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

Primary Purpose

Narcolepsy

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
sodium oxybate
Sponsored by
Orphan Medical
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Narcolepsy focused on measuring Narcolepsy, Sodium Oxybate, Cataplexy, Excessive Daytime Sleepiness, GHB, Gamma Hydroxybutyric Acid, Orphan Medical, Orphan Drug

Eligibility Criteria

16 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Sleep Disorders Center of Alabama, Inc.
  • Pacific Sleep Medicine Services
  • Stanford Sleep Disorders Clinic
  • St. Petersburg Sleep Disorders Center
  • Sleep Disorders Center of Georgia
  • Sleep Disorders Center--Division of Neurology
  • Peoria Pulmonary Associates, Ltd.
  • The Center for Sleep and Wake Disorders/Midwest Neurology
  • Fort Wayne Neurological Center
  • Fort Wayne Neurological Center
  • Chest Medicine Associates DBA/Sleep Medicine Specialists
  • LSU Health Science Center
  • Center for Sleep and Wake Disorders
  • Center for Sleep Diagnostics
  • Department of Neurology
  • Washington University -- Sleep Medicine Center
  • Sleep/Wake Center 7N2 -- Bellevue Hospital Center
  • The Sleep Center - Community General Hospital
  • Duke University Medical Center
  • Raleigh Neurology Associates
  • Central Carolina Neurology & Sleep
  • CSC Research -- Grove City Sleep Diagnostic Center
  • Southwest Cleveland Sleep Center
  • Lynn Health Science Institute
  • Clinical Pharmaceutical Trials, Inc.
  • Rhode Island Hospital - Division of Pulmonary, Sleep and Critical Care Medicine
  • Miriam Hospital
  • Charleston Pulmonary Associates PA
  • Low Country Lung and Critical Care PA
  • Palmetto Baptist Medical Center Sleep Disorders Center
  • Charleston Pulmonary Associates PA
  • Sleep Medicine Assoc PLLC -- Summit Medical Center
  • The Houston Sleep Center
  • Sleep Medicine Associates of Texas
  • Sleep Disorders Center -- Eastern Virginia Medical School -- Sentara General Hospital
  • VCU Health System MCV Hospitals - Sleep Disorders Center
  • Swedish Sleep Medicine Institute
  • Canadian Sleep Institute
  • Vancouver Hospital -- Sleep Disorders Clinic
  • Saint John Regional Hospital -- Somnology Program
  • The Ottawa Hospital Sleep Centre -- Ottawa Hospital -- Civic Campus -- Sleep Laboratory
  • The Sleep Disorders Clinic of the Centre for Sleep and Chronobiology
  • Brain & Sleep Diagnostic Center
  • Sleep Disorder Centre -- Hopital du Sacre-Coeur
  • Neurologische Poliklinik - Universitats Spital Zurich

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
November 13, 2002
Last Updated
June 23, 2005
Sponsor
Orphan Medical
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1. Study Identification

Unique Protocol Identification Number
NCT00049803
Brief Title
Safety and Efficacy of Xyrem Oral Solution (Sodium Oxybate) Compared With Placebo in Narcoleptic Patients
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2004
Overall Recruitment Status
Completed
Study Start Date
December 2000 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
April 2004 (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
Orphan Medical

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Narcolepsy
Keywords
Narcolepsy, Sodium Oxybate, Cataplexy, Excessive Daytime Sleepiness, GHB, Gamma Hydroxybutyric Acid, Orphan Medical, Orphan Drug

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
Double
Allocation
Randomized
Enrollment
200 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
sodium oxybate

10. Eligibility

Sex
All
Minimum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
William Houghton, MD
Organizational Affiliation
Orphan Medical
Official's Role
Study Chair
First Name & Middle Initial & Last Name & Degree
Harry N Cook, MBA, RPh
Organizational Affiliation
Orphan Medical
Official's Role
Study Director
Facility Information:
Facility Name
Sleep Disorders Center of Alabama, Inc.
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35213
Country
United States
Facility Name
Pacific Sleep Medicine Services
City
La Jolla
State/Province
California
ZIP/Postal Code
92037-1205
Country
United States
Facility Name
Stanford Sleep Disorders Clinic
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
St. Petersburg Sleep Disorders Center
City
St. Petersburg
State/Province
Florida
ZIP/Postal Code
33707
Country
United States
Facility Name
Sleep Disorders Center of Georgia
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Facility Name
Sleep Disorders Center--Division of Neurology
City
Evanston
State/Province
Illinois
ZIP/Postal Code
60201
Country
United States
Facility Name
Peoria Pulmonary Associates, Ltd.
City
Peoria
State/Province
Illinois
ZIP/Postal Code
61603
Country
United States
Facility Name
The Center for Sleep and Wake Disorders/Midwest Neurology
City
Danville
State/Province
Indiana
ZIP/Postal Code
46122
Country
United States
Facility Name
Fort Wayne Neurological Center
City
Fort Wayne
State/Province
Indiana
ZIP/Postal Code
46804
Country
United States
Facility Name
Fort Wayne Neurological Center
City
Fort Wayne
State/Province
Indiana
ZIP/Postal Code
46805
Country
United States
Facility Name
Chest Medicine Associates DBA/Sleep Medicine Specialists
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40217
Country
United States
Facility Name
LSU Health Science Center
City
Shreveport
State/Province
Louisiana
ZIP/Postal Code
71130
Country
United States
Facility Name
Center for Sleep and Wake Disorders
City
Chevy Chase
State/Province
Maryland
ZIP/Postal Code
20815
Country
United States
Facility Name
Center for Sleep Diagnostics
City
Newton
State/Province
Massachusetts
ZIP/Postal Code
02459
Country
United States
Facility Name
Department of Neurology
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01608
Country
United States
Facility Name
Washington University -- Sleep Medicine Center
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63108
Country
United States
Facility Name
Sleep/Wake Center 7N2 -- Bellevue Hospital Center
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
The Sleep Center - Community General Hospital
City
Syracuse
State/Province
New York
ZIP/Postal Code
13215
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Raleigh Neurology Associates
City
Raleigh
State/Province
North Carolina
ZIP/Postal Code
27607
Country
United States
Facility Name
Central Carolina Neurology & Sleep
City
Salisbury
State/Province
North Carolina
ZIP/Postal Code
28144
Country
United States
Facility Name
CSC Research -- Grove City Sleep Diagnostic Center
City
Grove City
State/Province
Ohio
ZIP/Postal Code
43123
Country
United States
Facility Name
Southwest Cleveland Sleep Center
City
Middleburg Heights
State/Province
Ohio
ZIP/Postal Code
44130
Country
United States
Facility Name
Lynn Health Science Institute
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73112
Country
United States
Facility Name
Clinical Pharmaceutical Trials, Inc.
City
Tulsa
State/Province
Oklahoma
ZIP/Postal Code
74104
Country
United States
Facility Name
Rhode Island Hospital - Division of Pulmonary, Sleep and Critical Care Medicine
City
Providence
State/Province
Rhode Island
ZIP/Postal Code
02903
Country
United States
Facility Name
Miriam Hospital
City
Providence
State/Province
Rhode Island
ZIP/Postal Code
02906
Country
United States
Facility Name
Charleston Pulmonary Associates PA
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29403
Country
United States
Facility Name
Low Country Lung and Critical Care PA
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29406-7108
Country
United States
Facility Name
Palmetto Baptist Medical Center Sleep Disorders Center
City
Columbia
State/Province
South Carolina
ZIP/Postal Code
29201
Country
United States
Facility Name
Charleston Pulmonary Associates PA
City
Mount Pleasant
State/Province
South Carolina
ZIP/Postal Code
29464
Country
United States
Facility Name
Sleep Medicine Assoc PLLC -- Summit Medical Center
City
Hermitage
State/Province
Tennessee
ZIP/Postal Code
37076
Country
United States
Facility Name
The Houston Sleep Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77024
Country
United States
Facility Name
Sleep Medicine Associates of Texas
City
Plano
State/Province
Texas
ZIP/Postal Code
75093
Country
United States
Facility Name
Sleep Disorders Center -- Eastern Virginia Medical School -- Sentara General Hospital
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23507
Country
United States
Facility Name
VCU Health System MCV Hospitals - Sleep Disorders Center
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23235
Country
United States
Facility Name
Swedish Sleep Medicine Institute
City
Seattle
State/Province
Washington
ZIP/Postal Code
98122
Country
United States
Facility Name
Canadian Sleep Institute
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2X2A8
Country
Canada
Facility Name
Vancouver Hospital -- Sleep Disorders Clinic
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6T2B5
Country
Canada
Facility Name
Saint John Regional Hospital -- Somnology Program
City
Saint John
State/Province
New Brunswick
ZIP/Postal Code
E2L4L2
Country
Canada
Facility Name
The Ottawa Hospital Sleep Centre -- Ottawa Hospital -- Civic Campus -- Sleep Laboratory
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1Y 4E9
Country
Canada
Facility Name
The Sleep Disorders Clinic of the Centre for Sleep and Chronobiology
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5T 3A9
Country
Canada
Facility Name
Brain & Sleep Diagnostic Center
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M8X2W2
Country
Canada
Facility Name
Sleep Disorder Centre -- Hopital du Sacre-Coeur
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H4J1C5
Country
Canada
Facility Name
Neurologische Poliklinik - Universitats Spital Zurich
City
Zurich
ZIP/Postal Code
CH-8091
Country
Switzerland

12. IPD Sharing Statement

Citations:
PubMed Identifier
11833860
Citation
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.
Results Reference
background
PubMed Identifier
2281247
Citation
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.
Results Reference
background
PubMed Identifier
8506453
Citation
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.
Results Reference
background
PubMed Identifier
12627729
Citation
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.
Results Reference
background
PubMed Identifier
32356515
Citation
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.
Results Reference
derived
PubMed Identifier
27807903
Citation
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.
Results Reference
derived
PubMed Identifier
27778193
Citation
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.
Results Reference
derived

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

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