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

NCT ID: NCT02221869

Last Updated: 2019-04-30

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 106 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-10-01
• Study Completion Date: 2019-01-25

Brief Summary

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

Conditions

Narcolepsy With Cataplexy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Xyrem

Active Xyrem at a dose ≤9 g/night

Group Type: EXPERIMENTAL

Xyrem

Intervention Type: DRUG

Xyrem Placebo

Xyrem placebo at a volume and regimen equivalent to the stable dose of Xyrem.

Group Type: PLACEBO_COMPARATOR

Xyrem

Intervention Type: DRUG

Interventions

Xyrem

Intervention Type: DRUG

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

7. Be willing to spend 2 additional nights in the clinic for PK evaluation

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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

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• Minimum Eligible Age: 7 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Jazz Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

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

Countries

United States; France; Italy; Netherlands

References

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.

Reference Type: DERIVED

PMID: 37942930 (View on PubMed)

Lecendreux 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.

Reference Type: DERIVED

PMID: 35689598 (View on PubMed)

Wang 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.

Reference Type: DERIVED

PMID: 34920345 (View on PubMed)

Plazzi 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.

Reference Type: DERIVED

PMID: 30169321 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

13-005

Identifier Type: -

Identifier Source: org_study_id