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

Completed · Phase 3 · Has a placebo group

Conditions studied: Narcolepsy With Cataplexy

In brief

The purpose of this trial is to assess the efficacy and safety of Xyrem in pediatrics subjects with narcolepsy that includes cataplexy.

Key facts

Study ID: NCT02221869
Run by: Jazz Pharmaceuticals
People needed: 106
Starts: 2014-10-01
Expected to finish: 2019-01-25
Last updated by the study team: 2019-04-30

Who can join

Age: 7 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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)
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)
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
Have parent(s)/guardian(s) who have given informed consent for his/her/their child's participation in the study
Be willing to spend the required number of nights (2 to 3) in a sleep laboratory for PSG evaluations
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:
Be willing to spend 2 additional nights in the clinic for PK evaluation

You may not qualify if…

Inability to understand assent or follow study instructions for any reason, in the opinion of the Investigator
Parent(s) or guardian(s) unable to comply with the requirements of the study for any reason, in the opinion of the Investigator
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
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)
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
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:

Where it is running

Miller Children's Hospital - Long Beach — Long Beach, California, United States
SDS Clinical Trials, Inc. — Orange, California, United States
Stanford Sleep Medicine Center — Redwood City, California, United States
Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
The U-M Sleep Disorders Center — Ann Arbor, Michigan, United States
Montefiore Medical Center — The Bronx, New York, United States
Duke Children's Hospital — Durham, North Carolina, United States
ARSM Research, LLC — Huntersville, North Carolina, United States
Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
University Hospitals Cleveland Medical Center — Cleveland, Ohio, United States
Nationwide Children's Hospital — Columbus, Ohio, United States
SleepMed of SC — Columbia, South Carolina, United States
Greenville Health System — Greenville, South Carolina, United States
UT/LeBonheur Neuroscience Institute — Memphis, Tennessee, United States
Todd Swick, MD, PA — Houston, Texas, United States
Children's Hospital of The King's Daughters — Norfolk, Virginia, United States
Seattle Children's Hospital — Seattle, Washington, United States
Hospital Robert Debre — Paris, France
Dipartimento di Scienze Biomediche e Biomotorie — Bologna, Italy
Sleep Wake Center SEIN Heemstede — Heemstede, North Holland, Netherlands

Full record on ClinicalTrials.gov

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