their current regimen, per the investigator's judgement. Only Xyrem will be substituted with XYWAV, with dose and regimen of any concomitant anticataplectics or stimulants remaining unchanged throughout the study. Sex and Contraceptive/Barrier Requirements Participant is male or female A female participant is eligible to participate if she is not pregnant or breastfeeding, and one of the following conditions applies: Is a woman of non-childbearing potential (WONCBP) as defined in Appendix 3 OR Is a WOCBP and using a contraceptive method that is highly effective (with a failure rate of < 1% per year), preferably with low user dependency, as described in Appendix 3, during the study Intervention period and for at least 7 days after the last dose of study intervention. The investigator should evaluate the potential for contraceptive method failure (eg, noncompliance, recently initiated) in relationship to the first dose of study intervention. A WOCBP must have a negative highly sensitive pregnancy test (serum) during screening. The investigator is responsible for review of medical history, menstrual history, and recent sexual activity to decrease the risk for inclusion of a woman with an early undetected pregnancy. Informed Consent Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. Exclusion Criteria: Medical Conditions Have a diagnosis of narcolepsy, secondary to another medical condition (eg, central nervous system injury or lesion) Are currently prescribed a Xyrem regimen exceeding a dose of 9 grams nightly, or any single dose in excess of 6 grams. Have been diagnosed with restless leg syndrome (RLS) requiring treatment other than iron supplements Exhibit succinic semi-aldehyde dehydrogenase deficiency (SSADH) Have uncontrolled hypothyroidism Have a history of seizures, excluding early childhood non-pathological febrile seizures Have a history of head trauma associated with loss of consciousness in the past 5 years, or if the event occurred more than 5 years prior to screening and the participant experiences sequelae due to the event Show evidence of untreated or inadequately treated sleep-disordered breathing including: Presence of clinically significant and untreated obstructive or central sleep apnea (as determined by the investigator or documented previously); or one of the following: Apnea index (AI) >10 if on Obstructive Sleep Apnea (OSA) treatment or untreated, or Clinically significant hypoventilation, or Noncompliance with primary OSA therapy Note: "Non-compliance" is defined as positive airway pressure use of <4 hours per night on <70% of nights (<5 of 7 nights/week) per historical report (with investigator concurrence) of use of an oral appliance on <70% of nights (≥5 of 7 nights/week), or receipt of an effective surgical intervention for OSA symptoms. Experience parasomnias (eg, sleep walking, REM Sleep Behavior Disorder, etc.) considered by the investigator to negatively impact the conduct of the study. Parasomnia events associated with physical injury to the participant (or others) shall be discussed with the sponsor Medical Monitor. Meet criteria for current major depression based on clinical interview Have any clinically relevant medical, behavioral, or psychiatric disorder (other than narcolepsy) that is associated with excessive sleepiness Have a history or presence of bipolar disorder, bipolar related disorders, schizophrenia, schizophrenia spectrum disorders, or other psychotic disorders according to DSM-5 criteria Have a 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 participant's safety and/or interfere with the conduct of the study, in the opinion of the investigator Display relevant suicidality as indicated by Columbia Suicide Severity Rating Scale (C-SSRS) evaluation at screening Display moderate to severe depression as indicated by the Participant Health Questionnaire - 9 (PHQ-9) at screening Are a female participant who is pregnant or breastfeeding Prior/Concomitant Therapy Have undergone treatment with any prohibited central nervous system (CNS) agents, including but not limited to benzodiazepines, non-benzodiazepine anxiolytics/ hypnotics/sedatives, neuroleptics, opioids, barbiturates, phenytoin, ethosuximide, or MCT inhibitors, eg, 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 Monitors. Prior/Concurrent Clinical Study Experience Received any other investigational drug within 30 days or five half-lives (whichever is longer) prior to screening, or plan to use an investigational drug (other than the study intervention) during the study.
Facility Information:
Facility Name
Wright Clinical Research, LLC
City
Alabaster
State/Province
Alabama
ZIP/Postal Code
35007
Country
United States
Facility Name
Sleep Disorders Center of Alabama
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35213
Country
United States
Facility Name
Pulmonary Associates of the Southeast, PC
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35243
Country
United States
Facility Name
Santa Monica Clinical Trials
City
Los Angeles
State/Province
California
ZIP/Postal Code
90025
Country
United States
Facility Name
Southern California Institute For Respiratory Diseases, Inc./ Tower Sleep Medicine
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
Southern California Institute For Respiratory Diseases, Inc.
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
Stanford University- Sleep Medicine
City
Redwood City
State/Province
California
ZIP/Postal Code
94063
Country
United States
Facility Name
SDS Clinical Trials, Inc
City
Santa Ana
State/Province
California
ZIP/Postal Code
92705
Country
United States
Facility Name
Delta Waves, Inc.
City
Colorado Springs
State/Province
Colorado
ZIP/Postal Code
80918
Country
United States
Facility Name
Pulmonary Disease Specialists, PA d/b/a PDS Research
City
Kissimmee
State/Province
Florida
ZIP/Postal Code
34741
Country
United States
Facility Name
Clinical Research of West Florida, Inc
City
Tampa
State/Province
Florida
ZIP/Postal Code
33606
Country
United States
Facility Name
Florida Pediatric Research Institute, LLC
City
Winter Park
State/Province
Florida
ZIP/Postal Code
32789
Country
United States
Facility Name
Sleep Practitioners LLC
City
Macon
State/Province
Georgia
ZIP/Postal Code
31210
Country
United States
Facility Name
Clinical Research Institute
City
Stockbridge
State/Province
Georgia
ZIP/Postal Code
30281
Country
United States
Facility Name
The Center for Sleep & Wake Disorders
City
Chevy Chase
State/Province
Maryland
ZIP/Postal Code
20815
Country
United States
Facility Name
WMed Center for Clinical Research
City
Kalamazoo
State/Province
Michigan
ZIP/Postal Code
49008
Country
United States
Facility Name
Minnesota Lung Center
City
Edina
State/Province
Minnesota
ZIP/Postal Code
55435
Country
United States
Facility Name
Clayton Sleep Institute, LLC
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63123
Country
United States
Facility Name
Clinical Research of Gastonia
City
Gastonia
State/Province
North Carolina
ZIP/Postal Code
28054
Country
United States
Facility Name
Intrepid Research LLC
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45245
Country
United States
Facility Name
Cleveland Clinic, Sleep Disorders Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Ohio Sleep Medicine Institute
City
Dublin
State/Province
Ohio
ZIP/Postal Code
43017
Country
United States
Facility Name
Geisinger Clinic
City
Danville
State/Province
Pennsylvania
ZIP/Postal Code
17822
Country
United States
Facility Name
Bogan Sleep Consultants, LLC
City
Columbia
State/Province
South Carolina
ZIP/Postal Code
29201
Country
United States
Facility Name
Medical University of South Carolina, Department of Psychiatry and Behavioral Sciences
City
North Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Clinical Research of Rock Hill
City
Rock Hill
State/Province
South Carolina
ZIP/Postal Code
29732
Country
United States
Facility Name
FutureSearch Trials of Neurology
City
Austin
State/Province
Texas
ZIP/Postal Code
78731
Country
United States
Facility Name
Sleep Therapy & Research Center
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States

12. IPD Sharing Statement

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An Interventional Safety Switch Study (Segue Study) of XYWAV in Narcolepsy

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