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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

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02221869
First Posted: August 21, 2014
Last Update Posted: September 18, 2017
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.
Information provided by (Responsible Party):
Jazz Pharmaceuticals
  Purpose
The purpose of this trial is to assess the efficacy and safety of Xyrem in pediatrics subjects with narcolepsy that includes cataplexy.

Condition Intervention Phase
Narcolepsy With Cataplexy Drug: Xyrem Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Jazz Pharmaceuticals:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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


Other Outcome Measures:
  • 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.


Enrollment: 97
Study Start Date: September 2014
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: November 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Xyrem
Active Xyrem at a dose ≤9 g/night
Drug: Xyrem
Placebo Comparator: Xyrem Placebo
Xyrem placebo at a volume and regimen equivalent to the stable dose of Xyrem.
Drug: Xyrem

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   7 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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:

  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02221869


  Show 35 Study Locations
Sponsors and Collaborators
Jazz Pharmaceuticals
  More Information

Additional Information:
Responsible Party: Jazz Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02221869     History of Changes
Other Study ID Numbers: 13-005
First Submitted: July 28, 2014
First Posted: August 21, 2014
Last Update Posted: September 18, 2017
Last Verified: September 2017

Additional relevant MeSH terms:
Narcolepsy
Cataplexy
Disorders of Excessive Somnolence
Sleep Disorders, Intrinsic
Dyssomnias
Sleep Wake Disorders
Nervous System Diseases
Mental Disorders
Sodium Oxybate
Adjuvants, Anesthesia
Anesthetics, Intravenous
Anesthetics, General
Anesthetics
Central Nervous System Depressants
Physiological Effects of Drugs