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
1 other identifier
interventional
106
4 countries
20
Brief Summary
The purpose of this trial is to assess the efficacy and safety of Xyrem in pediatrics subjects with narcolepsy that includes cataplexy.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Oct 2014
Typical duration for phase_3
20 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 28, 2014
CompletedFirst Posted
Study publicly available on registry
August 21, 2014
CompletedStudy Start
First participant enrolled
October 1, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
January 25, 2019
CompletedResults Posted
Study results publicly available
April 30, 2019
CompletedApril 30, 2019
April 1, 2019
2.3 years
July 28, 2014
November 23, 2018
April 26, 2019
Conditions
Outcome Measures
Primary Outcomes (1)
Change in Weekly Number of Cataplexy Attacks
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.
From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
Secondary Outcomes (4)
Clinical Global Impression of Change (CGIc) for Cataplexy Severity
From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
Change in the Epworth Sleepiness Scale (ESS) (CHAD) Score
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 (2 weeks)
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
From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
Study Arms (2)
Xyrem
EXPERIMENTALActive Xyrem at a dose ≤9 g/night
Xyrem Placebo
PLACEBO COMPARATORXyrem placebo at a volume and regimen equivalent to the stable dose of Xyrem.
Interventions
Eligibility Criteria
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-naĂ¯ve 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
- \. 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (20)
Miller Children's Hospital - Long Beach
Long Beach, California, 90806, United States
SDS Clinical Trials, Inc.
Orange, California, 92868, United States
Stanford Sleep Medicine Center
Redwood City, California, 94063, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
The U-M Sleep Disorders Center
Ann Arbor, Michigan, 48109, United States
Montefiore Medical Center
The Bronx, New York, 10467, United States
Duke Children's Hospital
Durham, North Carolina, 27710, United States
ARSM Research, LLC
Huntersville, North Carolina, 28078, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
SleepMed of SC
Columbia, South Carolina, 29201, United States
Greenville Health System
Greenville, South Carolina, 29615, United States
UT/LeBonheur Neuroscience Institute
Memphis, Tennessee, 38105, United States
Todd Swick, MD, PA
Houston, Texas, 77063, United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Hospital Robert Debre
Paris, 75019, France
Dipartimento di Scienze Biomediche e Biomotorie
Bologna, 40139, Italy
Sleep Wake Center SEIN Heemstede
Heemstede, North Holland, 2103 SW, Netherlands
Related Publications (4)
Dauvilliers Y, Lammers GJ, Lecendreux M, Maski K, Kansagra S, Black J, Parvataneni R, Chen A, Wang YG, Plazzi G. Effect of sodium oxybate on body mass index in pediatric patients with narcolepsy. J Clin Sleep Med. 2024 Mar 1;20(3):445-454. doi: 10.5664/jcsm.10912.
PMID: 37942930DERIVEDLecendreux M, Plazzi G, Dauvilliers Y, Rosen CL, Ruoff C, Black J, Parvataneni R, Guinta D, Wang YG, Mignot E. Long-term safety and maintenance of efficacy of sodium oxybate in the treatment of narcolepsy with cataplexy in pediatric patients. J Clin Sleep Med. 2022 Sep 1;18(9):2217-2227. doi: 10.5664/jcsm.10090.
PMID: 35689598DERIVEDWang YG, Menno D, Chen A, Steininger TL, Morris S, Black J, Profant J, Johns MW. Validation of the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD) questionnaire in pediatric patients with narcolepsy with cataplexy aged 7-16 years. Sleep Med. 2022 Jan;89:78-84. doi: 10.1016/j.sleep.2021.11.003. Epub 2021 Nov 11.
PMID: 34920345DERIVEDPlazzi 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.
PMID: 30169321DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Director, Disclosure & Transparency
- Organization
- Jazz Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 28, 2014
First Posted
August 21, 2014
Study Start
October 1, 2014
Primary Completion
February 1, 2017
Study Completion
January 25, 2019
Last Updated
April 30, 2019
Results First Posted
April 30, 2019
Record last verified: 2019-04