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
Condition: Narcolepsy With Cataplexy
MeSH terms: Narcolepsy; Cataplexy
Intervention: Xyrem
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 106.0
Sex: All
Minimum age: 7 Years
Maximum age: 17 Years
Healthy volunteers: No

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

Eligibility 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:

        -