Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43871   clinical trials with a EudraCT protocol, of which   7290   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2014-001389-93
    Sponsor's Protocol Code Number:13-005
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-06-19
    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 ConcernedFrance - ANSM
    A.2EudraCT number2014-001389-93
    A.3Full title of the trial
    A Double-Blind, Placebo-Controlled, Randomized-Withdrawal,
    Multicenter Study of the Efficacy and Safety of Xyrem with an Open- Label Pharmacokinetic Evaluation and Safety Extension in Pediatric 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 Efficacy and Safety of Xyrem in Pediatric Subjects with Narcolepsy with Cataplexy
    A.4.1Sponsor's protocol code number13-005
    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
    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
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJazz Pharmaceuticals
    B.5.2Functional name of contact pointGrace Wang, Senior Director
    B.5.3 Address:
    B.5.3.1Street Address3180 Porter Drive
    B.5.3.2Town/ cityPalo Alto- California
    B.5.3.3Post codeCA 94304
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16504962687
    B.5.5Fax number+16504962681
    B.5.6E-mailgrace.wang@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 Yes
    D.2.1.1.1Trade name Xyrem
    D.2.1.1.2Name of the Marketing Authorisation holderUCB Pharma
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 nameXyrem (sodium oxybate) oral solution
    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 INNNA
    D.3.9.1CAS number 502-85-2
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive nameSODIUM OXYBATE
    D.3.9.4EV Substance CodeSUB14731MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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
    Narcolepsy with Cataplexy
    E.1.1.1Medical condition in easily understood language
    Narcolepsy with Cataplexy
    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 18.0
    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 No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary objectives are:
    1) To evaluate the efficacy of Xyrem (sodium oxybate) oral solution in the treatment of cataplexy in pediatric subjects with narcolepsy
    2) To evaluate the safety of Xyrem in the treatment of cataplexy in pediatric subjects with narcolepsy for up to one year
    E.2.2Secondary objectives of the trial
    Secondary objectives are:
    1) To evaluate the efficacy of Xyrem in the treatment of excessive daytime sleepiness (EDS) in pediatric subjects with narcolepsy with cataplexy
    2) To characterize the pharmacokinetics (PK) of Xyrem in pediatric subjects (ages 7-17 years) with narcolepsy with cataplexy
    3) To evaluate the safety of titrating Xyrem in pediatric subjects to an effective and tolerable dose
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Open-label PK evaluation
    E.3Principal inclusion criteria
    1. Male or female subjects aged 7-16 years at Visit 2 for subjects on Xyrem at study entry and at Visit 1.1 for Xyrem-naïve subjects (to ensure subjects are <18 years of age at the end of the study)
    2. Have a primary diagnosis of narcolepsy with cataplexy that meets International Classification of Sleep Disorders (ICSD)-2 or ICSD-3 criteria, whichever was in effect at the time of the diagnosis
    3. Be positive for the Human Leukocyte Antigen (HLA) DQB1:0602 haplotype, determined prior to the study or as part of the study screening procedures
    4. Have given documented assent indicating that he/she was aware of the investigational nature of the study and the required procedures and restrictions efore participation in any protocol-related activities
    5. Have parent(s)/guardian(s) who have given informed consent for his/her/their child’s participation in the study
    6. 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
    7. Be willing to spend the required number of nights (2 to 3) in a sleep laboratory for PSG evaluations
    8. If currently treated with Xyrem, must have been taking unchanged doses (twice nightly dosing no higher than 9 g/night) of Xyrem, and stimulants, if applicable, for the treatment of narcolepsy symptoms for at least 2 months prior to screening
    9. If currently treated with Xyrem, must have demonstrated clinical improvement of cataplexy per Investigator’s clinical judgment
    10. Have agreed to abstain from caffeinated products during PSG and PK Nights
    11. Any female subject of child-bearing potential must be willing to use a method of contraception, deemed medically acceptable by the Investigator, or agree to abstain from sexual intercourse for the duration of the study and for 30 days after study termination
    12. Any male subject who is sexually active with a female partner must be willing to use a method of contraception, deemed medically acceptable by the Investigator, or agree to abstain from sexual intercourse for the duration of the study and for 30 days after study termination

    In addition to the above inclusion criteria, subjects participating in the PK evaluation must meet the following inclusion criteria:
    13. Be willing to spend 2 additional nights in the clinic for PK evaluation
    14. Have been taking Xyrem alone or Xyrem and stimulants for the treatment of narcolepsy symptoms for at least 2 months prior to screening. Xyrem and, if
    applicable, stimulants must have been taken at unchanged doses and regimens for the prior 2 months
    15. Have given additional documented assent and consent by the parent(s) or guardian(s) indicating awareness of the investigational nature of the PK evaluation and the required procedures and restrictions before participation in any protocol-related activities
    16. Have sufficient blood volume for PK sampling based on body weight in accordance with Seattle Children’s Hospital guideline (Appendix 3) or, for any particular investigational site, Institutional Review Board (IRB) eligibility guidelines for pediatric blood collection relevant to that site
    E.4Principal exclusion criteria
    1. Not able to understand assent or follow study instructions, in the Investigator's opinion
    2. Parent(s)/guardian(s) unable to comply with the study requirements, in the Investigator's opinion
    3. Previously treated with Xyrem and discontinued because of lack of efficacy and/or tolerability issues
    4. Narcolepsy secondary to another medical condition, e.g., CNS injury or lesion
    5. Restless leg syndrome requiring treatment other than iron supplements
    6. Succinic semi-aldehyde dehydrogenase deficiency
    7. Uncontrolled hypothyroidism
    8. History of seizures, excluding early childhood non-pathological febrile seizures
    9. History of head trauma associated with loss of consciousness
    10. Evidence of sleep-disordered breathing including:
    a. Presence of clinically significant obstructive or central sleep apnea as determined by the Inv. or documented previously; or
    b. Obstructive AHI >5 for subjects 7-11 years of age or obstructive AHI >10 for subjects 12-17 years of age; or
    c. Oxygen saturation nadir ≤85% at night; or
    d. Clinically significant hypoventilation
    11. Oxygen saturation level <95% for at least 5 minutes on room air as measured by pulse oximetry while fully awake during daytime monitoring, or subjects with known or suspected respiratory difficulty, or any condition that may compromise a subject’s breathing. If oxygen saturation values lower than 95% are observed at study sites at high geographic elevations and are acceptable to the Investigator, enrollment of the subject requires permission from the Medical Monitor
    12. Past or current major thought disorder, e.g., schizophrenia, paranoia, mania, etc.
    13. Recent history of clinically significant parasomnia (e.g., sleep walking, REM behavior disorder, etc.) that would negatively affect the conduct of the study
    14. Current suicidal risk as determined from history or Columbia Suicide Severity Rating
    Scale or history of suicide attempt
    15. A T-score at or above 65 on the Children’s Depression Inventory 2nd Edition Self-
    Report Short Version
    16. A T-score above 65 on the Multidimensional Anxiety Scale for Children 10 item Anxiety Index 17. Other documented clinically significant condition (including an unstable medical condition, chronic disease other than narcolepsy with cataplexy, porphyria, or history or presence of another neurological disorder) that might affect the subject’s safety and/or interfere with the conduct of the study in the Inv.'s opinion
    18. An ECG with clinically significant deviation(s) from normal, or clinically significant physical examination findings, as determined by the Inv.
    19. Any clinically significant lab abnormality as determined by the Inv.
    20. A positive pregnancy test result at screening (pregnancy tests will be performed for any female subject who has reached menarche)
    21. 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)
    22. Treatment with benzodiazepines, non-benzodiazepine anxiolytics/
    hypnotics/sedatives, neuroleptics, opioids, barbiturates, diclofenac, valproate,
    phenytoin, ethosuximide within 2 weeks prior to enrollment (discontinuation for the purpose of study enrollment is permitted only if considered safe by the Inv. and approved by the Medical Monitor)
    23. Treatment with any other medications that have anticataplectic effect (e.g., serotonin–
    norepinephrine reuptake inhibitors [SNRIs], selective serotonin reuptake inhibitors
    [SSRIs], or tricyclic antidepressants [TCAs]) within 1 month before Screening
    24. Current treatment with oral isotretinoin
    25. Inability to fast for 2 hours before the first dose through 4 hours after the last dose of
    Xyrem on PSG and PK nights
    26. Lack of parental (or legal guardian) commitment to ensuring home situation is safe
    for Xyrem use, in the opinion of Investigator
    27. Received any investigational drug within 30 days or 5 half-lives (whichever is longer) before Screening
    28. Allergy to any components of topical, local anesthetics that might be used for blood collection (not applicable if numbing agents will not be used)
    29. Allergy or sensitivity to malic acid, sucralose, or ingredients in the study drug formulation and/or the flavorant, if used
    30. Unsafe for the subject to receive placebo treatment for 2 weeks, in the Investigator's opinion
    E.5 End points
    E.5.1Primary end point(s)
    Primary efficacy endpoints:
    - Change in weekly number of cataplexy attacks.
    E.5.1.1Timepoint(s) of evaluation of this end point
    From the last 2 weeks of the Stable-Dose Period to the 2 weeks of the Double-Blind Treatment Period
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints:
    - Clinical Global Impression of Change (CGIc) for cataplexy severity
    - Change in the Epworth Sleepiness Scale for Children and Adolescents (ESS [CHAD]) score
    - CGIc for narcolepsy overall
    - Change in Quality of Life (QoL) (SF-10)
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Clinical Global Impression of Change (CGIc) for cataplexy severity: from the end of the Stable-Dose Period to the end of the Double-Blind Treatment Period
    - Change in the Epworth Sleepiness Scale for Children and Adolescents (ESS [CHAD]) score: from the end of the Stable-Dose Period to the end of the Double-Blind Treatment Period
    - CGIc for narcolepsy overall: from the end of the Stable-Dose Period to the end of the Double-Blind Treatment Period
    - Change in Quality of Life (QoL) (SF-10): from the end of the Stable-Dose Period to the end of the Double-Blind Treatment Period
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    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) Yes
    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 Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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
    Finland
    France
    Italy
    Netherlands
    United States
    E.8.7Trial has a data monitoring committee Yes
    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 months0
    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 months0
    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 Yes
    F.1.1Number of subjects for this age range: 100
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 20
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 80
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Minors
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 100
    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)
    None
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2014-12-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-01-13
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-01-31
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sat May 04 01:20:18 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA