Trial Outcomes & Findings for A Study of the Safety and Effectiveness of ADX-N05 for Excessive Daytime Sleepiness in Subjects With Narcolepsy (NCT NCT01681121)
NCT ID: NCT01681121
Last Updated: 2021-05-28
Results Overview
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.
COMPLETED
PHASE2
93 participants
Baseline up to Week 12/Last Assessment post-dose.
2021-05-28
Participant Flow
Participant milestones
| Measure |
ADX-N05
ADX-N05 to be taken once a day for 12 weeks
ADX-N05: 150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
|
Placebo
Placebo to match ADX-N05 to be taken once a day for 12 weeks
|
|---|---|---|
|
Overall Study
STARTED
|
44
|
49
|
|
Overall Study
COMPLETED
|
36
|
38
|
|
Overall Study
NOT COMPLETED
|
8
|
11
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of the Safety and Effectiveness of ADX-N05 for Excessive Daytime Sleepiness in Subjects With Narcolepsy
Baseline characteristics by cohort
| Measure |
ADX-N05
n=44 Participants
ADX-N05 to be taken once a day for 12 weeks
ADX-N05: 150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
|
Placebo
n=49 Participants
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Placebo: 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
|
Total
n=93 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
41 Years
STANDARD_DEVIATION 12.33 • n=5 Participants
|
36.7 Years
STANDARD_DEVIATION 11.71 • n=7 Participants
|
38.7 Years
STANDARD_DEVIATION 12.13 • n=5 Participants
|
|
Sex: Female, Male
Female
|
30 Participants
n=5 Participants
|
30 Participants
n=7 Participants
|
60 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
33 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline up to Week 12/Last Assessment post-dose.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.
Outcome measures
| Measure |
ADX-N05
n=40 Participants
ADX-N05 to be taken once a day for 12 weeks
ADX-N05: 150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
|
Placebo
n=45 Participants
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Placebo: 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
|
|---|---|---|
|
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.
|
12.8 minutes
Standard Deviation 10.28
|
2.1 minutes
Standard Deviation 7.88
|
PRIMARY outcome
Timeframe: Week 12The 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.
Outcome measures
| Measure |
ADX-N05
n=43 Participants
ADX-N05 to be taken once a day for 12 weeks
ADX-N05: 150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
|
Placebo
n=47 Participants
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Placebo: 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
|
|---|---|---|
|
Number of Participants With Improved Clinical Global Impression of Change (CGI-C) Scores for ADX-N05 vs. Placebo at Last Assessment
|
37 Participants
|
18 Participants
|
SECONDARY outcome
Timeframe: Baseline up to Week 4 post-dose.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.
Outcome measures
| Measure |
ADX-N05
n=40 Participants
ADX-N05 to be taken once a day for 12 weeks
ADX-N05: 150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
|
Placebo
n=45 Participants
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Placebo: 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
|
|---|---|---|
|
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
|
9.5 minutes
Standard Deviation 8.38
|
1.4 minutes
Standard Deviation 7.59
|
SECONDARY outcome
Timeframe: Baseline up to Week 4 post-dose.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.
Outcome measures
| Measure |
ADX-N05
n=40 Participants
ADX-N05 to be taken once a day for 12 weeks
ADX-N05: 150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
|
Placebo
n=45 Participants
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Placebo: 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
|
|---|---|---|
|
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
Trial 1
|
11.7 minutes
Standard Deviation 12.50
|
1.6 minutes
Standard Deviation 9.86
|
|
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
Trial 2
|
9.7 minutes
Standard Deviation 12.51
|
1.0 minutes
Standard Deviation 9.01
|
|
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
Trial 3
|
9.9 minutes
Standard Deviation 11.44
|
1.3 minutes
Standard Deviation 9.86
|
|
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
Trial 4
|
6.8 minutes
Standard Deviation 10.56
|
1.8 minutes
Standard Deviation 7.64
|
|
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
Trial 5
|
5.4 minutes
Standard Deviation 9.10
|
-4.0 minutes
Standard Deviation 12.28
|
SECONDARY outcome
Timeframe: Baseline up to Week 12/Last Assessment post-dose.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).
Outcome measures
| Measure |
ADX-N05
n=40 Participants
ADX-N05 to be taken once a day for 12 weeks
ADX-N05: 150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
|
Placebo
n=45 Participants
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Placebo: 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
|
|---|---|---|
|
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
Trial 1
|
15.5 minutes
Standard Deviation 13.84
|
2.6 minutes
Standard Deviation 10.65
|
|
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
Trial 2
|
13.4 minutes
Standard Deviation 15.31
|
2.7 minutes
Standard Deviation 10.97
|
|
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
Trial 3
|
11.4 minutes
Standard Deviation 12.62
|
2.0 minutes
Standard Deviation 10.83
|
|
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
Trial 4
|
10.8 minutes
Standard Deviation 13.85
|
1.0 minutes
Standard Deviation 6.92
|
|
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
Trial 5
|
8.2 minutes
Standard Deviation 12.73
|
-1.6 minutes
Standard Deviation 13.12
|
SECONDARY outcome
Timeframe: Baseline up to Week 4 post-dose.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.
Outcome measures
| Measure |
ADX-N05
n=40 Participants
ADX-N05 to be taken once a day for 12 weeks
ADX-N05: 150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
|
Placebo
n=45 Participants
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Placebo: 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
|
|---|---|---|
|
Change From Baseline in Epworth Sleepiness Scale (ESS) Scores for ADX-N05 vs. Placebo at Week 4
|
-5.6 score on a scale
Standard Deviation 5.28
|
-2.4 score on a scale
Standard Deviation 4.60
|
SECONDARY outcome
Timeframe: Baseline up to Week 12/Last Assessment post-dose.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.
Outcome measures
| Measure |
ADX-N05
n=43 Participants
ADX-N05 to be taken once a day for 12 weeks
ADX-N05: 150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
|
Placebo
n=47 Participants
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Placebo: 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
|
|---|---|---|
|
Change From Baseline in ESS Scores for ADX-N05 vs. Placebo at Last Assessment
|
-8.5 score on a scale
Standard Deviation 5.75
|
-2.5 score on a scale
Standard Deviation 5.13
|
SECONDARY outcome
Timeframe: Week 4The 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.
Outcome measures
| Measure |
ADX-N05
n=40 Participants
ADX-N05 to be taken once a day for 12 weeks
ADX-N05: 150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
|
Placebo
n=45 Participants
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Placebo: 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
|
|---|---|---|
|
Number of Participants With Improved Clinical Global Impression of Change (CGI-C) Scores for ADX-N05 vs. Placebo at Week 4
|
32 Participants
|
23 Participants
|
SECONDARY outcome
Timeframe: Week 4The 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.
Outcome measures
| Measure |
ADX-N05
n=40 Participants
ADX-N05 to be taken once a day for 12 weeks
ADX-N05: 150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
|
Placebo
n=45 Participants
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Placebo: 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
|
|---|---|---|
|
Number of Participants With Improved Patient Global Impression Change (PGI-C) Scores for ADX-N05 vs. Placebo at Week 4
|
33 Participants
|
20 Participants
|
SECONDARY outcome
Timeframe: Week 12/Last AssessmentThe 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.
Outcome measures
| Measure |
ADX-N05
n=43 Participants
ADX-N05 to be taken once a day for 12 weeks
ADX-N05: 150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
|
Placebo
n=47 Participants
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Placebo: 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
|
|---|---|---|
|
Number of Participants With Improved PGI-C Scores for ADX-N05 vs. Placebo at Last Assessment
|
40 Participants
|
18 Participants
|
Adverse Events
ADX-N05
Placebo
Serious adverse events
| Measure |
ADX-N05
n=44 participants at risk
ADX-N05 to be taken once a day for 12 weeks
ADX-N05: 150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
|
Placebo
n=49 participants at risk
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Placebo: 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
|
|---|---|---|
|
Psychiatric disorders
Conversion Disorder
|
2.3%
1/44 • Number of events 1
|
0.00%
0/49
|
|
Hepatobiliary disorders
Cholecystitis Acute
|
2.3%
1/44 • Number of events 1
|
0.00%
0/49
|
Other adverse events
| Measure |
ADX-N05
n=44 participants at risk
ADX-N05 to be taken once a day for 12 weeks
ADX-N05: 150 mg once a day for 4 weeks followed by 300 mg once a day for 8 weeks
|
Placebo
n=49 participants at risk
Placebo to match ADX-N05 to be taken once a day for 12 weeks
Placebo: 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
|
|---|---|---|
|
Nervous system disorders
Headache
|
15.9%
7/44
|
10.2%
5/49
|
|
Nervous system disorders
Insomnia
|
13.6%
6/44
|
2.0%
1/49
|
|
Nervous system disorders
Dizziness
|
6.8%
3/44
|
2.0%
1/49
|
|
Nervous system disorders
Initial Insomnia
|
4.5%
2/44
|
4.1%
2/49
|
|
Nervous system disorders
Middle Insomnia
|
4.5%
2/44
|
0.00%
0/49
|
|
Gastrointestinal disorders
Nausea
|
13.6%
6/44
|
6.1%
3/49
|
|
Gastrointestinal disorders
Diarrhoea
|
11.4%
5/44
|
6.1%
3/49
|
|
Infections and infestations
Upper Respirstory Tract Infection
|
4.5%
2/44
|
8.2%
4/49
|
|
Infections and infestations
Nasophanyngitis
|
4.5%
2/44
|
2.0%
1/49
|
|
Psychiatric disorders
Anxiety
|
11.4%
5/44
|
0.00%
0/49
|
|
Psychiatric disorders
Agitation
|
6.8%
3/44
|
0.00%
0/49
|
|
Psychiatric disorders
Bruxism
|
6.8%
3/44
|
0.00%
0/49
|
Additional Information
Director, Disclosure & Transparency
Jazz Pharmaceuticals
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER