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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

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ClinicalTrials.gov Identifier: NCT02221869
Recruitment Status : Completed
First Posted : August 21, 2014
Results First Posted : April 30, 2019
Last Update Posted : April 30, 2019
Sponsor:
Information provided by (Responsible Party):
Jazz Pharmaceuticals

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

Condition or disease Intervention/treatment Phase
Narcolepsy With Cataplexy Drug: Xyrem Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 106 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Actual Study Start Date : October 1, 2014
Actual Primary Completion Date : February 2017
Actual Study Completion Date : January 25, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Club Drugs

Arm Intervention/treatment
Experimental: Xyrem
Active Xyrem at a dose ≤9 g/night
Drug: Xyrem
Placebo Comparator: Xyrem Placebo
Xyrem placebo at a volume and regimen equivalent to the stable dose of Xyrem.
Drug: Xyrem



Primary Outcome Measures :
  1. Change in Weekly Number of Cataplexy Attacks [ Time Frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks) ]
    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.


Secondary Outcome Measures :
  1. Clinical Global Impression of Change (CGIc) for Cataplexy Severity [ Time Frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks) ]

    CGIc for cataplexy severity from the end of the Stable Dose Period to the end of the Double-blind Treatment Period.

    The CGIc is a 7-point scale ranging from "very much improved" to "very much worse." A score of 0 = no change, a score of 3 = very much improved, and a score of -3 = very much worse.


  2. Change in the Epworth Sleepiness Scale (ESS) (CHAD) Score [ Time Frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks) ]

    Change in the ESS (CHAD) score from the end of the Stable Dose Period to the end of the Double-blind Treatment Period.

    The ESS is a self-administered questionnaire with 8 questions. It provides a measure of a person's general level of daytime sleepiness, or their average sleep propensity in daily life. In the ESS for children and adolescents (CHAD), certain activities were modified. Each activity is scored on a scale ranging from 0-3, with 0 = would never fall asleep, and 3 = high chance of falling asleep. The total score ranges from 0-24, with a higher number representing an increased propensity for sleepiness.


  3. CGIc for Narcolepsy Overall [ Time Frame: 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.

    The CGIc is a 7-point scale ranging from "very much improved" to "very much worse." A score of 0 = no change, a score of 3 = very much improved, and a score of -3 = very much worse.


  4. 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 [ Time Frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks) ]

    The SF-10 Health Survey for Children is a parent-completed survey that contains 10 questions adapted from the Child Health Questionnaire. The SF-10 is intended to produce physical and psychosocial health summary measures. Each of the 10 questions responses is scored with a point value from 1 to 6 (1 is the worst possible condition and 6 is the best possible condition). The SF-10 physical and psychosocial measures are scored such that higher scores indicate more favorable functioning.

    The questions and associated point values are separated into the Physical Health (PHS-10 domain) and Psychosocial Health (PSS-10 domain). The sums of the scores in each domain are standardized using the mean and standard deviation from a normal population (2006 sample). The standardized scores are transformed to norm based scoring (NBS) metric. Through NBS, scale scores are standardized to a mean of 50 and SD of 10 in the combined U.S. general population and clinical samples. NBS scores are reported




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Ages Eligible for Study:   7 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
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:

-


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02221869


Locations
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United States, California
Miller Children's Hospital - Long Beach
Long Beach, California, United States, 90806
SDS Clinical Trials, Inc.
Orange, California, United States, 92868
Stanford Sleep Medicine Center
Redwood City, California, United States, 94063
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Michigan
The U-M Sleep Disorders Center
Ann Arbor, Michigan, United States, 48109
United States, New York
Montefiore Medical Center
Bronx, New York, United States, 10467
United States, North Carolina
Duke Children's Hospital
Durham, North Carolina, United States, 27710
ARSM Research, LLC
Huntersville, North Carolina, United States, 28078
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States, 44106
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, South Carolina
SleepMed of SC
Columbia, South Carolina, United States, 29201
Greenville Health System
Greenville, South Carolina, United States, 29615
United States, Tennessee
UT/LeBonheur Neuroscience Institute
Memphis, Tennessee, United States, 38105
United States, Texas
Todd Swick, MD, PA
Houston, Texas, United States, 77063
United States, Virginia
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States, 23507
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
France
Hospital Robert Debre
Paris, France, 75019
Italy
Dipartimento di Scienze Biomediche e Biomotorie
Bologna, Italy, 40139
Netherlands
Sleep Wake Center SEIN Heemstede
Heemstede, Noord Holland, Netherlands, 2103 SW
Sponsors and Collaborators
Jazz Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Jazz Pharmaceuticals:
Study Protocol  [PDF] February 23, 2017
Statistical Analysis Plan  [PDF] January 19, 2018


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Jazz Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02221869     History of Changes
Other Study ID Numbers: 13-005
First Posted: August 21, 2014    Key Record Dates
Results First Posted: April 30, 2019
Last Update Posted: April 30, 2019
Last Verified: April 2019

Additional relevant MeSH terms:
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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