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



Status:Active, not recruiting
Healthy:No
Age Range:7 - 17
Updated:6/29/2018
Start Date:October 1, 2014
End Date:June 2019

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


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:
We found this trial at
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3550 Jerome Avenue
Bronx, New York 10467
(718) 920-4321
Principal Investigator: Michael Thorpy, MD
Phone: 718-920-4841
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1720 2nd Ave S
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(205) 934-4011 
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
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Phone: 513-636-0607
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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700 Childrens Drive
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Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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3100 SW 62nd Ave
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601 Children's Lane
Norfolk, Virginia 23507
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Principal Investigator: Michael Strunc, MD
Phone: 757-668-7732
Children's Hospital of The King's Daughters Children
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South 34th Street
Philadelphia, Pennsylvania 19104
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Ann Arbor, Michigan 48109
Principal Investigator: Fauziya Hassan, MD
Phone: 734-615-4302
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13123 E 16th Ave
Aurora, Colorado 80045
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Principal Investigator: Ann Halbower, MD
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Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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Austin, Texas 78703
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Principal Investigator: Kiran Maski, MD
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Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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225 E Chicago Ave
Chicago, Illinois 60611
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Columbia, South Carolina 29201
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Dallas, Texas 75235
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Durham, North Carolina 27710
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Greenville, South Carolina 29615
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Houston, Texas 77063
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Huntersville, North Carolina 28078
Principal Investigator: Thomas Stern, MD
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Long Beach, California 90806
Principal Investigator: Gary Feldman, MD
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Louisville, Kentucky 40217
Principal Investigator: David Winslow, MD
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777 Washington Avenue
Memphis, Tennessee 38105
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80 Avenue Augustin Fliche
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Orange, California 92868
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Redwood City, California 94063
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San Antonio, Texas 78258
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Principal Investigator: Maida Chen, MD
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Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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