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    Summary
    EudraCT Number:2016-000426-20
    Sponsor's Protocol Code Number:15-006
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-01-30
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2016-000426-20
    A.3Full title of the trial
    A Double-Blind, Placebo-Controlled, Randomized-Withdrawal, Multicenter Study of the Efficacy and Safety of JZP-258 in Subjects with Narcolepsy with Cataplexy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of the Effect of JZP-258 in Patients with Cataplexy and Narcolepsy
    A.3.2Name or abbreviated title of the trial where available
    JZP-258 Efficacy Study
    A.4.1Sponsor's protocol code number15-006
    A.5.4Other Identifiers
    Name:IND Number:49,641
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorJazz Pharmaceuticals Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportJazz Pharmaceuticals Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJazz Pharmaceuticals Inc.
    B.5.2Functional name of contact pointMario D'Achille
    B.5.3 Address:
    B.5.3.1Street Address1818 Market Street, Suite 2350
    B.5.3.2Town/ cityPhiladelphia
    B.5.3.3Post codePA 19103
    B.5.3.4CountryUnited States
    B.5.4Telephone number1215710 8317
    B.5.6E-mailmario.dachille@jazzpharma.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameJZP-258, 500mg/ml oral solution
    D.3.2Product code JZP-258
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNoxybate (4-hydroxybutanoic acid, γ- hydroxybutyrate, 4-hydroxybutyrate, or GHB)
    D.3.9.1CAS number 502-85-2
    D.3.9.3Other descriptive nameoxybate mixed salt solution: sodium oxybate, potassium oxybate, calcium oxybate, magnesium oxybate
    D.3.10 Strength
    D.3.10.1Concentration unit g/ml gram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.5
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral solution
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Treatment of cataplexy in narcolepsy
    Treatment of excessive daytime sleepiness (EDS) in narcolepsy
    E.1.1.1Medical condition in easily understood language
    Sleep Disorder
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10015595
    E.1.2Term Excessive daytime sleepiness
    E.1.2System Organ Class 100000004852
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10007738
    E.1.2Term Cataplexy and narcolepsy
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of JZP-258 in the treatment of cataplexy in subjects with narcolepsy
    E.2.2Secondary objectives of the trial
    Key Secondary Objective
    • To evaluate the efficacy of JZP-258 in the treatment of excessive daytime sleepiness (EDS) in subjects with narcolepsy .

    Secondary Objective
    • To evaluate the safety of JZP-258 in the treatment of subjects with narcolepsy with cataplexy

    Exploratory Objective
    • To characterize the conversion from non-Xyrem anticataplectic treatment regimens to JZP-258 in subjects with narcolepsy with cataplexy.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Main study
    1. Male or female subjects between 18 and 70 years of age, inclusive at screening.

    2. 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.

    3. Have a history of having at least 14 cataplexy attacks in a typical 2-week period and clinically significant symptoms of EDS prior to any narcolepsy treatment.

    4. Treatment status (Pre-randomization Group) at study entry:
    a) Have been taking Xyrem at unchanged doses (twice or thrice nightly dosing no higher than a total of 9 g/night), for the treatment of cataplexy in narcolepsy for at least 2 months prior to screening; or
    b) Have been taking Xyrem at unchanged doses (twice or thrice nightly dosing no higher than a total of 9 g/night), and another anticataplectic (TCA, SNRI, SSRI, atomoxetine, or other) for the treatment of cataplexy in narcolepsy for at least 2 months prior to screening; or
    c) Treated with a non-Xyrem anticataplectic (TCA, SNRI, SSRI, atomoxetine, or other) and not treated with Xyrem; or
    d) Not treated with any agent with anticataplectic properties.

    5. If currently treated with Xyrem, must have documented clinical improvement of cataplexy and EDS per Investigator's clinical judgment.

    6. 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.

    7. Have used a medically acceptable method of contraception* for at least 2 months prior to the first dose of study drug and consent to use a medically acceptable method of contraception throughout the entire study period and for 90 days after the study is completed.

    8. Willing and able to comply with the study design schedule and other requirements.

    9. Willing and able to provide written informed consent.
    Open Label
    1.Completion of the JZP-258 double-blind treatment and completion of Visit 16
    2.Is able, in the opinion of the investigator, to take JZP-258 for an additional 24 weeks
    3.Agrees to continue to use a medically acceptable method of contraception throughout the entire study period and for 90 days after the Open-Label Extension is completed.
    4.Willing and able to comply with the study design schedule and other requirements.
    5.Willing and able to provide written informed consent for Open-Label Extension.
    6.If currently being treated with Xyrem, the subject's total twice nightly Xyrem dose must be no higher than 9 g/night
    E.4Principal exclusion criteria
    Main Study
    1. Narcolepsy secondary to another medical condition(e.g., CNS injury or lesion).

    2. Restless leg syndrome (RLS) requiring treatment other than iron supplements.

    3. Succinic semi-aldehyde dehydrogenase deficiency (SSADH).

    4. Uncontrolled hypothyroidism.

    5. History of seizures, excluding early childhood non-pathological febrile seizures.

    6. 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 subject has sequelae due to the event.

    7. Evidence of untreated or inadequately treated sleep-disordered breathing including:
    a. Presence of clinically significant and untreated obstructive or central sleep apnea as determined by the Investigator or documented previously; or documentation of one of the following:
    b. Apnea index (AI) >10 if on OSA treatment or untreated; or
    c. Clinically significant hypoventilation; or
    d. Noncompliance with primary OSA therapy. (Compliance defined as positive airway pressure use of ≥4 hours per night on ≥70% of nights [≥5 of 7 nights/week], 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).

    8. Parasomnias (e.g., sleep walking, REM Sleep Behavior Disorder, etc.) felt by the investigator to negatively affect the conduct of the study. Parasomnia events associated with physical injury to the subject (or others) shall be discussed with the Medical Monitor.

    9. Meets criteria for current major depression by clinical interview.

    10. Any other clinically relevant medical, behavioral, or psychiatric disorder other than narcolepsy that is associated with excessive sleepiness.

    11. History or presence of bipolar disorder, bipolar related disorders, schizophrenia, schizophrenia spectrum disorders, or other psychotic disorders according to DSM-5 criteria.

    12. 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.

    13. A current electrocardiogram (ECG) with clinically significant deviation(s) from normal, or clinically significant physical examination findings, as determined by the Investigator at screening.

    14. Any current clinically significant laboratory abnormality as determined by the Investigator at screening.

    15. Is a female subject who is pregnant, nursing or lactating.

    16. A positive urine drug screen for benzodiazepines or drugs of abuse, a positive alcohol test, a history of substance abuse including alcohol abuse, or unwillingness to refrain from consuming alcohol during the study (if the subject takes prescribed amphetamines, a positive result for amphetamines will not exclude the subject).

    17. Treatment with any central nervous system sedating agents including but not limited to benzodiazepines, non-benzodiazepine anxiolytics /hypnotics/sedatives, neuroleptics, opioids, barbiturates, phenytoin, ethosuximide, or the monocarboxylate transporter (MCT) inhibitor valproate, within 2 weeks prior to enrollment (Day 1) (discontinuation for the purpose of study enrollment is permitted only if considered safe by the Investigator and approved by the Medical Monitor).

    18. 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.

    19. Current treatment with oral isotretinoin.

    20. Received any investigational drug within 30 days or 5 half-lives (whichever is longer) before the Screening Visit.

    21. Allergy or sensitivity to malic acid, sucralose or ingredients in the study drug formulation or placebo.

    22. Unsafe for the subject to receive placebo treatment for 2 weeks, in the opinion of the Investigator.
    Open Label
    1.Meet Exclusion Criteria 1 through 19 from the Main Study at Visits 18 and 19.
    2.Received any investigational drug (with the exception of JZP 258) within 30 days or 5 half-lives (whichever is longer) before the Screening Visit.
    3.Allergy or sensitivity to malic acid, sucralose or ingredients in the study drug formulation.
    E.5 End points
    E.5.1Primary end point(s)
    Change in weekly number of cataplexy attacks from the two weeks of the Stable-Dose Period to the two weeks of the Double-Blind Randomized-Withdrawal Period
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of stable-dose period to end of double- blind randomized-withdrawal period (2 weeks)
    E.5.2Secondary end point(s)
    • Change in the Epworth Sleepiness Scale (ESS) score from the end of the Stable- Dose Period to the end of the Double-Blind Randomized-Withdrawal Period.
    • PGIc for narcolepsy overall at the end of the Double-Blind Randomized-Withdrawal Period.
    • CGIc for narcolepsy overall at the end of the Double-Blind Randomized-Withdrawal Period.
    • Change in QoL (SF-36) from the end of the Stable-Dose Period to the end of the Double-Blind Randomized-Withdrawal Period.
    • QoL (EuroQol 5 Dimensions Self-Report Questionnaire) from the end of the Stable-Dose Period to the end of the Double-Blind Randomized-
    Withdrawal Period.
    E.5.2.1Timepoint(s) of evaluation of this end point
    End of stable-dose period to end of double-blind randomized-withdrawal period (2 weeks)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Followed by a 24-week Open-Label Extension study
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA60
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Belgium
    Croatia
    Czech Republic
    Denmark
    Finland
    France
    Germany
    Italy
    Netherlands
    Poland
    Spain
    Sweden
    Switzerland
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 167
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 18
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 185
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    JZP-258 will not be available to the subject after their participation in the study has ended.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-03-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-05-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-07-10
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