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 currently recruiting participants. (see Contacts and Locations)
Verified July 2015 by Jazz Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
Jazz Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT02221869
First received: July 28, 2014
Last updated: November 12, 2015
Last verified: July 2015
  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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, 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 ] [ Designated as safety issue: No ]
    Change in weekly number of cataplexy attacks (2 weeks). Two week double-blind comparison

  • Xyrem Safety In Participants [ Time Frame: Up to 52 weeks ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    Overnight PK sampling for evaluation of AUC, Cmax, Tmax for subjects willing to participate in this part of the study.


Estimated Enrollment: 100
Study Start Date: September 2014
Estimated Primary Completion Date: November 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Xyrem Drug: Xyrem
Placebo Comparator: Xyrem Placebo Drug: Xyrem

  Eligibility

Ages Eligible for Study:   7 Years to 17 Years   (Child)
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02221869

Contacts
Contact: Jazz Pharmaceuticals 650 496 3777

  Show 46 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 
Study First Received: July 28, 2014
Last Updated: November 12, 2015
Health Authority: United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on July 26, 2016