Trial Outcomes & Findings for Safety of L1-79 in Adolescent and Adult Males With Autism (NCT NCT02947048)
NCT ID: NCT02947048
Last Updated: 2024-08-21
Results Overview
Number of Participants with On-treatment Adverse Events. To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
42 participants
Primary outcome timeframe
4 weeks
Results posted on
2024-08-21
Participant Flow
Participant milestones
| Measure |
100 mg Open
open-label lead-in L1-79 100 mg tid
|
100 mg Blinded
blinded and randomized L1-79 100 mg tid
|
Placebo (100 mg Cohort)
blinded and randomized placebo tid
|
200 mg Open
open-label lead-in L1-79 200 mg tid
|
200 mg Blinded
blinded and randomized L1-79 200 mg tid
|
Placebo (200 mg Cohort)
blinded and randomized placebo tid
|
|---|---|---|---|---|---|---|
|
Overall Study
STARTED
|
5
|
10
|
5
|
6
|
11
|
5
|
|
Overall Study
Safety Population
|
5
|
10
|
5
|
6
|
11
|
5
|
|
Overall Study
Efficacy Population
|
0
|
9
|
4
|
0
|
11
|
5
|
|
Overall Study
COMPLETED
|
5
|
10
|
5
|
5
|
10
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
1
|
1
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety of L1-79 in Adolescent and Adult Males With Autism
Baseline characteristics by cohort
| Measure |
100 mg Open
n=5 Participants
open-label lead-in 100 mg L1-79 t.i.d.
L1-79
|
100 mg Blinded
n=10 Participants
blinded and randomized 100 mg L1-79 t.i.d.
L1-79
|
Placebo Blinded (100 mg Cohort)
n=5 Participants
blinded and randomized placebo t.i.d.
Placebo (100 mg cohort)
|
200 mg Open
n=6 Participants
open-label lead-in 200 mg L1-79 t.i.d.
L1-79
|
200 mg Blinded
n=11 Participants
blinded and randomized 200 mg L1-79 t.i.d.
L1-79
|
Placebo Blinded (200 mg Cohort)
n=5 Participants
blinded and randomized placebo t.i.d.
Placebo (200 mg cohort)
|
Total
n=42 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|---|
|
Age, Continuous
|
16.2 years
n=99 Participants
|
16.1 years
n=107 Participants
|
15.3 years
n=206 Participants
|
15.8 years
n=7 Participants
|
16.4 years
n=31 Participants
|
16.6 years
n=30 Participants
|
16.1 years
n=3 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=3 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
6 Participants
n=7 Participants
|
11 Participants
n=31 Participants
|
5 Participants
n=30 Participants
|
42 Participants
n=3 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=30 Participants
|
3 Participants
n=3 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
5 Participants
n=7 Participants
|
9 Participants
n=31 Participants
|
5 Participants
n=30 Participants
|
35 Participants
n=3 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=31 Participants
|
0 Participants
n=30 Participants
|
4 Participants
n=3 Participants
|
|
Region of Enrollment
United States
|
5 participants
n=99 Participants
|
10 participants
n=107 Participants
|
5 participants
n=206 Participants
|
6 participants
n=7 Participants
|
11 participants
n=31 Participants
|
5 participants
n=30 Participants
|
42 participants
n=3 Participants
|
PRIMARY outcome
Timeframe: 4 weeksPopulation: Safety Population. Note that all Placebo data (open-label, first cohort and second cohort) is combined for the safety population. However for the efficacy population the open-label data is excluded and placebo arms from the first cohort and second cohort are presented separately because in the first cohort questionnaires were filled out at home by patients and in the second cohort they were completed in clinic with study staff.
Number of Participants with On-treatment Adverse Events. To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
Outcome measures
| Measure |
100 mg Open
n=5 Participants
open-label lead-in 100 mg L1-79 tid
|
100 mg Blinded
n=10 Participants
blinded and randomized 100 mg L1-79 tid
|
200 mg Open
n=6 Participants
open-label lead-in 200 mg L1-79 tid
|
200 mg Blinded
n=11 Participants
blinded and randomized 200 mg L1-79 tid
|
Placebo Blinded (100 mg Cohort)
n=5 Participants
blinded and randomized placebo tid (100 mg cohort)
|
Placebo Blinded (200 mg Cohort)
n=5 Participants
blinded and randomized placebo tid (200 mg cohort)
|
|---|---|---|---|---|---|---|
|
Adverse Events
cough
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Adverse Events
any adverse event
|
2 participants
|
1 participants
|
2 participants
|
6 participants
|
5 participants
|
5 participants
|
|
Adverse Events
diarrhea
|
0 participants
|
0 participants
|
0 participants
|
3 participants
|
0 participants
|
0 participants
|
|
Adverse Events
fatigue
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Adverse Events
pyrexia
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Adverse Events
seasonal allergy
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Adverse Events
ear infection
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Adverse Events
nasopharyngitis
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Adverse Events
otitis externa
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Adverse Events
amylase increased
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Adverse Events
blood thyroid stimulating hormone increased
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Adverse Events
crystal urine present
|
0 participants
|
0 participants
|
0 participants
|
2 participants
|
0 participants
|
0 participants
|
|
Adverse Events
generalized tonic-clonic seizure
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Adverse Events
syncope
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Adverse Events
agitation
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Adverse Events
anxiety
|
1 participants
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Adverse Events
confusional state
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Adverse Events
intentional self-injury
|
2 participants
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Adverse Events
irritability
|
2 participants
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Adverse Events
urinary incontinence
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
SECONDARY outcome
Timeframe: Week 0 and Week 4Population: Efficacy Population Note that for the efficacy population open-label data is excluded and placebo arms from the first cohort and second cohort are presented separately because in the first cohort questionnaires were filled out at home by patients and in the second cohort they were completed in clinic with study staff.
The Clinical Global Impression (CGI) has two components: the CGI-Severity (CGI-S), which rates illness severity, and the CGI-Improvement (CGI-I), which rates the change in illness severity from baseline. The CGI-S is rated on the following seven-point scale with 1 being normal and 7 being among the most extremely ill subjects. The CGI-I score is rated on a seven-point scale with 1 being very much improved and 7 being very much worse. Higher values of the CGI-S score indicate a "worse" state. CGI-I scores of 5 or greater indicate decline, CGI I scores of 3 or lower indicate improvement, and a CGI-I score of 4 indicates no change.
Outcome measures
| Measure |
100 mg Open
n=9 Participants
open-label lead-in 100 mg L1-79 tid
|
100 mg Blinded
n=4 Participants
blinded and randomized 100 mg L1-79 tid
|
200 mg Open
n=10 Participants
open-label lead-in 200 mg L1-79 tid
|
200 mg Blinded
n=4 Participants
blinded and randomized 200 mg L1-79 tid
|
Placebo Blinded (100 mg Cohort)
blinded and randomized placebo tid (100 mg cohort)
|
Placebo Blinded (200 mg Cohort)
blinded and randomized placebo tid (200 mg cohort)
|
|---|---|---|---|---|---|---|
|
Change From Baseline in Clinical Global Impression Scale (CGI)
Change from Baseline in Clinical Global Impression - Severity
|
-0.5 score on a scale
Standard Error 0.2
|
-0.2 score on a scale
Standard Error 0.3
|
-0.8 score on a scale
Standard Error 0.3
|
-0.3 score on a scale
Standard Error 0.4
|
—
|
—
|
|
Change From Baseline in Clinical Global Impression Scale (CGI)
Clinical Global Impression - Improvement
|
3.0 score on a scale
Standard Error 0.3
|
2.8 score on a scale
Standard Error 0.4
|
2.8 score on a scale
Standard Error 0.2
|
2.8 score on a scale
Standard Error 0.4
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 0 and Week 4Population: Efficacy Population Note that for the efficacy population open-label data is excluded and placebo arms from the first cohort and second cohort are presented separately because in the first cohort questionnaires were filled out at home by patients and in the second cohort they were completed in clinic with study staff.
The Vineland Adaptive Behavior Scales, Second Edition (VABS-II) indicates adaptive functioning and consists of four major domains. Only the communication and socialization domains were done in this study. The VABS-II data is comprised of Socialization. Socialization Standard Scores may range from 20 to 160, with a population mean of 100 and a standard deviation of 15. Lower Vineland Adaptive Behavior Socialization Standard Scores indicate a "worse" state. A positive value of change from baseline indicates improvement.
Outcome measures
| Measure |
100 mg Open
n=5 Participants
open-label lead-in 100 mg L1-79 tid
|
100 mg Blinded
n=2 Participants
blinded and randomized 100 mg L1-79 tid
|
200 mg Open
n=7 Participants
open-label lead-in 200 mg L1-79 tid
|
200 mg Blinded
n=2 Participants
blinded and randomized 200 mg L1-79 tid
|
Placebo Blinded (100 mg Cohort)
blinded and randomized placebo tid (100 mg cohort)
|
Placebo Blinded (200 mg Cohort)
blinded and randomized placebo tid (200 mg cohort)
|
|---|---|---|---|---|---|---|
|
Change From Baseline in Vineland Adaptive Behavior Scale - 2nd Edition Socialization Standard Score
|
-4.2 score on a scale
Standard Deviation 12.1
|
0.0 score on a scale
Standard Deviation 0.0
|
7.6 score on a scale
Standard Deviation 11.9
|
1.5 score on a scale
Standard Deviation 2.1
|
—
|
—
|
SECONDARY outcome
Timeframe: Week 0 and Week 4Population: Efficacy Population Note that for the efficacy population open-label data is excluded and placebo arms from the first cohort and second cohort are presented separately because in the first cohort questionnaires were filled out at home by patients and in the second cohort they were completed in clinic with study staff.
The Vineland Adaptive Behavior Scales, Second Edition (VABS-II) indicates adaptive functioning and consists of four major domains. Only the communication and socialization domains were done in this study. The VABS-II is comprised of Communication. Communication Standard Scores may range from 20 to 160, with a population mean of 100 and a standard deviation of 15. Lower Vineland Adaptive Behavior Communication Standard Scores indicate a "worse" state. A positive value of change from baseline indicates improvement.
Outcome measures
| Measure |
100 mg Open
n=8 Participants
open-label lead-in 100 mg L1-79 tid
|
100 mg Blinded
n=3 Participants
blinded and randomized 100 mg L1-79 tid
|
200 mg Open
n=7 Participants
open-label lead-in 200 mg L1-79 tid
|
200 mg Blinded
n=2 Participants
blinded and randomized 200 mg L1-79 tid
|
Placebo Blinded (100 mg Cohort)
blinded and randomized placebo tid (100 mg cohort)
|
Placebo Blinded (200 mg Cohort)
blinded and randomized placebo tid (200 mg cohort)
|
|---|---|---|---|---|---|---|
|
Change From Baseline in Vineland Adaptive Behavior Scale - 2nd Edition Communication Standard Score
|
10.1 score on a scale
Standard Deviation 16.6
|
7.7 score on a scale
Standard Deviation 10.8
|
2.7 score on a scale
Standard Deviation 4.3
|
3.0 score on a scale
Standard Deviation 2.8
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 0 and Week 4Population: Efficacy Population Note that for the efficacy population open-label data is excluded and placebo arms from the first cohort and second cohort are presented separately because in the first cohort questionnaires were filled out at home by patients and in the second cohort they were completed in clinic with study staff.
The Autism Diagnostic Observation Schedule 2nd Edition (ADOS-2) is the "Gold Standard" for assessing severity of ASD and position on the autism spectrum. The ADOS-2 is a semi-structured, standardized assessment of: Communication, Social interaction, Play, and Restricted and repetitive behaviors. The total scores range from 15 to 60, and the cut-off score to determine autism is 30. More specifically, a score of \<30 is classified as non-autism, a score of 30-36 is classified as mild to moderate autism, and a score of ≥37 is classified as severe autism. A higher ADOS-2 value indicates a "worse" state. A negative value of change from baseline indicates an improvement.
Outcome measures
| Measure |
100 mg Open
n=9 Participants
open-label lead-in 100 mg L1-79 tid
|
100 mg Blinded
n=4 Participants
blinded and randomized 100 mg L1-79 tid
|
200 mg Open
n=10 Participants
open-label lead-in 200 mg L1-79 tid
|
200 mg Blinded
n=4 Participants
blinded and randomized 200 mg L1-79 tid
|
Placebo Blinded (100 mg Cohort)
blinded and randomized placebo tid (100 mg cohort)
|
Placebo Blinded (200 mg Cohort)
blinded and randomized placebo tid (200 mg cohort)
|
|---|---|---|---|---|---|---|
|
Change From Baseline in the Autism Diagnostic Observation Schedule 2nd Edition (ADOS-2) Overall Total Score
|
-0.6 score on a scale
Standard Deviation 1.7
|
0.3 score on a scale
Standard Deviation 1.0
|
-1.0 score on a scale
Standard Deviation 2.2
|
-0.3 score on a scale
Standard Deviation 3.0
|
—
|
—
|
SECONDARY outcome
Timeframe: Week 0 and Week 4Population: Efficacy Population Note that for the efficacy population open-label data is excluded and placebo arms from the first cohort and second cohort are presented separately because in the first cohort questionnaires were filled out at home by patients and in the second cohort they were completed in clinic with study staff.
Social Responsiveness Scale - 2nd edition (SRS-2) identifies social impairment associated with ASD and quantifies its severity. Raw and T-scores will be collected for SRS-2 Total, Social Awareness, Social Cognition, Social Communication, Social Motivation, Restricted Interest and Repetitive Behavior, DSM-5 Social Communication, and DSM-5 Restrictive Interest and Repetitive Behavior. Each domain has a varied but similar ranges of possible scores from 32 points-114 points. All T-scores have a mean of 50 points with a standard deviation of 10 points. A higher SRS-2 total T-score indicates greater impairment. A negative change from baseline indicates improvement.
Outcome measures
| Measure |
100 mg Open
n=8 Participants
open-label lead-in 100 mg L1-79 tid
|
100 mg Blinded
n=4 Participants
blinded and randomized 100 mg L1-79 tid
|
200 mg Open
n=10 Participants
open-label lead-in 200 mg L1-79 tid
|
200 mg Blinded
n=4 Participants
blinded and randomized 200 mg L1-79 tid
|
Placebo Blinded (100 mg Cohort)
blinded and randomized placebo tid (100 mg cohort)
|
Placebo Blinded (200 mg Cohort)
blinded and randomized placebo tid (200 mg cohort)
|
|---|---|---|---|---|---|---|
|
Change From Baseline in the Social Responsiveness Scale - 2nd Edition (SRS-2) Total T-score
total T-score
|
-0.4 score on a scale
Standard Deviation 6.2
|
-3.8 score on a scale
Standard Deviation 9.6
|
-8.6 score on a scale
Standard Deviation 11.1
|
-9.3 score on a scale
Standard Deviation 9.8
|
—
|
—
|
|
Change From Baseline in the Social Responsiveness Scale - 2nd Edition (SRS-2) Total T-score
social awareness T-score
|
1.0 score on a scale
Standard Deviation 11.3
|
-0.8 score on a scale
Standard Deviation 1.5
|
-2.8 score on a scale
Standard Deviation 10.3
|
-8.0 score on a scale
Standard Deviation 14.9
|
—
|
—
|
|
Change From Baseline in the Social Responsiveness Scale - 2nd Edition (SRS-2) Total T-score
social cognition T-score
|
0.4 score on a scale
Standard Deviation 5.4
|
-2.3 score on a scale
Standard Deviation 5.3
|
-11.4 score on a scale
Standard Deviation 20.9
|
-7.5 score on a scale
Standard Deviation 14.1
|
—
|
—
|
|
Change From Baseline in the Social Responsiveness Scale - 2nd Edition (SRS-2) Total T-score
social communication T-score
|
1.1 score on a scale
Standard Deviation 7.1
|
-3.0 score on a scale
Standard Deviation 9.0
|
-8.9 score on a scale
Standard Deviation 11.4
|
-9.3 score on a scale
Standard Deviation 12.6
|
—
|
—
|
|
Change From Baseline in the Social Responsiveness Scale - 2nd Edition (SRS-2) Total T-score
social motivation T-score
|
-1.4 score on a scale
Standard Deviation 9.2
|
0.8 score on a scale
Standard Deviation 12.8
|
-11.5 score on a scale
Standard Deviation 12.9
|
-6.8 score on a scale
Standard Deviation 4.9
|
—
|
—
|
|
Change From Baseline in the Social Responsiveness Scale - 2nd Edition (SRS-2) Total T-score
RRB T-score
|
-0.4 score on a scale
Standard Deviation 9.3
|
-9.8 score on a scale
Standard Deviation 14.0
|
-8.7 score on a scale
Standard Deviation 13.5
|
-7.3 score on a scale
Standard Deviation 7.2
|
—
|
—
|
|
Change From Baseline in the Social Responsiveness Scale - 2nd Edition (SRS-2) Total T-score
DSM-5 social communication interaction T-score
|
0.6 score on a scale
Standard Deviation 5.4
|
-2.3 score on a scale
Standard Deviation 8.9
|
-8.3 score on a scale
Standard Deviation 10.6
|
-9.0 score on a scale
Standard Deviation 11.5
|
—
|
—
|
|
Change From Baseline in the Social Responsiveness Scale - 2nd Edition (SRS-2) Total T-score
DSM-5 RRB T-score
|
-0.4 score on a scale
Standard Deviation 9.3
|
-9.8 score on a scale
Standard Deviation 14.0
|
-8.7 score on a scale
Standard Deviation 13.5
|
-7.3 score on a scale
Standard Deviation 7.2
|
—
|
—
|
SECONDARY outcome
Timeframe: Week 0 to Week 4Population: Efficacy Population Note that for the efficacy population open-label data is excluded and placebo arms from the first cohort and second cohort are presented separately because in the first cohort questionnaires were filled out at home by patients and in the second cohort they were completed in clinic with study staff.
The Aberrant Behavior Checklist - Community (ABC-C) is a 58-symptom checklist for assessing problem behaviors of children and adults with developmental disabilities (intellectual disability, ASD, cerebral palsy, epilepsy) that resolve onto five subscales. The subscales and the respective number of items are as follows: (a) irritability (15 items, scores range from 0-45), (b) lethargy/social withdrawal (16 items, scores range from 0-48), (c) stereotypic behavior (7 items, scores range from 0-21), (d) hyperactivity/noncompliance (16 items, scores range from 0-48), and (e) inappropriate speech (4 items, scores range from 0-12). Higher ABC-C subscale scores indicates a "worse" outcome. A negative value of change from baseline indicates improvement.
Outcome measures
| Measure |
100 mg Open
n=9 Participants
open-label lead-in 100 mg L1-79 tid
|
100 mg Blinded
n=4 Participants
blinded and randomized 100 mg L1-79 tid
|
200 mg Open
n=10 Participants
open-label lead-in 200 mg L1-79 tid
|
200 mg Blinded
n=4 Participants
blinded and randomized 200 mg L1-79 tid
|
Placebo Blinded (100 mg Cohort)
blinded and randomized placebo tid (100 mg cohort)
|
Placebo Blinded (200 mg Cohort)
blinded and randomized placebo tid (200 mg cohort)
|
|---|---|---|---|---|---|---|
|
Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C)
inappropriate speech
|
0.0 score on a scale
Standard Deviation 1.7
|
-1.3 score on a scale
Standard Deviation 1.2
|
-2.1 score on a scale
Standard Deviation 2.6
|
-2.5 score on a scale
Standard Deviation 4.0
|
—
|
—
|
|
Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C)
irritability and agitation
|
-0.9 score on a scale
Standard Deviation 2.0
|
-5.3 score on a scale
Standard Deviation 3.8
|
-3.1 score on a scale
Standard Deviation 6.5
|
-4.5 score on a scale
Standard Deviation 4.8
|
—
|
—
|
|
Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C)
lethargy and social withdrawal
|
-1.6 score on a scale
Standard Deviation 6.7
|
-3.5 score on a scale
Standard Deviation 6.8
|
-5.4 score on a scale
Standard Deviation 6.2
|
-8.0 score on a scale
Standard Deviation 4.1
|
—
|
—
|
|
Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C)
stereotypic behavior
|
1.9 score on a scale
Standard Deviation 3.3
|
-1.0 score on a scale
Standard Deviation 2.9
|
-2.0 score on a scale
Standard Deviation 2.9
|
-2.8 score on a scale
Standard Deviation 1.7
|
—
|
—
|
|
Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C)
hyperactivity and noncompliance
|
0.4 score on a scale
Standard Deviation 3.9
|
-6.7 score on a scale
Standard Deviation 3.1
|
-4.6 score on a scale
Standard Deviation 9.4
|
-5.5 score on a scale
Standard Deviation 6.0
|
—
|
—
|
SECONDARY outcome
Timeframe: Week 0 and Week 4Population: Efficacy Population Note that for the efficacy population open-label data is excluded and placebo arms from the first cohort and second cohort are presented separately because in the first cohort questionnaires were filled out at home by patients and in the second cohort they were completed in clinic with study staff.
The RBS-R is a 43-item self-report questionnaire that is used to measure the breadth of repetitive behavior in children, adolescents, and adults with Autism Spectrum disorders. Behaviors are rated on a 4-point scale: 0-Behavior does not occur, 1-Behavior occurs and is a mild problem, 2-Behavior occurs and is a moderate problem, 3-Behavior occurs and is a severe problem. The total score is the sum of all 6 subscale scores and ranges from 0-127, stereotypic behavior ranges from 0-18, self-injurious behavior ranges from 0-24, compulsive behavior ranges from 0-24, ritualistic behavior ranges from 0-18, sameness behavior ranges from 0-33 and restricted behaviors ranges from 0-12. A higher RBS-R score indicates a "worse" state. A negative value of change from baseline indicates an improvement.
Outcome measures
| Measure |
100 mg Open
n=9 Participants
open-label lead-in 100 mg L1-79 tid
|
100 mg Blinded
n=3 Participants
blinded and randomized 100 mg L1-79 tid
|
200 mg Open
n=10 Participants
open-label lead-in 200 mg L1-79 tid
|
200 mg Blinded
n=4 Participants
blinded and randomized 200 mg L1-79 tid
|
Placebo Blinded (100 mg Cohort)
blinded and randomized placebo tid (100 mg cohort)
|
Placebo Blinded (200 mg Cohort)
blinded and randomized placebo tid (200 mg cohort)
|
|---|---|---|---|---|---|---|
|
Change From Baseline in the Repetitive Behavior Scale - Revised (RBS-R) Total Score
total score
|
-3.2 score on a scale
Standard Deviation 7.8
|
-9.3 score on a scale
Standard Deviation 12.7
|
-17.6 score on a scale
Standard Deviation 24.9
|
-16.5 score on a scale
Standard Deviation 6.0
|
—
|
—
|
|
Change From Baseline in the Repetitive Behavior Scale - Revised (RBS-R) Total Score
stereotypic behavior
|
-0.6 score on a scale
Standard Deviation 1.7
|
-2.3 score on a scale
Standard Deviation 4.0
|
-1.9 score on a scale
Standard Deviation 2.9
|
-0.5 score on a scale
Standard Deviation 2.6
|
—
|
—
|
|
Change From Baseline in the Repetitive Behavior Scale - Revised (RBS-R) Total Score
self-injurious behavior
|
-2.2 score on a scale
Standard Deviation 5.3
|
-2.3 score on a scale
Standard Deviation 4.9
|
-2.0 score on a scale
Standard Deviation 4.0
|
-2.3 score on a scale
Standard Deviation 3.3
|
—
|
—
|
|
Change From Baseline in the Repetitive Behavior Scale - Revised (RBS-R) Total Score
compulsive behavior
|
-1.2 score on a scale
Standard Deviation 3.6
|
-0.7 score on a scale
Standard Deviation 0.6
|
-3.0 score on a scale
Standard Deviation 5.1
|
-2.5 score on a scale
Standard Deviation 1.7
|
—
|
—
|
|
Change From Baseline in the Repetitive Behavior Scale - Revised (RBS-R) Total Score
ritualistic behavior
|
0.1 score on a scale
Standard Deviation 2.8
|
0.0 score on a scale
Standard Deviation 0.0
|
-3.4 score on a scale
Standard Deviation 4.2
|
-6.0 score on a scale
Standard Deviation 2.6
|
—
|
—
|
|
Change From Baseline in the Repetitive Behavior Scale - Revised (RBS-R) Total Score
sameness behavior
|
-0.7 score on a scale
Standard Deviation 2.0
|
-2.3 score on a scale
Standard Deviation 3.2
|
-4.6 score on a scale
Standard Deviation 7.3
|
-3.3 score on a scale
Standard Deviation 1.5
|
—
|
—
|
|
Change From Baseline in the Repetitive Behavior Scale - Revised (RBS-R) Total Score
restricted behavior
|
1.3 score on a scale
Standard Deviation 2.6
|
-1.7 score on a scale
Standard Deviation 1.5
|
-2.7 score on a scale
Standard Deviation 2.9
|
-2.0 score on a scale
Standard Deviation 1.6
|
—
|
—
|
Adverse Events
100 mg Open
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
100 mg Blinded
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
200 mg Open
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
200 mg Blinded
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Placebo Blinded (100 mg Cohort)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Placebo Blinded (200 mg Cohort)
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
100 mg Open
n=5 participants at risk
open-label lead-in 100 mg L1-79 tid
|
100 mg Blinded
n=10 participants at risk
blinded and randomized 100 mg L1-79 tid
|
200 mg Open
n=6 participants at risk
open-label lead-in 200 mg L1-79 tid
|
200 mg Blinded
n=11 participants at risk
blinded and randomized 200 mg L1-79 tid
|
Placebo Blinded (100 mg Cohort)
n=5 participants at risk
blinded and randomized placebo tid (100 mg cohort)
|
Placebo Blinded (200 mg Cohort)
n=5 participants at risk
blinded and randomized placebo tid (200 mg cohort)
|
|---|---|---|---|---|---|---|
|
Gastrointestinal disorders
diarrhoea
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
27.3%
3/11 • Number of events 3 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
General disorders
fatigue
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
16.7%
1/6 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/11 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
General disorders
pyrexia
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
16.7%
1/6 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/11 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Immune system disorders
seasonal allergy
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
16.7%
1/6 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/11 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Infections and infestations
ear infections
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
9.1%
1/11 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Infections and infestations
nasopharyngitis
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/11 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
20.0%
1/5 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Infections and infestations
otitis externa
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
9.1%
1/11 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Investigations
amylase increased
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/11 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
20.0%
1/5 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Investigations
blood TSH increased
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
9.1%
1/11 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Investigations
crystal urine present
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
18.2%
2/11 • Number of events 2 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Nervous system disorders
generalized tonic-clonic seizure
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
9.1%
1/11 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Nervous system disorders
syncope
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
10.0%
1/10 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/11 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Psychiatric disorders
agitation
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/11 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
20.0%
1/5 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Psychiatric disorders
anxiety
|
20.0%
1/5 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/11 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Psychiatric disorders
confusional state
|
20.0%
1/5 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/11 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Psychiatric disorders
intentional self injury
|
40.0%
2/5 • Number of events 2 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/11 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
20.0%
1/5 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Psychiatric disorders
irritability
|
40.0%
2/5 • Number of events 2 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/11 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Renal and urinary disorders
urinary incontinence
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/6 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/11 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
20.0%
1/5 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/10 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
16.7%
1/6 • Number of events 1 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/11 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
0.00%
0/5 • From the time of screening to the end of the study (28-day treatment period with 4-week post-treatment follow-up)
To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place