Safety and Efficacy of Xyrem Oral Solution (Sodium Oxybate) Compared With Placebo in Narcoleptic Patients

NCT ID: NCT00049803

Last Updated: 2005-06-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

200 participants

Study Classification

INTERVENTIONAL

Study Start Date

2000-12-31

Study Completion Date

2004-04-30

Brief Summary

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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.

Detailed Description

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Conditions

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Narcolepsy

Keywords

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Narcolepsy Sodium Oxybate Cataplexy Excessive Daytime Sleepiness GHB Gamma Hydroxybutyric Acid Orphan Medical Orphan Drug

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Interventions

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sodium oxybate

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Have signed \& dated informed consent before beginning protocol procedures.
* Willing \& able to complete entire trial as described in protocol.
* 16 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.

Exclusion Criteria

* 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.
Minimum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Orphan Medical

INDUSTRY

Sponsor Role lead

Principal Investigators

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William Houghton, MD

Role: STUDY_CHAIR

Orphan Medical

Harry N Cook, MBA, RPh

Role: STUDY_DIRECTOR

Orphan Medical

Locations

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Sleep Disorders Center of Alabama, Inc.

Birmingham, Alabama, United States

Site Status

Pacific Sleep Medicine Services

La Jolla, California, United States

Site Status

Stanford Sleep Disorders Clinic

Stanford, California, United States

Site Status

St. Petersburg Sleep Disorders Center

St. Petersburg, Florida, United States

Site Status

Sleep Disorders Center of Georgia

Atlanta, Georgia, United States

Site Status

Sleep Disorders Center--Division of Neurology

Evanston, Illinois, United States

Site Status

Peoria Pulmonary Associates, Ltd.

Peoria, Illinois, United States

Site Status

The Center for Sleep and Wake Disorders/Midwest Neurology

Danville, Indiana, United States

Site Status

Fort Wayne Neurological Center

Fort Wayne, Indiana, United States

Site Status

Fort Wayne Neurological Center

Fort Wayne, Indiana, United States

Site Status

Chest Medicine Associates DBA/Sleep Medicine Specialists

Louisville, Kentucky, United States

Site Status

LSU Health Science Center

Shreveport, Louisiana, United States

Site Status

Center for Sleep and Wake Disorders

Chevy Chase, Maryland, United States

Site Status

Center for Sleep Diagnostics

Newton, Massachusetts, United States

Site Status

Department of Neurology

Worcester, Massachusetts, United States

Site Status

Washington University -- Sleep Medicine Center

St Louis, Missouri, United States

Site Status

Sleep/Wake Center 7N2 -- Bellevue Hospital Center

New York, New York, United States

Site Status

The Sleep Center - Community General Hospital

Syracuse, New York, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Raleigh Neurology Associates

Raleigh, North Carolina, United States

Site Status

Central Carolina Neurology & Sleep

Salisbury, North Carolina, United States

Site Status

CSC Research -- Grove City Sleep Diagnostic Center

Grove City, Ohio, United States

Site Status

Southwest Cleveland Sleep Center

Middleburg Heights, Ohio, United States

Site Status

Lynn Health Science Institute

Oklahoma City, Oklahoma, United States

Site Status

Clinical Pharmaceutical Trials, Inc.

Tulsa, Oklahoma, United States

Site Status

Rhode Island Hospital - Division of Pulmonary, Sleep and Critical Care Medicine

Providence, Rhode Island, United States

Site Status

Miriam Hospital

Providence, Rhode Island, United States

Site Status

Charleston Pulmonary Associates PA

Charleston, South Carolina, United States

Site Status

Low Country Lung and Critical Care PA

Charleston, South Carolina, United States

Site Status

Palmetto Baptist Medical Center Sleep Disorders Center

Columbia, South Carolina, United States

Site Status

Charleston Pulmonary Associates PA

Mt. Pleasant, South Carolina, United States

Site Status

Sleep Medicine Assoc PLLC -- Summit Medical Center

Hermitage, Tennessee, United States

Site Status

The Houston Sleep Center

Houston, Texas, United States

Site Status

Sleep Medicine Associates of Texas

Plano, Texas, United States

Site Status

Sleep Disorders Center -- Eastern Virginia Medical School -- Sentara General Hospital

Norfolk, Virginia, United States

Site Status

VCU Health System MCV Hospitals - Sleep Disorders Center

Richmond, Virginia, United States

Site Status

Swedish Sleep Medicine Institute

Seattle, Washington, United States

Site Status

Canadian Sleep Institute

Calgary, Alberta, Canada

Site Status

Vancouver Hospital -- Sleep Disorders Clinic

Vancouver, British Columbia, Canada

Site Status

Saint John Regional Hospital -- Somnology Program

Saint John, New Brunswick, Canada

Site Status

The Ottawa Hospital Sleep Centre -- Ottawa Hospital -- Civic Campus -- Sleep Laboratory

Ottawa, Ontario, Canada

Site Status

The Sleep Disorders Clinic of the Centre for Sleep and Chronobiology

Toronto, Ontario, Canada

Site Status

Brain & Sleep Diagnostic Center

Toronto, Ontario, Canada

Site Status

Sleep Disorder Centre -- Hopital du Sacre-Coeur

Montreal, Quebec, Canada

Site Status

Neurologische Poliklinik - Universitats Spital Zurich

Zurich, , Switzerland

Site Status

Countries

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United States Canada Switzerland

References

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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.

Reference Type BACKGROUND
PMID: 11833860 (View on PubMed)

Scrima 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.

Reference Type BACKGROUND
PMID: 2281247 (View on PubMed)

Lammers 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.

Reference Type BACKGROUND
PMID: 8506453 (View on PubMed)

A 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.

Reference Type BACKGROUND
PMID: 12627729 (View on PubMed)

Husain 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.

Reference Type DERIVED
PMID: 32356515 (View on PubMed)

Roth 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.

Reference Type DERIVED
PMID: 27807903 (View on PubMed)

Bogan 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.

Reference Type DERIVED
PMID: 27778193 (View on PubMed)

Other Identifiers

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OMC-SXB-15

Identifier Type: -

Identifier Source: org_study_id