Trial Outcomes & Findings for Efficacy and Safety of Tasimelteon Compared With Placebo in Totally Blind Subjects With Non-24-Hour Sleep-Wake Disorder (NCT NCT01163032)
NCT ID: NCT01163032
Last Updated: 2014-10-16
Results Overview
Entrainment is a measure of synchronization of the master body clock to the 24-hour day. The circadian period (τ) was calculated using urinary aMT6s collected over four 48 hour periods , collected approximately 1 week apart for 4 separate weeks, during the screening and month 1 of the randomization phase of the trial. Entrainment was defined as having a post-baseline τ value less than 24.1 and a 95% CI that included 24.0.
COMPLETED
PHASE3
136 participants
1 month
2014-10-16
Participant Flow
\*Various category for the Randomization Phase: 4 patients in each treatment group discontinued due to study termination by the sponsor and 1 patient in the tasimelteon group discontinued due to travel across multiple time zones
Participant milestones
| Measure |
Tasimelteon (Randomized)
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
Open Label Tasimelteon
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
|---|---|---|---|
|
Randomization Phase
STARTED
|
42
|
42
|
0
|
|
Randomization Phase
COMPLETED
|
32
|
30
|
0
|
|
Randomization Phase
NOT COMPLETED
|
10
|
12
|
0
|
|
Open Label Extension Phase
STARTED
|
0
|
0
|
55
|
|
Open Label Extension Phase
COMPLETED
|
0
|
0
|
39
|
|
Open Label Extension Phase
NOT COMPLETED
|
0
|
0
|
16
|
Reasons for withdrawal
| Measure |
Tasimelteon (Randomized)
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
Open Label Tasimelteon
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
|---|---|---|---|
|
Randomization Phase
Withdrawal by Subject
|
2
|
3
|
0
|
|
Randomization Phase
Adverse Event
|
3
|
3
|
0
|
|
Randomization Phase
Protocol Violation
|
0
|
1
|
0
|
|
Randomization Phase
Various*
|
5
|
4
|
0
|
|
Randomization Phase
Unsatisfactory Therapeutic Effect
|
0
|
1
|
0
|
|
Open Label Extension Phase
Adverse Event
|
0
|
0
|
3
|
|
Open Label Extension Phase
Withdrawal by Subject
|
0
|
0
|
3
|
|
Open Label Extension Phase
Protocol Violation
|
0
|
0
|
1
|
|
Open Label Extension Phase
Study Termination
|
0
|
0
|
7
|
|
Open Label Extension Phase
Unsatisfactory Therapeutic Effect
|
0
|
0
|
2
|
Baseline Characteristics
Efficacy and Safety of Tasimelteon Compared With Placebo in Totally Blind Subjects With Non-24-Hour Sleep-Wake Disorder
Baseline characteristics by cohort
| Measure |
Tasimelteon (Randomized)
n=42 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=42 Participants
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
Open Label Tasimelteon
n=52 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Total
n=136 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
50.8 years
STANDARD_DEVIATION 12.63 • n=5 Participants
|
50.7 years
STANDARD_DEVIATION 13.15 • n=7 Participants
|
50.37 years
STANDARD_DEVIATION 13.17 • n=5 Participants
|
50.6 years
STANDARD_DEVIATION 12.91 • n=4 Participants
|
|
Age, Customized
Rand to OLE
|
42.00 years
STANDARD_DEVIATION 5.66 • n=5 Participants
|
54.00 years
STANDARD_DEVIATION NA • n=7 Participants
|
NA years
STANDARD_DEVIATION NA • n=5 Participants
|
46.00 years
STANDARD_DEVIATION 8.00 • n=4 Participants
|
|
Sex/Gender, Customized
Rand to OLE (Female)
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
0 participants
n=4 Participants
|
|
Sex/Gender, Customized
Rand to OLE (Male)
|
2 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
3 participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
18 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
25 Participants
n=5 Participants
|
60 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
24 Participants
n=5 Participants
|
25 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
76 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 1 monthPopulation: Intent-to-Treat (ITT) Population: all subjects randomized into the study that have τ calculated post-randomization.
Entrainment is a measure of synchronization of the master body clock to the 24-hour day. The circadian period (τ) was calculated using urinary aMT6s collected over four 48 hour periods , collected approximately 1 week apart for 4 separate weeks, during the screening and month 1 of the randomization phase of the trial. Entrainment was defined as having a post-baseline τ value less than 24.1 and a 95% CI that included 24.0.
Outcome measures
| Measure |
Tasimelteon (Randomized)
n=40 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=38 Participants
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
|---|---|---|
|
Proportion of Patients Entrained as Assessed by Urinary aMT6
|
20 percentage of patients
|
2.6 percentage of patients
|
PRIMARY outcome
Timeframe: 6 monthsPopulation: Analysis Population: all patients in the ITT population that had at least 70% of 1 circadian cycle of nighttime total sleep data reported during each phase (screening and post-randomization)
Clinical response is defined as the coincident demonstration of entrainment (aMT6) and a score ≥ 3 on the Non-24 Clinical Response Scale (N24CRS). N24CRS measures improvement in sleep-wake measures and overall functioning (LQ-nTST, UQ-dTSD, MoST and CGI-C). Each assessment is scored as a 1 or 0 depending on the pre-specified threshold (see below). LQ-nTST: \>45 minutes increase in average nighttime sleep duration; UQ-dTSD: \>45 minutes decrease in average daytime sleep duration; MoST: \>30 minutes increase and a standard deviation \<2 hours during double-masked phase (6 months); CGI-C: \<2.0 from the average of D112 and Day 183 compared to baseline For patients randomized to tasimelteon 20 mg and who also participated in the screening phase of Study 3203 (month 7 of treatment), the screening τ from Study 3203 was used if the patient did not become entrained in Study 3201 but did become entrained during the screening phase of Study 3203.
Outcome measures
| Measure |
Tasimelteon (Randomized)
n=38 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=34 Participants
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
|---|---|---|
|
Proportion of Patients With a Clinical Response: Entrainment of aMT6 and Score of ≥ 3 on N24CRS
|
23.7 percentage of patients
|
0 percentage of patients
|
SECONDARY outcome
Timeframe: 1 monthPopulation: Intent-to-Treat (ITT) Population: all subjects randomized into the study that have τ calculated post-randomization.
Entrainment is a measure of synchronization of the master body clock to the 24-hour day. The circadian period (τ) was calculated using urinary cortisol collected over four 48 hour periods, approximately 1 week apart for 4 separate weeks, during the screening and month 1 of the randomization phase of the trial. Entrainment was defined as having a post-baseline τ value less than 24.1 and a 95% CI that included 24.0.
Outcome measures
| Measure |
Tasimelteon (Randomized)
n=40 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=38 Participants
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
|---|---|---|
|
Proportion of Patients Entrained as Assessed by Urinary Cortisol
|
17.5 percentage of patients
|
2.6 percentage of patients
|
SECONDARY outcome
Timeframe: Day 112 and 183Population: Analysis Population: all patients in the ITT population that had at least 70% of 1 circadian cycle of nighttime total sleep data reported during each phase (screening and post-randomization)
CGI-C scores range from 1 (very much improved) to 7 (very much worse). The average post-randomization score was obtained for each patient by averaging the last 2 scheduled assessments (Day D112 and Day D183). Lower number indicates improvement.
Outcome measures
| Measure |
Tasimelteon (Randomized)
n=38 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=34 Participants
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
|---|---|---|
|
Average Clinical Global Impression of Change (CGI-C)
|
2.6 score
Standard Error 0.20
|
3.4 score
Standard Error 0.21
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: Analysis Population: all patients in the ITT population that had at least 70% of 1 circadian cycle of nighttime total sleep data reported during each phase (screening and post-randomization)
The sleep/wake response represents measurement of the combined improvement in the nighttime sleep duration and daytime sleep duration. Individuals that have an improvement in nighttime sleep and daytime sleep, defined as an increase of 90 minutes or more in the lower quartile of subjective nighttime total sleep time (LQ-nTST) and a decrease of 90 minutes or more in the upper quartile of daytime total sleep duration (UQ-dTSD) are considered to be a responder.
Outcome measures
| Measure |
Tasimelteon (Randomized)
n=38 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=34 Participants
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
|---|---|---|
|
Proportion of Responders With a Combined Sleep/Wake Response for LQ-nTST (≥ 90 Minutes) and UQ-dTSD (≤ 90 Minutes)
|
13.2 percentage of patients
|
2.9 percentage of patients
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: Analysis Population: all patients in the ITT population that had at least 70% of 1 circadian cycle of nighttime total sleep data reported during each phase (screening and post-randomization)
LQ-nTST measures the difference in average nighttime sleep during the patient's worst 25% of nights (shortest total nighttime sleep) between the randomized phase (6 months)and the screening phase (\~ 6 weeks). The higher number indicates improvement.
Outcome measures
| Measure |
Tasimelteon (Randomized)
n=38 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=34 Participants
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
|---|---|---|
|
Average Lower Quartile of Nights of Nighttime Total Sleep Time (LQ-nTST)
|
56.80 minutes
Standard Error 9.305
|
17.08 minutes
Standard Error 9.702
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: Analysis Population: all patients in the ITT population that had at least 70% of 1 circadian cycle of nighttime total sleep data reported during each phase (screening and post-randomization)
UQ-dTSD measures the difference in average daytime sleep during the patient's worst 25% of days (longest total daytime sleep) between the randomized phase (6 months) and the screening phase (\~ 6 weeks). Lower number indicates improvement.
Outcome measures
| Measure |
Tasimelteon (Randomized)
n=38 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=34 Participants
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
|---|---|---|
|
Average Upper Quartile of Days of Subjective Daytime Sleep Duration (UQ-dTSD)
|
-46.48 minutes
Standard Error 6.595
|
-17.87 minutes
Standard Error 6.889
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: Analysis Population: all patients in the ITT population that had at least 70% of 1 circadian cycle of nighttime total sleep data reported during each phase (screening and post-randomization)
Midpoint of Sleep Timing (MoST) is the measurement of the average midpoint of sleep time relative to bedtime. The average MoST value will trend to 0 as an individual's sleep becomes more fragmented. Improvement is defined as an increase in the average.
Outcome measures
| Measure |
Tasimelteon (Randomized)
n=38 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=34 Participants
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
|---|---|---|
|
Average Midpoint of Sleep (MoST)
|
35.00 minutes
Standard Error 5.313
|
14.48 minutes
Standard Error 5.547
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: One subject who rolled into the OLE from the randomized phase (tasimelteon) experienced an unrelated TEAE during the OLE phase.
Adverse events were recorded in the source documents from the time of the patient's informed consent signature until the end of the patient's study participation. An AE was defined as any untoward medical occurrence in a clinical investigation patient who does not necessarily have causal relationship with treatment.
Outcome measures
| Measure |
Tasimelteon (Randomized)
n=54 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
|---|---|---|
|
Number of Patients With a Treatment Emergent Adverse Event (Open Label Extension Phase Only)
|
37 participants
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 6 monthsPopulation: Analysis Population: all patients in the ITT population that had at least 70% of 1 circadian cycle of nighttime total sleep data reported during each phase (screening and post-randomization)
Clinical response is defined as the coincident demonstration of entrainment (aMT6) and a score ≥ 2 on the Non-24 Clinical Response Scale (N24CRS) which includes LQ-nTST, UQ-dTSD, MoST and CGI-C assessments. Each assessment is scored as a 1 or 0 depending on the pre-specified threshold (see below). LQ-nTST: \>45 minutes increase in average nighttime sleep duration; UQ-dTSD: \>45 minutes decrease in average daytime sleep duration; MoST: \>30 minutes increase and a standard deviation \<2 hours during double-masked phase (6 months); CGI-C: \<2.0 from the average of D112 and Day 183 compared to baseline For patients randomized to tasimelteon 20 mg and who also participated in the screening phase of Study 3203 (month 7 of treatment), the screening τ from Study 3203 was used if the patient did not become entrained in Study 3201 but did become entrained during the screening phase of Study 3203.
Outcome measures
| Measure |
Tasimelteon (Randomized)
n=38 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=34 Participants
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
|---|---|---|
|
Proportion of Patients With a Clinical Response: Entrainment of aMT6 and Score of ≥ 2 on N24CRS
|
28.9 percentage of patients
|
0 percentage of patients
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 6 monthsPopulation: Analysis Population: all patients in the ITT population that had at least 70% of 1 circadian cycle of nighttime total sleep data reported during each phase (screening and post-randomization)
Non-24 Clinical Response Scale (N24CRS) was a 4-item scale which includes LQ-nTST, UQ-dTSD, MoST and CGI-C assessments. Each assessment is scored as a 1 or 0 depending on the pre-specified threshold (see below). LQ-nTST: \>45 minutes increase in average nighttime sleep duration; UQ-dTSD: \>45 minutes decrease in average daytime sleep duration; MoST: \>30 minutes increase and a standard deviation \<2 hours during double-masked phase (6 months); CGI-C: \<2.0 from the average of D112 and Day 183 compared to baseline
Outcome measures
| Measure |
Tasimelteon (Randomized)
n=38 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=34 Participants
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
|---|---|---|
|
Proportion of Patients With a Clinical Response (Score of ≥ 3 on N24CRS)
|
28.9 percentage of patients
|
2.9 percentage of patients
|
POST_HOC outcome
Timeframe: 6 monthsPopulation: Analysis Population: all patients in the ITT population that had at least 70% of 1 circadian cycle of nighttime total sleep data reported during each phase (screening and post-randomization)
Responder analysis with responder defined as an increase of 45 minutes or more in (LQ-nTST) and a decrease of 45 minutes or more in (UQ-dTSD).
Outcome measures
| Measure |
Tasimelteon (Randomized)
n=38 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=34 Participants
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
|---|---|---|
|
Proportion of Responders With a Combined Sleep/Wake Response for LQ-nTST (≥ 45 Minutes) and UQ-dTSD (≤ 45 Minutes)
|
31.6 percentage of patients
|
8.8 percentage of patients
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 6 monthsPopulation: Analysis Population: all patients in the ITT population that had at least 70% of 1 circadian cycle of nighttime total sleep data reported during each phase (screening and post-randomization)
Non-24 Clinical Response Scale (N24CRS) was a 4-item scale which includes LQ-nTST, UQ-dTSD, MoST and CGI-C assessments. Each assessment is scored as a 1 or 0 depending on the pre-specified threshold (see below). LQ-nTST: \>45 minutes increase in average nighttime sleep duration; UQ-dTSD: \>45 minutes decrease in average daytime sleep duration; MoST: \>30 minutes increase and a standard deviation \<2 hours during double-masked phase (6 months); CGI-C: \<2.0 from the average of D112 and Day 183 compared to baseline
Outcome measures
| Measure |
Tasimelteon (Randomized)
n=38 Participants
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=34 Participants
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
|---|---|---|
|
Proportion of Patients With a Clinical Response (Score of ≥ 2 on N24CRS)
|
57.9 percentage of patients
|
20.6 percentage of patients
|
Adverse Events
Tasimelteon (Randomized)
Placebo (Randomized)
Open Label Tasimelteon
Serious adverse events
| Measure |
Tasimelteon (Randomized)
n=42 participants at risk
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=42 participants at risk
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
Open Label Tasimelteon
n=54 participants at risk
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
|---|---|---|---|
|
Injury, poisoning and procedural complications
Procedural Pain
|
0.00%
0/42 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
2.4%
1/42 • Number of events 1 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/54 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Gastrointestinal disorders
Small Intestinal Obstruction
|
0.00%
0/42 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
2.4%
1/42 • Number of events 1 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/54 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Nervous system disorders
Syncope
|
2.4%
1/42 • Number of events 1 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/42 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/54 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute Lymphocytic Leukaemia
|
2.4%
1/42 • Number of events 1 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/42 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/54 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Hepatobiliary disorders
Cholecystitis
|
2.4%
1/42 • Number of events 1 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/42 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/54 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Infections and infestations
Diverticulitis
|
2.4%
1/42 • Number of events 1 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/42 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/54 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Nervous system disorders
Serotonin Syndrome
|
0.00%
0/42 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/42 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
1.9%
1/54 • Number of events 1 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Nervous system disorders
Transient Ischaemic Attack
|
0.00%
0/42 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/42 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
1.9%
1/54 • Number of events 1 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
Other adverse events
| Measure |
Tasimelteon (Randomized)
n=42 participants at risk
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
Placebo (Randomized)
n=42 participants at risk
Placebo capsules, PO daily for 6 months
Placebo: Placebo capsules, PO daily for 6 months
|
Open Label Tasimelteon
n=54 participants at risk
20 mg tasimelteon capsules, PO daily for 6 months
tasimelteon: 20 mg tasimelteon capsules, PO daily for 6 months
|
|---|---|---|---|
|
Gastrointestinal disorders
Nausea
|
2.4%
1/42 • Number of events 1 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
7.1%
3/42 • Number of events 3 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/54 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Infections and infestations
Nasopharyngitis
|
7.1%
3/42 • Number of events 3 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
9.5%
4/42 • Number of events 4 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
9.3%
5/54 • Number of events 6 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Infections and infestations
Urinary tract infection
|
7.1%
3/42 • Number of events 3 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
2.4%
1/42 • Number of events 1 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
9.3%
5/54 • Number of events 6 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Investigations
Alanine aminotransferase increased
|
9.5%
4/42 • Number of events 4 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
4.8%
2/42 • Number of events 2 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
3.7%
2/54 • Number of events 2 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Investigations
Asparate aminotransferase increased
|
7.1%
3/42 • Number of events 3 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
4.8%
2/42 • Number of events 2 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
5.6%
3/54 • Number of events 3 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Investigations
Gamma-glutamyltransferase increased
|
0.00%
0/42 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
4.8%
2/42 • Number of events 2 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
5.6%
3/54 • Number of events 3 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Nervous system disorders
Headache
|
16.7%
7/42 • Number of events 11 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
7.1%
3/42 • Number of events 3 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
7.4%
4/54 • Number of events 4 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Psychiatric disorders
Abnormal dreams
|
4.8%
2/42 • Number of events 2 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/42 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
5.6%
3/54 • Number of events 3 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
General disorders
Oedema peripheral
|
7.1%
3/42 • Number of events 3 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
4.8%
2/42 • Number of events 2 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/54 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
|
Infections and infestations
Upper respiratory tract infection
|
7.1%
3/42 • Number of events 3 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
0.00%
0/42 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
3.7%
2/54 • Number of events 2 • 1st dose to 30 days following last administration of study treatment
55 subjects enrolled into the Open Label phase (including 3 randomized subjects who rolled into the OLE). One subject enrolled in the Open Label phase but did not take any study drug.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place