A Study of the Safety and Effectiveness of ADX-N05 for Excessive Daytime Sleepiness in Subjects With Narcolepsy
Primary Purpose
Narcolepsy
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
ADX-N05
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Narcolepsy
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of narcolepsy
- Good general health
- Willing and able to comply with the study design and schedule and other requirements
Exclusion Criteria:
- If female, pregnant or lactating
- Customary bedtime later than midnight
- History of significant medical condition, behavioral, or psychiatric disorder (including suicidal ideation), or surgical history
- Any other clinically relevant medical, behavioral or psychiatric disorder other than narcolepsy that is associated with excessive sleepiness
- History of significant cardiovascular disease
- Body mass index > 34
- Excessive caffeine use - > 600 mg/day of caffeine or > 6 cups of coffee/day
- History of alcohol or drug abuse within the past 2 years
- Nicotine dependence that has an effect on sleep
Sites / Locations
- Sleep Disorders Center of Alabama
- Pulmonary Associates
- Stanford Sleep Medicine Center
- Pacific Research Network
- Sleep-Alertness Disorders Center
- PAB Clinical Research
- Clinical Research Group of St. Petersburg
- NeuroTrials Research, Inc.
- Sleep Disorders Center of Georgia
- SleepMed of Central Georgia
- Chicago Research Center
- Community Research
- Kentucky Research Group
- The Center for Sleep and Wake Disorders
- Pulmonary and Critical Care Associates of Baltimore
- Neurocare, Inc.
- Minnesota Lung Center and Sleep Institute
- Washington University
- Rex Sleep Disorders Center
- Wake Research Associates
- Mercy St. Vincent Medical Center
- Center for Sleep Medicine
- SleepMed of South Carolina
- Future Search Trials of Neurology
- Sleep Medicine Associates of Texas
- Todd J. Swick, MD, PA
- Metroplex Pulmonary and Sleep Center
- Sleep Therapy and Research Center
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
ADX-N05
Placebo
Arm Description
ADX-N05 to be taken once a day for 12 weeks
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Outcomes
Primary Outcome Measures
Change From Baseline in the Average Sleep Latency Time (in Minutes) as Determined From the Maintenance of Wakefulness Test (MWT) for ADX-N05 300 mg vs. Placebo at Last Assessment.
The MWT is a validated objective measure of the ability to stay awake for a defined period of time. The primary analysis was a comparison of treatments vs. control groups on change from Baseline to last available post-Baseline assessment (Week 12/Last Assessment) in the average sleep latency time (in minutes) averaged across the first four trials of the MWT using a two-sample t-test.
Number of Participants With Improved Clinical Global Impression of Change (CGI-C) Scores for ADX-N05 vs. Placebo at Last Assessment
The CGI-C scale rated the change in the participant's condition as compared to the Baseline visit on a 7-point scale ranging from a minimum of "Very much improved" to a maximum of "Very much worse." The proportion of subjects experiencing at least minimal improvement on the CGI-C was calculated and summarized for each of the treatment groups at Week 4 and the last available post-Baseline assessment (Week 12/Last Assessment). The CGI-C scale consists of the following ratings: 1-Very Much improved, 2-Much improved, 3-Minimally improved, 4-No change, 5-Minimally worse, 6-Much worse, 7-Very much worse; a rating of 1 indicates a better outcome, and a rating of 7 indicates a worse outcome. Improvement was defined as a CGI-rating of 1, 2, or 3.
Secondary Outcome Measures
Change From Baseline in the Average Sleep Latency Time (in Minutes) as Determined From the Maintenance of Wakefulness Test (MWT) (Average of the First Four Trials) Following Four Weeks of Treatment With ADX-N05 150 mg vs. Placebo
The MWT is a validated objective measure of the ability to stay awake for a defined period of time. The MWT consisted of five 40-minute trials separated by 2 hour intervals.
This secondary analysis repeated the primary analysis for effects at the end of Week 4.
Change From Baseline in Sleep Latency Time (in Minutes) as Determined From Each of the 5 Individual MWT Trials for ADX-N05 vs. Placebo at Week 4
The MWT is a validated objective measure of the ability to stay awake for a defined period of time. The MWT consisted of five 40-minute trials separated by 2 hour intervals.
This secondary analysis repeated the primary analysis for effects for the five MWT trials analyzed separately at Week 4.
Change From Baseline in Sleep Latency Time (in Minutes) as Determined From Each of the 5 Individual MWT Trials for ADX-N05 vs. Placebo at Last Assessment
The MWT is a validated objective measure of the ability to stay awake for a defined period of time. The MWT consisted of five 40-minute trials separated by 2 hour intervals.
This secondary analysis repeated the primary analysis for effects for the five MWT trials analyzed separately at the last available post-Baseline assessment (Week 12/Last Assessment).
Change From Baseline in Epworth Sleepiness Scale (ESS) Scores for ADX-N05 vs. Placebo at Week 4
The ESS is a questionnaire intended to measure daytime sleepiness. In this test, participants answer questions with regard to the level of sleepiness they experienced over approximately the 7 days prior to the assessment while performing eight common, non-stimulating activities. The ESS total score range is 1 to 24. Each activity is rated on a 4-point scale ranging from a minimum of "would never doze" to a maximum of "a high chance of dozing." Thus, the ESS scale range is as follows: 0=would never doze, 1=slight chance of dozing, 2=moderate chance of dozing, 3=high chance of dozing; 0 indicates a better outcome, and 3 indicates a worse outcome. A negative mean change value indicates a decrease in score from baseline and an improvement in daytime sleepiness.
Change From Baseline in ESS Scores for ADX-N05 vs. Placebo at Last Assessment
The ESS is a questionnaire intended to measure daytime sleepiness. In this test, participants answer questions with regard to the level of sleepiness they experienced over approximately the 7 days prior to the assessment while performing eight common, non-stimulating activities. The ESS total score range is 1 to 24. Each activity is rated on a 4-point scale ranging from a minimum of "would never doze" to a maximum of "a high chance of dozing." Thus, the ESS scale range is as follows: 0=would never doze, 1=slight chance of dozing, 2=moderate chance of dozing, 3=high chance of dozing; 0 indicates a better outcome, and 3 indicates a worse outcome. A negative mean change value indicates a decrease in score from baseline and an improvement in daytime sleepiness.
Number of Participants With Improved Clinical Global Impression of Change (CGI-C) Scores for ADX-N05 vs. Placebo at Week 4
The CGI-C scale rated the change in the participant's condition as compared to the Baseline visit on a 7-point scale ranging from a minimum of "Very much improved" to a maximum of "Very much worse." The proportion of subjects experiencing at least minimal improvement on the CGI-C was calculated and summarized for each of the treatment groups at Week 4 and the last available post-Baseline assessment (Week 12/Last Assessment). The CGI-C scale consists of the following ratings: 1-Very Much improved, 2-Much improved, 3-Minimally improved, 4-No change, 5-Minimally worse, 6-Much worse, 7-Very much worse; a rating of 1 indicates a better outcome, and a rating of 7 indicates a worse outcome. Improvement was defined as a CGI-rating of 1, 2, or 3.
Number of Participants With Improved Patient Global Impression Change (PGI-C) Scores for ADX-N05 vs. Placebo at Week 4
The Patient Global Impression - Change (PGI-C) scale was completed by the subject at the Weeks 1, 2, 4, 6, 8, and 12 visits. The CGI-C scale rated the change in the participant's condition as compared to the Baseline visit on a 7-point scale ranging from a minimum of "Very much improved" to a maximum of "Very much worse." The proportion of subjects experiencing at least minimal improvement on the CGI-C was calculated and summarized for each of the treatment groups at Week 4 and the last available post-Baseline assessment (Week 12/Last Assessment). The CGI-C scale consists of the following ratings: 1-Very Much improved, 2-Much improved, 3-Minimally improved, 4-No change, 5-Minimally worse, 6-Much worse, 7-Very much worse; a rating of 1 indicates a better outcome, and a rating of 7 indicates a worse outcome. Improvement was defined as a CGI-rating of 1, 2, or 3.
Number of Participants With Improved PGI-C Scores for ADX-N05 vs. Placebo at Last Assessment
The Patient Global Impression - Change (PGI-C) scale was completed by the subject at the Weeks 1, 2, 4, 6, 8, and 12 visits. The CGI-C scale rated the change in the participant's condition as compared to the Baseline visit on a 7-point scale ranging from a minimum of "Very much improved" to a maximum of "Very much worse." The proportion of subjects experiencing at least minimal improvement on the CGI-C was calculated and summarized for each of the treatment groups at Week 4 and the last available post-Baseline assessment (Week 12/Last Assessment). The CGI-C scale consists of the following ratings: 1-Very Much improved, 2-Much improved, 3-Minimally improved, 4-No change, 5-Minimally worse, 6-Much worse, 7-Very much worse; a rating of 1 indicates a better outcome, and a rating of 7 indicates a worse outcome. Improvement was defined as a CGI-rating of 1, 2, or 3.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01681121
Brief Title
A Study of the Safety and Effectiveness of ADX-N05 for Excessive Daytime Sleepiness in Subjects With Narcolepsy
Official Title
A Twelve-week, Double-blind, Placebo-controlled, Randomized, Parallel-group, Multi-center Study of the Safety and Efficacy of ADX-N05 in the Treatment of Excessive Daytime Sleepiness in Subjects With Narcolepsy
Study Type
Interventional
2. Study Status
Record Verification Date
May 2021
Overall Recruitment Status
Completed
Study Start Date
September 2012 (undefined)
Primary Completion Date
August 2013 (Actual)
Study Completion Date
August 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Jazz Pharmaceuticals
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
This is a study to evaluate the safety and effectiveness of ADX-N05 compared to placebo in the treatment of excessive daytime sleepiness in adults with narcolepsy.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Narcolepsy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
93 (Actual)
8. Arms, Groups, and Interventions
Arm Title
ADX-N05
Arm Type
Experimental
Arm Description
ADX-N05 to be taken once a day for 12 weeks
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Intervention Type
Drug
Intervention Name(s)
ADX-N05
Intervention Description
150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
One capsule placebo to match ADX-N05 to be taken for 4 weeks followed by 2 capsules placebo to match ADX-N05 to be taken for 8 weeks
Primary Outcome Measure Information:
Title
Change From Baseline in the Average Sleep Latency Time (in Minutes) as Determined From the Maintenance of Wakefulness Test (MWT) for ADX-N05 300 mg vs. Placebo at Last Assessment.
Description
The MWT is a validated objective measure of the ability to stay awake for a defined period of time. The primary analysis was a comparison of treatments vs. control groups on change from Baseline to last available post-Baseline assessment (Week 12/Last Assessment) in the average sleep latency time (in minutes) averaged across the first four trials of the MWT using a two-sample t-test.
Time Frame
Baseline up to Week 12/Last Assessment post-dose.
Title
Number of Participants With Improved Clinical Global Impression of Change (CGI-C) Scores for ADX-N05 vs. Placebo at Last Assessment
Description
The CGI-C scale rated the change in the participant's condition as compared to the Baseline visit on a 7-point scale ranging from a minimum of "Very much improved" to a maximum of "Very much worse." The proportion of subjects experiencing at least minimal improvement on the CGI-C was calculated and summarized for each of the treatment groups at Week 4 and the last available post-Baseline assessment (Week 12/Last Assessment). The CGI-C scale consists of the following ratings: 1-Very Much improved, 2-Much improved, 3-Minimally improved, 4-No change, 5-Minimally worse, 6-Much worse, 7-Very much worse; a rating of 1 indicates a better outcome, and a rating of 7 indicates a worse outcome. Improvement was defined as a CGI-rating of 1, 2, or 3.
Time Frame
Week 12
Secondary Outcome Measure Information:
Title
Change From Baseline in the Average Sleep Latency Time (in Minutes) as Determined From the Maintenance of Wakefulness Test (MWT) (Average of the First Four Trials) Following Four Weeks of Treatment With ADX-N05 150 mg vs. Placebo
Description
The MWT is a validated objective measure of the ability to stay awake for a defined period of time. The MWT consisted of five 40-minute trials separated by 2 hour intervals.
This secondary analysis repeated the primary analysis for effects at the end of Week 4.
Time Frame
Baseline up to Week 4 post-dose.
Title
Change From Baseline in Sleep Latency Time (in Minutes) as Determined From Each of the 5 Individual MWT Trials for ADX-N05 vs. Placebo at Week 4
Description
The MWT is a validated objective measure of the ability to stay awake for a defined period of time. The MWT consisted of five 40-minute trials separated by 2 hour intervals.
This secondary analysis repeated the primary analysis for effects for the five MWT trials analyzed separately at Week 4.
Time Frame
Baseline up to Week 4 post-dose.
Title
Change From Baseline in Sleep Latency Time (in Minutes) as Determined From Each of the 5 Individual MWT Trials for ADX-N05 vs. Placebo at Last Assessment
Description
The MWT is a validated objective measure of the ability to stay awake for a defined period of time. The MWT consisted of five 40-minute trials separated by 2 hour intervals.
This secondary analysis repeated the primary analysis for effects for the five MWT trials analyzed separately at the last available post-Baseline assessment (Week 12/Last Assessment).
Time Frame
Baseline up to Week 12/Last Assessment post-dose.
Title
Change From Baseline in Epworth Sleepiness Scale (ESS) Scores for ADX-N05 vs. Placebo at Week 4
Description
The ESS is a questionnaire intended to measure daytime sleepiness. In this test, participants answer questions with regard to the level of sleepiness they experienced over approximately the 7 days prior to the assessment while performing eight common, non-stimulating activities. The ESS total score range is 1 to 24. Each activity is rated on a 4-point scale ranging from a minimum of "would never doze" to a maximum of "a high chance of dozing." Thus, the ESS scale range is as follows: 0=would never doze, 1=slight chance of dozing, 2=moderate chance of dozing, 3=high chance of dozing; 0 indicates a better outcome, and 3 indicates a worse outcome. A negative mean change value indicates a decrease in score from baseline and an improvement in daytime sleepiness.
Time Frame
Baseline up to Week 4 post-dose.
Title
Change From Baseline in ESS Scores for ADX-N05 vs. Placebo at Last Assessment
Description
The ESS is a questionnaire intended to measure daytime sleepiness. In this test, participants answer questions with regard to the level of sleepiness they experienced over approximately the 7 days prior to the assessment while performing eight common, non-stimulating activities. The ESS total score range is 1 to 24. Each activity is rated on a 4-point scale ranging from a minimum of "would never doze" to a maximum of "a high chance of dozing." Thus, the ESS scale range is as follows: 0=would never doze, 1=slight chance of dozing, 2=moderate chance of dozing, 3=high chance of dozing; 0 indicates a better outcome, and 3 indicates a worse outcome. A negative mean change value indicates a decrease in score from baseline and an improvement in daytime sleepiness.
Time Frame
Baseline up to Week 12/Last Assessment post-dose.
Title
Number of Participants With Improved Clinical Global Impression of Change (CGI-C) Scores for ADX-N05 vs. Placebo at Week 4
Description
The CGI-C scale rated the change in the participant's condition as compared to the Baseline visit on a 7-point scale ranging from a minimum of "Very much improved" to a maximum of "Very much worse." The proportion of subjects experiencing at least minimal improvement on the CGI-C was calculated and summarized for each of the treatment groups at Week 4 and the last available post-Baseline assessment (Week 12/Last Assessment). The CGI-C scale consists of the following ratings: 1-Very Much improved, 2-Much improved, 3-Minimally improved, 4-No change, 5-Minimally worse, 6-Much worse, 7-Very much worse; a rating of 1 indicates a better outcome, and a rating of 7 indicates a worse outcome. Improvement was defined as a CGI-rating of 1, 2, or 3.
Time Frame
Week 4
Title
Number of Participants With Improved Patient Global Impression Change (PGI-C) Scores for ADX-N05 vs. Placebo at Week 4
Description
The Patient Global Impression - Change (PGI-C) scale was completed by the subject at the Weeks 1, 2, 4, 6, 8, and 12 visits. The CGI-C scale rated the change in the participant's condition as compared to the Baseline visit on a 7-point scale ranging from a minimum of "Very much improved" to a maximum of "Very much worse." The proportion of subjects experiencing at least minimal improvement on the CGI-C was calculated and summarized for each of the treatment groups at Week 4 and the last available post-Baseline assessment (Week 12/Last Assessment). The CGI-C scale consists of the following ratings: 1-Very Much improved, 2-Much improved, 3-Minimally improved, 4-No change, 5-Minimally worse, 6-Much worse, 7-Very much worse; a rating of 1 indicates a better outcome, and a rating of 7 indicates a worse outcome. Improvement was defined as a CGI-rating of 1, 2, or 3.
Time Frame
Week 4
Title
Number of Participants With Improved PGI-C Scores for ADX-N05 vs. Placebo at Last Assessment
Description
The Patient Global Impression - Change (PGI-C) scale was completed by the subject at the Weeks 1, 2, 4, 6, 8, and 12 visits. The CGI-C scale rated the change in the participant's condition as compared to the Baseline visit on a 7-point scale ranging from a minimum of "Very much improved" to a maximum of "Very much worse." The proportion of subjects experiencing at least minimal improvement on the CGI-C was calculated and summarized for each of the treatment groups at Week 4 and the last available post-Baseline assessment (Week 12/Last Assessment). The CGI-C scale consists of the following ratings: 1-Very Much improved, 2-Much improved, 3-Minimally improved, 4-No change, 5-Minimally worse, 6-Much worse, 7-Very much worse; a rating of 1 indicates a better outcome, and a rating of 7 indicates a worse outcome. Improvement was defined as a CGI-rating of 1, 2, or 3.
Time Frame
Week 12/Last Assessment
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of narcolepsy
Good general health
Willing and able to comply with the study design and schedule and other requirements
Exclusion Criteria:
If female, pregnant or lactating
Customary bedtime later than midnight
History of significant medical condition, behavioral, or psychiatric disorder (including suicidal ideation), or surgical history
Any other clinically relevant medical, behavioral or psychiatric disorder other than narcolepsy that is associated with excessive sleepiness
History of significant cardiovascular disease
Body mass index > 34
Excessive caffeine use - > 600 mg/day of caffeine or > 6 cups of coffee/day
History of alcohol or drug abuse within the past 2 years
Nicotine dependence that has an effect on sleep
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
David Ward, MD
Organizational Affiliation
Aerial BioPharma, LLC
Official's Role
Study Director
Facility Information:
Facility Name
Sleep Disorders Center of Alabama
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35213
Country
United States
Facility Name
Pulmonary Associates
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85006
Country
United States
Facility Name
Stanford Sleep Medicine Center
City
Redwood City
State/Province
California
ZIP/Postal Code
94063
Country
United States
Facility Name
Pacific Research Network
City
San Diego
State/Province
California
ZIP/Postal Code
92103
Country
United States
Facility Name
Sleep-Alertness Disorders Center
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80012
Country
United States
Facility Name
PAB Clinical Research
City
Brandon
State/Province
Florida
ZIP/Postal Code
33511
Country
United States
Facility Name
Clinical Research Group of St. Petersburg
City
Saint Petersburg
State/Province
Florida
ZIP/Postal Code
33707
Country
United States
Facility Name
NeuroTrials Research, Inc.
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Facility Name
Sleep Disorders Center of Georgia
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Facility Name
SleepMed of Central Georgia
City
Macon
State/Province
Georgia
ZIP/Postal Code
31201
Country
United States
Facility Name
Chicago Research Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60634
Country
United States
Facility Name
Community Research
City
Crestview Hills
State/Province
Kentucky
ZIP/Postal Code
41017
Country
United States
Facility Name
Kentucky Research Group
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40217
Country
United States
Facility Name
The Center for Sleep and Wake Disorders
City
Chevy Chase
State/Province
Maryland
ZIP/Postal Code
20815
Country
United States
Facility Name
Pulmonary and Critical Care Associates of Baltimore
City
Towson
State/Province
Maryland
ZIP/Postal Code
21204
Country
United States
Facility Name
Neurocare, Inc.
City
Newton
State/Province
Massachusetts
ZIP/Postal Code
02459
Country
United States
Facility Name
Minnesota Lung Center and Sleep Institute
City
Edina
State/Province
Minnesota
ZIP/Postal Code
55435
Country
United States
Facility Name
Washington University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63108
Country
United States
Facility Name
Rex Sleep Disorders Center
City
Raleigh
State/Province
North Carolina
ZIP/Postal Code
27607
Country
United States
Facility Name
Wake Research Associates
City
Raleigh
State/Province
North Carolina
ZIP/Postal Code
27612
Country
United States
Facility Name
Mercy St. Vincent Medical Center
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43623
Country
United States
Facility Name
Center for Sleep Medicine
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19118
Country
United States
Facility Name
SleepMed of South Carolina
City
Columbia
State/Province
South Carolina
ZIP/Postal Code
29201
Country
United States
Facility Name
Future Search Trials of Neurology
City
Austin
State/Province
Texas
ZIP/Postal Code
78731
Country
United States
Facility Name
Sleep Medicine Associates of Texas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
Facility Name
Todd J. Swick, MD, PA
City
Houston
State/Province
Texas
ZIP/Postal Code
77063
Country
United States
Facility Name
Metroplex Pulmonary and Sleep Center
City
McKinney
State/Province
Texas
ZIP/Postal Code
75069
Country
United States
Facility Name
Sleep Therapy and Research Center
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
27166238
Citation
Ruoff C, Swick TJ, Doekel R, Emsellem HA, Feldman NT, Rosenberg R, Bream G, Khayrallah MA, Lu Y, Black J. Effect of Oral JZP-110 (ADX-N05) on Wakefulness and Sleepiness in Adults with Narcolepsy: A Phase 2b Study. Sleep. 2016 Jul 1;39(7):1379-87. doi: 10.5665/sleep.5968.
Results Reference
derived
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A Study of the Safety and Effectiveness of ADX-N05 for Excessive Daytime Sleepiness in Subjects With Narcolepsy
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