Safety and Efficacy of Xyrem Oral Solution (Sodium Oxybate) Compared With Placebo in Narcoleptic Patients
Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Center Trial Comparing the Effects of Orally Administered Xyrem (Sodium Oxybate) With Placebo for the Treatment of Narcolepsy
1 other identifier
interventional
200
3 countries
45
Brief Summary
The initial portion of the protocol involves discontinuing any medications for cataplexy that the patient may be taking. Subsequently, the patient is prescribed a dose of oral solution of study drug or placebo over a 10-11 week period. During the trial, narcolepsy symptoms will be evaluated. Participants are allowed to continue using stimulant medications at constant doses during the study. A total of 1 to 3 daytime visits in addition to 4 overnight visits to the sleep center will be required to complete the study.
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 Dec 2000
Typical duration for phase_3
45 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
Study Start
First participant enrolled
December 1, 2000
CompletedFirst Submitted
Initial submission to the registry
November 13, 2002
CompletedFirst Posted
Study publicly available on registry
November 15, 2002
CompletedStudy Completion
Last participant's last visit for all outcomes
April 1, 2004
CompletedJune 24, 2005
July 1, 2004
November 13, 2002
June 23, 2005
Conditions
Keywords
Interventions
Eligibility Criteria
You may qualify if:
- Have signed \& dated informed consent before beginning protocol procedures.
- Willing \& able to complete entire trial as described in protocol.
- years of age or older.
- Have a history and presenting symptoms of excessive daytime sleepiness.
- Have a history of cataplexy localizable to a specific muscle group(s) or part(s) of body during which the patient is lucid (not experiencing an inadvertent nap or micro sleep).
- Have valid PSG \& MSLT scores (collected during an overnight test) within last five years and a current diagnosis of narcolepsy according to the following criteria established by the American Sleep Disorders Association: (1) Recurrent daytime naps or lapses into sleep occur almost daily for at least 3 months; (2) Sudden bilateral loss of postural muscle tone occurs in association with intense emotion (cataplexy); (3) Polysomnography demonstrates one or more of the following: (a) Sleep latency less than 10 minutes; (b) REM sleep latency less than 20 minutes; (c) An MSLT that demonstrates a mean sleep latency of less than 5 minutes; (d) Two or more sleep-onset REM periods
- Females who are surgically sterile, two years post-menopausal, or if of child-bearing potential, using a medically accepted method of birth control and agree to continue use of this method for the duration of the trial.
- In the opinion of the investigator, have adequate support for the duration of trial to include transportation to and from trial site. In addition, if in the investigator's assessment it is clinically indicated, the patient is willing to not operate a car or heavy machinery for the duration of the trial or for as long as the investigator deems clinically indicated.
You may not qualify if:
- Received gamma-hydroxybutyrate in the last 30 days.
- Have taken any investigational therapy within 30-day period prior to initial screening visit for this trial.
- Patients taking fluoxetine (Prozac).
- Have been diagnosed with sleep apnea syndrome, defined as an Apnea Index \> 10 per hour or an Apnea Hypopnea Index greater than 15 per hour, or have any other cause of daytime sleepiness, and have any other disorder(s) that can be considered a primary cause of excessive daytime sleepiness.
- Taking hypnotics, tranquilizers, antihistamines (except for non-sedating antihistamines), benzodiazepines or clonidine at the start of the baseline period. Patients taking anticonvulsants are not eligible to participate even if willing to washout anticonvulsants for the trial.
- Experiencing unstable cardiovascular, endocrine, neoplastic (excluding localized basal cell carcinoma), gastrointestinal, hematologic, hepatic, immunologic, metabolic, neurological (other than narcolepsy/cataplexy), pulmonary, and/or renal disease which would place the patient at risk during the trial or compromise objectives outlined in the protocol.
- Have psychiatric disorders, major affective or psychotic disorders, or other problems that, in the investigator's opinion, would preclude the patient's participation and completion of this trial or compromise reliable representation of subjective symptoms.
- Have current or recent (within one year) history of a substance use disorder including alcohol abuse as defined by DSM-IV.
- Serum creatinine greater than 2.0 mg/dL, abnormal liver function tests (SGOT \[AST\] or SGPT \[ALT\] more than twice the upper limit of normal), or elevated serum bilirubin (more than 1.5 times upper limit of normal), or pre-trial ECG results demonstrating clinically significant arrhythmias, greater than a first degree AV block or a history of myocardial infarction within last six months.
- Have an occupation that requires variable shift work or routine night shift.
- Have a clinically significant history of seizure disorder, a history of clinically significant head trauma (i.e., concussion resulting in clinically significant loss of consciousness) or past invasive intracranial surgery, and are taking anticonvulsant medications.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Orphan Medicallead
Study Sites (45)
Sleep Disorders Center of Alabama, Inc.
Birmingham, Alabama, 35213, United States
Pacific Sleep Medicine Services
La Jolla, California, 92037-1205, United States
Stanford Sleep Disorders Clinic
Stanford, California, 94305, United States
St. Petersburg Sleep Disorders Center
St. Petersburg, Florida, 33707, United States
Sleep Disorders Center of Georgia
Atlanta, Georgia, 30342, United States
Sleep Disorders Center--Division of Neurology
Evanston, Illinois, 60201, United States
Peoria Pulmonary Associates, Ltd.
Peoria, Illinois, 61603, United States
The Center for Sleep and Wake Disorders/Midwest Neurology
Danville, Indiana, 46122, United States
Fort Wayne Neurological Center
Fort Wayne, Indiana, 46804, United States
Fort Wayne Neurological Center
Fort Wayne, Indiana, 46805, United States
Chest Medicine Associates DBA/Sleep Medicine Specialists
Louisville, Kentucky, 40217, United States
LSU Health Science Center
Shreveport, Louisiana, 71130, United States
Center for Sleep and Wake Disorders
Chevy Chase, Maryland, 20815, United States
Center for Sleep Diagnostics
Newton, Massachusetts, 02459, United States
Department of Neurology
Worcester, Massachusetts, 01608, United States
Washington University -- Sleep Medicine Center
St Louis, Missouri, 63108, United States
Sleep/Wake Center 7N2 -- Bellevue Hospital Center
New York, New York, 10016, United States
The Sleep Center - Community General Hospital
Syracuse, New York, 13215, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Raleigh Neurology Associates
Raleigh, North Carolina, 27607, United States
Central Carolina Neurology & Sleep
Salisbury, North Carolina, 28144, United States
CSC Research -- Grove City Sleep Diagnostic Center
Grove City, Ohio, 43123, United States
Southwest Cleveland Sleep Center
Middleburg Heights, Ohio, 44130, United States
Lynn Health Science Institute
Oklahoma City, Oklahoma, 73112, United States
Clinical Pharmaceutical Trials, Inc.
Tulsa, Oklahoma, 74104, United States
Rhode Island Hospital - Division of Pulmonary, Sleep and Critical Care Medicine
Providence, Rhode Island, 02903, United States
Miriam Hospital
Providence, Rhode Island, 02906, United States
Charleston Pulmonary Associates PA
Charleston, South Carolina, 29403, United States
Low Country Lung and Critical Care PA
Charleston, South Carolina, 29406-7108, United States
Palmetto Baptist Medical Center Sleep Disorders Center
Columbia, South Carolina, 29201, United States
Charleston Pulmonary Associates PA
Mt. Pleasant, South Carolina, 29464, United States
Sleep Medicine Assoc PLLC -- Summit Medical Center
Hermitage, Tennessee, 37076, United States
The Houston Sleep Center
Houston, Texas, 77024, United States
Sleep Medicine Associates of Texas
Plano, Texas, 75093, United States
Sleep Disorders Center -- Eastern Virginia Medical School -- Sentara General Hospital
Norfolk, Virginia, 23507, United States
VCU Health System MCV Hospitals - Sleep Disorders Center
Richmond, Virginia, 23235, United States
Swedish Sleep Medicine Institute
Seattle, Washington, 98122, United States
Canadian Sleep Institute
Calgary, Alberta, T2X2A8, Canada
Vancouver Hospital -- Sleep Disorders Clinic
Vancouver, British Columbia, V6T2B5, Canada
Saint John Regional Hospital -- Somnology Program
Saint John, New Brunswick, E2L4L2, Canada
The Ottawa Hospital Sleep Centre -- Ottawa Hospital -- Civic Campus -- Sleep Laboratory
Ottawa, Ontario, K1Y 4E9, Canada
The Sleep Disorders Clinic of the Centre for Sleep and Chronobiology
Toronto, Ontario, M5T 3A9, Canada
Brain & Sleep Diagnostic Center
Toronto, Ontario, M8X2W2, Canada
Sleep Disorder Centre -- Hopital du Sacre-Coeur
Montreal, Quebec, H4J1C5, Canada
Neurologische Poliklinik - Universitats Spital Zurich
Zurich, CH-8091, Switzerland
Related Publications (7)
A randomized, double blind, placebo-controlled multicenter trial comparing the effects of three doses of orally administered sodium oxybate with placebo for the treatment of narcolepsy. Sleep. 2002 Feb 1;25(1):42-9.
PMID: 11833860BACKGROUNDScrima L, Hartman PG, Johnson FH Jr, Thomas EE, Hiller FC. The effects of gamma-hydroxybutyrate on the sleep of narcolepsy patients: a double-blind study. Sleep. 1990 Dec;13(6):479-90. doi: 10.1093/sleep/13.6.479.
PMID: 2281247BACKGROUNDLammers GJ, Arends J, Declerck AC, Ferrari MD, Schouwink G, Troost J. Gammahydroxybutyrate and narcolepsy: a double-blind placebo-controlled study. Sleep. 1993 Apr;16(3):216-20. doi: 10.1093/sleep/16.3.216.
PMID: 8506453BACKGROUNDA 12-month, open-label, multicenter extension trial of orally administered sodium oxybate for the treatment of narcolepsy. Sleep. 2003 Feb 1;26(1):31-5.
PMID: 12627729BACKGROUNDHusain AM, Bujanover S, Ryan R, Scheckner B, Black J, Profant J. Incidence and duration of common, early-onset adverse events occurring during 2 randomized, placebo-controlled, phase 3 studies of sodium oxybate in participants with narcolepsy. J Clin Sleep Med. 2020 Sep 15;16(9):1469-1474. doi: 10.5664/jcsm.8530.
PMID: 32356515DERIVEDRoth T, Dauvilliers Y, Guinta D, Alvarez-Horine S, Dynin E, Black J. Effect of sodium oxybate on disrupted nighttime sleep in patients with narcolepsy. J Sleep Res. 2017 Aug;26(4):407-414. doi: 10.1111/jsr.12468. Epub 2016 Nov 3.
PMID: 27807903DERIVEDBogan R, Swick T, Mamelak M, Kovacevic-Ristanovic R, Lai C, Black J, Villa KF, Montplaisir J. Evaluation of Quality of Life in Patients With Narcolepsy Treated With Sodium Oxybate: Use of the 36-Item Short-Form Health Survey in a Clinical Trial. Neurol Ther. 2016 Dec;5(2):203-213. doi: 10.1007/s40120-016-0053-5. Epub 2016 Oct 24.
PMID: 27778193DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
William Houghton, MD
Orphan Medical
- STUDY DIRECTOR
Harry N Cook, MBA, RPh
Orphan Medical
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
November 13, 2002
First Posted
November 15, 2002
Study Start
December 1, 2000
Study Completion
April 1, 2004
Last Updated
June 24, 2005
Record last verified: 2004-07