Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

A 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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02221869
Recruitment Status : Completed
First Posted : August 21, 2014
Results First Posted : April 30, 2019
Last Update Posted : April 30, 2019
Sponsor:
Information provided by (Responsible Party):
Jazz Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE July 28, 2014
First Posted Date  ICMJE August 21, 2014
Results First Submitted Date  ICMJE November 23, 2018
Results First Posted Date  ICMJE April 30, 2019
Last Update Posted Date April 30, 2019
Actual Study Start Date  ICMJE October 1, 2014
Actual Primary Completion Date February 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 26, 2019)
Change in Weekly Number of Cataplexy Attacks [ Time Frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks) ]
Double-blind comparison of the change in weekly number of cataplexy attacks from the last 2 weeks of the Stable Dose Period to the 2 weeks of the Double-blind Treatment Period.
Original Primary Outcome Measures  ICMJE
 (submitted: August 19, 2014)
  • Efficacy of Xyrem [ Time Frame: 2 Weeks ]
    Change in weekly number of cataplexy attacks (2 weeks). Two week double-blind comparison
  • Xyrem Safety In Participants [ Time Frame: Up to 52 weeks ]
    Adverse events will be summarized by treatment Group and by dose.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 26, 2019)
  • Clinical Global Impression of Change (CGIc) for Cataplexy Severity [ Time Frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks) ]
    CGIc for cataplexy severity from the end of the Stable Dose Period to the end of the Double-blind Treatment Period. The CGIc is a 7-point scale ranging from "very much improved" to "very much worse." A score of 0 = no change, a score of 3 = very much improved, and a score of -3 = very much worse.
  • Change in the Epworth Sleepiness Scale (ESS) (CHAD) Score [ Time Frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks) ]
    Change in the ESS (CHAD) score from the end of the Stable Dose Period to the end of the Double-blind Treatment Period. The ESS is a self-administered questionnaire with 8 questions. It provides a measure of a person's general level of daytime sleepiness, or their average sleep propensity in daily life. In the ESS for children and adolescents (CHAD), certain activities were modified. Each activity is scored on a scale ranging from 0-3, with 0 = would never fall asleep, and 3 = high chance of falling asleep. The total score ranges from 0-24, with a higher number representing an increased propensity for sleepiness.
  • CGIc for Narcolepsy Overall [ Time Frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks) ]
    CGIc for narcolepsy overall from the end of the Stable Dose Period to the end of the Double-blind Treatment Period. The CGIc is a 7-point scale ranging from "very much improved" to "very much worse." A score of 0 = no change, a score of 3 = very much improved, and a score of -3 = very much worse.
  • Change in Quality of Life (QoL; SF-10 Physical and Psychosocial Summary Score) From the End of the Stable Dose Period to the End of the Double-blind Treatment Period [ Time Frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks) ]
    The SF-10 Health Survey for Children is a parent-completed survey that contains 10 questions adapted from the Child Health Questionnaire. The SF-10 is intended to produce physical and psychosocial health summary measures. Each of the 10 questions responses is scored with a point value from 1 to 6 (1 is the worst possible condition and 6 is the best possible condition). The SF-10 physical and psychosocial measures are scored such that higher scores indicate more favorable functioning. The questions and associated point values are separated into the Physical Health (PHS-10 domain) and Psychosocial Health (PSS-10 domain). The sums of the scores in each domain are standardized using the mean and standard deviation from a normal population (2006 sample). The standardized scores are transformed to norm based scoring (NBS) metric. Through NBS, scale scores are standardized to a mean of 50 and SD of 10 in the combined U.S. general population and clinical samples. NBS scores are reported
Original Secondary Outcome Measures  ICMJE
 (submitted: August 19, 2014)
To Evaluate the efficacy of xyrem in the treatment of excessive daytimne sleepiness (EDS) in pediatric subjects with narcolepsy with cataplexy [ Time Frame: 2 Weeks ]
Clinical Global Impression of Change (CGIc) for cataplexy severity. Change in the Epworth Sleepiness Scale for CHildren and Adolescents (ESS [CHAD}) score
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures
 (submitted: August 19, 2014)
Overnight PK Sampling [ Time Frame: 2 Nights ]
Overnight PK sampling for evaluation of AUC, Cmax, Tmax for subjects willing to participate in this part of the study.
 
Descriptive Information
Brief Title  ICMJE A 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
Official Title  ICMJE Not Provided
Brief Summary The purpose of this trial is to assess the efficacy and safety of Xyrem in pediatrics subjects with narcolepsy that includes cataplexy.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Narcolepsy With Cataplexy
Intervention  ICMJE Drug: Xyrem
Study Arms  ICMJE
  • Experimental: Xyrem
    Active Xyrem at a dose ≤9 g/night
    Intervention: Drug: Xyrem
  • Placebo Comparator: Xyrem Placebo
    Xyrem placebo at a volume and regimen equivalent to the stable dose of Xyrem.
    Intervention: Drug: Xyrem
Publications * Plazzi G, Ruoff C, Lecendreux M, Dauvilliers Y, Rosen CL, Black J, Parvataneni R, Guinta D, Wang YG, Mignot E. Treatment of paediatric narcolepsy with sodium oxybate: a double-blind, placebo-controlled, randomised-withdrawal multicentre study and open-label investigation. Lancet Child Adolesc Health. 2018 Jul;2(7):483-494. doi: 10.1016/S2352-4642(18)30133-0. Epub 2018 May 21.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 19, 2018)
106
Original Enrollment  ICMJE Not Provided
Actual Study Completion Date  ICMJE January 25, 2019
Actual Primary Completion Date February 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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 or, with the permission of the Medical Monitor, completes a Multiple Sleep Latency Test (MSLT) during Screening to confirm the diagnosis of Type 1 narcolepsy by ICSD-3 criteria (i.e., the subject meets all other ICSD-3 criteria for Type 1 narcolepsy)
  3. Have given documented assent indicating that he/she was aware of the investigational nature of the study and the required procedures and restrictions before participation in any protocol-related activities
  4. Have parent(s)/guardian(s) who have given informed consent for his/her/their child's participation in the study
  5. Be willing to spend the required number of nights (2 to 3) in a sleep laboratory for PSG evaluations
  6. 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

In addition to the above inclusion criteria, subjects participating in the PK evaluation must meet the following inclusion criteria:

7. Be willing to spend 2 additional nights in the clinic for PK evaluation

-

Exclusion Criteria:

  1. Inability to understand assent or follow study instructions for any reason, in the opinion of the Investigator
  2. Parent(s) or guardian(s) unable to comply with the requirements of the study for any reason, in the opinion of the Investigator
  3. Other documented clinically significant condition (including an unstable medical condition, chronic disease other than narcolepsy with cataplexy, 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 opinion of the Investigator
  4. 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 Investigator and approved by the Medical Monitor)
  5. 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
  6. Unsafe for the subject to receive placebo treatment for 2 weeks, in the opinion of the Investigator

In addition to the above exclusion criteria, subjects participating in the PK evaluation must not demonstrate the following:

-

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 7 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France,   Italy,   Netherlands,   United States
Removed Location Countries Finland
 
Administrative Information
NCT Number  ICMJE NCT02221869
Other Study ID Numbers  ICMJE 13-005
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Jazz Pharmaceuticals
Study Sponsor  ICMJE Jazz Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Jazz Pharmaceuticals
Verification Date April 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP